异基因造血干细胞移植治疗儿童重型再生障碍性贫血相关并发症的临床研究

Clinical Study of Related Complications of Severe Aplastic Anemia Children Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

目的:分析异基因造血干细胞移植(allo-HSCT)治疗重型再生障碍性贫血(SAA)儿童后的并发症的临床特征。方法:分析2017.09~2023.05在新疆维吾尔自治区人民医院接受allo-HSCT的52名SAA儿童患者(≤14岁)的临床资料。结果:HID-HSCT和MSD-HSCT总生存率是没有统计学差异的(88.6% vs 100.0%,P = 0.154)。最常见的并发症是移植物抗宿主病(GVHD),分别有14例(26.9%)和9例(17.3%)患者发生II-IV度和III-IV度aGVHD,死亡的4例患者中有3例合并有IV度aGVHD,通过单因素分析没有发现III-IV度aGVHD的危险因素。累计有4 (8.1%)例患者发生cGVHD,广泛型和局限型GVHD各2例。其余的常见并发症包括原发性植入功能不良、可逆性后部脑病综合征和出血性膀胱炎分别有2例(3.8%),3例(5.8%)和7例(13.5%)。最常见的病毒感染是巨细胞病毒(CMV)感染,共12例(23.1%)患者,其中4例(7.8%)患者进展为CMV肠炎。我们发现II-IV度aGVHD (P = 0.001)和III-IV度aGVHD (P < 0.001)是CMV感染的危险因素。结论:allo-HSCT治疗儿童SAA有不错的疗效,但移植后相关并发症仍然是影响患者预后的主要因素之一。

Objective: To analyze the clinical features of SAA children undergoing allo-HSCT. Methods: The clinical data of 52 SAA children (≤14 years old) who received allo-HSCT in the People’s Hospital of Xinjiang Uygur Autonomous Region from September 2017 to May 2019 were analyzed. Results: There was no significant difference in overall survival between HID-HSCT and MSD-HSCT (88.6% vs 100.0%, P = 0.154). The most common complication was graft-versus-host disease (GVHD), with grade II-IV and III-IV aGVHD occurring in 14 patients (26.9%) and 9 patients (17.3%), respectively. Among the 4 patients who died, 3 patients had grade IV aGVHD. No risk factors for grade III-IV aGVHD were found by single factor analysis. There were 4 (8.1%) patients with cGVHD, 2 patients with extensive GVHD and 2 patients with localized GVHD. Other common complications included primary poor graft function, posterior reversible encephalopathy syndrome, PRES, and hemorrhagic cystitis had 2 (3.8%), 3 (5.8%), and 7 (13.5%) cases, respectively. The most common viral infection was cytomegalovirus (CMV) activation, with 12 patients (23.1%), of whom 4 patients (7.8%) progressed to CMV enteritis. We found that grade II-IV aGVHD (P = 0.001) and grade III-IV aGVHD (P < 0.001) were risk factors for CMV activation. Conclusion: Allo-HSCT has good efficacy in pediatric SAA, but post-transplant related complications remain one of the main factors affecting patient outcomes.