Table 3.
Selected randomized treatment trials for myelodysplastic syndrome (MDS)
| Treatment | N | Disease Risk Category |
Positive outcomes | Toxicity |
|---|---|---|---|---|
| Azacitidine [16] 75mg/m2 SQ × 7days Q 4 weeks vs. supportive care |
191 | IPSS Int-1/Int-2/High | Improved response rate (23 vs. 5%) Improved time to AML or death (21 vs. 13 months) Decreased AML transformation (15 vs. 38%) Improved QOL (physical function, symptoms, psychological state) |
Grade 3-4 myelosuppression (43-58%) Infection (20%) |
| Azacitidine [15] 75mg/m2 SQ × 7days Q 4 weeks vs. conventional care (supportive, low-dose cytarabine, intensive chemotherapy) |
358 | IPSS Int-2 or High | Overall survival (median 24.5 vs. 15 months) | Myelosuppression |
| Decitabine 15mg/m2 IV ever 8 hours for 3 days Q 6 weeks vs. supportive care [20] | 170 | IPSS Intermediate or High | Response rate (17 vs. 0%) Improved QOL (global health, fatigue, dyspnea) |
Dose reductions/delays (35%) Grade 4 myelosuppression (>50%) |
| Lenalidomide 10mg/day days 1-21 vs.5mg/day days 1-28 vs. placebo on 28 day cycle [21] | 205 | MDS with del5q31 IPSS Low or Int-1 RBC transfusion dependence |
RBC transfusion independence for ≥26 weeks (56.1 vs. 42.6 vs. 5.9%) RBC transfusion independence >8 weeks associated with decreased risk of death and AML progression |
Myelosuppression in first 2 cycles DVT (5.8%) in 10mg group |
Abbreviations: SQ= subcutaneous; Q= every; IV=intravenous; vs.= versus; int=intermediate; FAB= French-American-British; RA= refractory anemia; RARS= refractory anemia with ringed sideroblasts; IPSS= international prognostic scoring system; CMML= chronic myelomonocytic leukemia; RBC= red blood cell; ECOG= Eastern Cooperative Oncology Group; PS=performance status; AML= acute myeloid leuekemia; QOL= quality of life; CR= complete remission; DVT=deep venous thrombosis.