Advanced Systemic Mastocytosis with associated haematological neoplasm: Treatment with avapritinib can facilitate successful bridge to allogeneic haematopoietic cell transplant

IF 3.2 4区医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Current Research in Translational Medicine Pub Date : 2023-07-01 DOI:10.1016/j.retram.2023.103398

P Sriskandarajah , D P McLornan , C Oni , AJ Wilson , C Woodley , M Ciesielska , K Raj , R Dillon , M Ethell , J Chacko , K Orchard , DH Radia

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引用次数: 4

Abstract

Advanced systemic mastocytosis (AdvSM) is a rare, life-limiting mast cell (MC) neoplasm, with approximately 70% patients having an associated haematological neoplasm (AHN). Avapritinib, a selective tyrosine kinase inhibitor targeting KIT D816V, has shown potent activity translating clinically into durable responses in the phase 1 EXPLORER (NCT02561988) and phase 2 PATHFINDER (NCT03580655) studies.

We report three patients with AdvSM-AHN on avapritinib who achieved complete remission (CR) of SM and were successfully bridged to allogeneic haematopoietic cell transplant (allo-HCT). Two cases additionally highlight the risk of clonal evolution within the AHN component and requirement for close monitoring while on targeted therapy.

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伴有相关血液肿瘤的晚期系统性肥大细胞增多症：阿伐普替尼治疗可促进成功桥接异基因造血细胞移植。

晚期系统性肥大细胞增多症（AdvSM）是一种罕见的、限制生命的肥大细胞（MC）肿瘤，约70%的患者患有相关的血液肿瘤（AHN）。Avapritinib是一种靶向KIT D816V的选择性酪氨酸激酶抑制剂，在1期EXPLORER（NCT02561988）和2期PATHFINDER（NCT03580655）研究中显示出强大的活性，可将临床转化为持久反应。我们报告了三名接受阿伐普利替尼治疗的AdvSM AHN患者，他们获得了SM的完全缓解（CR），并成功桥接至异基因造血细胞移植（allo-HCT）。另外两个病例强调了AHN成分中克隆进化的风险，以及在靶向治疗时需要密切监测。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Current Research in Translational Medicine Biochemistry, Genetics and Molecular Biology-General Biochemistry,Genetics and Molecular Biology

CiteScore

7.00

自引率

4.90%

发文量

审稿时长

45 days

期刊介绍： Current Research in Translational Medicine is a peer-reviewed journal, publishing worldwide clinical and basic research in the field of hematology, immunology, infectiology, hematopoietic cell transplantation, and cellular and gene therapy. The journal considers for publication English-language editorials, original articles, reviews, and short reports including case-reports. Contributions are intended to draw attention to experimental medicine and translational research. Current Research in Translational Medicine periodically publishes thematic issues and is indexed in all major international databases (2017 Impact Factor is 1.9). Core areas covered in Current Research in Translational Medicine are: Hematology, Immunology, Infectiology, Hematopoietic, Cell Transplantation, Cellular and Gene Therapy.