用于骨髓衰竭综合征的脐血移植：现状
Cord blood transplantation for bone marrow failure syndromes: state of art.
作者信息
Pagliuca Simona, Ruggeri Annalisa, Peffault de Latour Régis
机构信息
Hematology and Transplantation Unit, Saint Louis Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.
Department of Pediatric Hematology and Oncology, IRCCS Ospedale Pediatrico Bambino Gesù, Roma, Italy.
出版信息
Stem Cell Investig. 2019 Dec 5;6:39. doi: 10.21037/sci.2019.10.04. eCollection 2019.
Abstract
Hematopoietic stem cell transplantation (HSCT) and immunosuppressive therapy (IST) represent the milestones of the treatment algorithm for idiopathic and inherited bone marrow failure (BMF) disorders. However, patients lacking a suitable donor or failing IST still have a poor prognosis. Cord blood transplantation (CBT) has extended the possibility of HSCT for many patients in case of the absence of an eligible donor, and although in the last years, this procedure is less used in several hematological diseases, it remains an option for the treatment of patients with BMF syndromes. Nevertheless, optimization of conditioning regimen and cord blood unit selection is warranted to reduce the risk of graft failure and transplant-related mortality. This review summarizes the state of art of CBT in the field of BMF diseases, focusing on historical and recent issues in idiopathic aplastic anemia and inherited disorders.
摘要
造血干细胞移植（HSCT）和免疫抑制治疗（IST）是特发性和遗传性骨髓衰竭（BMF）疾病治疗方案中的里程碑。然而，缺乏合适供体或IST治疗失败的患者预后仍然很差。在没有合适供体的情况下，脐血移植（CBT）为许多患者提供了进行HSCT的可能性，尽管在过去几年中，该方法在几种血液系统疾病中的应用较少，但它仍然是治疗BMF综合征患者的一种选择。尽管如此，仍有必要优化预处理方案和脐血单位的选择，以降低移植物失败和移植相关死亡率的风险。本综述总结了BMF疾病领域中CBT的现状，重点关注特发性再生障碍性贫血和遗传性疾病的历史和近期问题。
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