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. 2023 Jul;12(14):14892-14901.
doi: 10.1002/cam4.6120. Epub 2023 May 22.

Clinical outcomes after CPX-351 in patients with high-risk acute myeloid leukemia: A comparison with a matched cohort from the Spanish PETHEMA registry

Teresa Bernal  1   2 , Ainhoa Fernández Moreno  1   2 , Almudena de LaIglesia  3 , Celina Benavente  4 , Ana García-Noblejas  5 , Daniel García Belmonte  6 , Rosalía Riaza  7 , Olga Salamero  8 , Maria Angeles Foncillas  9 , Alicia Roldán  10 , Víctor Noriega Concepción  11 , Laura Llorente González  12 , Juan Miguel Bergua Burgués  13 , Soraya Lorente de Uña  14 , Gabriela Rodríguez-Macías  15 , Adolfo de la Fuente Burguera  16 , Maria José García Pérez  17 , Jose Luis López-Lorenzo  18 , Pilar Martínez  19 , Concepción Aláez  20 , Marta Callejas  21 , Carmen Martínez-Chamorro  22 , José Rifón Roca  23 , Lourdes Amador Barciela  24 , Armando V Mena Durán  25 , Karoll Gómez Correcha  26 , Esperanza Lavilla Rubira  27 , María Luz Amigo  28 , Ferran Vall-Llovera  29 , Ana Garrido  30 , María García-Fortes  31 , Dunia de Miguel Llorente  32 , Anastasia Aules Leonardo  33 , Carlos Cervero  34 , Rosa Coll Jordá  35 , Manuel M Pérez-Encinas  36 , Marta Polo Zarzuela  4 , Angela Figuera  5 , Guillermo Rad  2 , David Martínez-Cuadrón  37 , Pau Montesinos  37
Affiliations

Clinical outcomes after CPX-351 in patients with high-risk acute myeloid leukemia: A comparison with a matched cohort from the Spanish PETHEMA registry

Teresa Bernal et al. Cancer Med. 2023 Jul.

Abstract

Background: CPX-351 is approved for the treatment of therapy related acute myeloid leukemia (t-AML) and AML with myelodysplastic related changes (MRC-AML). The benefits of this treatment over standard chemotherapy has not been addressed in well matched cohorts of real-life patients.

Methods: Retrospective analysis of AML patients treated with CPX-351 as per routine practice. A propensity score matching (PSM) was used to compare their main outcomes with those observed in a matched cohort among 765 historical patients receiving intensive chemotherapy (IC), all of them reported to the PETHEMA epidemiologic registry.

Results: Median age of 79 patients treated with CPX-351 was 67 years old (interquartile range 62-71), 53 were MRC-AML. The complete remission (CR) rate or CR without recovery (CRi) after 1 or 2 cycles of CPX-351 was 52%, 60-days mortality 18%, measurable residual disease <0.1% in 54% (12 out of 22) of them. Stem cell transplant (SCT) was performed in 27 patients (34%), median OS was 10.3 months, and 3-year relapse incidence was 50%. Using PSM, we obtained two comparable cohorts treated with CPX-351 (n = 52) or IC (n = 99), without significant differences in CR/CRi (60% vs. 54%) and median OS (10.3 months vs. 9.1 months), although more patients were bridged to SCT in the CPX-351 group (35% vs. 12%). The results were confirmed when only 3 + 7 patients were included in the historical cohort. In multivariable analyses, SCT was associated with better OS (HR 0.33 95% CI: 0.18-0.59), p < 0.001.

Conclusion: Larger post-authorization studies may provide evidence of the clinical benefits of CPX-351 for AML in the real-life setting.

Keywords: acute myeloid leukemia; clinical observations; intensive chemotherapy; real-world.

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Figures

FIGURE 1
FIGURE 1
Flow chart of patients treated with CPX‐351.
FIGURE 2
FIGURE 2
Overall survival according to induction chemotherapy.
FIGURE 3
FIGURE 3
Cumulative incidence of relapse and death in the matched cohort.
See this image and copyright information in PMC

References

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