UO Ematologia e terapie cellulari, IRCCS Ospedale Policlinico San Martino, Genoa, Italy.
UO Ematologia e terapie cellulari, IRCCS Ospedale Policlinico San Martino, Genoa, Italy.
Handb Clin Neurol. 2024;202:117-134. doi: 10.1016/B978-0-323-90242-7.00012-2.
Autologous hematopoietic stem cell transplantation (aHSCT) is a complex process, designed to replace the blood and lymphoid systems of a patient with hematopoietic stem cells (HSCs) that have been previously collected and cryopreserved, derived from the same patient. The rationale of aHSCT in neurologic diseases is to eliminate self-reacting cell clones and induce self-tolerance through a profound renewal of the immune system. The steps analyzed in this chapter are conditioning, HSCs infusion, supportive care, and monitoring. Before transplantation, ablation of the hemato-lymphopoietic system is achieved with chemotherapy; this stage is known as the conditioning regimen. The EBMT guidelines support the use of "intermediate intensity" regimens, either cyclophosphamide 200mg/kg or BEAM (bis-chloroethyl-nitrosourea, etoposide, cytarabine, and melphalan), in combination with serotherapy that consists of rabbit antithymocyte globulin (ATG) in most protocols. The infusion of HSC is performed through a central intravenous line, after being thawed at 37°C using either a water bath or a heat bath; in this phase, the prevention and management of infusion-related adverse events are crucial. The supportive care consists mainly of infection prophylaxis and treatment, administration of blood product transfusions, and nutritional and electrolyte support. The monitoring phase is focused on hematologic recovery and monitoring for early and late complications of aHSCT.
自体造血干细胞移植(aHSCT)是一个复杂的过程,旨在通过先前采集并冷冻保存的、源自同一患者的造血干细胞(HSCs)来替代患者的血液和淋巴系统。aHSCT 在神经疾病中的原理是通过免疫系统的深度更新来消除自身反应性细胞克隆并诱导自身耐受。本章分析的步骤包括预处理、HSCs 输注、支持性护理和监测。在移植前,通过化疗实现造血淋巴系统的消融;这一阶段称为预处理方案。EBMT 指南支持使用“中度强度”方案,即环磷酰胺 200mg/kg 或 BEAM(双氯乙基亚硝脲、依托泊苷、阿糖胞苷和马法兰),结合大多数方案中的兔抗胸腺细胞球蛋白(ATG)血清疗法。HSC 通过中心静脉导管输注,在 37°C 下解冻,使用水浴或热浴;在这一阶段,预防和管理输注相关不良事件至关重要。支持性护理主要包括感染预防和治疗、血液制品输注、营养和电解质支持。监测阶段主要集中在造血恢复和监测 aHSCT 的早期和晚期并发症上。