Table 2.
Current approved therapies and ongoing clinical trials for acute myeloid leukemia (AML) with myelodysplasia-related changes (AML-MRC).
| Current Treatment Options for Patients with AML-MRC | ||
|---|---|---|
| Treatment | Year FDA Approved for AML | Outcomes in AML-MRC Patients |
| Approved with a specific indication for AML-MRC patients | ||
| Liposomal daunorubicin-cytarabine | 2017 | CR/CRi rate 48% mOS* 19.15 months |
| Approved for AML but not with a specific indication for AML-MRC patients | ||
| Standard 7 + 3 | First reported in 1973 [49] | CR/CRi rate 33% mOS 11.58 months |
| Hypomethylating agents (HMAs) | ||
| Azacitidine | 2004 (MDS; no specific FDA approval for AML) [50] | CR/CRi rate 24.8% mOS 8.9 months |
| Decitabine | 2006 (MDS; no specific FDA approval for AML) [51] | CR/CRi rate 74% No specific OS analysis |
| Venetoclax combinations | ||
| Venetoclax with HMA | 2018 | CR/CRi rate 67% (note: only secondary AML patients analyzed) |
| Venetoclax with low-dose cytarabine | 2018 | No specific analysis in AML-MRC group |
| Allogeneic stem cell transplant | First reported in 1957 [52] | Similar to patients without AML-MRC |
| Ongoing Clinical Trials for Patients with AML-MRC ^ | ||
| Study Title | Trial Details | NCT Number |
| CPX-351 for the Treatment of Secondary Acute Myeloid Leukemia in Patients Younger Than 60 Years Old | Phase 2 Newly diagnosed Includes AML-MRC, secondary AML, t-AML patients |
NCT04269213 |
| CPX-351 and Glasdegib for Newly Diagnosed Acute Myelogenous Leukemia With MDS Related Changes or Therapy-related Acute Myeloid Leukemia | Phase 2 Newly diagnosed Includes AML-MRC and t-AML patients |
NCT04231851 |
| A Study of Azacitidine for Patients With Int/High -Risk MDS and AML-MRC | Phase 3 Includes intermediate- and high-risk MDS patients and AML-MRC patients with less than 30% blasts |
NCT03978364 |
| Pevonedistat, Cytarabine, and Idarubicin in Treating Patients With Acute Myeloid Leukemia | Phase 1b/2 Newly diagnosed Includes AML-MRC and t-AML patients |
NCT03330821 |
| Integrating Geriatric Assessment and Genetic Profiling to Personalize Therapy Selection in Older Adults With Acute Myeloid Leukemia | Phase 2 Newly diagnosed Age ≥60 Includes de novo AML, secondary AML, t-AML, other AML equivalent such as myeloid sarcoma, MDS in transformation to AML, or high-grade treatment-related myeloid neoplasm |
NCT03226418 |
* mOS—median OS; ^ all information obtained from clinicaltrials.gov.