Study | Number of patients | Median age (years) | Diagnosis | Treatment | Results | Common toxicity |
---|---|---|---|---|---|---|
Raffoux et al., 2010 [64] | 65 | 72 | AML (85%) or high-risk MDS | 5-AZA 75 mg/m2. VPA 35 to 50 mg/kg po day 1 to 7. ATRA 45 mg/m2 po day 8 to 28. Six cycles. | After six cycles, 34 patients survived: 13 patients (38%) CR, two patients (6%) PR and 14 patients (41%) had stable disease. Median OS was 12.4 months. | Confusion 33 events, infection 76 events. |
Blum et al., 2007 [63] | 25 | 70 | AML | Decitabine 15 to 20 mg/m2/d iv days 1 to 10 every 28 days. VPA 15 to 20 mg/kg days 5 to 21 in ten patients. | Response rate was 44%: four patients CR, four patients CRi and three patients PR (AML criteria). Survival not reported. | Neutropenic fever (64%), fatigue and infection (both 48%) were most common. |
Soriano et al., 2007 [65] | 53 | 69 | AML (92%) or high-risk MDS | 5-AZA 75 mg/m2/d. VPA 50 to 75 mg/kg/d days 1 to 7. ATRA 45 mg/m2 days 3 to 5. Treatment repeated every 3 weeks. | Overall response rate was 42%: 12 patients (22%) CR. Survival not reported. | Two events of grade IV and 11 events of grade III non-hematological toxicity; mainly fatigue or other neurotoxicities. |
Garcia-Manero et al., 2006 [66] | 54 | 60 | AML (89%) or MDS | Decitabine 15 mg/m2/d iv. VPA 20 to 50 mg/kg days 1 to 10. Treatment repeated every fourth week. | Twelve patients (22%) had responses: 10 patients CR and two patients CRp. OS 6 months (0.6 to 20.2 months). | Fatigue, nausea and diarrhoea were the most common non-hematological toxicities. |
Maslak et al., 2006 [67] | 10 | 66.5 | AML (80%) or MDS | 5-AZA 75 mg/m2/d days 1 to 7. Sodium phenylbutyrate 200 mg/kg/d iv days 8 to 12. Treatment repeated every 21 to 28 days. | Three patients (30%) PR and two patients (20%) had stable disease. Duration of response was 45 days (37 to 136 days). Survival not reported. | Three patients had neutropenic fever. Nausea, dizziness and fatigue were common. |