骨髓纤维化
Janus激酶2
医学
贾纳斯激酶
体质症状
骨髓增生性肿瘤
原发性血小板增多症
鲁索利替尼
真性红细胞增多症
内科学
骨髓
细胞因子
受体
疾病
作者
Kazuhiko Ikeda,Koki Ueda
出处
期刊:The Lancet
[Elsevier]
日期:2023-01-01
卷期号:401 (10373): 248-249
被引量:2
标识
DOI:10.1016/s0140-6736(23)00171-x
摘要
Myelofibrosis is a progressive chronic myeloproliferative neoplasm driven by Janus kinase and signal transducer and activator of transcription (JAK–STAT) pathway activation, mostly due to mutations in JAK2, MPL, and CALR. 1 Tefferi A Primary myelofibrosis: 2021 update on diagnosis, risk-stratification and management. Am J Hematol. 2021; 96: 145-162 Crossref PubMed Scopus (100) Google Scholar Myelofibrosis arises de novo (primary myelofibrosis) or secondarily from polycythaemia vera and essential thrombocythaemia. Myelofibrosis presents with constitutional symptoms, such as fever, night sweats, and fatigue, accompanied by splenomegaly and either cytopenias or cytoses. The most frequent cytopenia is anaemia, which correlates with a worse prognosis. 2 Passamonti F Giorgino T Mora B et al. A clinical-molecular prognostic model to predict survival in patients with post polycythemia vera and post essential thrombocythemia myelofibrosis. Leukemia. 2017; 31: 2726-2731 Crossref PubMed Scopus (175) Google Scholar , 3 Guglielmelli P Lasho TL Rotunno G et al. MIPSS70: mutation-enhanced international prognostic score system for transplantation-age patients with primary myelofibrosis. J Clin Oncol. 2018; 36: 310-318 Crossref PubMed Scopus (253) Google Scholar , 4 Reynolds SB Pettit K New approaches to tackle cytopenic myelofibrosis. Hematology Am Soc Hematol Educ Program. 2022; 2022: 235-244 Crossref PubMed Scopus (0) Google Scholar Momelotinib versus danazol in symptomatic patients with anaemia and myelofibrosis (MOMENTUM): results from an international, double-blind, randomised, controlled, phase 3 studyTreatment with momelotinib, compared with danazol, resulted in clinically significant improvements in myelofibrosis-associated symptoms, anaemia measures, and spleen response, with favourable safety. These findings support the future use of momelotinib as an effective treatment in patients with myelofibrosis, especially in those with anaemia. Full-Text PDF
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