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
10.3760/cma.j.cn121090-20240129-00043
- VernacularTitle:阿伐替尼治疗异基因造血干细胞移植后分子生物学阳性的伴KIT突变CBF-AML的疗效及安全性
- Author:
Juan WANG
1
;
Yingling ZU
;
Ruirui GUI
;
Zhen LI
;
Yanli ZHANG
;
Jian ZHOU
Author Information
1. 郑州大学附属肿瘤医院，河南省肿瘤医院血液科，郑州 450008
- Keywords:
Core binding factors;
Leukemia, myeloid, acute;
Hematopoietic stem cell transplantation;
Avapritinib;
Gene, KIT
- From:
Chinese Journal of Hematology
2024;45(8):761-766
- CountryChina
- Language:Chinese
-
Abstract:
Objective: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) .Methods: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.Results: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 neutropenia in two cases, thrombocytopenia in two cases, and anemia in one case. The non-hematological adverse reactions were nausea in two cases, edema in one case, and memory loss in one case, all of which were grades 1-2.Conclusion:Avapritinib was effective for molecular biologically positive CBF-AML patients with KIT mutation after allo-HSCT. The main adverse reaction was myelosuppression, which could generally be tolerated.
