Table 5.
Study | Number of patients (median age) and protocol | Patient characteristics | Clinical results | Survival |
---|---|---|---|---|
Silverman LR et al. (Reference [119]) Phase III randomized study of Azacitidine (AZA) vs. Best supportive care (BSC). Crossover allowed. |
191 (68 years) AZA 75 mg/m2/d SQ for 7 days every 28 days Vs. BSC |
IPSS AZA/SC %: Low 2%/6% INT-1 26%/20% INT-2 11%/16% High 9%/10% |
ORR: AZA arm 60% (CR 7%, PR 16%, and HI 37%) Vs. BSC arm 5%
|
|
Fenaux P et al. (Reference [118]) Phase III randomized study of Azacitidine (AZA) Vs. Conventional care regimen (CCR). Crossover NOT allowed. |
358 (AZA 69 years; CCR 70 years) AZA 75 mg/m2/d SQ for 7 days every 28 days Vs. CCR (best supportive care [BSC], low dose cytarabine, or intensive chemotherapy) |
IPSS AZA/CCR %: Low excluded INT-1 3%/7% INT-2 43%/39% High 46%/48% |
|
|
Kantarjian H et al. (Reference [117]) Phase III randomized study of Decitabine (DAC) vs. Best supportive care (BSC) |
170 (70) DAC 15 mg/m2 IV q 8 h for 3 days every 6 weeks Vs. BSC |
IPSS DAC/BSC%: Low excluded INT-1 31%/30% INT-2 43%/44% High 26%/26% |
|
|
Lubbert M et al. (Reference [120]) Phase III randomized study of decitabine (DAC) Vs. Best Supportive Care (BSC) in elderly patients deemed ineligible for intensive chemotherapy |
233 (70) DAC 15 mg/m2 IV over 4 h 3 times a day for 3 days every 6 weeks or BSC |
IPSS DAC/BSC%: Low excluded INT-1 6.7%/7% INT-2 53.8%/55.3% High 38.7%/36.8%
|
|
|
BSC: Best supportive care; IPSS: International Prognostic Scoring System; INT-1: Intermediate-1; INT-2: Intermediate 2; ORR: Overall response rate; CR: Complete remission; PR: Partial remission; HI: Hematologic improvement; OS: Overall survival; CCR: Conventional care regimen; AML: Acutemyeloid leukemia; PFS: Progression-free survival.