原发性噬血细胞性淋巴组织细胞增生症的造血干细胞移植
Haematopoietic Stem Cell Transplantation for Primary Haemophagocytic Lymphohistiocytosis.
作者信息
Lehmberg Kai, Moshous Despina, Booth Claire
机构信息
Division of Paediatric Stem Cell Transplantation and Immunology, University Medical Centre Hamburg Eppendorf, Hamburg, Germany.
Department of Immunohematology, Necker-Enfants Malades Hospital, APHP, and Imagine Institute, Inserm U 1163, Descartes University, Paris Sorbonne Cité, Paris, France.
出版信息
Front Pediatr. 2019 Oct 25;7:435. doi: 10.3389/fped.2019.00435. eCollection 2019.
Abstract
Haematopoietic stem cell transplantation currently remains the only curative treatment of primary forms of haemophagocytic lymphohistiocytosis (HLH). Rapid diagnosis, efficient primary treatment of hyperinflammation, and conditioning regimens tailored to this demanding condition have substantially improved prognosis in the past 40 years. However, refractory hyperinflammation, central nervous system (CNS) involvement, unavailability of matched donors, susceptibility to conditioning-related toxicities, and a high frequency of mixed chimaerism remain a challenge in a substantial proportion of patients. Gene therapeutic approaches for several genetic defects of primary HLH are being developed at pre-clinical and translational levels.
摘要
造血干细胞移植目前仍然是原发性噬血细胞性淋巴组织细胞增生症（HLH）唯一的治愈性治疗方法。在过去40年里，快速诊断、高效的高炎症初始治疗以及针对这种严苛病症量身定制的预处理方案已显著改善了预后。然而，难治性高炎症、中枢神经系统（CNS）受累、缺乏匹配的供体、对预处理相关毒性的易感性以及高频率的混合嵌合体现象在相当一部分患者中仍然是一项挑战。针对原发性HLH的几种基因缺陷的基因治疗方法正在临床前和转化层面进行研发。
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