Long-term outcome and evaluation of organ function in pediatric patients undergoing haploidentical and matched related hematopoietic cell transplantation for sickle cell disease.
Long-term outcome and evaluation of organ function in pediatric patients undergoing haploidentical and matched related hematopoietic cell transplantation for sickle cell disease.
复制标题
接受单倍体相合和匹配相关造血细胞移植治疗镰状细胞病的儿科患者的长期结果和器官功能评估。
DOI：
10.1016/j.bbmt.2013.02.010
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发表时间：
2013-05
期刊：
影响因子：
--
通讯作者：
Leung W
中科院分区：
文献类型：
--
作者：
Dallas MH;Triplett B;Shook DR;Hartford C;Srinivasan A;Laver J;Ware R;Leung W
HL A血型相合的亲缘供者(MRD)造血干细胞移植(HSCT)是治疗镰状细胞病(SCD)的一种成熟的治疗方法；然而，在SCD的HSCT中使用包括半相合供者在内的替代供者的经验有限。我们报告了在圣犹大儿童研究医院接受亲缘供者HSCT治疗SCD的22名儿童患者的长期结果，无论是清髓性同胞MRD HSCT(n=14)还是降低强度的父母半相合供者HSCT(n=8)。MRD供者的平均年龄为11.0±3.9岁，半相合供者为9.0±5.0岁。MRD组的中位随访期为9.0±2.3年，总存活率为93%，复发/移植失败率为0%；单倍体相合供者组的中位随访期为7.4±2.4年，总存活率为75%，无病存活率为38%，疾病复发率为38%。我们报告了接受MRD或半相合供者HSCT治疗严重SCD的患者的长期血液学反应和器官功能。我们的数据显示了持续植入的HSCT后的长期血液学改善，并证实HSCT对SCD的常见并发症提供了长期的保护，包括中风、肺动脉高压、急性胸部和肾病，无论供体来源如何。
HLA-matched related donor (MRD) hematopoietic stem cell transplantation (HSCT) is a well-established therapy for patients with sickle cell disease (SCD); however, experience using alternative donors, including haploidentical donors, in HSCT for SCD is limited. We report the long-term outcomes of 22 pediatric patients who underwent related donor HSCT for SCD at St. Jude Children’s Research Hospital, either a myeloablative sibling MRD HSCT (n = 14) or reduced-intensity parental haploidentical donor HSCT (n = 8). The median patient age was 11.0 ± 3.9 years in the MRD graft recipients and 9.0 ± 5.0 years in the haploidentical donor graft recipients. The median follow-up was 9.0 ± 2.3 years, with an overall survival (OS) of 93% and a recurrence/graft failure rate of 0%, for the MRD cohort and 7.4 ± 2.4 years, with an OS of 75%, disease-free survival of 38%, and disease recurrence of 38%, for the haploidentical donor cohort. We report the long-term hematologic response and organ function in patients undergoing MRD or haploidentical donor HSCT for severe SCD. Our data demonstrate long-term hematologic improvements after HSCT with sustained engraftment, and confirm that HSCT offers long-term protection from common complications of SCD, including stroke, pulmonary hypertension, acute chest, and nephropathy, regardless of donor source.