肝移植后的哈普洛干细胞移植治疗状细胞疾病与相关的肝功能障碍:一个案例系列
Ali D Alahmari1, Saad Alghamdi2, Reem Alasbali3
1Hematology, Stem cell transplant & Cellular therapy department, Cancer Centre of Excellence, King Faisal Specialist Hospital and Research Centre, Riyadh, 11211, Saudi Arabia. aalahmari7@kfshrc.edu.sa.
Communications medicine
|August 15, 2025
概括
双倍单双血型造血干细胞移植 (haplo-HCT) 与活体供体肝移植 (LDLT) 结合,为患有末期肝病的严重状细胞病 (SCD) 患者提供了一种可行的治疗方法.
科学领域:
- 血液学 血液学 血液学
- 移植免疫学 移植免疫学
- 肝病学 肝病学是一种肝病学.
背景情况:
- 状细胞疾病 (SCD) 导致严重的并发症,如中风和肝脏疾病,导致早期死亡.
- 造血干细胞移植 (HCT) 是治愈的,但有风险,特别是对于患有并发症的患者.
- 结合HCT和固体器官移植 (SOT) 可能有利于严重的SCD患者器官功能障碍.
研究的目的:
- 报告首次使用双重正体肝移植和单双相同的HCT (haplo-HCT) 在患有晚期肝硬化的严重SCD患者中.
- 评估这种综合方法的可行性和结果.
主要方法:
- 采用了双重正体肝移植,随后从相关的捐赠者获得了哈普洛-HCT.
- 采用非骨髓衰变调节方案和移植后环胺 (PTCy).
主要成果:
- 两名患者在Haplo-HCT后实现了稳定的肝脏全移植功能和完整的供体移植.
- 在这两种情况下,通过诱导免疫耐受性来实现成功的免疫抑制撤销.
结论:
- 双倍哈普洛-HCT和活体供体肝移植 (LDLT) 是符合条件的SCD患者具有末期肝病的可行治疗选择.
- 这种综合方法表明了复杂的SCD病例中治疗治疗的潜力.
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