Indication and management of allogeneic haematopoietic stem-cell transplantation in myelofibrosis: updated recommendations by the EBMT/ELN International Working Group

Nicolaus Kröger, Andrea Bacigalupo, Tiziano Barbui, Markus Ditschkowski, Nico Gagelmann, Martin Griesshammer, Vikas Gupta, Nada Hamad, Claire Harrison, Juan Carlos Hernandez-Boluda, Steffen Koschmieder, Tania Jain, John Mascarenhas, Ruben Mesa, Uday R Popat, Francesco Passamonti, Nicola Polverelli, Alessandro Rambaldi, Marie Robin, Rachel B Salit, Giovanni Barosi
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Abstract

New options for medical therapy and risk scoring systems containing molecular data are leading to increased complexity in the management of patients with myelofibrosis. To inform patients’ optimal care, we updated the 2015 guidelines on indications for and management of allogeneic haematopoietic stem-cell transplantation (HSCT) with the support of the European Society for Blood and Marrow Transplantation (EBMT) and European LeukemiaNet (ELN). New recommendations were produced using a consensus-building methodology after a comprehensive review of articles released from January, 2015 to December, 2022. Seven domains and 18 key questions were selected through a series of questionnaires using a Delphi process. Key recommendations in this update include: patients with primary myelofibrosis and an intermediate-2 or high-risk Dynamic International Prognostic Scoring System score, or a high-risk Mutation-Enhanced International Prognostic Score Systems (MIPSS70 or MIPSS70-plus) score, or a low-risk or intermediate-risk Myelofibrosis Transplant Scoring System score should be considered candidates for allogeneic HSCT. All patients who are candidates for allogeneic HSCT with splenomegaly greater than 5 cm below the left costal margin or splenomegaly-related symptoms should receive a spleen-directed treatment, ideally with a JAK-inhibitor; HLA-matched sibling donors remain the preferred donor source to date. Reduced intensity conditioning and myeloablative conditioning are both valid options for patients with myelofibrosis. Regular post-transplantation driver mutation monitoring is recommended to detect and treat early relapse with donor lymphocyte infusion. In a disease where evidence-based guidance is scarce, these recommendations might help clinicians and patients in shared decision making.

同种异体造血干细胞移植治疗骨髓纤维化的适应症和管理:EBMT/ELN国际工作组的最新建议
医学治疗的新选择和包含分子数据的风险评分系统正在导致骨髓纤维化患者管理的复杂性增加。为了告知患者最佳护理,我们在欧洲血液和骨髓移植协会(EBMT)和欧洲白血病网(ELN)的支持下更新了2015年同种异体造血干细胞移植(HSCT)的适应症和管理指南。在对2015年1月至2022年12月发布的文章进行全面审查后,采用建立共识的方法提出了新的建议。采用德尔菲法,选取了7个领域和18个关键问题。本次更新的主要建议包括:原发性骨髓纤维化患者和中-2或高风险动态国际预后评分系统评分,或高风险突变增强国际预后评分系统(MIPSS70或MIPSS70 +)评分,或低风险或中风险骨髓纤维化移植评分系统评分,应考虑进行同种异体HSCT的候选人。所有脾肿大在左肋缘以下大于5cm或脾肿大相关症状的同种异体造血干细胞移植候选患者应接受脾定向治疗,理想情况下使用jak抑制剂;迄今为止,hla匹配的兄弟姐妹供体仍然是首选的供体来源。降低强度调节和清髓调节都是骨髓纤维化患者的有效选择。建议定期监测移植后驱动突变,以发现和治疗供体淋巴细胞输注的早期复发。在缺乏循证指导的疾病中,这些建议可能有助于临床医生和患者共同决策。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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