Data_Sheet_1_Short- and long-term outcome of allogeneic stem cell transplantation in infants: A single-center experience over 20 years.PDF

IntroductionAllogeneic hematopoietic stem cell transplantation (allo-HSCT) is a treatment method for a wide range of malignant and non-malignant diseases. Infants constitute a distinct patient group, especially due to their organ immaturity and differences in drug metabolism. The present paper aims to analyse the short- and long-term outcomes after allo-HSCT in infants.
Material and methodsIn the study period, 67 patients under 12 months of age underwent allo-HSCT. This study is a retrospective analysis of patient medical records, in the form of paper and electronic documentation.
ResultsThe probability of 5-year OS was 69% and 72% in patients with malignant and non-malignant diseases, respectively. The allo-HSCT from a matched donor was associated with improved OS in comparison to haploidentical donor (0.8 vs. 0.58%, p = 0.0425). The overall incidence of acute graft-vs.-host disease (aGVHD) was 59.3%, and grade III–IV aGVHD was diagnosed in 23% of patients. The 100-day non-relapse mortality (NRM) in the study cohort was 17.9%, while the 5-year NRM was 26.9%. Among the causes of NRM, infections occurred in 83.3% of patients, and aGVHD in 16.3% of individuals. Twenty-two children (32.8%) required hospitalization in the pediatric intensive care unit (PICU). The median length of PICU hospitalization was 6 days (range 1 to 12 days). Late sequelae diagnosed during post-transplant surveillance included ocular disorders in 26.8% of patients, cardiac complications in 4.4%, as well as endocrinopathy with short stature (<3rd percentile) in 37.2% and overt hypothyroidism in 35.4%. In the long-term perspective, 83.3% of survivors were able to attend a regular school.
ConclusionsImprovements in unrelated donor availability, and better supportive care resulted in better outcomes. Management of infant allo-HSCT recipients requires the formation of multi-disciplinary specialist teams. In addition, the role of parental empowerment must be acknowledged; for example, in speech therapy and rehabilitation.

## 引言
异基因造血干细胞移植（Allogeneic Hematopoietic Stem Cell Transplantation，简称allo-HSCT）是一类可用于治疗多种恶性与非恶性疾病的治疗手段。婴儿是一类特殊的患者群体，这主要源于其器官发育未成熟以及药物代谢特征存在差异。本研究旨在分析婴儿接受异基因造血干细胞移植后的短期与长期转归。

## 材料与方法
研究周期内，共有67名年龄不足12个月的患者接受了异基因造血干细胞移植。本研究为回顾性分析，分析对象为纸质与电子形式留存的患者病历资料。

## 结果
恶性疾病患者与非恶性疾病患者的5年总生存期（Overall Survival，OS）率分别为69%与72%。与单倍体相合供者来源的移植相比，匹配供者来源的异基因造血干细胞移植可改善患者总生存期（0.8 vs. 0.58%，p = 0.0425）。急性移植物抗宿主病（acute graft-versus-host disease，aGVHD）的总体发生率为59.3%，其中23%的患者被诊断为Ⅲ~Ⅳ级急性移植物抗宿主病。本研究队列的100天非复发死亡率（Non-relapse Mortality，NRM）为17.9%，5年非复发死亡率为26.9%。非复发死亡的诱因中，感染占比83.3%，急性移植物抗宿主病占比16.3%。22名患儿（占比32.8%）需入住儿科重症监护室（Pediatric Intensive Care Unit，PICU）接受治疗。患儿入住儿科重症监护室的中位时长为6天（范围1~12天）。移植后随访期间确诊的晚期后遗症包括：眼部疾病（占比26.8%）、心脏并发症（占比4.4%），以及以身材矮小（低于第3百分位）为表现的内分泌疾病（占比37.2%）和显性甲状腺功能减退症（占比35.4%）。从长期随访结果来看，83.3%的存活患儿可正常就读普通学校。

## 结论
无关供者来源可及性的提升以及更完善的支持治疗，使患者转归得到改善。对婴儿异基因造血干细胞移植受者的诊疗管理，需要组建多学科专业团队。此外，必须重视家长赋能的作用，例如在言语治疗与康复训练领域。