Background:Haploidentical hematopoietic cell transplantations (haplo-HCTs) with post-transplant cyclophosphamide (PT-Cy) are standard practice, but complications causing morbidity and mortality are not well described.Methods:The aim of this prospective non-interventional multicenter study was to document frequency of potential non-infectious and infection-related complications and main transplant outcomes after the first unmanipulated haplo-HCT with PT-Cy between 2017 and 2019 in 129 adult patients with hematological malignancies. The median follow-up was 37.3 months [95% CI: 34.3–39.7].Results:The cumulative incidence (CI) of acute graft versus host disease (aGvHD) at day +100 was 22.4% grade II-IV [95% CI: 15.5–30.1] and 8.8% grade III-IV [95% CI: 4.6–14.6], respectively. The cumulative incidence of chronic GvHD (cGvHD) at 24 months was 25.8% [95% CI: 18.5–33.6]; extensive cGvHD was 10.9% [95% CI: 6.3–17.1], respectively. The most frequent non-infectious complications for the whole study population were mucositis—37.5% (n= 48); renal insufficiency—18% (n= 23); and cardiovascular complications—10.9% (n= 14). The following infection-related complications were diagnosed: bacterial in 84 (65.1%), viral in 66 (51.6%), and fungal in 24 (18.6%) recipients. Two-year OS was 58.1% [95% CI: 50.2–67.3]; NRM—27.1% [95% CI: 19.7–35]; PFS—50.4% [95% CI: 42.5–59.8]; and GRFS—38.8% [95% CI: 31.2–48.1]. About 50% of all deaths were directly caused by infection or infection-related conditions.Conclusions:Disease remission status at transplant significantly affected PFS, chronic GvHD, and GRFS. Although clinical applications of haplo-HCT with PTCy are widespread, the study confirms the need to reduce infection-related mortality after this type of GvHD prophylaxis.
背景：采用移植后环磷酰胺（PT-Cy）的单倍体相合造血细胞移植（haplo-HCT）已成为标准治疗方案，但其导致发病率和死亡率的并发症尚未得到充分描述。方法：本前瞻性非干预性多中心研究旨在记录2017年至2019年间129例血液系统恶性肿瘤成年患者首次接受未经处理的haplo-HCT联合PT-Cy治疗后，潜在非感染性和感染相关并发症的发生频率及主要移植结局。中位随访时间为37.3个月[95% CI: 34.3–39.7]。结果：移植后第+100天急性移植物抗宿主病（aGvHD）的累积发生率（CI）分别为：II-IV级22.4% [95% CI: 15.5–30.1]和III-IV级8.8% [95% CI: 4.6–14.6]。24个月时慢性GvHD（cGvHD）的累积发生率为25.8% [95% CI: 18.5–33.6]，广泛型cGvHD为10.9% [95% CI: 6.3–17.1]。全研究人群中最常见的非感染性并发症为黏膜炎（37.5%，n=48）、肾功能不全（18%，n=23）和心血管并发症（10.9%，n=14）。感染相关并发症诊断情况为：细菌感染84例（65.1%）、病毒感染66例（51.6%）、真菌感染24例（18.6%）。两年总生存率（OS）为58.1% [95% CI: 50.2–67.3]；非复发死亡率（NRM）为27.1% [95% CI: 19.7–35]；无进展生存率（PFS）为50.4% [95% CI: 42.5–59.8]；无移植物抗宿主病/无复发生存率（GRFS）为38.8% [95% CI: 31.2–48.1]。约50%的死亡病例直接由感染或感染相关病症导致。结论：移植时疾病缓解状态显著影响PFS、慢性GvHD和GRFS。尽管haplo-HCT联合PTCy的临床应用广泛，本研究证实需要降低此类GvHD预防方案后的感染相关死亡率。
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