在患有天生的免疫缺陷的儿童中进行血造干细胞移植:一个单一中心的经验
Özlem Arman Bilir1, Betül Karaatmaca2, İkbal Ok Bozkaya3
1Department of Pediatric Hematology Oncology & Bone Marrow Transplantation Unit, University of Health Sciences Ankara Bilkent City Hospital, Ankara, Turkey.
Scandinavian journal of immunology
|January 9, 2025
概括
造血干细胞移植 (HSCT) 为患有天生的免疫错误 (IEI) 的儿科患者提供了更好的生存率. 早期诊断和优化移植方案对于降低移植相关死亡率和提高患者生活质量至关重要.
科学领域:
- 免疫学 免疫学 免疫学
- 儿科血液学 儿科血液学
- 移植医学 移植医学
背景情况:
- 免疫的先天性错误 (IEI) 涵盖了一系列影响免疫系统的疾病.
- 造血干细胞移植 (HSCT) 是严重IEI的潜在治愈治疗方法.
- 在儿科IEI人群中HSCT的结果需要持续评估.
研究的目的:
- 以回顾分析经过HSCT的IEI儿科患者的结果.
- 评估不同IEI类别的整体存活率 (OS) 和移植相关死亡率 (TRM).
- 确定影响HSCT成功的因素.
主要方法:
- 在2011年至2023年期间接受HSCT的61名IEI儿科患者的回顾性分析.
- 将患者分为原发性免疫缺陷障碍 (PIDD),原发性免疫失调障碍 (PIRD) 和细胞数量或功能 (CDP) 的先天性缺陷.
- 评估生存率,TRM和随访数据.
主要成果:
- 总生存率 (OS) 为70%,随访时间中位数为51个月.
- 移植后100天的生存率为80%,整体移植相关死亡率 (TRM) 为29%.
- 根据IEI类别的TRM率有所不同:PIRD为42%,PIDD为22.5%,CDP为27%.
结论:
- 早期诊断和HSCT显著改善了儿科IEI患者的生存率.
- 为了减少TRM,需要不断改进HSCT协议.
- 改善IEI患者移植后的生活质量仍然是一个关键目标.
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