Key points
- Azacitidine is injected under the skin or into a vein for 7 days in a row, and the course is repeated every 28 days [1].
- In high-risk MDS, it helped patients live longer than usual treatments did [2].
- In older patients with AML, together with venetoclax it helped them live longer [3].
- It takes months to work; at least 4 to 6 cycles are needed before judging the response.
What is this drug and how does it work?
Azacitidine helps faulty blood-forming cells behave more normally again and reduces the number of cancer cells [1].
For physicians
A cytidine analogue; inhibits DNA methyltransferase and causes DNA hypomethylation, with direct cytotoxicity at higher doses [1].
Which cancers is it used for?
- Myelodysplastic syndromes (MDS) [1, 2]
- Acute myeloid leukaemia in patients who cannot have intensive chemotherapy, with venetoclax [3]
- Juvenile myelomonocytic leukaemia [1]
For physicians
| Disease | Use | Key evidence |
|---|---|---|
| Higher-risk MDS | First line [1] | AZA-001: median survival 24.5 vs 15 months with conventional care [2] |
| AML unfit for intensive treatment | With venetoclax | VIALE-A: median survival 14.7 vs 9.6 months [3] |
How it is taken and dosing
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7 days in a row, as an injection under the skin (abdomen, thigh or upper arm) or into a vein, repeated every 28 days. An anti-sickness medicine is given before each injection [1]. Rotate the injection sites under the skin.
For physicians
Standard dosing (FDA label)
Azacitidine
MDS and AMLCycle length: Every 28 days
MDS and AML
| Drug | Dose | Route | Schedule |
|---|---|---|---|
| Azacitidine | 75 mg/m² daily | Subcutaneous or IV | Days 1 to 7 |
The first-cycle dose for all patients is 75 mg/m²; if there is no benefit after 2 cycles and no toxicity other than nausea, it may be increased to 100 mg/m². Later doses are adjusted to blood counts and kidney function. The oral form (Onureg) is not interchangeable with the injection [1]. With venetoclax: the Aza/Ven regimen.
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.
Side effects
The most common side effects: nausea, vomiting, low blood cells and platelets, fever, diarrhoea or constipation, redness at the injection site and bruising [1].
For physicians
Common adverse reactions (above 30%, subcutaneous): nausea, anaemia, thrombocytopenia, vomiting, pyrexia, leucopenia, diarrhoea, injection-site erythema, constipation, neutropenia and ecchymosis [1].
Serious side effects
Serious side effects: very low white cells and platelets (especially in the first cycles), liver damage in people with severe liver disease, kidney damage and tumour lysis [1].
For physicians
When should I call my doctor?
- A temperature of 38 °C or higher
- Bleeding or a lot of bruising
- Passing less urine or swelling
Interactions, pregnancy and precautions
- Pregnancy: this drug may harm an unborn baby; use effective contraception during treatment and tell your doctor straight away if you become pregnant. Do not breastfeed during treatment; men should use effective contraception for 3 months after the last dose [1].
Frequently asked questions
Why haven't I improved after one or two cycles?
Azacitidine works slowly; most responses are seen after several cycles [1].
Related cancer types
Treatment regimens (physicians)
References
3 referencesShow all references, 1 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
- 2
- 3
This page is based on reputable scientific sources and was last updated on 27 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.
Printed from the website; the full and up-to-date version: https://drdabiri.com/en/drugs/azacitidine/