Leukemia

Key points

Leukemia has four main types: AML, ALL, CML and CLL; each has a completely different course, treatment and prognosis ^{[1, 2, 3]}.
An accurate diagnosis today is not made with blood counts and a microscope alone; it depends on flow cytometry, cytogenetics and molecular testing (such as FLT3, NPM1, BCR::ABL1 and TP53) ^{[2, 3]}.
In children with ALL, five-year survival in trials of the US Children’s Oncology Group has reached about 90% ^[4].
In CML, most patients in the chronic phase live for many years with good disease control on oral tyrosine kinase inhibitors (such as imatinib), and some can stop the drug under medical supervision ^{[5, 6, 21]}.
Newer treatments such as venetoclax and BTK inhibitors (such as ibrutinib), FLT3 inhibitors and blinatumomab, and, in selected patients, menin inhibitors and CAR T-cell therapy, have transformed the treatment landscape ^{[7, 8, 9, 10, 11, 28, 29, 30]}.

What is leukemia?

Leukemia is a group of cancers that start in the blood-forming cells of the bone marrow. In this disease, immature or abnormal blood cells multiply out of control, take the place of healthy cells in the bone marrow and enter the bloodstream. The result is a fall in red blood cells (anemia), healthy white blood cells (a higher risk of infection) and platelets (bleeding and bruising).

Leukemias are classified by two features: the cell type (myeloid or lymphoid) and the speed of progression (acute or chronic). The current World Health Organization classification (fifth edition) relies more than before on the genetic features of the cancer cells ^[1]. For an overview of all blood cancers (leukemia, lymphoma and myeloma), see the blood cancers page.

The four main types of leukemia

Type	Cell of origin	Disease course	Key feature
Acute myeloid leukemia (AML)	Myeloid precursors	Acute; needs immediate treatment	Mainly a disease of adults and older people; treatment is tailored to genetic mutations (such as FLT3 and NPM1) ^[2]
Acute lymphoblastic leukemia (ALL)	Lymphoid precursors (B or T)	Acute; needs immediate treatment	The most common cancer of childhood; in children the chance of cure is high ^{[4, 13]}
Chronic myeloid leukemia (CML)	Hematopoietic stem cell with the abnormal BCR::ABL1 gene	Chronic; usually diagnosed in the chronic phase	Targeted oral drugs (TKIs) have turned it into a controllable disease ^[5]
Chronic lymphocytic leukemia (CLL)	Mature B lymphocytes	Chronic; often slow-growing	Many patients initially need only monitoring ^[3]

Rarer types also exist, such as acute promyelocytic leukemia (APL) and hairy cell leukemia, each with its own specific treatment.

Signs and symptoms

The symptoms of leukemia mostly come from a shortage of healthy blood cells and the buildup of cancer cells in the body. In acute leukemias, symptoms usually appear and worsen over a few days to a few weeks; chronic leukemias (especially CLL), by contrast, often cause no symptoms and are found by chance on a routine blood test ^{[2, 3]}.

Severe tiredness, pale skin and shortness of breath on exertion (due to anemia)
Fever and frequent or long-lasting infections
Easy bruising, bleeding from the gums or nose, tiny red spots on the skin (petechiae)
Bone or joint pain (especially in children with ALL)
Enlarged lymph nodes, spleen or liver; a feeling of fullness on the left side of the abdomen
Night sweats, unexplained weight loss and fever with no obvious cause

For patients

When to see a doctor urgently

For patients

When to see a doctor urgently

A combination of fever, unusual bruising or bleeding, and severe tiredness that has developed over a few days to a few weeks calls for an urgent complete blood count (CBC). Acute leukemias are medical emergencies, and a delay in diagnosis can be dangerous. Anyone receiving chemotherapy should report any fever to their care team straight away.

Risk factors

In most patients, no specific cause of leukemia is found. However, some risk factors are known:

Previous chemotherapy or radiotherapy for another cancer; the World Health Organization classification has a separate category for myeloid neoplasms after cytotoxic therapy ^[1].
Inherited genetic predisposition and some genetic syndromes; for example, Down syndrome increases the risk of ALL in children ^{[1, 13]}.
Older age: AML and CLL are mainly diseases of older people; the median age of patients in the major trials of these two diseases has been over 70 years ^{[7, 14]}.
Previous blood disorders such as myelodysplastic syndromes, which can progress to AML ^[2].

Diagnosis and classification

Steps in the diagnosis and classification of leukemia

1
Complete blood count (CBC) and peripheral blood smear
The first step is to check the blood cell counts and look at the cells under a microscope. The presence of blasts (immature cells) in the blood raises suspicion of acute leukemia. In CLL, the diagnosis requires at least 5,000 monoclonal B lymphocytes per microliter of blood ^[3].
2
Bone marrow aspiration and biopsy
For AML and ALL, a bone marrow sample determines the percentage of blasts. In AML, the usual threshold is 20% or more blasts, but in subtypes with a defining genetic abnormality a lower percentage is also enough for the diagnosis ^{[1, 2]}.
3
Flow cytometry (immunophenotyping)
This test examines markers on the cell surface and identifies the type of leukemia (myeloid, B-lymphoid or T-lymphoid). Flow cytometry is also used to confirm that the lymphocytes are monoclonal in CLL and to measure measurable residual disease (MRD) ^{[2, 3]}.
4
Cytogenetics and molecular testing
Testing the chromosomes and mutations is the most important tool for determining prognosis and choosing treatment: FLT3, NPM1, IDH1/2 and TP53 in AML; the Philadelphia chromosome (BCR::ABL1) in ALL and CML; and IGHV mutation status, 17p deletion and TP53 mutation in CLL ^{[2, 3, 5]}.
5
Additional assessment before treatment
Depending on the situation: examination of the cerebrospinal fluid in ALL, assessment of heart, liver and kidney function, viral tests (such as hepatitis B) and tissue typing (HLA) for a possible transplant ^{[2, 3]}.

Acute myeloid leukemia (AML)

Treatment regimens and doses: index30 regimens

AML is an aggressive disease that is fatal within a few weeks to a few months without treatment. The choice of treatment depends on three factors: the patient’s age and physical fitness (ability to tolerate intensive chemotherapy), genetic risk and the presence of targetable mutations.

Genetic risk classification (ELN 2022)

The 2022 European LeukemiaNet (ELN) recommendations place patients into three groups based on cytogenetic and molecular findings ^[2]:

Favorable: for example, t(8;21), inv(16), NPM1 mutation without FLT3-ITD, and bZIP mutations in the CEBPA gene.
Intermediate: for example, FLT3-ITD mutation (regardless of allelic ratio) and t(9;11).
Adverse: for example, complex karyotype, KMT2A rearrangements, TP53 mutation and myelodysplasia-related mutations (such as ASXL1, RUNX1 and SRSF2).

This classification was developed in patients receiving intensive chemotherapy. In 2024, the ELN proposed a separate classification for patients receiving less-intensive therapy (such as venetoclax + azacitidine) ^[32].

Treatment options for AML

Intensive “7+3” chemotherapy

For physically fit patients, remission induction consists of 7 days of cytarabine and 3 days of an anthracycline such as daunorubicin, followed by consolidation therapy (high-dose cytarabine or a transplant) ^{[2, 8]}.

Venetoclax + azacitidine

For older patients or those unable to tolerate intensive chemotherapy, combining venetoclax with azacitidine more than doubled the remission rate compared with azacitidine alone in the VIALE-A trial and significantly prolonged survival ^{[7, 28]}.

Acute promyelocytic leukemia (APL): tretinoin + arsenic

APL is a distinct subtype of AML with the PML::RARA fusion gene and has its own treatment ^[1]. In the phase 3 APL0406 trial in low- to intermediate-risk patients, the chemotherapy-free combination of tretinoin (ATRA) and arsenic trioxide produced complete remission in all evaluable patients; it caused less hematologic toxicity and fewer infections but more liver toxicity than tretinoin plus chemotherapy ^[25], and with long-term follow-up it achieved better event-free survival and overall survival ^[38].

FLT3 inhibitors

In patients with a FLT3 mutation, intensive chemotherapy and, when possible, an allogeneic transplant are the mainstay of treatment ^[2]. After transplantation, maintenance with the oral drug sorafenib more than halved the risk of relapse and improved overall survival in a phase 3 trial in FLT3-ITD-positive patients ^[29]. Adding FLT3 inhibitors to induction chemotherapy is also beneficial: midostaurin in the RATIFY trial and quizartinib in the QuANTUM-First trial (in FLT3-ITD-positive patients) improved survival ^{[8, 15]}.

Allogeneic stem cell transplantation

For patients with adverse-risk or relapsed disease who are fit enough for a transplant, a transplant from a donor is the most important way to reduce the risk of relapse ^[2].

Menin inhibitors

In relapsed or refractory leukemia, salvage chemotherapy and, when possible, an allogeneic transplant are the main options ^[2]. For relapsed or refractory disease with a KMT2A rearrangement or an NPM1 mutation, the new oral drugs revumenib and ziftomenib are used. “Differentiation syndrome” is an important side effect of this class and requires close monitoring ^{[9, 16, 17]}. Based on the AUGMENT-101 trial, revumenib is approved for relapsed or refractory acute leukemia with a KMT2A translocation in patients aged one year and older ^[16].

For physicians

Shared safety points

WBC before starting: start venetoclax, revumenib and ziftomenib only when the WBC count is below 25×10⁹/L (cytoreduction if needed) ^{[39, 43, 44]}.
QTc prolongation: with quizartinib, arsenic trioxide and revumenib, obtain an ECG before starting and regularly during treatment, and correct potassium and magnesium; the monitoring frequency is given in the notes of all three regimens, and the thresholds for interruption or dose reduction in the notes for quizartinib and for tretinoin + arsenic trioxide ^{[41, 42, 43]}.
Differentiation syndrome: occurs with tretinoin and arsenic trioxide (APL) and with menin inhibitors (revumenib and ziftomenib) and requires close monitoring; its prevention and treatment are described in the tretinoin + arsenic trioxide notes, and its treatment in the ziftomenib notes ^{[9, 16, 17, 42]}.

For physiciansRegimens and dosing in newly diagnosed AML (induction, consolidation and maintenance), 5 regimens · Regimen and dosing in acute promyelocytic leukemia (APL), 1 regimen · +1 more

For physicians

Regimens and dosing in newly diagnosed AML (induction, consolidation and maintenance)

7+3 (cytarabine + daunorubicin or idarubicin) followed by high-dose cytarabine

Newly diagnosed AML (non-APL) in patients fit for intensive chemotherapy

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Induction
Cytarabine	100 or 200 mg/m² per day	IV, continuous infusion	Days 1–7
Daunorubicin	60 mg/m² per day	IV	Days 1–3
Induction (instead of daunorubicin)
Idarubicin	12 mg/m² per day	IV	Days 1–3
Consolidation
High-dose cytarabine	1.5 to 3 g/m² every 12 hours	IV	Days 1, 3 and 5

Cycle length: Induction: 1 cycle and, if needed, a second cycle (7+3 or 5+2); consolidation: 28-day cycles
Duration: Up to 2 induction cycles, then up to 4 consolidation cycles or allogeneic transplantation according to ELN risk

These doses are the chemotherapy backbone of the RATIFY trial (daunorubicin 60 + cytarabine 200 mg/m²; consolidation with cytarabine 3 g/m²) and the QuANTUM-First trial (cytarabine 100 or 200, daunorubicin 60 or idarubicin 12; consolidation 1.5 to 3 g/m²) ^{[15, 40, 41]}. In FLT3-mutated AML, midostaurin or (in FLT3-ITD) quizartinib is added to this same backbone (following regimens). The decision between continued consolidation and allogeneic transplantation in first remission is based on ELN 2022 genetic risk ^[2].

