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
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.
CITA DEL ARTÍCULO Cancer Med. 2023 May 22. doi: 10.1002/cam4.6120