The efficacy and safety of avapritinib in the treatment of molecular biologically positive core binding factor-acute myeloid leukemia with KIT mutation after allogeneic hematopoietic stem cell transplantation

• Published in: Zhōnghuá xuèyèxué zázhì 2024-08, Vol.45 (8), p.761
• Main Authors: Wang, J, Zu, Y L, Gui, R R, Li, Z, Zhang, Yanli, Zhou, J
• Format: Article
• Language: Chinese
• Subjects: Adult; Female; Hematopoietic Stem Cell Transplantation - methods; Humans; Leukemia, Myeloid, Acute - genetics; Leukemia, Myeloid, Acute - therapy; Male; Middle Aged; Mutation; Proto-Oncogene Proteins c-kit - genetics; Pyrazines - administration & dosage; Pyrazines - adverse effects; Pyrazoles; Pyrroles; Retrospective Studies; Transplantation, Homologous; Triazines
• ISSN: 0253-2727
• DOI: 10.3760/cma.j.cn121090-20240129-00043

To investigate the efficacy and safety of avapritinib in the treatment of molecular biologically positive core binding factor-acute myeloid leukemia (CBF-AML) with KIT mutation after allogeneic hematopoietic stem cell transplantation (allo-HSCT) . We retrospectively analyzed the clinical data of six patients with molecular biologically positive CBF-AML with KIT mutation after allo-HSCT, who were treated with avapritinib at Henan Cancer Hospital from December 2021 to March 2023, and evaluated the efficacy and safety of avapritinib. After 1 month of treatment with avapritinib, the transcription level of the fusion gene decreased in six patients, and the transcription level decreased by ≥1 log in five patients. In four patients who received avapritinib for ≥3 months, the fusion gene turned negative, and the median time to turn negative was 2.0 (range: 1.0-3.0) months. Up to the end of follow-up, four patients had no recurrence. The most common adverse reaction of avapritinib was myelosuppression, including neutr