Evidence: RATIFY (CALGB 10603) (2017)

7+3 + midostaurin (RATIFY)

Newly diagnosed AML with a FLT3 mutation (ITD or TKD) detected by an approved test, fit for intensive chemotherapy (patients aged 18 to 60 years in RATIFY)

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Induction (up to 2 cycles)
Daunorubicin	60 mg/m² per day	IV	Days 1–3
Cytarabine	200 mg/m² per day	IV, continuous infusion	Days 1–7
Midostaurin	50 mg twice daily with food	Oral	Days 8–21
Consolidation (up to 4 cycles)
High-dose cytarabine	3 g/m² every 12 hours	IV	Days 1, 3 and 5
Midostaurin	50 mg twice daily with food	Oral	Days 8–21
Maintenance in RATIFY
Midostaurin	50 mg twice daily	Oral	Continuous, up to 12 cycles of 28 days

Cycle length: 28-day induction and consolidation cycles
Duration: Up to 2 induction cycles + up to 4 consolidation cycles (12-cycle maintenance only in the trial protocol)

Give prophylactic antiemetics before each dose; take the drug with food, approximately 12 hours apart. The FDA label approves midostaurin only in combination with induction and consolidation; single-agent maintenance was part of the RATIFY protocol, and patients who proceeded to transplantation discontinued study treatment ^{[15, 40]}.

Evidence: RATIFY (CALGB 10603) (2017)

7+3 + quizartinib (QuANTUM-First)

Newly diagnosed FLT3-ITD-positive AML (patients up to 75 years of age in the trial), only if QTcF ≤450 msCycle length: 28-day cycles

Newly diagnosed FLT3-ITD-positive AML (patients up to 75 years of age in the trial), only if QTcF ≤450 ms

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Induction (up to 2 cycles)
Cytarabine + daunorubicin or idarubicin	Cytarabine 100 or 200 mg/m²; daunorubicin 60 or idarubicin 12 mg/m²	IV	Cytarabine Days 1–7; anthracycline Days 1–3
Quizartinib	35.4 mg once daily	Oral	Days 8–21 (in a second 5+2 induction: Days 6–19)
Consolidation (up to 4 cycles)
High-dose cytarabine	1.5 to 3 g/m² every 12 hours	IV	Days 1, 3 and 5
Quizartinib	35.4 mg once daily	Oral	Days 6–19
Maintenance
Quizartinib	26.5 mg once daily, then 53 mg once daily	Oral	Days 1–14 of the first cycle at 26.5 mg; from Day 15, 53 mg if QTcF ≤450 ms, continuous

Cycle length: 28-day cycles
Duration: Up to 2 induction cycles, up to 4 consolidation cycles and up to 36 maintenance cycles (or until progression/unacceptable toxicity)

ECG before starting and weekly during induction and consolidation; during maintenance, weekly for at least one month after starting or after a dose increase. Discontinue QT-prolonging drugs as far as possible. QTcF 481–500 ms: reduce the dose without interrupting; above 500: interrupt and resume at a lower dose once below 450; if QTcF above 500 occurred during induction or consolidation, maintenance should continue at 26.5 mg; recurrent QTcF above 500 or torsades de pointes: discontinue permanently. Maintenance starts after count recovery (neutrophils above 500 and platelets above 50,000 per microliter); stop 7 days before the transplant conditioning regimen, and in the trial, transplanted patients started maintenance after recovering from the transplant ^{[8, 41]}.

Evidence: QuANTUM-First (2023)

Venetoclax + azacitidine (VIALE-A)

Newly diagnosed AML in patients aged 75 years or older or with comorbidities that preclude intensive chemotherapyCycle length: Every 28 days

Newly diagnosed AML in patients aged 75 years or older or with comorbidities that preclude intensive chemotherapy

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Cycle 1: ramp-up
Venetoclax	100 mg (Day 1), 200 mg (Day 2), 400 mg (Day 3)	Oral, with food	Days 1–3
Continuation
Venetoclax	400 mg once daily	Oral, with food	Days 1–28 of each cycle
All cycles
Azacitidine	75 mg/m² per day	SC or IV	Days 1–7

Cycle length: Every 28 days
Duration: Until disease progression or unacceptable toxicity

TLS prophylaxis with hydration and a uric acid–lowering agent from before the first dose and throughout the ramp-up; check potassium, uric acid, phosphorus, calcium and creatinine before each new dose, 6 to 8 hours afterward and 24 hours after reaching the final dose. Interactions: with posaconazole, ramp up 10, 20, 50 and then 70 mg; with other strong CYP3A inhibitors, ramp up 10, 20, 50 and then 100 mg; with a moderate CYP3A inhibitor, reduce the dose by at least 50%. Cytopenias before remission are usually not a reason to interrupt treatment (a bone marrow assessment is recommended); grade 4 neutropenia or thrombocytopenia after remission lasting 7 days or longer: delay the next cycle until recovery to grade 1 or 2, and if it recurs, shorten venetoclax administration in each cycle by 7 days (e.g., 21 days instead of 28). Label alternatives: venetoclax 400 mg with decitabine 20 mg/m² IV Days 1–5, or venetoclax 600 mg with cytarabine 20 mg/m² SC Days 1–10. In VIALE-A, febrile neutropenia occurred in 42% vs 19% ^{[7, 39]}.

Evidence: VIALE-A (2024), VIALE-A (2020)

Sorafenib maintenance after allogeneic transplantation

FLT3-ITD AML after allogeneic transplantation, in composite complete remission before and after transplantation, aged 18 to 60 years, ECOG 0–2, with hematologic recovery by Day 60 after transplantationCycle length: Continuous

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Sorafenib	400 mg twice daily (adjusted within 200 to 800 mg per day according to toxicity)	Oral	Start between Day 30 and Day 60 after transplantation

Cycle length: Continuous
Duration: Until Day 180 after transplantation

This use (after transplantation) is not in the sorafenib FDA label and is based on a Chinese phase 3 trial; at five-year follow-up it was not associated with an increase in non-relapse mortality or chronic GVHD ^{[29, 45, 62]}.

Evidence: Xuan 2023 (2023)

Doses follow the trial protocol, guideline or official drug label (source given in the notes of each regimen). Before prescribing, check blood counts, renal and hepatic function, drug interactions and the patient’s condition, and adjust the dose according to the drug label.

For physicians

Regimen and dosing in acute promyelocytic leukemia (APL)

Tretinoin (ATRA) + arsenic trioxide (APL0406)

Newly diagnosed acute promyelocytic leukemia (APL), low to intermediate risk (WBC ≤10,000 per microliter)

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Induction (1 cycle)
Arsenic trioxide	0.15 mg/kg once daily	IV	Daily until bone marrow remission (maximum 60 days)
Tretinoin	22.5 mg/m² twice daily (45 mg/m² per day)	Oral	Daily until bone marrow remission (maximum 60 days)
Consolidation (4 cycles of 8 weeks)
Arsenic trioxide	0.15 mg/kg once daily	IV	5 days per week, Weeks 1–4 of each 8-week cycle
Tretinoin	22.5 mg/m² twice daily	Oral	Weeks 1, 2, 5 and 6 of each 8-week cycle (Weeks 5 and 6 are omitted in the fourth cycle)

Cycle length: Induction: daily until bone marrow remission (maximum 60 days); consolidation: 8-week cycles
Duration: 1 induction cycle + 4 consolidation cycles

Differentiation syndrome prophylaxis with prednisone 0.5 mg/kg per day from Day 1 until the end of induction. If differentiation syndrome occurs: temporarily withhold arsenic trioxide (and, in severe cases, tretinoin), give dexamethasone 10 mg IV every 12 hours for at least 3 days and until symptoms resolve, then resume at 50% of the dose. ECG before starting and weekly; keep potassium above 4 mEq/L and magnesium above 1.8 mg/dL; QTc above 450 ms (men) or 460 ms (women): interrupt and resume at half the dose after correction. Hepatotoxicity (bilirubin more than 3 times, or AST or ALP more than 5 times the upper limit of normal): interrupt and resume at 50% of the dose. During induction, coagulation tests, blood counts and blood chemistry at least 2 to 3 times per week. The tretinoin dose is rounded to the nearest 10 mg ^{[25, 38, 42]}.

Before prescribing, check each dose against the drug label, laboratory tests and the patient’s condition.

For physicians

Regimens and dosing in relapsed or refractory AML

Revumenib (AUGMENT-101)

Relapsed or refractory acute leukemia with a KMT2A translocation (aged 1 year and older), or relapsed or refractory NPM1-mutated AML with no other satisfactory optionCycle length: Continuous

Relapsed or refractory acute leukemia with a KMT2A translocation (aged 1 year and older), or relapsed or refractory NPM1-mutated AML with no other satisfactory option

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Weight 40 kg or more
Revumenib	270 mg twice daily (with a strong CYP3A4 inhibitor: 160 mg twice daily)	Oral, on an empty stomach or with a low-fat meal	Continuous
Weight less than 40 kg
Revumenib	160 mg/m² twice daily (with a strong CYP3A4 inhibitor: 95 mg/m²)	Oral	Continuous

Cycle length: Continuous
Duration: Until progression or unacceptable toxicity; in the absence of progression, at least 6 months to allow time for a response

ECG before starting, at least weekly for the first 4 weeks and then monthly (QTc prolongation); blood counts, electrolytes and liver enzymes before starting and monthly. Differentiation syndrome is an important adverse effect (grade ≥3 in 16.0% in AUGMENT-101); grade ≥3 QTc prolongation occurred in 13.8%. In patients at risk of central nervous system relapse, concurrent intrathecal prophylaxis is recommended ^{[9, 16, 43]}.

Evidence: AUGMENT-101 (2025), AUGMENT-101 (2025)

Ziftomenib (KOMET-001)

Relapsed or refractory NPM1-mutated AML in adults with no other satisfactory optionCycle length: Continuous

Relapsed or refractory NPM1-mutated AML in adults with no other satisfactory option

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Ziftomenib	600 mg once daily	Oral, on an empty stomach (1 hour before or 2 hours after a meal)	Continuous

Cycle length: Continuous
Duration: Until progression or unacceptable toxicity; at least 6 months in the absence of progression

Avoid proton pump inhibitors; if an H2 antagonist cannot be avoided, take ziftomenib 2 hours before or 10 hours after it. If differentiation syndrome is suspected: interrupt the drug; systemic corticosteroids and hemodynamic monitoring for at least 3 days; control noninfectious leukocytosis with hydroxyurea. Dose reduction for recurrent grade ≥3 toxicity: 400 mg and then 200 mg; inability to tolerate 200 mg: discontinue permanently ^{[17, 44]}.

