全身性肥大细胞增多症
肿瘤
医学
骨髓增生性肿瘤
造血
血液学
酪氨酸激酶抑制剂
肥大细胞
肿瘤科
内科学
癌症研究
免疫学
干细胞
病理
生物
骨髓
癌症
骨髓纤维化
遗传学
作者
Priya Sriskandarajah,Donal McLornan,Clare Oni,Alfred J. Wilson,Cheryl M. Woodley,Monika Ciesielska,Kavita Raj,Richard Dillon,Mark Ethell,Joseph Chacko,Kim Orchard,D. H. Radia
标识
DOI:10.1016/j.retram.2023.103398
摘要
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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