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骨髓纤维化与异基因移植:关键点与挑战

Myelofibrosis and allogeneic transplantation: critical points and challenges.

作者信息

Ranalli Paola, Natale Annalisa, Guardalupi Francesco, Santarone Stella, Cantò Chiara, La Barba Gaetano, Di Ianni Mauro

机构信息

Hematology Unit, Pescara Hospital, Pescara, Italy.

Department of Medicine and Aging Sciences, University of Chieti-Pescara, Chieti, Italy.

出版信息

Front Oncol. 2024 Jun 20;14:1396435. doi: 10.3389/fonc.2024.1396435. eCollection 2024.

Abstract

New available drugs allow better control of systemic symptoms associated with myelofibrosis (MF) and splenomegaly but they do not modify the natural history of progressive and poor prognosis disease. Thus, hematopoietic stem cell transplantation (HSCT) is still considered the only available curative treatment for patients with MF. Despite the increasing number of procedures worldwide in recent years, HSCT for MF patients remains challenging. An increasingly complex network of the patient, disease, and transplant-related factors should be considered to understand the need for and the benefits of the procedure. Unfortunately, prospective trials are often lacking in this setting, making an evidence-based decision process particularly arduous. In the present review, we will analyze the main controversial points of allogeneic transplantation in MF, that is, the development of more sophisticated models for the identification of eligible patients; the need for tools offering a more precise definition of expected outcomes combining comorbidity assessment and factors related to the procedure; the decision-making process about the best transplantation time; the evaluation of the most appropriate platform for curative treatment; the impact of splenomegaly; and splenectomy on outcomes.

摘要

新出现的可用药物能更好地控制与骨髓纤维化(MF)和脾肿大相关的全身症状,但它们并不能改变这种进行性且预后不良疾病的自然病程。因此,造血干细胞移植(HSCT)仍然被认为是MF患者唯一可用的治愈性治疗方法。尽管近年来全球范围内该手术的数量不断增加,但对MF患者进行HSCT仍然具有挑战性。需要考虑一个由患者、疾病和移植相关因素组成的日益复杂的网络,以理解该手术的必要性和益处。不幸的是,在这种情况下往往缺乏前瞻性试验,这使得基于证据的决策过程格外艰巨。在本综述中,我们将分析MF异基因移植的主要争议点,即开发更复杂的模型以识别 eligible 患者;需要能结合合并症评估和与手术相关因素更精确地定义预期结果的工具;关于最佳移植时间的决策过程;对治愈性治疗最合适平台的评估;脾肿大的影响;以及脾切除术对结果的影响。 (注:eligible 一词原文未翻译完整,可能是有遗漏信息,正常翻译为“符合条件的” )

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