A phase 2, multicenter, clinical trial of CPX-351 in older patients with secondary or high-risk acute myeloid leukemia : PETHEMA-LAMVYX
Rodríguez-Arbolí, Eduardo (Hospital Universitario Virgen del Rocío (Sevilla, Andalusia))
Rodríguez-Veiga, Rebeca (Hospital Universitari i Politècnic La Fe (València))
Soria-Saldise, Elena (Hospital Universitario Virgen del Rocío (Sevilla, Andalusia))
Bergua Burgues, Juan Miguel (Hospital San Pedro de Alcántara (Càceres))
Caballero Velazquez, Teresa (Hospital Universitario Virgen del Rocío (Sevilla, Andalusia))
Arnán, Montserrat (Hospital Duran i Reynals (Barcelona))
Vives Polo, Susana (Institut Germans Trias i Pujol. Hospital Universitari Germans Trias i Pujol)
Serrano, Josefina (Hospital Universitario Reina Sofía (Còrdova, Espanya))
Bernal, Teresa (Hospital Universitario Central de Asturias)
Martínez-Sánchez, Pilar (Hospital Universitario 12 de Octubre (Madrid))
Tormo, Mar (Hospital Clínic Universitari (València))
Rodríguez-Medina, Carlos (Hospital Universitario de Gran Canaria Dr. Negrín)
Herrera-Puente, Pilar (Hospital Universitario Ramón y Cajal (Madrid))
Lavilla, Esperanza (Hospital Universitario Lucus Augusti (Lugo))
Boluda, Blanca (Hospital Universitari i Politècnic La Fe (València))
Acuña-Cruz, Evelyn (Hospital Universitari i Politècnic La Fe (València))
Cano, Isabel (Hospital Universitari i Politècnic La Fe (València))
Cáceres, Sara (Hospital San Pedro de Alcántara)
Ballesteros, Juan (Vivia Biotech. S.L. (Madrid))
Falantes, José Francisco (Hospital Universitario Virgen del Rocío (Sevilla, Andalusia))
Martínez-Cuadrón, David (Hospital Universitari i Politècnic La Fe (València))
Pérez-Simón, José A. (Hospital Universitario Virgen del Rocío (Sevilla, Andalusia))
Montesinos, Pau (Hospital Universitari i Politècnic La Fe (València))

Date:	2025
Abstract:	Background: LAMVYX was a multicenter, single-arm, phase 2 trial designed to validate the safety and efficacy of CPX-351 in patients aged 60-75 years with newly diagnosed, secondary acute myeloid leukemia and to generate evidence on key issues not addressed in the preceding regulatory pivotal trial. Methods: The primary end point of the study was the complete remission (CR)/CR with incomplete hematologic recovery (CRi) rate after induction. Eligible patients were recommended to undergo allogeneic hematopoietic stem cell transplantation after the first consolidation cycle. Alternatively, patients could undergo up to six maintenance cycles with CPX-351. Results: Twenty-nine patients (49%; 95% exact confidence interval [CI], 37%-62%) patients achieved a CR/CRi after one or two cycles of induction, with a measurable residual disease negativity rate of 67% as assessed by centralized, multiparameter flow cytometry. Among patients who had serial next-generation sequencing analyses available, clearance of somatic mutations that were present at diagnosis was achieved in 7 (35%). The median follow-up among survivors was 16. 8 months (range, 8. 7-24. 3 months). The median event-free survival was 3. 0 months (95% CI, 1. 4-7. 3 months), and the median overall survival was 7. 4 months (95% CI, 3. 7-12. 7 months). In landmark analyses at day +100 from diagnosis, the 1-year overall and event-free survival rate among patients who underwent allogeneic hematopoietic stem cell transplantation was 70% (95% CI, 47%-100%) and 70% (95% CI, 47%-100%), respectively. The corresponding values were 89% (95% CI, 71%-100%) and 44% (95% CI, 21%-92%), respectively, for patients who entered the maintenance phase. No significant longitudinal changes were observed in severity index or quality-of-life visual analog scale scores. Conclusions: The current data provide novel insights that might inform the clinical positioning and optimal use of CPX-351, complementing previous results (ClinicalTrials. gov identifier NCT04230239).
Note:	Altres ajuts: Juan Rods Clinician Scientist Grant (JR23/00067)
Rights:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original.
Language:	Anglès
Document:	Article ; recerca ; Versió publicada
Subject:	Acute myeloid leukemia ; Allogeneic hematopoietic stem cell transplantation ; Maintenance therapy ; Measurable residual disease ; Quality of life
Published in:	Cancer, Vol. 131 Núm. 1 (January 2025) , ISSN 1097-0142

DOI: 10.1002/cncr.35618
PMID: 39476204

15 p, 1.4 MB

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Research literature > UAB research groups literature > Research Centres and Groups (research output) > Health sciences and biosciences > Institut d'Investigació en Ciencies de la Salut Germans Trias i Pujol (IGTP)
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