关键词： chronic granulomatous disease immune deficiencies pediatric allogeneic transplant reduced-intensity conditioning

Mesh ： Alemtuzumab / administration & dosage Child Child, Preschool Follow-Up Studies Graft vs Host Disease / prevention & control Granulomatous Disease, Chronic / pathology therapy Hematopoietic Stem Cell Transplantation / methods Humans Infant Melphalan / administration & dosage Myeloablative Agonists / administration & dosage Prognosis Prospective Studies Thiotepa / administration & dosage Transplantation Conditioning / methods Vidarabine / administration & dosage analogs & derivatives

来  源：   DOI：10.1002/pbc.28030   PDF(Sci-hub)

Abstract：
Chronic granulomatous disease (CGD) is an immune deficiency characterized by defective neutrophil function and increased risk of life-threatening infections. Allogeneic hematopoietic cell transplantation is curative for CGD, and conditioning regimen impacts transplant-related outcomes. We report a single-center prospective study (NCT01821781) of four patients with CGD transplanted using a reduced-intensity conditioning regimen (RIC) containing alemtuzumab, fludarabine, melphalan, and thiotepa. Patients had early immune reconstitution with low incidence of infections. Disease-free survival was 75% at a median of five years after transplant. This RIC regimen presents an alternative approach for transplant of patients with CGD who may not tolerate busulfan-based conditioning.
摘要：
慢性肉芽肿病（CGD）是一种免疫缺陷，其特征是中性粒细胞功能缺陷和危及生命的感染风险增加。异基因造血细胞移植治疗CGD,和条件治疗方案影响移植相关结果。我们报告了一项单中心前瞻性研究（NCT01821781），使用包含阿仑珠单抗的低强度预处理方案（RIC）移植了四名CGD患者，氟达拉滨,melphalan,还有Thiotepa.患者早期免疫重建，感染发生率低。移植后5年的中位无病生存率为75%。此RIC方案为可能不耐受白消安基础的CGD患者的移植提供了一种替代方法。
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