Evidence: KOMET-001 (2025)

Before prescribing, check each dose against the drug label, laboratory tests and the patient’s condition.

Acute lymphoblastic leukemia (ALL)

ALL is the most common cancer of childhood and also occurs in adults ^[13]. Standard treatment consists of several phases of combination chemotherapy (induction, consolidation, intensification and maintenance) lasting about two to three years in total, together with prevention of central nervous system involvement ^[13].

Children: The treatment of childhood ALL is one of the greatest successes of modern medicine. Among more than 21,000 children and adolescents enrolled in US Children’s Oncology Group (COG) trials, five-year survival rose from 83.7% in 1990–1994 to 90.4% in 2000–2005 ^[4].

Adolescents and young adults: In the CALGB 10403 study in patients aged 17 to 39 years, a regimen using the same doses and schedule as the pediatric protocol was associated with low treatment-related mortality and much better outcomes than historical controls, and it was put forward as a standard backbone for treating this age group ^[35].

Factors that determine risk: age, white blood cell count, genetic features (such as the Philadelphia chromosome) and, most importantly, response to treatment as measured by MRD ^{[13, 33]}.

New treatments for ALL

Philadelphia-positive ALL: adding a tyrosine kinase inhibitor

In Philadelphia chromosome–positive ALL, a tyrosine kinase inhibitor such as imatinib or dasatinib is used together with chemotherapy or corticosteroids ^{[13, 34, 36]}. In the randomized GRAAPH-2005 trial, imatinib with reduced-intensity chemotherapy led to fewer deaths during induction and a higher complete remission rate than imatinib with the intensive hyper-CVAD regimen; allogeneic transplantation in first remission was associated with better overall survival ^[34]. In the PhALLCON trial, ponatinib with reduced-intensity chemotherapy produced more MRD-negative complete remissions than imatinib ^[19], and in the phase 2 D-ALBA study, the chemotherapy-free regimen of dasatinib followed by blinatumomab produced complete remission in almost all patients ^[36].

First-line blinatumomab

Blinatumomab is a bispecific antibody that links T lymphocytes to CD19-positive leukemia cells. In the E1910 trial in adults aged 30 to 70 years in MRD-negative remission, adding it to consolidation chemotherapy significantly improved survival ^[10].

Inotuzumab ozogamicin

This antibody–drug conjugate targets CD22. In the INO-VATE trial in relapsed ALL, the complete remission rate was more than double that with standard chemotherapy; hepatic veno-occlusive disease (VOD) is an important side effect ^[18].

CAR T-cell therapy

In children and young adults with relapsed or refractory ALL, tisagenlecleucel produced remission within three months in 81% of patients in the ELIANA trial ^[11]. In adults, brexucabtagene autoleucel produced complete remission or complete remission with incomplete hematologic recovery in 71% of patients in the ZUMA-3 trial ^[20].

For physicians

Shared safety points

Cytokine release syndrome and neurologic toxicity: boxed warning for blinatumomab and for both CAR T-cell products (tisagenlecleucel and brexucabtagene autoleucel); for CAR T-cell therapy, secondary hematologic malignancies are also included in the boxed warning ^{[49, 51, 52]}.
Before CAR T-cell infusion: premedication with acetaminophen and an H1 antihistamine; confirm that tocilizumab is available before infusion ^{[51, 52]}.

For physiciansRegimens and dosing in Philadelphia-negative ALL, 2 regimens · Regimens and dosing in Philadelphia-positive ALL, 3 regimens · +1 more

For physicians

Regimens and dosing in Philadelphia-negative ALL

Pediatric regimen in adolescents and young adults (CALGB 10403; identical to one arm of COG AALL0232)

Newly diagnosed Ph-negative ALL (B or T) in patients aged 17 to 39 years, ECOG 0–2

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Induction (course I)
Prednisone	60 mg/m² per day in 2 divided doses	Oral or IV	Days 1–28
Vincristine	1.5 mg/m² (maximum 2 mg)	IV	Days 1, 8, 15 and 22
Daunorubicin	25 mg/m²	IV	Days 1, 8, 15 and 22
Pegaspargase	2500 units/m²	IM or IV	Day 4
Intrathecal cytarabine; intrathecal methotrexate	Cytarabine 70 mg; methotrexate 15 mg	Intrathecal	Cytarabine Day 1; methotrexate Days 8 and 29 (in CNS3, also Days 15 and 22)
Maintenance (12-week cycles)
Vincristine	1.5 mg/m² (maximum 2 mg)	IV	Days 1, 29 and 57
Maintenance
Dexamethasone	6 mg/m² per day in 2 divided doses	Oral or IV	Days 1–5, 29–33 and 57–61
Mercaptopurine	75 mg/m² per day	Oral	Days 1–84
Methotrexate	20 mg/m² weekly	Oral	Days 8, 15, 22, 29, 36, 43, 50, 57, 64, 71 and 78 (Day 29 is omitted in the first 4 cycles)
Methotrexate	15 mg	Intrathecal	Day 1 (in the first 4 cycles, also Day 29)

Cycle length: 28-day induction; maintenance in 12-week cycles
Duration: Induction → consolidation → interim maintenance → delayed intensification → maintenance until 3 years (men) or 2 years (women) from the start of interim maintenance

During induction, allopurinol 300 mg per day. Consolidation (course II): cyclophosphamide 1000 mg/m² Days 1 and 29; cytarabine 75 mg/m² IV or SC Days 1–4, 8–11, 29–32 and 36–39; mercaptopurine 60 mg/m² Days 1–14 and 29–42; vincristine Days 15, 22, 43 and 50; pegaspargase 2500 units/m² Days 15 and 43; intrathecal methotrexate Days 1, 8, 15 and 22. Interim maintenance (course III): IV methotrexate starting at 100 mg/m² and escalating by 50 mg/m² with each dose (Days 1, 11, 21, 31 and 41), pegaspargase Days 2 and 22, and intrathecal methotrexate Days 1 and 31. Delayed intensification (course IV): vincristine Days 1, 8, 43 and 50; dexamethasone 10 mg/m² per day on Days 1–7 and 15–21; pegaspargase Day 4 (or 5 or 6) and Day 43; cyclophosphamide 1000 mg/m² Day 29; cytarabine 75 mg/m² Days 29–32 and 36–39; thioguanine 60 mg/m² Days 29–42; intrathecal methotrexate Days 1, 29 and 36. In T-ALL, prophylactic cranial irradiation of 24 Gy during the first maintenance cycle, and 18 Gy for CNS involvement at diagnosis. The doses are taken from Figure 1 of the paper; use the full protocol for complete details (dose modifications and monitoring) ^[35].

Blinatumomab in consolidation (E1910)

Philadelphia-negative CD19-positive B-ALL in the consolidation phase of multiphase chemotherapy; in E1910, adults aged 30 to 70 years in MRD-negative CR or CRiCycle length: Every 42 days

Philadelphia-negative CD19-positive B-ALL in the consolidation phase of multiphase chemotherapy; in E1910, adults aged 30 to 70 years in MRD-negative CR or CRi

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Each blinatumomab cycle
Blinatumomab	45 kg or more: 28 mcg per day; less than 45 kg: 15 mcg/m² per day (maximum 28 mcg per day)	IV, continuous infusion by pump	Days 1–28, then 14 days off

Cycle length: Every 42 days
Duration: In E1910: 2 blinatumomab cycles → 3 cycles of consolidation chemotherapy → third blinatumomab cycle → fourth chemotherapy cycle → fourth blinatumomab cycle; then maintenance until 2.5 years from the start of intensification

Premedication: dexamethasone 20 mg IV within 1 hour before the first dose of each cycle. Hospitalization is recommended for the first 3 days of the first cycle and the first 2 days of the second cycle. Intrathecal prophylaxis before and during treatment. Other label schedules: MRD-positive ALL (≥0.1%): 28 mcg per day, 1 induction cycle + up to 3 consolidation cycles (premedication with prednisone 100 mg IV); relapsed or refractory ALL: first cycle 9 mcg per day on Days 1–7 and 28 mcg per day on Days 8–28, then 28 mcg per day for up to 2 induction cycles, 3 consolidation cycles and up to 4 continued-therapy cycles (28 days of infusion + 56 days off), with hospitalization for the first 9 days ^{[10, 49]}.

Evidence: E1910 (2024)

For physicians

Regimens and dosing in Philadelphia-positive ALL

Imatinib + reduced-intensity chemotherapy (GRAAPH-2005)

Newly diagnosed Philadelphia-positive ALL in adults (median age 47 years)

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Induction
Imatinib	High dose (exact figure not stated in the abstract); FDA label dose for adult Ph+ ALL: 600 mg per day	Oral, with food	Daily
Reduced-intensity chemotherapy	Per the GRAAPH-2005 protocol

Duration: After the second cycle, allogeneic transplantation if a donor is available

The chemotherapy doses of this protocol could not be verified in the sources available to us; use the full protocol text ^{[34, 47]}.

Ponatinib + reduced-intensity chemotherapy (PhALLCON)

Newly diagnosed Philadelphia-positive (Ph+) ALL in adultsEach drug’s schedule in the table

Newly diagnosed Philadelphia-positive (Ph+) ALL in adults

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Induction (cycles 1–3)
Ponatinib	30 mg once daily	Oral	Continuous
Vincristine	1.4 mg/m² (maximum 2 mg)	IV	Days 1 and 14, every 28 days
Dexamethasone	Under 60 years: 40 mg; 60 years and older: 20 mg	Oral	Days 1–4 and 11–14, every 28 days
Consolidation (cycles 4, 6 and 8)
Methotrexate	Under 60 years: 1000 mg/m²; 60 years and older: 250 mg/m²	IV	Day 1 of cycles 4, 6 and 8 (each cycle 28 days)
Consolidation (cycles 5, 7 and 9)
Cytarabine	Under 60 years: 1000 mg/m²; 60 years and older: 250 mg/m², every 12 hours	IV	Days 1, 3 and 5 of cycles 5, 7 and 9 (each cycle 28 days)
Maintenance (cycles 10–20)
Vincristine	1.4 mg/m² (maximum 2 mg)	IV	Day 1, every 28 days
Prednisone	Under 60 years: 200 mg; 60 to 69 years: 100 mg; 70 years and older: 50 mg	Oral	Days 1–5, every 28 days

Cycle schedule: Chemotherapy in 28-day cycles; ponatinib daily and continuously
Duration: 20 cycles with chemotherapy (ponatinib continues through consolidation and maintenance); in the trial, followed by single-agent ponatinib until relapse, transplantation or toxicity

After induction, once MRD-negative complete remission is achieved (BCR::ABL1/ABL1 ≤0.01%), the ponatinib dose is reduced to 15 mg, and it returns to 30 mg if MRD negativity is lost. Boxed warning: arterial occlusive events, venous thromboembolism, heart failure and hepatotoxicity; monitor cardiovascular risk factors and liver enzymes. Continuing ponatinib alone after chemotherapy, and the imatinib 600 mg comparator, are not FDA-approved regimens ^{[19, 46]}.

