Savaşan Süreyya, Özdemir Öner, Gadgeel Manisha
Pediatric Hematology/Oncology/Bone Marrow Transplantation, Children's Hospital of Michigan, Detroit, USA.
Pediatrics, Central Michigan University, Mt. Pleasant, USA.
Cureus. 2023 Sep 8;15(9):e44919. doi: 10.7759/cureus.44919. eCollection 2023 Sep.
Hematopoietic stem cells (HSCs) give rise to mast cells (MCs), and a relative increase in bone marrow (BM) MC is common in various BM failure (BMF) conditions. Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal HSC disorder, frequently associated with BMF, characterized by decreased expression of glycosylphosphatidylinositol membrane anchor for complement regulatory proteins. Eculizumab, a monoclonal antibody that blocks complement factor 5, successfully controls PNH symptoms. In this study, we, for the first time, demonstrated PNH-type culture-grown BM MC (c-BMMC) using flow cytometry in two BMF patients and monitored population size during eculizumab therapy. Further research may unravel the properties of PNH-type c-BMMC.
造血干细胞（HSCs）可分化为肥大细胞（MCs），在各种骨髓衰竭（BMF）病症中，骨髓（BM）MC相对增多的情况较为常见。阵发性睡眠性血红蛋白尿症（PNH）是一种获得性克隆性造血干细胞疾病，常与BMF相关，其特征为补体调节蛋白的糖基磷脂酰肌醇膜锚定物表达降低。依库珠单抗是一种阻断补体因子5的单克隆抗体，可成功控制PNH症状。在本研究中，我们首次利用流式细胞术在两名BMF患者中证实了PNH型培养生长的BM MC（c-BMMC），并在依库珠单抗治疗期间监测其群体大小。进一步的研究可能会揭示PNH型c-BMMC的特性。
