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我如何治疗有移植适应证的骨髓纤维化患者。

How I treat transplant-eligible patients with myelofibrosis.

机构信息

Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.

出版信息

Blood. 2023 Nov 16;142(20):1683-1696. doi: 10.1182/blood.2023021218.

Abstract

Despite the approval of Janus kinase inhibitors and novel agents for patients with myelofibrosis (MF), disease-modifying responses remain limited, and hematopoietic stem cell transplantation (HSCT) remains the only potentially curative treatment option. The number of HSCTs for MF continues to increase worldwide, but its inherent therapy-related morbidity and mortality limit its use for many patients. Furthermore, patients with MF often present at an older age, with cytopenia, splenomegaly, and severe bone marrow fibrosis, posing challenges in managing them throughout the HSCT procedure. Although implementation of molecular analyses enabled improved understanding of disease mechanisms and subsequently sparked development of novel drugs with promising activity, prospective trials in the HSCT setting are often lacking, making an evidence-based decision process particularly difficult. To illustrate how we approach patients with MF with respect to HSCT, we present 3 different clinical scenarios to capture relevant aspects that influence our decision making regarding indication for, or against, HSCT. We describe how we perform HSCT according to different risk categories and, furthermore, discuss our up-to-date approach to reduce transplant-related complications. Last, we show how to harness graft-versus-MF effects, particularly in the posttransplant period to achieve the best possible outcomes for patients.

摘要

尽管已经批准了 Janus 激酶抑制剂和新型药物用于骨髓纤维化 (MF) 患者,但疾病缓解反应仍然有限,造血干细胞移植 (HSCT) 仍然是唯一潜在的治愈性治疗选择。全球范围内接受 HSCT 治疗的 MF 患者数量持续增加,但该治疗方法固有的治疗相关发病率和死亡率限制了其在许多患者中的应用。此外,MF 患者通常年龄较大,伴有细胞减少症、脾肿大和严重的骨髓纤维化,这给 HSCT 过程中的管理带来了挑战。尽管实施分子分析使我们能够更好地了解疾病机制,并随后开发出具有良好疗效的新型药物,但 HSCT 环境中的前瞻性试验往往缺乏,使得基于证据的决策过程特别困难。为了说明我们如何在 HSCT 方面处理 MF 患者,我们提出了 3 种不同的临床情况,以捕捉影响我们对 HSCT 适应证或禁忌证决策的相关方面。我们描述了如何根据不同的风险类别进行 HSCT,并进一步讨论了我们目前降低移植相关并发症的方法。最后,我们展示了如何利用移植物抗 MF 效应,特别是在移植后阶段,为患者实现最佳治疗效果。

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