骨髓纤维化的治疗:新旧策略

IF 3 Q2 Medicine Clinical Medicine Insights-Blood Disorders Pub Date : 2017-03-08 eCollection Date: 2017-01-01 DOI:10.1177/1179545X17695233
Alessandra Iurlo, Daniele Cattaneo
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引用次数: 0

摘要

骨髓纤维化(MF)是一种BCR-ABL1阴性骨髓增生性肿瘤,主要表现为反应性骨髓纤维化、髓外造血、贫血、肝脾肿大、体征、白血病进展和生存期缩短。因此,这种恶性肿瘤仍然是治愈性治疗的孤儿;事实上,唯一能明确证明对疾病进展有影响的治疗方法是异体造血干细胞移植,但只有少数患者有资格接受这种强化治疗。然而,最近,JAK2突变的发现也促进了小分子JAK1/2抑制剂的开发,其中第一个抑制剂--鲁索利替尼(ruxolitinib)已在美国和欧洲获准用于治疗MF。在这篇文章中,我们报告了在早期临床前和临床试验中,以及随后在日常临床实践中,被证明对MF患者有效的新旧治疗策略,特别是关于贫血、脾肿大、铁超载和异基因干细胞移植等主题。
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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Treatment of Myelofibrosis: Old and New Strategies.

Myelofibrosis (MF) is a BCR-ABL1-negative myeloproliferative neoplasm that is mainly characterised by reactive bone marrow fibrosis, extramedullary haematopoiesis, anaemia, hepatosplenomegaly, constitutional symptoms, leukaemic progression, and shortened survival. As such, this malignancy is still orphan of curative treatments; indeed, the only treatment that has a clearly demonstrated impact on disease progression is allogeneic haematopoietic stem cell transplantation, but only a minority of patients are eligible for such intensive therapy. However, more recently, the discovery of JAK2 mutations has also led to the development of small-molecule JAK1/2 inhibitors, the first of which, ruxolitinib, has been approved for the treatment of MF in the United States and Europe. In this article, we report on old and new therapeutic strategies that proved effective in early preclinical and clinical trials, and subsequently in the daily clinical practice, for patients with MF, particularly concerning the topics of anaemia, splenomegaly, iron overload, and allogeneic stem cell transplantation.

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来源期刊
CiteScore
3.70
自引率
0.00%
发文量
0
审稿时长
8 weeks
期刊最新文献
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