Selected randomized controlled trials and multicenter studies evaluating single-agent parenteral azacitidine and decitabine in myelodysplastic syndromes and AML
| Study . | Study population . | Intervention . | Primary end point . | Primary end point results . |
|---|---|---|---|---|
| MDS | ||||
| Silverman et al, 200223 | MDS classified by FAB criteria | Azacitidine 75 mg/m2 once daily for 7 d every 4 wk vs supportive care | Response defined by CR, PR, or improvement in hematopoiesis/transfusion requirements | Response rate: 60% vs 5% (P < .0001) |
| Fenaux et al, 200925 | IPSS intermediate-2/high-risk MDS classified by FAB criteria as RAEB, RAEB in transformation, or CMML with ≥10% bone marrow blasts and WBC ≤13 × 109/L | Azacitidine 75 mg/m2 once daily for 7 d every 4 wk vs conventional therapy (best supportive care, low-dose cytarabine, or intensive chemotherapy) | Overall survival | Median overall survival: 24.5 mo vs 15.0 mo (P = .0001) |
| Kantarjian et al, 200624 | MDS with an IPSS score ≥0.5 | Decitabine 15 mg/m2 every 8 h for 3 d every 6 wk vs best supportive care | ORR by IWG criteria and time to AML transformation or death | ORR: 17% vs 0% (P < .001); median time to AML transformation or death: 12.1 mo vs 7.8 mo (P = .16) |
| Steensma et al, 200927 | MDS of any FAB subtype including CMML | Decitabine 20 mg/m2 once daily for 5 d every 4 wk | ORR by IWG criteria | Overall response rate: 32% (95% CI, 23%-43%); overall improvement rate (ORR + HI): 51% (95% CI, 40-61%) |
| Lübbert et al, 201126 | IPSS Intermediate/high-risk MDS in patients ≥60 y ineligible for intensive chemotherapy | Decitabine 15 mg/m2 every 8 h for 3 d every 6 wk vs best supportive care | Overall survival | Median overall survival: 10.1 mo vs 8.5 mo (P = .38) |
| AML | ||||
| Dombret et al, 201533 | Newly diagnosed AML with ≥30% bone marrow blasts in adults ≥65 y | Azacitidine vs conventional therapy (best supportive care, low-dose cytarabine, or standard induction chemotherapy) | Overall survival | Median overall survival: 10.4 mo vs 6.5 mo (P = .1009) |
| Kantarjian et al, 201232 | Newly diagnosed AML in adults ≥65 y | Decitabine vs treatment choice (best supportive care or low-dose cytarabine) | Overall survival | Median overall survival: 7.7 mo vs 5.0 mo (P = .108) |
| Study . | Study population . | Intervention . | Primary end point . | Primary end point results . |
|---|---|---|---|---|
| MDS | ||||
| Silverman et al, 200223 | MDS classified by FAB criteria | Azacitidine 75 mg/m2 once daily for 7 d every 4 wk vs supportive care | Response defined by CR, PR, or improvement in hematopoiesis/transfusion requirements | Response rate: 60% vs 5% (P < .0001) |
| Fenaux et al, 200925 | IPSS intermediate-2/high-risk MDS classified by FAB criteria as RAEB, RAEB in transformation, or CMML with ≥10% bone marrow blasts and WBC ≤13 × 109/L | Azacitidine 75 mg/m2 once daily for 7 d every 4 wk vs conventional therapy (best supportive care, low-dose cytarabine, or intensive chemotherapy) | Overall survival | Median overall survival: 24.5 mo vs 15.0 mo (P = .0001) |
| Kantarjian et al, 200624 | MDS with an IPSS score ≥0.5 | Decitabine 15 mg/m2 every 8 h for 3 d every 6 wk vs best supportive care | ORR by IWG criteria and time to AML transformation or death | ORR: 17% vs 0% (P < .001); median time to AML transformation or death: 12.1 mo vs 7.8 mo (P = .16) |
| Steensma et al, 200927 | MDS of any FAB subtype including CMML | Decitabine 20 mg/m2 once daily for 5 d every 4 wk | ORR by IWG criteria | Overall response rate: 32% (95% CI, 23%-43%); overall improvement rate (ORR + HI): 51% (95% CI, 40-61%) |
| Lübbert et al, 201126 | IPSS Intermediate/high-risk MDS in patients ≥60 y ineligible for intensive chemotherapy | Decitabine 15 mg/m2 every 8 h for 3 d every 6 wk vs best supportive care | Overall survival | Median overall survival: 10.1 mo vs 8.5 mo (P = .38) |
| AML | ||||
| Dombret et al, 201533 | Newly diagnosed AML with ≥30% bone marrow blasts in adults ≥65 y | Azacitidine vs conventional therapy (best supportive care, low-dose cytarabine, or standard induction chemotherapy) | Overall survival | Median overall survival: 10.4 mo vs 6.5 mo (P = .1009) |
| Kantarjian et al, 201232 | Newly diagnosed AML in adults ≥65 y | Decitabine vs treatment choice (best supportive care or low-dose cytarabine) | Overall survival | Median overall survival: 7.7 mo vs 5.0 mo (P = .108) |
HI, hematologic improvement; IWG, International Working Group; RAEB, refractory anemia with excess blasts; PR, partial remission; WBC, white blood cell count.