Use of Venetoclax in Patients with Relapsed or Refractory Acute Myeloid Leukemia: The PETHEMA Registry Experience
Jorge Labrador, Miriam Saiz‐Rodríguez, Dunia de Miguel, Almudena de Laiglesia, Carlos Rodríguez‐Medina, María‐Belén Vídriales, Manuel Pérez‐Encinas, María José Sánchez-Sánchez, Rebeca Cuello, Alicia Roldán, Susana Vives, Gonzalo Benzo-Callejo, Mercedes Colorado, María García‐Fortes, María José Sayas, C Olivier, Isabel Recio, Diego Conde-Royo, Álvaro Bienert-García, Maria Vahí, Carmen Muñoz-García, Cristina Seri-Merino, Mar Tormo, Ferran Vall‐Llovera, María Ángeles Foncillas, David Martínez‐Cuadrón, Miguel Á. Sanz, Pau Montesinos
Abstract
The effectiveness of venetoclax (VEN) in relapsed or refractory acute myeloid leukemia (RR-AML) has not been well established. This retrospective, multicenter, observational database studied the effectiveness of VEN in a cohort of 51 RR-AML patients and evaluated for predictors of response and overall survival (OS). The median age was 68 years, most were at high risk, 61% received ≥2 therapies for AML, 49% had received hypomethylating agents, and ECOG was ≥2 in 52%. Complete remission (CR) rate, including CR with incomplete hematological recovery (CRi), was 12.4%. Additionally, 10.4% experienced partial response (PR). The CR/CRi was higher in combination with azacitidine (AZA; 17.9%) than with decitabine (DEC; 6.7%) and low-dose cytarabine (LDAC; 0%). Mutated NPM1 was associated with increased CR/CRi. Median OS was 104 days (95% CI: 56–151). For the combination with AZA, DEC, and LDAC, median OS was 120 days, 104 days, and 69 days, respectively; p = 0.875. Treatment response and ECOG 0 influenced OS in a multivariate model. A total of 28% of patients required interruption of VEN because of toxicity. Our real-life series describes a marginal probability of CR/CRi and poor OS after VEN-based salvage. Patients included had very poor-risk features and were heavily pretreated. The small percentage of responders did not reach the median OS.