Hematology, University Hospital Basel, Basel, Switzerland.
University Center for Immunology, University Hospital and Immunodeficiency Laboratory, Department of Biomedicine, University of Basel, Basel, Switzerland.
Anticancer Res. 2022 Sep;42(9):4505-4509. doi: 10.21873/anticanres.15952.
BACKGROUND/AIM: We report three adult patients with primary immunodeficiency (PID) treated with reduced-intensity allogenic hematopoietic cell transplantation (HCT) with fludarabine/treosulfan conditioning and graft-versus-host disease (GvHD) prophylaxis with alemtuzumab and a calcineurin inhibitor.
Patient 1, a 51-year-old male, had common variable immunodeficiency (CVID) with protein-losing enteropathy. Patient 2 was a 29-year-old woman with STAT3 (signal transducer and activator of transcription 3)-dependent hyper-IgE syndrome (HIES). Patient 3 was a 25-year-old male with XIAP (X-linked inhibitor of apoptosis)-deficiency presenting as treatment-refractory granulomatous enteropathy. Engraftment occurred in all three patients, with 100% donor chimerism in blood. Two patients survived, whereas the patient with CVID died due to infection.
This series highlights issues of transplantation for PID in adults and treosulfan-based conditioning, which is feasible for PID patients; infectious complications are the major issue of concern.
背景/目的：我们报告了三例原发性免疫缺陷（PID）成人患者，他们接受了氟达拉滨/替佐沙胺预处理的低强度同种异体造血细胞移植（HCT），并用阿仑单抗和钙调神经磷酸酶抑制剂预防移植物抗宿主病（GvHD）。
患者 1 为 51 岁男性，患有伴有蛋白丢失性肠病的普通可变免疫缺陷（CVID）。患者 2 为 29 岁女性，患有依赖于 STAT3（信号转导和转录激活因子 3）的高 IgE 综合征（HIES）。患者 3 为 25 岁男性，患有 XIAP（凋亡抑制因子 X 连锁）缺陷，表现为治疗抵抗性肉芽肿性肠炎。所有三名患者均发生了植入，血液中均有 100%的供体嵌合体。两名患者存活，而患有 CVID 的患者因感染而死亡。
本系列强调了成人 PID 患者的移植问题和基于替佐沙胺的预处理，这对于 PID 患者是可行的；感染并发症是主要关注的问题。
