Venetoclax Therapy in a Heavily Treated Cohort of Patients with Relapsed or Refractory Acute Myeloid Leukemia: Update of the Pethema Registry Experience

Introduction The prognosis of patients with relapsed or refractory acute myeloid leukemia (RR-AML) is very poor, and treatment options are very limited. The exciting results of venetoclax (VEN) in untreated AML have led to its off-label use in RR-AML. However, evidence in RR-AML is still scarce and...

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Published in:Blood 2021-11, Vol.138 (Supplement 1), p.2325-2325
Main Authors: Labrador, Jorge, Saiz-Rodríguez, Miriam, De Miguel, Maria Dunia, De Laiglesia, Almudena, Rodriguez, Carlos, Belén Vidriales, María, Pérez-Encinas, Manuel, Sanchez, Maria Jose, Cuello, Rebeca, Roldán Pérez, Alicia, Vives, Susana, Benzo Callejo, Gonzalo, Araujo, Mercedes Colorado, García-Fortes, María, Sayas, Maria Jose, Olivier, Carmen, Recio, Isabel, Conde, Diego, Bienert Garcia, Alvaro, Vahi, Maria, Muñoz García, Carmen, Seri, Cristina, Tormo, Mar, Vall-llovera, Ferran, Foncillas, Maria Angeles, Martínez-Cuadrón, David, Sanz, Miguel A., Montesinos, Pau
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Language:English
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Summary:Introduction The prognosis of patients with relapsed or refractory acute myeloid leukemia (RR-AML) is very poor, and treatment options are very limited. The exciting results of venetoclax (VEN) in untreated AML have led to its off-label use in RR-AML. However, evidence in RR-AML is still scarce and the available data are mostly from retrospective and single-center studies. The aim of our study was to analyze the effectiveness of VEN use in patients with RR-AML reported to the PETHEMA AML epidemiological registry. Initial results were presented previously (Labrador J, et al. ASH 2020). Here, we report an updated analysis. Methods We conducted a retrospective, multicenter, observational study of a cohort of patients with AML-RR who were treated with venetoclax in the hospitals of the PETHEMA group. We evaluated efficacy, CR/CRi rate and overall survival (OS). We performed a descriptive analysis. Overall survival (OS) was calculated using the Kaplan-Meier method. Results Fifty-one patients were included, 33 men and 18 women, with a median age of 68 years (25-82). The main characteristics of the included patients are shown in Table 1. With a median follow-up of 167 days, 10/51 patients (19%) continued to receive VEN at the time of analyses. Patients received a median of 2 cycles (0-8). VEN was administered with azacitidine (AZA) in 59%, with decitabine (DEC) in 29% and with low-dose cytarabine (LDAC) in 12% of patients, respectively. The CR/CRi and partial response (PR) rates were 12.4% and 10.4%, respectively. The CR/RCi and overall response (ORR, CR/CRi+PR) was higher in patients receiving VEN+AZA (17.9% and 32.1%) than in those receiving DEC + VEN (6.7% and 13.3%) or LDAC + VEN (0%). The presence of NPM1 or CEBPA variants were the only two variables associated with increased CR/CRi with VEN in RR-AML. Median OS was 104 days (95% CI: 56 - 151) (Figure 1A), 120 days in combination with AZA, 104 days with DEC, and 69 days with LDAC; p=0.875. Treatment response (Figure 1B) and ECOG 0 were the only variables that influenced OS in a multivariate model adjusted for age and sex (Table 2). VEN-resistant patients who received subsequent salvage therapy had superior median OS (98 vs. 5 days, p=0.004).Twenty-eight percent of patients required discontinuation of VEN due to toxicity. Sixty-one percent of patients required admission, mainly due to infections (45%), 10% due to bleeding and other causes in 12%. One case of tumor lysis syndrome was described. Conclusions Our
ISSN:0006-4971
1528-0020
DOI:10.1182/blood-2021-150231