Division of Leukemia, Department of Hematology and Hematopoietic Cell Transplantation, City of Hope Medical Center, Duarte, California, USA.
Department of Pathology, City of Hope Medical Center, Duarte, California, USA.
Eur J Haematol. 2023 Feb;110(2):213-216. doi: 10.1111/ejh.13894. Epub 2022 Nov 15.
Systemic mastocytosis (SM) results from clonal proliferation of neoplastic mast cells that infiltrate bone marrow and other organs. A major subset of patients with SM has a clonally related myeloid neoplasm and the SM itself (SM-AMN). We evaluated the efficacy of hypomethylating agent and venetoclax (HMA-VEN) to target both the myeloid neoplasm and mast cell infiltrate in a patient with SM associated with acute myeloid leukemia arising from myelodysplastic syndrome and illustrate complete elimination of bone marrow mast cells and complete remission of MDS/AML. This case illustrates the potent activity of HMA-VEN both against the AMN as well as the associated SM.
系统性肥大细胞增多症 (SM) 是由异常增殖的肥大细胞浸润骨髓和其他器官引起的克隆性疾病。SM 患者的一个主要亚组存在与 SM 本身相关的克隆性髓系肿瘤（SM-AMN）。我们评估了低甲基化药物联合维奈托克（HMA-VEN）对骨髓肥大细胞浸润和髓系肿瘤的疗效，该患者患有由骨髓增生异常综合征发展而来的伴发急性髓系白血病的 SM，并阐明了骨髓肥大细胞的完全消除和 MDS/AML 的完全缓解。该病例说明了 HMA-VEN 对 AMN 和相关 SM 均具有强大的活性。
