在患有获得性低细胞骨髓衰竭的儿科患者中进行 Haploidentical 血造干细胞移植
Victor Quintero1, David Bueno-Sánchez1, Yasmina Mozo-Del-Castillo1
1Paediatric Haeamatology and Oncology Department, La Paz University Hospital, Madrid, Spain.
Transplantation and cellular therapy
|July 16, 2023
概括
顺序相同的干细胞移植为没有匹配供体的骨髓衰竭儿童提供了治疗方法. 虽然成功,但这些移植具有显著的毒性,包括移植相关的微血管病变.
科学领域:
- 儿科血液学 儿科血液学
- 干细胞移植 干细胞移植
- 骨髓衰竭综合征 骨髓衰竭综合征
背景情况:
- 在儿童中,未知原因的获得性低细胞骨髓衰竭 (AHBMF) 通常被诊断为严重的无形成性贫血 (SAA) 或儿童耐火性细胞衰减 (RCC).
- 缺乏匹配供体的AHBMF患者,并且不耐免疫抑制疗法 (IST) 需要其他治疗选择.
研究的目的:
- 评估 haploidentical 造血干细胞移植 (haplo-HSCT) 作为 AHBMF 的儿科患者的救援疗法的疗效和安全性.
- 分析接受SAA和RCC的Haplo-HSCT儿童的结果和并发症.
主要方法:
- 一个多中心的西班牙研究涉及11名儿科患者 (9个SAA,2个RCC) 接受了Haplo-HSCT.
- 患者接受了不同的淋巴缺血策略,SAA的强度降低和RCC的髓损伤.
- 低剂量放射治疗被列入SAA患者的条件.
主要成果:
- 所有患者都实现了移植.
- 病毒再激活 (8/11) 和急性GVHD (5/11) 是常见的. 慢性GVHD发生在4名长期幸存者中.
- 移植相关的微血管病变是SAA患者经常出现的并发症,影响了结果. 总生存率为81%,随访时间中位数为36个月.
结论:
- 哈普洛-HSCT是一种可行的治疗选择,适用于IST耐药的儿科AHBMF患者.
- 显著的毒性,特别是移植相关的微血管病变,需要仔细管理,以改善结果.
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