Table 4.
Clinical AML studies with GO after its withdrawal from the market (2010)
| Study | Phase | Population | Association | Results (efficacy) | Results (safety) | References | Status |
|---|---|---|---|---|---|---|---|
| Gemtuzumab ozogamicin | |||||||
| ALFA-0701 | III | De novo AML (age 50–70 years) GO 3 mg/m2 D1–D4–D7 | + IC vs. IC alone | No difference in CR (81% vs. 75%); > EFS (15.6 vs. 9.7 months) > RFS (28.1 vs. 11.4 months), > OS (34 vs. 19 months) | G ≥ 3 hemorrhage (22.9% vs. 9.5%); longer time to platelet recovery. SOS: 2/139 cases | [54] | Completed |
| MRC AML15 | III |
De novo AML (age 18–60 years) GO 3 mg/m2 D1 |
+ IC vs. IC alone (different regimens) |
No differences in CR, OS; > OS in favorable cytogenetic risk |
No > hematological or extra-hematological toxicities | [61] | Completed |
| SWOG S0106 (NCT00085709) | III | De novo AML (age 18–60 years) GO 6 mg/m2 D4 | + IC vs. IC alone | No differences in CR rate (69 vs. 70%), 5-year OS (46 vs. 50%), 5-year RFS (43 vs. 42%) | > Fatal toxicity (5.5 vs. 1.4%) | [50] | Completed |
| NCRI AML16 | III | De novo AML (unfit/> 60 years of age) GO 5 mg/m2 D1 | + LDAC vs. LDAC | > ORR (30 vs. 17%); no improved OS | No > hematological or extra-hematological toxicities | [62] | Completed |
| GOELAMS AML 2006 IR | III | De novo AML (age 18–60 years) GO 6 mg/m2 D4 | + IC vs. IC |
> EFS in GO arm (53.7 vs. 27%) in patients not undergoing allo-SCT No differences in CR rate (91 vs. 86.5%), early death (10 vs. 4.5%), OS (53 vs. 46%) |
> G3/4 hepatic toxicities in the GO arm (23 vs. 13%). No differences in TRM | [60] | Completed |
| AML-19 (NCT00091234) | III | De novo AML (unfit/> 60 years of age) GO 6 mg/m2 D1 and 3 mg/m2 D8 | vs. BSC | ORR 27%; mOS 4.9 vs. 3.5 m | No > hematological or extra-hematological toxicities | [51] | Completed |
Mechanism: recombinant humanized anti-CD33 antibody, which delivers the linked cytotoxic drug calicheamicin to CD33-expressing leukemic cells
Status: approved by FDA for treatment of newly diagnosed and R/R CD33-positive AML, alone or in combination with daunorubicin and cytarabine (September 1, 2017)
CR complete response, EFS event free survival, IC intensive chemotherapy, OS overall survival