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1
Primary myelofibrosis: 2013 update on diagnosis, risk-stratification, and management.原发性骨髓纤维化:2013 年诊断、风险分层和治疗更新。
Am J Hematol. 2013 Feb;88(2):141-50. doi: 10.1002/ajh.23384.
2
Myeloproliferative neoplasm (MPN) symptom assessment form total symptom score: prospective international assessment of an abbreviated symptom burden scoring system among patients with MPNs.骨髓增殖性肿瘤 (MPN) 症状评估表总症状评分:在 MPN 患者中前瞻性国际评估简化症状负担评分系统。
J Clin Oncol. 2012 Nov 20;30(33):4098-103. doi: 10.1200/JCO.2012.42.3863. Epub 2012 Oct 15.
3
Improving survival trends in primary myelofibrosis: an international study.原发性骨髓纤维化的生存趋势改善:一项国际研究。
J Clin Oncol. 2012 Aug 20;30(24):2981-7. doi: 10.1200/JCO.2012.42.0240. Epub 2012 Jul 23.
4
A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis.芦可替尼治疗骨髓纤维化的双盲、安慰剂对照试验。
N Engl J Med. 2012 Mar 1;366(9):799-807. doi: 10.1056/NEJMoa1110557.
5
JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis.芦可替尼与最佳可用疗法治疗骨髓纤维化的 JAK 抑制作用比较。
N Engl J Med. 2012 Mar 1;366(9):787-98. doi: 10.1056/NEJMoa1110556.
6
Safety and efficacy of everolimus, a mTOR inhibitor, as single agent in a phase 1/2 study in patients with myelofibrosis.在骨髓纤维化患者中进行的 1/2 期研究中,mTOR 抑制剂依维莫司作为单一药物的安全性和疗效。
Blood. 2011 Aug 25;118(8):2069-76. doi: 10.1182/blood-2011-01-330563. Epub 2011 Jul 1.
7
Safety and efficacy of TG101348, a selective JAK2 inhibitor, in myelofibrosis.选择性 JAK2 抑制剂 TG101348 治疗骨髓纤维化的安全性和疗效。
J Clin Oncol. 2011 Mar 1;29(7):789-96. doi: 10.1200/JCO.2010.32.8021. Epub 2011 Jan 10.
8
Myeloproliferative neoplasms: molecular pathophysiology, essential clinical understanding, and treatment strategies.骨髓增殖性肿瘤:分子病理生理学、必要的临床理解和治疗策略。
J Clin Oncol. 2011 Feb 10;29(5):573-82. doi: 10.1200/JCO.2010.29.8711. Epub 2011 Jan 10.
9
A phase-2 trial of low-dose pomalidomide in myelofibrosis.一项低剂量泊马度胺治疗骨髓纤维化的 2 期临床试验。
Leukemia. 2011 Feb;25(2):301-4. doi: 10.1038/leu.2010.254. Epub 2010 Nov 5.
10
Safety and efficacy of INCB018424, a JAK1 and JAK2 inhibitor, in myelofibrosis.INCB018424,一种 JAK1 和 JAK2 抑制剂,在骨髓纤维化中的安全性和疗效。
N Engl J Med. 2010 Sep 16;363(12):1117-27. doi: 10.1056/NEJMoa1002028.

修订后的骨髓纤维化反应标准:国际工作组-骨髓增生性肿瘤研究和治疗(IWG-MRT)和欧洲白血病网络(ELN)共识报告。

Revised response criteria for myelofibrosis: International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) and European LeukemiaNet (ELN) consensus report.

机构信息

Mayo Clinic, 200 First Street SW, Rochester, MN 55905, USA.

出版信息

Blood. 2013 Aug 22;122(8):1395-8. doi: 10.1182/blood-2013-03-488098. Epub 2013 Jul 9.

DOI:10.1182/blood-2013-03-488098
PMID:23838352
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC4828070/
Abstract

The current document is a revision of the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) criteria for treatment response in myelofibrosis (MF) and represents a collaborative effort by the IWG-MRT and the European LeukemiaNet to objectively assess the value of new drugs in inducing morphologic remission or improvement in MF-associated symptomatic burden (MF-SB). Some of the changes in the current revision include stricter definitions of red cell transfusion dependency and independency and consideration of the Myeloproliferative Neoplasm Symptom Assessment Form as a tool to quantify meaningful changes in disease-related symptoms. Six response categories are listed: complete remission (CR) and partial remission signify treatment effects that are consistent with disease modification, whereas drug-induced improvements in MF-SB were annotated as clinical improvement, anemia response, spleen response, or symptoms response. Additional criteria are provided for progressive disease, stable disease, and relapse. The document also includes recommendations for assessing cytogenetic and molecular remissions, without mandating their inclusion for CR assignment.

摘要

本文件是国际骨髓增生性肿瘤研究与治疗工作组(IWG-MRT)修订的骨髓纤维化(MF)治疗反应标准,代表了 IWG-MRT 和欧洲白血病网络之间的合作努力,旨在客观评估新药在诱导形态缓解或改善 MF 相关症状负担(MF-SB)方面的价值。本次修订中的一些变化包括更严格的红细胞输血依赖和独立性定义,并考虑使用骨髓增生性肿瘤症状评估表作为量化疾病相关症状有意义变化的工具。列出了六个反应类别:完全缓解(CR)和部分缓解表示与疾病修正一致的治疗效果,而药物诱导的 MF-SB 改善则被标注为临床改善、贫血反应、脾脏反应或症状反应。对于进行性疾病、稳定疾病和复发,还提供了其他标准。本文档还包括评估细胞遗传学和分子缓解的建议,但不强制要求将其纳入 CR 分配。