BACKGROUND Familial hemophagocytic lymphohistiocytosis (FHLH) is a potentially fatal disorder of immune regulation. Management includes chemotherapy followed by hematopoietic stem cell transplantation (HSCT). T-cell depleted (TCD)- haploidentical HSCT could be an option for those patients who do not have HLA matching family donor. OBJECTIVE To report on the outcome of TCD- haploidentical HSCT in FHLH patients transplanted in Sultan Qaboos University Hospital (SQUH). SUBJECTS AND METHODS This is a retrospective report on 12 cases with FHLH who received TCD- haploidentical HSCT in SQUH between Aug. 2010 and Dec. 2018. Epidemiological characteristics and details on the transplantation procedures and complications were collected from electronic patients' records. RESULTS Twelve patients with FHLH received TCD-haploidentical after myeloablative conditioning regimen, composed of (treosulfan/ thiotepa/ fludarabine/ATG) and Rituximab. The mean age at transplantation was 11.67±8 months. All patients had perforin gene mutations, except one patient who had UNC-13D mutation. Most patients received TCRαβ+/CD19+ depleted grafts for faster immune reconstitution. Seven patients (58.3%) have been cured with a mean follow-up duration of 3.44 years. Four patients died due to multi-organ failure secondary to gram negative sepsis. One patient had primary graft failure (GF), and two patients had mild graft versus host disease. Two patients had PCP pneumonitis, two had adenoviremia, while nine patients had CMV viremia. Amongst patients with CMV viremia two had evidence of disease (retinitis, enteritis). All patients with CMV viremia were treated successfully with foscarnet pre engraftment and ganciclovir post engraftment respectively. CONCLUSION TCD- haploidentical HSCT could be a viable option for patients with FHLH who do not have HLA-matching family donors. Infectious complications are the leading cause of death in that setting. CMV virmeia was the most frequently encountered infectious complication.

中文翻译：

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对于没有相配家庭供体的家族性吞噬性淋巴细胞组织细胞增多症患者，T细胞耗竭的单倍型造血干细胞移植：在阿曼的经验。

背景技术家族性噬血细胞淋巴组织细胞增生症（FHLH）是免疫调节的潜在致命疾病。管理包括化学疗法后进行造血干细胞移植（HSCT）。对于那些没有HLA匹配家庭供体的患者，可以选择T细胞耗竭（TCD）-单倍体HSCT。目的报告在苏丹卡布斯大学医院（SQUH）移植的FHLH患者中TCD-单倍体HSCT的结果。研究对象和方法这是一项回顾性报告，回顾性分析了2010年8月至2018年12月在SQUH接受TCD-单倍体HSCT的12例FHLH病例。从电子病历中收集了流行病学特征，移植程序和并发症的详细信息。结果12名FHLH患者在接受清髓治疗后接受了TCD单相治疗，治疗方案由（硫代硫丹/硫替泰/氟达拉滨/ ATG）和利妥昔单抗组成。移植的平均年龄为11.67±8个月。除一名患有UNC-13D突变的患者外，所有患者均具有穿孔素基因突变。大多数患者接受了TCRαβ+ / CD19 +耗竭的移植物，以加快免疫重建速度。七名患者（58.3％）已经治愈，平均随访时间为3.44年。4例患者因继发于革兰氏阴性败血症的多器官衰竭而死亡。一名患者患有原发性移植失败（GF），两名患者患有轻度移植物抗宿主病。两名患者患有PCP肺炎，两名患有腺病毒血症，而九名患者患有CMV病毒血症。在CMV病毒血症患者中，有两个有疾病迹象（视网膜炎，肠炎）。所有的CMV病毒血症患者分别在植入前使用膦甲酸和植入后更昔洛韦治疗成功。结论对于没有HLA匹配家庭供体的FHLH患者，TCD单倍体HSCT可能是可行的选择。在这种情况下，感染性并发症是导致死亡的主要原因。CMV病毒血症是最常见的感染性并发症。