Evidence: PhALLCON (2024)

Dasatinib followed by blinatumomab without chemotherapy (D-ALBA)

Newly diagnosed Philadelphia-positive ALL in adults (no upper age limit); investigational regimen (phase 2)

Newly diagnosed Philadelphia-positive ALL in adults (no upper age limit); investigational regimen (phase 2) ^{[36, 60]}

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Induction
Prednisone	Escalating dose up to 60 mg/m²	Oral	From Day -6 to Day 0, continued until Day 24 and tapered by Day 31
Dasatinib	140 mg once daily	Oral	Days 1–84
Post-induction
Blinatumomab	Per the D-ALBA protocol	IV, continuous infusion	4 weeks of infusion + 2 weeks off in each cycle

Cycle length: Blinatumomab: every 6 weeks
Duration: At least 2 and at most 5 blinatumomab cycles

The labeled dasatinib dose for adult Ph+ ALL is 140 mg ^[48]. Dasatinib may cause fluid retention and pleural effusion ^[48].

Before prescribing, check each dose against the drug label, laboratory tests and the patient’s condition.

For physicians

Regimens and dosing in relapsed or refractory B-ALL

Inotuzumab ozogamicin (INO-VATE)

Relapsed or refractory CD22-positive B-ALL (adults and children aged 1 year and older)

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Cycle 1 (all patients)
Inotuzumab ozogamicin	0.8 mg/m² (Day 1), 0.5 mg/m² (Days 8 and 15)	IV	Days 1, 8 and 15
Subsequent cycles, in CR or CRi
Inotuzumab ozogamicin	0.5 mg/m²	IV	Days 1, 8 and 15
Subsequent cycles, without CR or CRi
Inotuzumab ozogamicin	0.8 / 0.5 / 0.5 mg/m²	IV	Days 1, 8 and 15

Cycle length: Cycle 1: 21 days (up to 28 days in CR/CRi or to allow recovery from toxicity); subsequent cycles: 28 days
Duration: If proceeding to transplantation: 2 cycles (a third cycle if MRD-negative CR/CRi has not been reached after 2 cycles); without transplantation: up to 6 cycles; no CR/CRi within 3 cycles: discontinue

Premedication before each infusion: a corticosteroid, an antipyretic and an antihistamine. Boxed warning: hepatotoxicity, including hepatic veno-occlusive disease (VOD in 11% of INO-VATE patients), and increased non-relapse mortality after transplantation; monitor liver tests ^{[18, 50]}.

Evidence: INO-VATE ALL (2016)

Tisagenlecleucel (CAR T-cell therapy; ELIANA)

B-ALL that is refractory or in second or later relapse, in patients up to 25 years of ageCycle length: One-time

B-ALL that is refractory or in second or later relapse, in patients up to 25 years of age

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Lymphodepletion
Fludarabine	30 mg/m² per day	IV	4 days
Cyclophosphamide	500 mg/m² per day	IV	2 days (starting on the first day of fludarabine)
One-time infusion
Tisagenlecleucel	50 kg or less: 0.2 to 5.0 × 10⁶ CAR-positive viable T cells/kg; more than 50 kg: 0.1 to 2.5 × 10⁸ cells	IV	2 to 14 days after completion of lymphodepletion

Cycle length: One-time
Duration: One-time

Do not use a leukocyte-depleting filter; do not infuse in the presence of active infection ^{[11, 51]}.

Evidence: ELIANA (2018)

Brexucabtagene autoleucel (CAR T-cell therapy; ZUMA-3)

Relapsed or refractory B-ALL in adultsCycle length: One-time

Relapsed or refractory B-ALL in adults

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Lymphodepletion
Fludarabine	25 mg/m² over 30 minutes	IV	Days -4, -3 and -2 before infusion
Cyclophosphamide	900 mg/m² over 60 minutes	IV	Day -2 before infusion
One-time infusion
Brexucabtagene autoleucel	1 × 10⁶ CAR-positive viable T cells/kg (maximum 1 × 10⁸)	IV	Day 0

Cycle length: One-time
Duration: One-time

Give premedication 30 to 60 minutes before infusion: acetaminophen and diphenhydramine (or another H1 antihistamine) ^{[20, 52]}.

Evidence: ZUMA-3 (2021)

Before prescribing, check each dose against the drug label, laboratory tests and the patient’s condition.

Chronic myeloid leukemia (CML)

CML is caused by a chromosomal translocation that creates the BCR::ABL1 fusion gene (the Philadelphia chromosome). This gene makes a permanently active enzyme (a tyrosine kinase) that drives cell proliferation. Tyrosine kinase inhibitors (TKIs) target exactly this enzyme, which is why most patients in the chronic phase live for many years with good disease control ^{[5, 21]}.

In the landmark IRIS trial, ten-year survival among patients treated with imatinib was 83.3% ^[21]. Response to treatment is monitored at regular intervals with quantitative PCR testing for BCR::ABL1. According to the 2025 European LeukemiaNet (ELN) recommendations, if an unfavorable response is confirmed or side effects cannot be controlled by dose reduction, the drug is switched after adherence and BCR::ABL1 mutations have been checked; this decision should not be based on a single result ^[5].

Tyrosine kinase inhibitors in CML

Drug	Generation / mechanism	Clinical note
Imatinib	First generation	One of the standard first-line options, with the longest follow-up ^{[5, 21]}
Dasatinib, nilotinib, bosutinib	Second generation	Faster molecular response, but in trials they have not improved overall survival compared with imatinib; choice based on comorbidities and side effects ^[5]
Ponatinib	Third generation	Active against the resistant T315I mutation; the dose is reduced once a response is achieved ^[5]
Asciminib	Specific inhibitor of the ABL “myristoyl pocket”	In the first-line ASC4FIRST trial, it achieved more major molecular responses and fewer dose reductions due to side effects than other TKIs; long-term survival data are not yet available ^{[5, 22]}

The choice of drug should take into account age, cardiovascular, lung and liver disease, and drug interactions.

Treatment-free remission

In patients who have taken a TKI for at least 5 years (at least 4 years with a second-generation TKI) and have had a deep molecular response (MR4 or better) for more than 2 years, stopping the drug can be tried under close monitoring; about 40% to 50% of these patients remain off treatment ^[5]. Stopping the drug on your own without regular PCR monitoring is dangerous.

For physiciansTreatment regimens and dosing in CML, 6 regimens

For physicians

Treatment regimens and dosing in CML

Imatinib

Philadelphia-positive CML in adults (first line or later)Cycle length: Continuous

Philadelphia-positive CML in adults (first line or later)

Drug	Dose	Route	Schedule
Chronic phase
Imatinib	400 mg once daily	Oral, with food and a large glass of water	Continuous
Accelerated or blast phase
Imatinib	600 mg once daily	Oral	Continuous

Cycle length: Continuous
Duration: Until loss of response or unacceptable toxicity; stop only according to treatment-free remission criteria

In the absence of severe adverse reactions, a dose increase from 400 to 600 mg (chronic phase) or from 600 to 800 mg (400 mg twice daily; advanced phase) may be considered. Severe hepatic impairment: 300 mg per day ^{[21, 47]}.

Evidence: IRIS (2017)

Dasatinib

Philadelphia-positive CML in adultsCycle length: Continuous

Philadelphia-positive CML in adults

Drug	Dose	Route	Schedule
Chronic phase
Dasatinib	100 mg once daily	Oral, with or without food	Continuous
Accelerated or blast phase
Dasatinib	140 mg once daily	Oral	Continuous

Cycle length: Continuous
Duration: Until loss of response or unacceptable toxicity

Fluid retention and pleural effusion (sometimes severe), pulmonary hypertension and bleeding are important adverse effects; monitor blood counts regularly ^[48].

Nilotinib

Philadelphia-positive CML in adultsCycle length: Continuous

Philadelphia-positive CML in adults

Drug	Dose	Route	Schedule
Newly diagnosed chronic phase
Nilotinib	300 mg twice daily	Oral, on an empty stomach	Every 12 hours, continuous
Resistant or intolerant (chronic or accelerated phase)
Nilotinib	400 mg twice daily	Oral, on an empty stomach	Every 12 hours, continuous

Cycle length: Continuous
Duration: Until loss of response or unacceptable toxicity

No food from 2 hours before until 1 hour after each dose. Boxed warning for QT prolongation and sudden death: ECG before starting, 7 days after starting, periodically and after any dose change; correct hypokalemia and hypomagnesemia, and do not prescribe in long QT syndrome or uncorrected electrolyte abnormalities. Hepatic impairment at baseline: lower dose ^[53].

Bosutinib

Philadelphia-positive CML in adultsCycle length: Continuous

Philadelphia-positive CML in adults

Drug	Dose	Route	Schedule
Newly diagnosed chronic phase
Bosutinib	400 mg once daily	Oral, with food	Continuous
Resistant or intolerant to prior therapy (all phases)
Bosutinib	500 mg once daily	Oral, with food	Continuous

Cycle length: Continuous
Duration: Until progression or intolerance

If the response is inadequate and there are no grade ≥3 adverse reactions, the dose may be increased in 100 mg increments up to a maximum of 600 mg per day ^[54].

Ponatinib

Chronic-phase CML resistant or intolerant to at least 2 prior kinase inhibitors; T315I-mutated CML; accelerated or blast phase with no other kinase inhibitor optionCycle length: Continuous

Chronic-phase CML resistant or intolerant to at least 2 prior kinase inhibitors; T315I-mutated CML; accelerated or blast phase with no other kinase inhibitor option

Drug	Dose	Route	Schedule
Chronic phase
Ponatinib	45 mg once daily; reduce to 15 mg once BCR::ABL1 IS reaches ≤1%	Oral	Continuous
Accelerated or blast phase
Ponatinib	45 mg once daily	Oral	Continuous

Cycle length: Continuous
Duration: Until loss of response or toxicity; no hematologic response by 3 months: consider discontinuation

If response is lost after dose reduction, return to 30 or 45 mg (the previously tolerated dose). Boxed warning: arterial occlusive events (even in people aged 50 years or younger), venous thromboembolism, heart failure and hepatotoxicity ^{[5, 46]}.

Asciminib

Philadelphia-positive chronic-phase CML in adults: newly diagnosed (ASC4FIRST), previously treated, or with the T315I mutationCycle length: Continuous

Philadelphia-positive chronic-phase CML in adults: newly diagnosed (ASC4FIRST), previously treated, or with the T315I mutation

Drug	Dose	Route	Schedule
Newly diagnosed or previously treated
Asciminib	80 mg once daily or 40 mg twice daily	Oral, on an empty stomach	Continuous
T315I mutation
Asciminib	200 mg twice daily	Oral, on an empty stomach	Continuous

Cycle length: Continuous
Duration: As long as there is clinical benefit, or until unacceptable toxicity

No food from 2 hours before until 1 hour after each dose. Dose reduction for adverse reactions: 40 mg once daily or 20 mg twice daily (in T315I: 160 mg twice daily); inability to tolerate these doses: discontinue permanently. The FDA first-line approval is based on major molecular response (accelerated approval) ^{[22, 55]}.

