Facultad de Medicina, Universidad CES, Medellín, Colombia.
Department of Economics, Unversidad EAFIT, Medellín, Colombia.
Pediatr Transplant. 2020 Sep;24(6):e13774. doi: 10.1111/petr.13774. Epub 2020 Jul 17.
Major histocompatibility complex class II deficiency is a rare case of PID. Specific recommendations for hematopoietic stem cell transplant, the only curative treatment option, are still lacking. This meta-analysis aims to identify the factors associated with better prognosis in these patients. Thirteen articles reporting 63 patients with major histocompatibility complex class II deficiency that underwent hematopoietic stem cell transplant were included. The median age for hematopoietic stem cell transplant was 18 months. The most common source of transplant was bone marrow, with alternative sources as umbilical cord blood emerging during recent years. The highest proportion of engraftment was seen with umbilical cord. Engraftment was higher in patients with matched donors, with better overall survival in patients with reduced-intensity conditioning. Graft-vs-host disease developed in 65% of the patients, with grades I-II being the most frequently encountered. There was a higher mortality in patients with myeloablative conditioning and no engraftment. There was an inverse correlation between survival and stage of graft-vs-host disease. The main cause of mortality was infectious disease, mostly secondary to viral infections. Ideally, matched grafts should be used, and reduced-intensity conditioning should be considered to reduce early post-transplant complications. GVHD and viral prophylaxis are fundamental.
主要组织相容性复合体 II 类缺陷是一种罕见的 PID 病例。对于造血干细胞移植这一唯一的治疗选择，具体的推荐意见仍存在不足。本荟萃分析旨在确定与这些患者更好预后相关的因素。共纳入了 13 篇报道了 63 例接受造血干细胞移植的主要组织相容性复合体 II 类缺陷患者的文章。造血干细胞移植的中位年龄为 18 个月。最常见的移植来源是骨髓，近年来脐带血等替代来源也开始出现。脐带的植入率最高。与匹配供体相比，植入率更高，在接受减强度预处理的患者中总体生存率更好。65%的患者发生移植物抗宿主病，其中 I-II 级最常见。在接受清髓性预处理和未植入的患者中死亡率更高。存活与移植物抗宿主病的阶段呈反比关系。死亡的主要原因是传染病，主要继发于病毒感染。理想情况下，应使用匹配的移植物，并考虑采用减强度预处理以减少移植后早期并发症。GVHD 和病毒预防是基础。
