在SCID患者中,移植后晚期并发症随着时间的推移而增加:一次性免疫缺陷治疗联盟 (PIDTC) 的里程碑式研究
Hesham Eissa1, Monica S Thakar2, Ami J Shah3
1Division of Pediatric Hematology-Oncology-BMT, University of Colorado, Aurora, Wash.
The Journal of allergy and clinical immunology
|October 4, 2023
概括
针对严重联合免疫缺陷 (SCID) 的血造细胞移植 (HCT) 会导致严重的慢性和晚期影响 (CLE),影响存活率. 制定生存指南对于管理SCID HCT幸存者的长期结果至关重要.
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:
- 主要免疫缺陷治疗联盟 (PIDTC) 对美国和加拿大的儿童进行了血液细胞移植 (HCT) 的自然史研究.
- 严重综合免疫缺陷 (SCID) 是一种关键性疾病,需要HCT才能生存.
研究的目的:
- 调查HCT对SCID的结果.
- 在儿童SCID患者中评估HCT后的慢性和晚期影响 (CLE).
主要方法:
- 在1982年至2012年期间在32个PIDTC中心接受HCT的399名SCID患者的回顾性分析.
- 定义CLE为在HCT后2年之前存在的条件 (慢性) 或在2年后发展 (晚期).
- 资格要求HCT后至少2年生存,没有进一步的细胞疗法.
主要成果:
- 在HCT后15年,CLE的累积发病率达到41%,神经,神经发育和牙科问题是最常见的.
- 化疗条件与身高降低和内分泌/牙CLE增加有关.
- 任何CLE的存在显著增加了晚期死亡的风险 (HR 7.21).
结论:
- 在SCID HCT后的晚期发病率是显著的,并对整体存活率产生不利影响.
- 有证据支持为SCID HCT接受者量身定制的生存指南的制定.
- 长期监测和管理对于改善这些患者的治疗结果至关重要.
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