基于tandem调节的骨髓缩性HSCT用于患有耐火性骨髓性白血病的儿科患者
Jae Won Yoo1, Suejung Jo1, Seongkoo Kim2
1Division of Pediatric Hematology and Oncology, Department of Pediatrics, Catholic Hematology Hospital, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Republic of Korea.
基于协奏调节的骨髓衰变性造血干细胞移植 (HSCT) 对儿科耐火性骨髓性白血病有希望. 尽管存在严重的并发症,但这种方法在高风险患者中实现了良好的缓解和生存率.
科学领域:
- 血液学 血液学 血液学
- 儿科瘤学 儿科瘤学
- 干细胞移植 干细胞移植
背景情况:
- 全基性造血干细胞移植 (HSCT) 为患有耐火性髓性白血病的儿科患者提供了有限的存活率.
- 儿童中耐火性髓性白血病是一个重大的治疗挑战,需要新的治疗策略.
研究的目的:
- 为了评估基于双重调节的骨髓损伤性HSCT疗程的安全性和有效性.
- 为了评估患有耐火性髓性白血病的儿科患者接受这种密集的HSCT协议的结果.
主要方法:
- 对10名患有耐火性髓性白血病的儿科患者 (9例急性髓性白血病,1例青少年髓性单细胞白血病爆发期) 的回顾性分析.
- 患者接受了HSCT,然后进行了双重调节:FLAG (fludarabine,cytarabine,粒细胞殖民地刺激因子),其次是busulfan和melphalan.
- 所有患者在移植时处于非缓解状态.
主要成果:
- 中性粒细胞和血小板移植分别发生在10名和8名患者中.
- 在移植后的10名患者中,有9名患者实现了完全缓解 (CR).
- 估计的两年无白血病生存率为70.0±14.5%,具有显著的早期并发症,包括CMVDNAemia,静脉封闭性疾病和出血性囊炎.
结论:
- 基于串联调节的骨髓缩性HSCT在儿科耐火性髓性白血病中显示出令人鼓舞的缓解和存活率.
- 虽然并发症很明显,但该疗法在高风险人群中显示出改善结果的潜力.
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