Journal: Blood advances
Study type and population
- Multicenter, real-world cohort (2019–2022): 403 patients with AML with myelodysplasia-related changes (AML-MRC) or therapy-related AML treated with CPX-351 outside clinical trials.
- Fitness for intensive chemotherapy: Prospectively assessed using SIE/SIES/GITMO criteria (age, comorbidities, performance status): 328 (81.4%) classified as fit, 75 (18.6%) as unfit.
- ELN 2017 risk (396 fully characterized patients): 4% favorable, 41% intermediate, 55% adverse.
Efficacy
- Overall complete remission (CR) rate after induction: 57.1%.
- CR rates by fitness group: 57.3% (fit) vs 56% (unfit).
Survival and early mortality
- Divergence in outcomes despite similar CR:
- Median overall survival: 18 months (fit) vs 8 months (unfit).
- 28-day mortality: 4.6% (fit) vs 10.7% (unfit).
- 100-day mortality: 14.3% (fit) vs 32% (unfit).
Key implications for practice
- CPX-351 activity across fitness strata: Substantial activity with similar CR rates, but unfit patients have significantly worse early mortality and shorter survival.
- Value of SIE/SIES/GITMO fitness criteria: Effectively identify subgroups with distinct short- and long-term outcomes on CPX-351.
- Need for refined fitness tools: Supports refining or developing treatment-specific fitness tools to better select candidates for CPX-351 and optimize risk–benefit in older/comorbid AML-MRC and t-AML patients.