Evidence: ASC4FIRST (2026)

Chronic lymphocytic leukemia (CLL)

CLL is the most common leukemia in adults in Western countries and usually has a slow course. Starting treatment as soon as it is diagnosed brings no benefit; patients without symptoms in the early stages are followed with “active surveillance” (watch and wait) ^[3].

According to the iwCLL guidelines, treatment is started when “active disease” is present, including ^[3]:

Progressive bone marrow failure (hemoglobin below 10 g/dL or platelets below 100,000)
Massive or progressive enlargement of the spleen or lymph nodes
A rapid rise in lymphocytes (doubling in less than 6 months)
Troublesome general symptoms such as fever, night sweats or significant weight loss
Autoimmune anemia or low platelets that do not respond to standard treatment

Before treatment starts, testing for 17p deletion/TP53 mutation and IGHV mutation status is essential, because the results affect the choice of treatment ^[3].

Targeted treatments for CLL

BTK inhibitors (continuous treatment)

The oral drug ibrutinib is taken until progression or intolerance. In patients aged 65 years and older, progression-free survival with ibrutinib was markedly longer than with bendamustine–rituximab chemoimmunotherapy, and this advantage was also seen in patients with TP53 abnormalities ^[23]. In patients aged 70 years and younger, ibrutinib–rituximab also improved overall survival compared with FCR chemoimmunotherapy ^[37]. Atrial fibrillation and high blood pressure are important side effects of ibrutinib ^[23]. Second-generation BTK inhibitors such as zanubrutinib have fewer cardiac side effects; in the ALPINE trial in relapsed or refractory CLL, zanubrutinib achieved longer progression-free survival and fewer cardiac side effects than ibrutinib ^[12].

Fixed-duration targeted treatments

The all-oral, fixed-duration combination of ibrutinib + venetoclax is effective in both younger and older patients: in the FLAIR trial, this combination, with its duration set according to MRD, achieved longer progression-free survival than ibrutinib alone and than FCR ^[30], and in the GLOW trial in patients aged 65 years and older or with comorbidities, 15 cycles of treatment (3 cycles of ibrutinib followed by 12 cycles of the two-drug combination) markedly prolonged progression-free survival compared with chlorambucil–obinutuzumab ^[26]. The one-year combination of venetoclax + obinutuzumab also more than doubled median progression-free survival compared with chlorambucil–obinutuzumab in patients with comorbidities in the CLL14 trial ^[31]. In relapsed or refractory disease, venetoclax + rituximab (venetoclax for 2 years) improved progression-free survival and overall survival compared with bendamustine–rituximab in the MURANO trial ^[27].

Choosing between the two approaches

Continuous treatment with a BTK inhibitor or fixed-duration venetoclax-based treatment is chosen according to comorbidities (especially heart and kidney disease), genetic features and the patient’s preference ^{[3, 24]}. According to the 2026 European Hematology Association (EHA) guideline, fixed-duration targeted treatments are preferred over continuous treatment in patients without TP53 abnormalities, and continuous treatment is suggested for patients with TP53 abnormalities. For continuous treatment, acalabrutinib or zanubrutinib is preferred over ibrutinib because of fewer side effects, but patients whose disease is well controlled on ibrutinib without significant side effects do not need to switch; the fixed-duration combination of acalabrutinib and venetoclax is also a newer first-line option ^[24].

For physicians

Shared safety points

Tumor lysis syndrome with venetoclax (all fixed-duration regimens): low tumor burden (all lymph nodes less than 5 cm and absolute lymphocyte count less than 25×10⁹/L): oral hydration 1.5 to 2 L + allopurinol, outpatient; medium (any lymph node 5 to less than 10 cm, or lymphocytes ≥25×10⁹/L): in addition, consider IV hydration, and if CrCl is less than 80 mL/min, consider hospitalization for the 20 mg and 50 mg doses; high (any lymph node ≥10 cm, or lymphocytes ≥25×10⁹/L with a lymph node ≥5 cm): IV hydration at 150 to 200 mL per hour, allopurinol (and rasburicase if uric acid is elevated) and hospitalization for the 20 mg and 50 mg doses. Start allopurinol 2 to 3 days before venetoclax; laboratory tests before the dose and 6 to 8 and 24 hours after the 20 mg and 50 mg doses ^[39].
Venetoclax and CYP3A inhibitors: posaconazole and other strong CYP3A inhibitors are contraindicated at initiation and during the ramp-up; thereafter, reduce the venetoclax dose to 70 mg (posaconazole) or 100 mg (other strong inhibitors), and by at least 50% with a moderate inhibitor ^[39].
Rituximab and obinutuzumab: hepatitis B screening before starting is mandatory (boxed warning: HBV reactivation and PML) ^{[59, 61]}.

For physiciansRegimens and dosing in CLL: continuous BTK inhibitor therapy, 4 regimens · Regimens and dosing in CLL: fixed-duration targeted therapy, 4 regimens

For physicians

Regimens and dosing in CLL: continuous BTK inhibitor therapy

Ibrutinib monotherapy (A041202, RESONATE-2)

CLL/SLL requiring treatment (iwCLL), including with TP53 abnormalities; continuous treatmentCycle length: Continuous

CLL/SLL requiring treatment (iwCLL), including with TP53 abnormalities; continuous treatment

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Ibrutinib	420 mg once daily	Oral, with a glass of water	Continuous

Cycle length: Continuous
Duration: Until progression or unacceptable toxicity

Atrial fibrillation, heart failure and sudden death, hypertension (monitor blood pressure regularly) and bleeding (caution with anticoagulants and antiplatelet agents; withhold for 3 to 7 days before and after surgery depending on bleeding risk) are important adverse effects; monthly blood counts and liver function monitoring ^{[23, 56]}.

Evidence: Alliance A041202 (2024), RESONATE-2 (2015)

Ibrutinib + rituximab (E1912)

Untreated CLL in patients aged 70 years or younger without 17p deletion, with CrCl above 40 mL/minCycle length: Every 28 days

Untreated CLL in patients aged 70 years or younger without 17p deletion, with CrCl above 40 mL/min

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Ibrutinib	420 mg once daily	Oral	Continuous from cycle 1
From cycle 2 (6 cycles)
Rituximab	50 mg/m² on Day 1 and 325 mg/m² on Day 2 of cycle 2; then 500 mg/m²	IV	Day 1 of cycles 3–7

Cycle length: Every 28 days
Duration: Rituximab 6 cycles; ibrutinib until progression or toxicity

Rituximab boxed warning: fatal infusion-related reactions, HBV reactivation and PML ^{[37, 56, 61]}.

Evidence: E1912 (2022), E1912 (2019)

Zanubrutinib (ALPINE)

Untreated or relapsed/refractory CLL/SLL; continuous treatmentCycle length: Continuous

Untreated or relapsed/refractory CLL/SLL; continuous treatment

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Zanubrutinib	160 mg twice daily or 320 mg once daily	Oral, with or without food	Continuous

Cycle length: Continuous
Duration: Until progression or unacceptable toxicity

Severe hepatic impairment (Child-Pugh C): 80 mg twice daily. With CYP3A inhibitors or inducers, adjust the dose according to the label ^{[12, 57]}.

Evidence: ALPINE (2023)

Acalabrutinib ± obinutuzumab

CLL/SLL in adults; continuous treatmentCycle length: Every 28 days

CLL/SLL in adults; continuous treatment

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Acalabrutinib	100 mg every 12 hours	Oral, with or without food	Continuous from cycle 1
If combined (untreated CLL)
Obinutuzumab	Obinutuzumab CLL schedule: first obinutuzumab cycle 100 mg on Day 1, 900 mg on Day 2 and 1000 mg on Days 8 and 15; then 1000 mg on Day 1	IV	From cycle 2, 6 cycles in total

Cycle length: Every 28 days
Duration: Acalabrutinib until progression or toxicity; obinutuzumab 6 cycles

On days when both drugs are given, administer acalabrutinib before obinutuzumab. Avoid concomitant strong CYP3A inhibitors, and do not use in severe hepatic impairment ^{[58, 59]}.

For physicians

Regimens and dosing in CLL: fixed-duration targeted therapy

Fixed-duration ibrutinib + venetoclax (GLOW; FLAIR with MRD-guided duration)

Untreated CLL; in GLOW, patients aged 65 years and older, or aged 18 to 64 years with a CIRS score above 6 or CrCl below 70 mL/min, ECOG ≤2

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Cycles 1–3 (lead-in)
Ibrutinib	420 mg once daily	Oral	Continuous
Cycles 4–15
Ibrutinib	420 mg once daily	Oral	Continuous
Venetoclax	5-week ramp-up to 400 mg once daily	Oral, with food	Continuous

Cycle length: Ibrutinib daily and continuously from cycle 1; venetoclax daily and continuously from cycle 4 (28-day cycles)
Duration: 15 cycles in GLOW; in FLAIR, treatment duration was determined by MRD

The doses are from the GLOW abstract ^[26]; in FLAIR, treatment duration was guided by MRD monitoring, and the details are in the full text of the paper ^[30]. This combination is not in the US FDA label.

Evidence: FLAIR (2025), GLOW (2023), GLOW (2023)

Fixed-duration venetoclax + obinutuzumab (CLL14)

Untreated CLL (in CLL14, patients with comorbidities)Cycle length: Every 28 days

Untreated CLL (in CLL14, patients with comorbidities)

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Cycle 1
Obinutuzumab	100 mg (Day 1), 900 mg (Day 2), 1000 mg (Days 8 and 15)	IV	Days 1, 2, 8 and 15
Cycles 2–6
Obinutuzumab	1000 mg	IV	Day 1
From Day 22 of cycle 1: 5-week ramp-up
Venetoclax	20, 50, 100, 200 and then 400 mg (one step each week)	Oral, with food	Until Day 28 of cycle 2
Cycles 3–12
Venetoclax	400 mg once daily	Oral, with food	Continuous

Cycle length: Every 28 days
Duration: 12 cycles (obinutuzumab 6 cycles)

Give premedication before each obinutuzumab infusion; boxed warning: hepatitis B reactivation (screening mandatory) and PML ^{[14, 31, 59]}.

Evidence: CLL14 (2024), CLL14 (2019)

Fixed-duration acalabrutinib + venetoclax (AMPLIFY)

Untreated CLL/SLL in adultsCycle length: Every 28 days

Untreated CLL/SLL in adults

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
Cycles 1–14
Acalabrutinib	100 mg every 12 hours	Oral	Continuous
From cycle 3: 5-week ramp-up
Venetoclax	20, 50, 100, 200 and then 400 mg once daily (one step each week)	Oral, with food	Weeks 1–5
Until the end of cycle 14
Venetoclax	400 mg once daily	Oral	Continuous

Cycle length: Every 28 days
Duration: 14 cycles (about 13 months)

For grade 3 or 4 neutropenia or thrombocytopenia, interrupt the drug(s); on recurrence, resume venetoclax at one dose level lower ^{[24, 39, 58]}.

