Zhao X L, Zhang L N, Zhang X Y, Hao M Z, Chen S L, Wei J L, He Y, Han M Z, Jiang E L
State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China Tianjin Institutes of Health Science, Tianjin 301600, China.
Zhonghua Xue Ye Xue Za Zhi. 2024 Dec 14;45(12):1125-1128. doi: 10.3760/cma.cn121090-20240612-00221.
This study enrolled five patients with classic paroxysmal nocturnal hemoglobinuria (cPNH) who underwent allogeneic hematopoietic stem cell transplantation in our hospital from 2019 to 2023. All five patients were male, with a median age of 26 (range: 26-46) years. The median time from diagnosis to allo-HSCT was 5.5 (range: 3.6-18.0) years. The median PNH granulocyte clone size was 96.3% (ranged 90.0%-99.7%), and the median lactate dehydrogenase (LDH) level was 2 224 IU/L (8896-fold of the upper limit of normal). All patients were detected to have bone marrow hyperplasia by trephine biopsy. The stem cell source came from four haploidentical donors and one HLA-identical sibling donor. Among the five patients, three underwent myeloablative conditioning (MAC) and two underwent reduced-intensity conditioning (RIC) treatment. None of the patients experienced primary implantation failure. The neutrophil implantation time was 15 (range: 13-21) days, and the platelet implantation time was 24 (range: 13-60) days. The three patients developed grade II acute graft-versus-host disease (aGVHD). No patients developed grade Ⅲ/Ⅳ aGVHD. The two patients developed localized chronic GVHD (cGVHD), and no patients developed extensive cGVHD. PNH clones turned negative in all patients after 2 (range: 1-3) months of transplantation. At a median follow-up of 16 (range: 6-34) months, one patient died of relapse and infection, and the remaining four patients survived, of which two patients had discontinued all drugs.
本研究纳入了5例经典型阵发性睡眠性血红蛋白尿（cPNH）患者，他们于2019年至2023年在我院接受了异基因造血干细胞移植。所有5例患者均为男性，中位年龄为26岁（范围：26 - 46岁）。从诊断到异基因造血干细胞移植的中位时间为5.5年（范围：3.6 - 18.0年）。PNH粒细胞克隆大小的中位数为96.3%（范围90.0% - 99.7%），乳酸脱氢酶（LDH）水平的中位数为2224 IU/L（为正常上限的8896倍）。所有患者经骨髓活检均检测到骨髓增生。干细胞来源为4例单倍体相合供者和1例HLA全相合的同胞供者。5例患者中，3例接受了清髓性预处理（MAC），2例接受了减低强度预处理（RIC）治疗。所有患者均未发生原发性植入失败。中性粒细胞植入时间为15天（范围：13 - 21天），血小板植入时间为24天（范围：13 - 60天）。3例患者发生了Ⅱ级急性移植物抗宿主病（aGVHD）。无患者发生Ⅲ/Ⅳ级aGVHD。2例患者发生了局限性慢性移植物抗宿主病（cGVHD），无患者发生广泛性cGVHD。移植后2个月（范围：1 - 3个月）所有患者的PNH克隆均转为阴性。中位随访16个月（范围：6 - 34个月）时，1例患者死于复发和感染，其余4例患者存活，其中2例患者已停用所有药物。
