Intermediate-dose HAD Regimen for CEBPA Double-mutated AML
Summary
AML is highly heterogeneous in pathogenesis, and CEBPA double-mutated (CEBPAdm) AML is a common type of leukemia in China. Currently, no targeted therapies for CEBPAdm, and chemotherapy and transplantation are still the treatment options for CEBPA double-mutated AML. At present, the "3+7" treatment induction regimen of cytarabine combined with anthracyclines is still the first-line recommended regimen. In our retrospective study, the intermediate dose HAD regimen produced a 3-year RFS of 84.7% and a 3-year OS of 92.8% in CEBPAdm AML. Therefore, this project intends to confirm the efficacy of intermediate-dose HAD in the treatment of CEBPA double-mutated AML is superior to the conventional treatment regimen through the multi-center RCT study.
Timeline
- Start
- 2024-08-13
- Primary completion
- 2026-08-30
- Completion
- 2028-09-01
Publications
- Results Wei H, Wang Y, Zhou C, Lin D, Liu B, Liu K, Qiu S, Gong B, Li Y, Zhang G, Wei S, Gong X, Liu Y, Zhao X, Gu R, Mi Y, Wang J. Distinct genetic alteration profiles of acute myeloid leukemia between Caucasian and Eastern Asian population. J Hematol Oncol. 2018 Feb 10;11(1):18. doi: 10.1186/s13045-018-0566-8.
- Results Wei H, Zhou C, Liu B, Lin D, Li Y, Wei S, Gong B, Zhang G, Liu K, Gong X, Fang Q, Liu Y, Qiu S, Gu R, Song Z, Chen J, Yang M, Zhang J, Jin J, Wang Y, Mi Y, Wang J. The prognostic factors in acute myeloid leukaemia with double-mutated CCAAT/enhancer-binding protein alpha (CEBPAdm). Br J Haematol. 2022 May;197(4):442-451. doi: 10.1111/bjh.18113. Epub 2022 Mar 10.
- Results Wei H, Wang Y, Gale RP, Lin D, Zhou C, Liu B, Qiu S, Gu R, Li Y, Zhao X, Wei S, Gong B, Liu K, Gong X, Liu Y, Zhang G, Song Z, Wang Y, Li W, Mi Y, Wang J. Randomized Trial of Intermediate-dose Cytarabine in Induction and Consolidation Therapy in Adults with Acute Myeloid Leukemia. Clin Cancer Res. 2020 Jul 1;26(13):3154-3161. doi: 10.1158/1078-0432.CCR-19-3433. Epub 2020 Feb 6.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Comparator | Cytarabine | Small molecule | 100 mg/m2 | — |
| Comparator | Cytarabine | Small molecule | 1 g | — |
| Comparator | Cytarabine | Small molecule | 3 g | — |
| Comparator | Daunorubicin | Small molecule | 40 mg/m2 | — |
| Comparator | Daunorubicin | Small molecule | 60 mg/m2 | — |
| Subject | Homoharringtonine | Small molecule | 2 mg/m2 | — |
| Comparator | Idarubicin | Small molecule | 10 mg/m2 | — |
| Comparator | Idarubicin | Small molecule | 12 mg/m2 | — |
| Background | Cyclophosphamide | Other / unclassified | 350 mg/m2 | — |