Evidence: AMPLIFY (2025)

Venetoclax + rituximab (MURANO)

Relapsed or refractory CLLCycle length: Every 28 days

Relapsed or refractory CLL

Before prescribing: Shared safety points

Drug	Dose	Route	Schedule
5-week ramp-up
Venetoclax	20, 50, 100, 200 and then 400 mg (one step each week)	Oral, with food	Weeks 1–5
After 7 days of venetoclax 400 mg (6 cycles)
Rituximab	375 mg/m² in cycle 1; 500 mg/m² in cycles 2–6	IV	Day 1 of each cycle
Continuation
Venetoclax	400 mg once daily	Oral	24 months from Day 1 of rituximab cycle 1

Cycle length: Every 28 days
Duration: Venetoclax 24 months (after the ramp-up); rituximab 6 cycles

TLS prophylaxis, CYP3A interactions and hepatitis B screening as in “Shared safety points” above ^{[27, 39, 61]}.

Evidence: MURANO (2025), MURANO (2018)

Before prescribing, check each dose against the drug label, laboratory tests and the patient’s condition.

Allogeneic hematopoietic stem cell transplantation

In an allogeneic transplant, after conditioning chemotherapy (and sometimes radiotherapy), stem cells from a matched donor (a brother or sister, an unrelated donor or a half-matched donor) are infused. The main advantage is the immune “graft-versus-leukemia” effect. In AML, transplantation in first remission is recommended for patients with adverse risk and for many patients with intermediate risk ^[2]. It also plays an important role in high-risk ALL or ALL with an inadequate response (MRD-positive) and in advanced-phase CML ^{[5, 13]}. Important complications include graft-versus-host disease (GVHD) and infections, so the decision should be made at a specialized transplant center.

What should I do after diagnosis?

For patients

Patient guide: life during leukemia treatment1 / 5

1. What happens after diagnosis?

In acute leukemias, treatment usually starts within a few days, often with a stay in the hospital. Before it starts, genetic tests are done so that the best treatment can be chosen for your specific type of disease ^[2]. In chronic leukemias, there is usually more time to make decisions ^[3].

2. Preventing infection

Chemotherapy and the disease itself lower your white blood cells; for example, in the VIALE-A trial, infections occurred in 85% of patients in the venetoclax group ^[7]. Washing your hands often, staying away from sick people and crowds, keeping your mouth clean and eating only thoroughly cooked food are important. Never ignore a fever, and tell your care team right away.

3. Living with oral medication for CML

TKI medicines must be taken every day, exactly as prescribed. A PCR blood test is done at regular intervals to measure how well the treatment is working ^[5]. Talk to your doctor before taking any new medicine or herbal supplement, because drug interactions are common. Plan any pregnancy with your doctor in advance: men can usually continue their medicine, but in women the drug is stopped as soon as a pregnancy test is positive, and dasatinib must not be taken at any time during pregnancy ^[5].

4. What does “active surveillance” mean in CLL?

If your CLL is not causing symptoms, your doctor may recommend only regular tests and checkups. This does not mean you are being neglected; starting treatment early at this stage brings no benefit, and treatment will begin at the right time ^[3].

5. How is CAR T-cell therapy given?

First, your T lymphocytes are collected with a machine similar to a dialysis machine (leukapheresis) and engineered in the laboratory to recognize cancer cells. After a course of mild chemotherapy, the cells are given back to you. The main side effects are “cytokine release syndrome” (fever, low blood pressure) and neurologic side effects; in the ELIANA trial, cytokine release syndrome occurred in 77% of patients and was mostly manageable ^[11].

Treatment at a specialized center and clinical trials

Acute leukemias should be treated at centers that can provide molecular testing, supportive care (blood transfusions, infection control) and bone marrow transplantation. Taking part in a clinical trial is also a valuable option for many patients ^[2].

Frequently asked questions

Can leukemia be cured?

It depends on the type. Most children with ALL are cured ^[4]. Many patients with CML live for many years with good disease control on oral medication ^{[5, 21]}. In AML, the chance of cure depends on age and genetic features, and transplantation plays a key role in high-risk patients ^[2]. In low- to intermediate-risk acute promyelocytic leukemia (APL), the combination of tretinoin and arsenic trioxide gives very good results, with event-free survival above 97% at 50 months ^[38]. CLL is usually a chronic disease that can be controlled for years ^[3].

Is leukemia inherited?

Most cases of leukemia are not inherited and are not passed on to children. However, a small percentage of patients have an inherited genetic predisposition, to which the new World Health Organization classification gives special attention; this matters especially when choosing a related donor for a transplant ^[1].

What is the difference between leukemia and lymphoma?

Leukemia mainly affects the bone marrow and blood, whereas lymphoma usually appears as lumps in the lymph nodes. The boundary between the two sometimes overlaps; for example, CLL and small lymphocytic lymphoma (SLL) are one disease with two presentations ^[3]. For more information, see the blood cancers page.

What is MRD and why does it matter?

Measurable residual disease (MRD) refers to very small numbers of cancer cells that cannot be seen under a microscope but can be detected with flow cytometry or molecular methods. MRD is one of the strongest predictors of relapse and is used in decisions about continuing treatment or proceeding to transplantation ^{[2, 13, 33]}.

Can a person with CML ever stop taking the medication?

Yes. In some patients with a deep and stable molecular response, stopping the drug under close monitoring is possible, and about 40% to 50% of them stay off treatment in the long term ^{[5, 6]}. This decision should be made only with a hematologist’s advice and where regular PCR monitoring is available ^[5].

For physicians

Recent evidence and advances (for physicians)

For physicians4 key findings · AML

For physicians

97.3%

50-month event-free survival with tretinoin + arsenic trioxide in low- to intermediate-risk APL

Versus 80% with tretinoin + chemotherapy; final analysis of APL0406 ^[38]

73%

Three-year overall survival with a pediatric regimen in adolescents and young adults aged 17 to 39 years with ALL

Treatment-related mortality 3% (CALGB 10403) ^[35]

50%

CML patients free of molecular relapse 24 months after stopping a TKI

61% at 6 months; after at least 3 years of treatment and at least 1 year of deep molecular response (EURO-SKI) ^[6]

0.47

Hazard ratio for death with ibrutinib + rituximab vs FCR in untreated CLL (age 70 years or younger)

E1912 ^[37]

For physicians

AML

Classification and risk: ELN 2022 places FLT3-ITD in the intermediate-risk group regardless of allelic ratio and adds myelodysplasia-related mutations to the adverse-risk group; the response criteria have also been revised ^[2]. For less-intensive therapy, the ELN 2024 classification groups patients on the basis of TP53, FLT3-ITD, NRAS and KRAS mutations: for example, an NPM1 or IDH2 mutation without FLT3-ITD and without NRAS, KRAS and TP53 mutations falls into the favorable group, and a TP53 mutation into the adverse group ^[32].
VIALE-A: Venetoclax + azacitidine increased the composite complete remission rate from 28.3% to 66.4% ^[7]. In the final analysis, with a median follow-up of 43 months, median survival was 14.7 vs 9.6 months with azacitidine alone (HR for death 0.58), and two-year survival was 37.5% vs 16.9% ^[28].
FLT3: In RATIFY, midostaurin reduced the risk of death (HR 0.78) ^[15], and in QuANTUM-First, quizartinib increased median survival in FLT3-ITD-positive patients from 15.1 to 31.9 months versus placebo ^[8]. Post-transplant sorafenib maintenance in FLT3-ITD AML reduced the five-year incidence of relapse from 36.3% to 15.0%, increased five-year overall survival from 55.9% to 72.0% (HR 0.55) and increased five-year leukemia-free survival to 70.0% vs 49.0% ^[29].
APL (APL0406): In patients with a WBC count of 10,000 per microliter or less, the final analysis showed event-free survival at 50 months of 97.3% vs 80% and overall survival of 99.2% vs 92.6%, in favor of tretinoin and arsenic over tretinoin and chemotherapy ^[38].
Menin inhibitors: In the phase 2 AUGMENT-101 study in KMT2Ar, the CR+CRh rate was 22.8% and the overall response rate 63.2% ^[9]. In relapsed NPM1-mutated AML, revumenib achieved a CR/CRh rate of 23.4% and an overall response rate of 46.9% ^[16], and ziftomenib achieved a CR/CRh rate of 22% without clinically significant QTc prolongation ^[17].

ALL

Adolescents and young adults (CALGB 10403): In patients aged 17 to 39 years, the regimen using pediatric protocol doses and schedule was associated with 3% treatment-related mortality and yielded three-year overall survival of 73% and median event-free survival of 78 months (more than double that of historical controls) ^[35].
First-line immunotherapy (E1910): Immunotherapy has moved from the salvage setting to the first line; in adults aged 30 to 70 years in MRD-negative remission, adding blinatumomab to consolidation chemotherapy increased three-year survival from 68% to 85% (HR 0.41) ^[10].
Ph+ ALL: In GRAAPH-2005, imatinib with reduced-intensity chemotherapy, compared with imatinib + hyper-CVAD, resulted in fewer induction deaths and more complete remissions (98% vs 91%), with similar major molecular response; allogeneic transplantation in first remission was associated with better overall survival ^[34]. In PhALLCON, ponatinib with reduced-intensity chemotherapy achieved an MRD-negative complete remission rate of 34.4% vs 16.7% with imatinib ^[19]. In D-ALBA (phase 2), dasatinib followed by blinatumomab produced complete remission in 98% of patients, and overall survival at a median follow-up of 18 months was 95% ^[36]. Reduced-chemotherapy approaches based on imatinib ^[34] or ponatinib ^[19] and chemotherapy-free approaches with dasatinib ^[36] or ponatinib ^[63] combined with blinatumomab are expanding.
Relapsed B-ALL: In INO-VATE, the complete remission rate with inotuzumab ozogamicin was 80.7% vs 29.4% with standard chemotherapy ^[18].

CML

ELN 2025: The response terminology has changed to “favorable, warning and unfavorable,” dose reduction is preferred over switching drugs for managing side effects, and asciminib is a first-line option alongside imatinib and second-generation TKIs ^[5].
ASC4FIRST: The major molecular response rate at week 96 with first-line asciminib was 74.1% vs 52.0% with investigator-selected TKIs ^[22].
Treatment-free remission (EURO-SKI): In patients with at least 3 years of treatment and at least 1 year of deep molecular response, 61% remained free of molecular relapse at 6 months and 50% at 24 months ^[6].

