Trial | Study design | Patient population | Enrolled patients | Primary endpoint | Results | Reference |
ALFA-0701 | Randomized phase III | Patients aged 50–70 years with de novo AML | 280 | EFS | GO arm, median 17.3 months vs control arm, median 9.5 months; p=0.0002 | 137 138 |
EORTC-GIMEMA AML-19 | Randomized phase III | Patients aged 61 years or older with de novo AML unsuitable for intensive chemotherapy | 237 | OS | Median OS 4.9 months vs 3.6 months (HR, 0.69; 95% CI, 0.53 to 0.90; p=0.005) | 139 |
Mylo-France 1 | Single-arm, open label phase II | Patients aged 18 years or older with RR AML | 57 | CRR | CRR 26% and 7% CR with incomplete platelet recovery | 140 |
AAML0531 | Multicenter randomized phase III | Patients aged 0 to 29 years with newly diagnosed AML | 1022 | EFS,OS | GO+chemotherapy improved EFS (3 years: 53.1% v 46.9%, p=0.04) OS, 3 years: 69.4% v 65.4%,p=0.39 GO+chemotherapy versus chemotherapy alone | 141 |
AML, acute myeloid leukemia; CR, complete response; CRR, complete response rate; EFS, event-free survival; GO, Gemtuzumab ozogamicin; OS, overall survival; RR, relapsed/refractory.