Lenalidomide in the Myelodysplastic Syndrome with Chromosome 5q Deletion
- Study type
- Clinical trial
- Study name
- MDS-003
- Design
- Single-arm study with intention-to-treat analysis
- Sample size
- 148 patients
- Source
- N Engl J Med · 2006
- Trial registration
- NCT00065156
In plain words
In some patients with myelodysplastic syndrome, part of chromosome 5 is missing, which causes severe anaemia that needs frequent blood transfusions. In this study, 148 patients were treated with the drug lenalidomide, and about two thirds of them (67%) no longer needed transfusions. In many patients, the bone marrow and chromosome abnormalities also improved. The most common problem with treatment was a fall in white blood cells (neutrophils) and platelets, which sometimes meant the drug had to be stopped or its dose adjusted.
Key results
Treatment arms and regimens
Lenalidomide 10 mg for 21 days every 4 weeks or daily. Haematological, bone marrow and cytogenetic changes were assessed after 24 weeks of treatment.
Source of the regimen details: Abstract of the paper
Doses are given for the information of specialists, as in this study’s protocol, and are not a prescription. Every drug must be prescribed by the treating physician for the individual patient.
Study population
Patients with myelodysplastic syndrome with a chromosome 5q31 deletion, characterised by severe and often treatment-resistant anaemia. Other inclusion criteria are not given in the abstract.
Endpoints
Whether lenalidomide can reduce transfusion needs and suppress the abnormal 5q31-deleted clone. Haematological, bone marrow and cytogenetic changes were assessed after 24 weeks of treatment by intention-to-treat analysis.
Safety and side effects
Moderate to severe neutropenia (in 55% of patients) and thrombocytopenia (in 44%) were the most common reasons for treatment interruption or lenalidomide dose adjustment.
Clinical significance and place in guidelines
This study showed that in patients with MDS with a 5q31 deletion, lenalidomide can reduce transfusion needs and reverse cytological and cytogenetic abnormalities. The current status of drug approvals and guideline recommendations should be checked separately, because the abstract does not address them.
Limitations
Based on the abstract, the study is single-arm with no control group. Cytogenetic assessment was possible in only 85 patients and bone marrow cytological assessment in 106 patients. Neutropenia and thrombocytopenia were common reasons for treatment interruption or dose adjustment.
This summary was prepared by the editorial team from the published paper; the original paper is the final reference. Read the original abstract on PubMed.