CLL

Continuous BTK inhibitor therapy: In patients aged 65 years and older, 48-month progression-free survival with ibrutinib was 76% vs 47% with bendamustine–rituximab ^[23]; in patients aged 70 years and younger, ibrutinib–rituximab improved overall survival compared with FCR (HR 0.47) ^[37]; in ALPINE (relapsed or refractory), 24-month progression-free survival with zanubrutinib was 78.4% vs 65.9% with ibrutinib ^[12].
Fixed-duration therapy: In FLAIR, five-year progression-free survival with ibrutinib + venetoclax (MRD-guided duration) was 93.9% vs 79.0% with ibrutinib alone and 58.1% with FCR ^[30]; in GLOW, 42-month progression-free survival was 74.6% vs 24.8% with chlorambucil–obinutuzumab ^[26]; in CLL14, median progression-free survival with venetoclax + obinutuzumab was 76.2 vs 36.4 months ^[31]; in MURANO (relapsed or refractory), median progression-free survival with venetoclax + rituximab was 54.7 vs 17.0 months with bendamustine–rituximab, and seven-year overall survival was 69.6% vs 51.0% ^[27].
Summary: Fixed-duration targeted therapy (ibrutinib + venetoclax, including with MRD-guided duration, or venetoclax + an anti-CD20 antibody) and, for continuous therapy, second-generation BTKis with fewer cardiac adverse effects have changed standard practice ^{[12, 24, 30, 31]}.

Zanubrutinib or Ibrutinib in Relapsed or Refractory Chronic Lymphocytic Leukemia

N Engl J Med · 2023

Summary

Clinical trial · Phase 3

Azacitidine and Venetoclax in Previously Untreated Acute Myeloid Leukemia

N Engl J Med · 2020

Summary

Blood · 2018

Summary

References

63 references

Show all references, 32 with a study cardClose the reference list

Numbers in the text, such as ^[1], refer to these sources. Sources with a “Study card” button are summarised in the site’s evidence library.

1
The 5th edition of the World Health Organization Classification of Haematolymphoid Tumours: Myeloid and Histiocytic/Dendritic NeoplasmsKhoury JD, Solary E, Abla O, Akkari Y, Alaggio R, Apperley JF, et al · Leukemia · 2022;36:1703-1719
Study card DOI PubMed Back to text: 1 2 3 4 5 6 7
2
Diagnosis and management of AML in adults: 2022 recommendations from an international expert panel on behalf of the ELNDöhner H, Wei AH, Appelbaum FR, Craddock C, DiNardo CD, Dombret H, et al · Blood · 2022;140:1345-1377
Study card DOI PubMed Back to text: 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20 21
3
iwCLL guidelines for diagnosis, indications for treatment, response assessment, and supportive management of CLLHallek M, Cheson BD, Catovsky D, Caligaris-Cappio F, Dighiero G, Döhner H, et al · Blood · 2018;131:2745-2760
Study card DOI PubMed Back to text: 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16
4
Improved Survival for Children and Adolescents With Acute Lymphoblastic Leukemia Between 1990 and 2005: A Report From the Children's Oncology GroupHunger SP, Lu X, Devidas M, Camitta BM, Gaynon PS, Winick NJ, et al · J Clin Oncol · 2012;30:1663-1669
Study card DOI PubMed Back to text: 1 2 3 4
5
2025 European LeukemiaNet recommendations for the management of chronic myeloid leukemiaApperley JF, Milojkovic D, Cross NCP, Hjorth-Hansen H, Hochhaus A, Kantarjian H, et al · Leukemia · 2025;39:1797-1813
Study card DOI PubMed Back to text: 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18
6
Discontinuation of tyrosine kinase inhibitor therapy in chronic myeloid leukaemia (EURO-SKI): a prespecified interim analysis of a prospective, multicentre, non-randomised, trialSaussele S, Richter J, Guilhot J, Gruber FX, Hjorth-Hansen H, Almeida A, et al · Lancet Oncol · 2018;19:747-757
Study card DOI PubMed Back to text: 1 2 3 4
7
Azacitidine and Venetoclax in Previously Untreated Acute Myeloid LeukemiaDiNardo CD, Jonas BA, Pullarkat V, Thirman MJ, Garcia JS, Wei AH, et al · N Engl J Med · 2020;383:617-629
Study card DOI PubMed Back to text: 1 2 3 4 5 6
8
Quizartinib plus chemotherapy in newly diagnosed patients with FLT3-internal-tandem-duplication-positive acute myeloid leukaemia (QuANTUM-First): a randomised, double-blind, placebo-controlled, phase 3 trialErba HP, Montesinos P, Kim HJ, Patkowska E, Vrhovac R, Žák P, et al · Lancet · 2023;401:1571-1583
Study card DOI PubMed Back to text: 1 2 3 4 5
9
Menin Inhibition With Revumenib for KMT2A-Rearranged Relapsed or Refractory Acute Leukemia (AUGMENT-101)Issa GC, Aldoss I, Thirman MJ, DiPersio J, Arellano M, Blachly JS, et al · J Clin Oncol · 2025;43:75-84
Study card DOI PubMed Back to text: 1 2 3 4 5
10
Blinatumomab for MRD-Negative Acute Lymphoblastic Leukemia in AdultsLitzow MR, Sun Z, Mattison RJ, Paietta EM, Roberts KG, Zhang Y, et al · N Engl J Med · 2024;391:320-333
Study card DOI PubMed Back to text: 1 2 3 4
11
Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic LeukemiaMaude SL, Laetsch TW, Buechner J, Rives S, Boyer M, Bittencourt H, et al · N Engl J Med · 2018;378:439-448
Study card DOI PubMed Back to text: 1 2 3 4
12
Zanubrutinib or Ibrutinib in Relapsed or Refractory Chronic Lymphocytic LeukemiaBrown JR, Eichhorst B, Hillmen P, Jurczak W, Kaźmierczak M, Lamanna N, et al · N Engl J Med · 2023;388:319-332
Study card DOI PubMed Back to text: 1 2 3 4
13
Acute Lymphoblastic Leukemia in ChildrenHunger SP, Mullighan CG · N Engl J Med · 2015;373:1541-1552
Study card DOI PubMed Back to text: 1 2 3 4 5 6 7 8
14
Venetoclax and Obinutuzumab in Patients with CLL and Coexisting ConditionsFischer K, Al-Sawaf O, Bahlo J, Fink AM, Tandon M, Dixon M, et al · N Engl J Med · 2019;380:2225-2236
Study card DOI PubMed Back to text: 1 2
15
Midostaurin plus Chemotherapy for Acute Myeloid Leukemia with a FLT3 MutationStone RM, Mandrekar SJ, Sanford BL, Laumann K, Geyer S, Bloomfield CD, et al · N Engl J Med · 2017;377:454-464
Study card DOI PubMed Back to text: 1 2 3 4
16
Menin inhibition with revumenib for NPM1-mutated relapsed or refractory acute myeloid leukemia: the AUGMENT-101 studyArellano ML, Thirman MJ, DiPersio JF, Heiblig M, Stein EM, Schuh AC, et al · Blood · 2025;146:1065-1077
Study card DOI PubMed Back to text: 1 2 3 4 5
17
Ziftomenib in Relapsed or Refractory NPM1-Mutated AMLWang ES, Montesinos P, Foran J, Erba H, Rodríguez-Arbolí E, Fedorov K, et al · J Clin Oncol · 2025;43:3381-3390
Study card DOI PubMed Back to text: 1 2 3 4
18
Inotuzumab Ozogamicin versus Standard Therapy for Acute Lymphoblastic LeukemiaKantarjian HM, DeAngelo DJ, Stelljes M, Martinelli G, Liedtke M, Stock W, et al · N Engl J Med · 2016;375:740-753
Study card DOI PubMed Back to text: 1 2 3
19
Ponatinib vs Imatinib in Frontline Philadelphia Chromosome–Positive Acute Lymphoblastic LeukemiaJabbour E, Kantarjian HM, Aldoss I, Montesinos P, Leonard JT, Gómez-Almaguer D, et al · JAMA · 2024;331:1814-1823
Study card DOI PubMed Back to text: 1 2 3 4
20
KTE-X19 for relapsed or refractory adult B-cell acute lymphoblastic leukaemia: phase 2 results of the single-arm, open-label, multicentre ZUMA-3 studyShah BD, Ghobadi A, Oluwole OO, Logan AC, Boissel N, Cassaday RD, et al · Lancet · 2021;398:491-502
Study card DOI PubMed Back to text: 1 2
21
Long-Term Outcomes of Imatinib Treatment for Chronic Myeloid LeukemiaHochhaus A, Larson RA, Guilhot F, Radich JP, Branford S, Hughes TP, et al · N Engl J Med · 2017;376:917-927
Study card DOI PubMed Back to text: 1 2 3 4 5 6
22
Asciminib demonstrates superior efficacy and safety in newly diagnosed chronic myeloid leukemia in the ASC4FIRST trialCortes JE, Hughes TP, Wang J, Kim DW, Kim DDH, Mayer J, et al · Blood · 2026;147:1433-1446
Study card DOI PubMed Back to text: 1 2 3
23
Follow-up from the A041202 study shows continued efficacy of ibrutinib regimens for older adults with CLLWoyach JA, Perez Burbano G, Ruppert AS, Miller C, Heerema NA, Zhao W, et al · Blood · 2024;143:1616-1627
Study card DOI PubMed Back to text: 1 2 3 4
24
EHA Guidelines on management of chronic lymphocytic leukemia and Richter transformationEichhorst B, Ghia P, Bosch F, et al · HemaSphere · 2026;10:e70403
Study card DOI PubMed Back to text: 1 2 3 4
25
Retinoic acid and arsenic trioxide for acute promyelocytic leukemiaLo-Coco F, Avvisati G, Vignetti M, Thiede C, Orlando SM, Iacobelli S, et al · N Engl J Med · 2013;369:111-121
DOI PubMed Back to text: 1 2
26
Fixed-duration ibrutinib-venetoclax versus chlorambucil-obinutuzumab in previously untreated chronic lymphocytic leukaemia (GLOW): 4-year follow-up from a multicentre, open-label, randomised, phase 3 trialNiemann CU, Munir T, Moreno C, Owen C, Follows GA, Benjamini O, et al · Lancet Oncol · 2023;24:1423-1433
Study card DOI PubMed Back to text: 1 2 3
27
The MURANO study: final analysis and retreatment/crossover substudy results of VenR for patients with relapsed/refractory CLLKater AP, Harrup R, Kipps TJ, Eichhorst B, Owen CJ, Assouline S, et al · Blood · 2025;145:2733-2745
Study card DOI PubMed Back to text: 1 2 3
28
Long-term follow-up of VIALE-A: Venetoclax and azacitidine in chemotherapy-ineligible untreated acute myeloid leukemiaPratz KW, Jonas BA, Pullarkat V, Thirman MJ, Garcia JS, Döhner H, et al · Am J Hematol · 2024;99:615-624
Study card DOI PubMed Back to text: 1 2 3
29
Sorafenib maintenance after allogeneic haemopoietic stem-cell transplantation in patients with FLT3-ITD acute myeloid leukaemia: long-term follow-up of an open-label, multicentre, randomised, phase 3 trialXuan L, Wang Y, Yang K, Shao R, Huang F, Fan Z, et al · Lancet Haematol · 2023;10:e600-e611
Study card DOI PubMed Back to text: 1 2 3 4
30
Measurable Residual Disease-Guided Therapy for Chronic Lymphocytic LeukemiaMunir T, Girvan S, Cairns DA, Bloor A, Allsup D, Varghese AM, et al · N Engl J Med · 2025;393:1177-1190
Study card DOI PubMed Back to text: 1 2 3 4 5
31
Venetoclax-obinutuzumab for previously untreated chronic lymphocytic leukemia: 6-year results of the randomized phase 3 CLL14 studyAl-Sawaf O, Robrecht S, Zhang C, Olivieri S, Chang YM, Fink AM, et al · Blood · 2024;144:1924-1935
Study card DOI PubMed Back to text: 1 2 3 4
32
Genetic risk classification for adults with AML receiving less-intensive therapies: the 2024 ELN recommendationsDöhner H, DiNardo CD, Appelbaum FR, Craddock C, Dombret H, Ebert BL, et al · Blood · 2024;144:2169-2173
DOI PubMed Back to text: 1 2
33
Diagnosis, prognostic factors, and assessment of ALL in adults: 2024 ELN recommendations from a European expert panelGökbuget N, Boissel N, Chiaretti S, Dombret H, Doubek M, Fielding A, et al · Blood · 2024;143:1891-1902
Study card DOI PubMed Back to text: 1 2
34
Randomized study of reduced-intensity chemotherapy combined with imatinib in adults with Ph-positive acute lymphoblastic leukemiaChalandon Y, Thomas X, Hayette S, Cayuela JM, Abbal C, Huguet F, et al · Blood · 2015;125:3711-3719
DOI PubMed Back to text: 1 2 3 4 5
35
A pediatric regimen for older adolescents and young adults with acute lymphoblastic leukemia: results of CALGB 10403Stock W, Luger SM, Advani AS, Yin J, Harvey RC, Mullighan CG, et al · Blood · 2019;133:1548-1559
DOI PubMed Back to text: 1 2 3 4
36
Dasatinib-Blinatumomab for Ph-Positive Acute Lymphoblastic Leukemia in AdultsFoà R, Bassan R, Vitale A, Elia L, Piciocchi A, Puzzolo MC, et al · N Engl J Med · 2020;383:1613-1623
DOI PubMed Back to text: 1 2 3 4 5
37
Long-term outcomes for ibrutinib-rituximab and chemoimmunotherapy in CLL: updated results of the E1912 trialShanafelt TD, Wang XV, Hanson CA, Paietta EM, O'Brien S, Barrientos J, et al · Blood · 2022;140:112-120
Study card DOI PubMed Back to text: 1 2 3 4
38
Improved Outcomes With Retinoic Acid and Arsenic Trioxide Compared With Retinoic Acid and Chemotherapy in Non-High-Risk Acute Promyelocytic Leukemia: Final Results of the Randomized Italian-German APL0406 TrialPlatzbecker U, Avvisati G, Cicconi L, Thiede C, Paoloni F, Vignetti M, et al · J Clin Oncol · 2017;35:605-612
DOI PubMed Back to text: 1 2 3 4 5
39
VENCLEXTA (venetoclax) tablets — Prescribing InformationAbbVie Inc · FDA label · 2026
Source Back to text: 1 2 3 4 5 6
40
RYDAPT (midostaurin) capsules — Prescribing InformationNovartis Pharmaceuticals Corporation · FDA label · 2026
Source Back to text: 1 2
41
VANFLYTA (quizartinib) tablets — Prescribing InformationDaiichi Sankyo, Inc · FDA label · 2026
Source Back to text: 1 2 3
42
TRISENOX (arsenic trioxide) injection — Prescribing InformationCephalon, LLC · FDA label · 2026
Source Back to text: 1 2 3
43
REVUFORJ (revumenib) tablets — Prescribing InformationSyndax Pharmaceuticals, Inc · FDA label · 2026
Source Back to text: 1 2 3
44
KOMZIFTI (ziftomenib) capsules — Prescribing InformationKura Oncology, Inc · FDA label · 2025
Source Back to text: 1 2
45
Sorafenib maintenance in patients with FLT3-ITD acute myeloid leukaemia undergoing allogeneic haematopoietic stem-cell transplantation: an open-label, multicentre, randomised phase 3 trialXuan L, Wang Y, Huang F, et al · Lancet Oncol · 2020;21:1201-1212
DOI PubMed Back to text
46
ICLUSIG (ponatinib) tablets — Prescribing InformationTakeda Pharmaceuticals America, Inc · FDA label · 2026
Source Back to text: 1 2
47
GLEEVEC (imatinib mesylate) tablets — Prescribing InformationNovartis Pharmaceuticals Corporation · FDA label · 2026
Source Back to text: 1 2
48
SPRYCEL (dasatinib) tablets — Prescribing InformationE.R. Squibb & Sons, L.L.C · FDA label · 2026
Source Back to text: 1 2 3
49
BLINCYTO (blinatumomab) for injection — Prescribing InformationAmgen Inc · FDA label · 2026
Source Back to text: 1 2
50
BESPONSA (inotuzumab ozogamicin) for injection — Prescribing InformationWyeth Pharmaceuticals LLC (Pfizer) · FDA label · 2026
Source Back to text
51
KYMRIAH (tisagenlecleucel) suspension for intravenous infusion — Prescribing InformationNovartis Pharmaceuticals Corporation · FDA label · 2025
Source Back to text: 1 2 3
52
TECARTUS (brexucabtagene autoleucel) suspension for intravenous infusion — Prescribing InformationKite Pharma, Inc · FDA label · 2026
Source Back to text: 1 2 3
53
TASIGNA (nilotinib) capsules — Prescribing InformationNovartis Pharmaceuticals Corporation · FDA label · 2025
Source Back to text
54
BOSULIF (bosutinib) tablets and capsules — Prescribing InformationPfizer Laboratories · FDA label · 2026
Source Back to text
55
SCEMBLIX (asciminib) tablets — Prescribing InformationNovartis Pharmaceuticals Corporation · FDA label · 2026
Source Back to text
56
IMBRUVICA (ibrutinib) capsules, tablets and oral suspension — Prescribing InformationPharmacyclics LLC / Janssen Biotech · FDA label · 2025
Source Back to text: 1 2
57
BRUKINSA (zanubrutinib) capsules and tablets — Prescribing InformationBeOne Medicines · FDA label · 2026
Source Back to text
58
CALQUENCE (acalabrutinib) — Prescribing InformationAstraZeneca Pharmaceuticals LP · FDA label · 2026
Source Back to text: 1 2
59
GAZYVA (obinutuzumab) injection — Prescribing InformationGenentech, Inc · FDA label · 2026
Source Back to text: 1 2 3
60
D-ALBA: Frontline Sequential Dasatinib and Blinatumomab in Adult Philadelphia Positive Acute Lymphoblastic Leukemia (NCT02744768)GIMEMA · ClinicalTrials.gov · 2016
Source Back to text
61
RITUXAN (rituximab) injection — Prescribing InformationGenentech, Inc · FDA label · 2025
Source Back to text: 1 2 3
62
Sorafenib Maintenance for Prophylaxis of Leukemia Relapse in Allo-HSCT Recipients With FLT3-ITD Positive AML (NCT02474290)Nanfang Hospital, Southern Medical University · ClinicalTrials.gov · 2015
Source Back to text
63
Ponatinib and blinatumomab for Philadelphia chromosome-positive acute lymphoblastic leukaemia: a US, single-centre, single-arm, phase 2 trialJabbour E, Short NJ, Jain N, Huang X, Montalban-Bravo G, Banerjee P, et al · Lancet Haematol · 2023;10:e24-e34
DOI PubMed Back to text

