MDS 移植调理方案当前面临的挑战。
Current challenges in conditioning regimens for MDS transplantation.
发表日期：2024 Jul 10
作者：
A B Notarantonio, M Robin, M D'Aveni
来源：
BLOOD REVIEWS
摘要：
骨髓增生异常综合征（MDS）是一种异质性很强的克隆性疾病。患有“高风险”MDS（由特定的复发性遗传异常定义）的患者预后较差，因为进展为化疗敏感性低的继发性急性髓系白血病的风险很高。同种异体造血干细胞移植仍然是唯一能够提供持久疾病控制的治疗方法，因为供体免疫系统允许移植物抗MDS效应。就移植前的准备步骤而言，通过增加预处理方案强度来靶向恶性克隆仍然是一个激烈争论的问题。 MDS 主要在老年患者中诊断，并且已报道与常见清髓预处理方案相关的高毒性。过去 20 年来，人们一直在努力将新药物纳入预处理方案，以在不增加毒性的情况下实现最佳的恶性克隆控制。我们总结了这些回顾性和前瞻性研究，并评估了现有证据的局限性，以描述理想的调理方案。版权所有 © 2024 作者。由爱思唯尔有限公司出版。保留所有权利。
Myelodysplastic syndrome (MDS) is a very heterogeneous clonal disorder. Patients with "higher-risk" MDS, defined by specific recurrent genetic abnormalities, have a poor prognosis because of a high risk of progression to secondary acute myeloid leukemia with low chemosensitivity. Allogeneic hematopoietic stem cell transplantation remains the only treatment that offers durable disease control because the donor immune system allows graft-versus-MDS effects. In terms of preparation steps before transplantation, targeting the malignant clone by increasing the conditioning regimen intensity is still a matter of intense debate. MDS is mainly diagnosed in older patients, and high toxicity related to common myeloablative conditioning regimens has been reported. Efforts to include new drugs in the conditioning regimen to achieve the best malignant clone control without increasing toxicity have been made over the past 20 years. We summarized these retrospective and prospective studies and evaluated the limitations of the available evidence to delineate the ideal conditioning regimen.Copyright © 2024 The Author(s). Published by Elsevier Ltd.. All rights reserved.