Very-Low-Dose Decitabine Is Effective in Treating Intermediate- or High-Risk Myelodysplastic Syndrome

Area of Science:

• Hematology
• Oncology
• Clinical Pharmacology

Background:

• Standard decitabine dosing for myelodysplastic syndrome (MDS) involves 20 mg/m2/day for 5 days, associated with significant toxicities and costs.
• Investigating alternative, potentially less toxic and more cost-effective dosing strategies for MDS is crucial.

Purpose of the Study:

• To evaluate the efficacy, safety, and cost-effectiveness of a very-low-dose decitabine schedule in patients with intermediate- or high-risk MDS.
• To identify patient subgroups who may benefit most from this reduced-dose regimen.

Main Methods:

• Retrospective, multicenter analysis of 31 newly diagnosed MDS patients treated with decitabine monotherapy.
• The regimen administered was 6 mg/m2/day intravenously for 7 consecutive days, repeated every 4 weeks.
• Efficacy was assessed by complete remission, partial remission, and hematological improvement; safety by severe infections and bleedings; and cost analysis was performed.

Main Results:

• An overall response rate (ORR) of 67.7% was observed, with 32.3% achieving complete remission and 25.8% partial remission after a median follow-up of 4 months.
• Rates of severe infections were 21.7% and severe bleedings were 11.6%.
• The median cost per course decreased significantly with dose reduction. Bone marrow blast cells ≥10% and Charlson comorbidity index ≥1 were independent predictors of efficacy.

Conclusions:

• Very-low-dose decitabine demonstrates favorable efficacy, good tolerance, and reduced medical costs for intermediate- to high-risk MDS.
• Elderly patients with comorbidities or higher blast cell percentages may be ideal candidates for this regimen.