This page is based on reputable scientific sources and was last updated on 21 October 2026. The content of this website is for educational and informational purposes only and does not replace consultation, diagnosis or treatment by a specialist physician. Consult your treating physician about any treatment decision.

Key points

What is leukemia?

Signs and symptoms

When to see a doctor urgently

Risk factors

Diagnosis and classification

Steps in the diagnosis and classification of leukemia

Complete blood count (CBC) and peripheral blood smear

Bone marrow aspiration and biopsy

Flow cytometry (immunophenotyping)

Cytogenetics and molecular testing

Additional assessment before treatment

Acute myeloid leukemia (AML)

Genetic risk classification (ELN 2022)

Treatment options for AML

Intensive “7+3” chemotherapy

Venetoclax + azacitidine

Acute promyelocytic leukemia (APL): tretinoin + arsenic

FLT3 inhibitors

Allogeneic stem cell transplantation

Menin inhibitors

Regimens and dosing in newly diagnosed AML (induction, consolidation and maintenance)

7+3 (cytarabine + daunorubicin or idarubicin) followed by high-dose cytarabine

7+3 + midostaurin (RATIFY)

7+3 + quizartinib (QuANTUM-First)

Venetoclax + azacitidine (VIALE-A)

Sorafenib maintenance after allogeneic transplantation

Regimen and dosing in acute promyelocytic leukemia (APL)

Tretinoin (ATRA) + arsenic trioxide (APL0406)

Regimens and dosing in relapsed or refractory AML

Revumenib (AUGMENT-101)

Ziftomenib (KOMET-001)

Acute lymphoblastic leukemia (ALL)

New treatments for ALL

Philadelphia-positive ALL: adding a tyrosine kinase inhibitor

First-line blinatumomab

Inotuzumab ozogamicin

CAR T-cell therapy

Regimens and dosing in Philadelphia-negative ALL

Pediatric regimen in adolescents and young adults (CALGB 10403; identical to one arm of COG AALL0232)

Blinatumomab in consolidation (E1910)

Regimens and dosing in Philadelphia-positive ALL

Imatinib + reduced-intensity chemotherapy (GRAAPH-2005)

Ponatinib + reduced-intensity chemotherapy (PhALLCON)

Dasatinib followed by blinatumomab without chemotherapy (D-ALBA)

Regimens and dosing in relapsed or refractory B-ALL

Inotuzumab ozogamicin (INO-VATE)

Tisagenlecleucel (CAR T-cell therapy; ELIANA)

Brexucabtagene autoleucel (CAR T-cell therapy; ZUMA-3)

Chronic myeloid leukemia (CML)

Treatment regimens and dosing in CML

Imatinib

Dasatinib

Nilotinib

Bosutinib

Ponatinib

Asciminib

Chronic lymphocytic leukemia (CLL)

Targeted treatments for CLL

BTK inhibitors (continuous treatment)

Fixed-duration targeted treatments

Choosing between the two approaches

Regimens and dosing in CLL: continuous BTK inhibitor therapy

Ibrutinib monotherapy (A041202, RESONATE-2)

Ibrutinib + rituximab (E1912)

Zanubrutinib (ALPINE)

Acalabrutinib ± obinutuzumab

Regimens and dosing in CLL: fixed-duration targeted therapy

Fixed-duration ibrutinib + venetoclax (GLOW; FLAIR with MRD-guided duration)

Fixed-duration venetoclax + obinutuzumab (CLL14)

Fixed-duration acalabrutinib + venetoclax (AMPLIFY)

Venetoclax + rituximab (MURANO)

Allogeneic hematopoietic stem cell transplantation

What should I do after diagnosis?

1. What happens after diagnosis?

2. Preventing infection

3. Living with oral medication for CML

4. What does “active surveillance” mean in CLL?

5. How is CAR T-cell therapy given?