Venetoclax and azacitidine for relapsed and refractory pediatric acute myeloid leukemia: A single-institution experience

Abstract

Recently, the efficacy and safety of venetoclax/azacitidine (VEN/AZA) for adult acute myelogenous leukemia (AML) have been reported; however, only few reports focus on their efficacy in pediatric AML. Here, we report four cases of pediatric relapsed/refractory AML, who were treated with VEN/AZA. One patient experienced induction failure, whereas three patients relapsed after allogeneic hematopoietic stem cell transplantation (allo-HSCT). The median number of VEN/AZA courses was three (2–4). Complete hematological remission was achieved in two patients after the first course and in all four patients after the second course; three patients subsequently received allo-HSCT. Grade 4 neutropenia was observed in nine out of 12 (75%) courses. Neutropenia of less than 100/µL was common in the first course; however, it was only in one of eight courses in subsequent courses. No serious non-hematological toxicities were observed. All four patients were successfully remitted and side effects other than myelosuppression were minimal. This suggests that VEN/AZA may be a promising and novel treatment option for induction therapy in relapsed/refractory pediatric AML and could be used safely even for patients in poor conditions after allo-HSCT.