Department of Pediatric Immunology and Allergy, Bahcelievler Medical Park Hospital, Istinye University School of Medicine, Istanbul, Turkey.
Department of Pediatric Hematology-Oncology, Bahcelievler Medical Park Hospital, Altinbas University School of Medicine, Istanbul, Turkey.
Pediatr Transplant. 2021 Nov;25(7):e14063. doi: 10.1111/petr.14063. Epub 2021 Jun 6.
Primary immunodeficiency diseases (PID) are characterized by the occurrence of frequent infections and are caused by many genetic defects. Hematopoietic stem cell transplantation (HSCT) is the only curative treatment option for the majority of PID. As a Pediatric Hematology-Oncology-Immunology Transplantation Unit, we wanted to present our HSCT experience regarding treatment of primary immunodeficiency diseases.
58 patients were included in the study between January 2014 and June 2019. We searched 9/10 or 10/10 matched-related donor (MRD) firstly, in the absence of fully matched-related donor. We screened matched unrelated donor (MUD) from donor banks. MRD was used in 24 (41.3%) patients, MUD in 20 (34.4%) patients, and haploidentical donors in 14 (24.1%) patients. Demographic data, HSCT characteristics, and outcome were evaluated. While 16 patients had severe combined immunodeficiency (SCID), the remaining was non-SCID.
Of the 58 patients, 38 were male and 20 were female. Median age at transplantation was 12 months (range: 2.5-172 months). Combined immunodeficiencies consisted 67.2% of patients. Mean follow-up time was 27 months (6 months-5 years). Median neutrophil, lymphocyte, and thrombocyte engraftment days were similar in comparison of both donor type and stem cell source. The most common complication was acute GvHD in 15 (25.8%) patients. In total, five patients (31%) belonging to the SCID group and 10 patients (23.8%) belonging to the non-SCID group died. Our total mortality rate was 15 (25.8%) in all patients.
We would like to present our HSCT experiences as a pediatric immunology transplantation center. Existing severe infections before transplantation period, BCGitis, and CMV are important issues of transplantation in Turkey. However, the follow-up time is shorter than some studies, our results regarding complications and survival are similar to previous reports.
原发性免疫缺陷病（PID）的特征是频繁发生感染，由许多遗传缺陷引起。造血干细胞移植（HSCT）是大多数 PID 的唯一治愈治疗选择。作为儿科血液学-肿瘤学-免疫学移植单位，我们想介绍我们在治疗原发性免疫缺陷病方面的 HSCT 经验。
2014 年 1 月至 2019 年 6 月期间，我们共纳入 58 例患者。我们首先寻找 9/10 或 10/10 匹配相关供体（MRD），在没有完全匹配相关供体的情况下。我们从供体库中筛选匹配的无关供体（MUD）。MRD 用于 24 例（41.3%）患者，MUD 用于 20 例（34.4%）患者，单倍体相合供体用于 14 例（24.1%）患者。评估了人口统计学数据、HSCT 特征和结果。16 例患者患有严重联合免疫缺陷（SCID），其余患者为非 SCID。
58 例患者中，男性 38 例，女性 20 例。移植时的中位年龄为 12 个月（范围：2.5-172 个月）。联合免疫缺陷占患者的 67.2%。中位中性粒细胞、淋巴细胞和血小板植入天数在比较两种供体类型和干细胞来源时相似。最常见的并发症是急性移植物抗宿主病（GVHD），共 15 例（25.8%）。总共，5 例（31%）SCID 组和 10 例（23.8%）非 SCID 组患者死亡。我们所有患者的总死亡率为 15 例（25.8%）。
我们想作为儿科免疫学移植中心介绍我们的 HSCT 经验。在土耳其，移植前存在严重感染、卡介苗（BCG）病和巨细胞病毒（CMV）是移植的重要问题。然而，随访时间比一些研究短，我们关于并发症和生存率的结果与之前的报告相似。
