修订国际工作组 - 欧洲白血病网骨髓纤维化贫血反应标准的提案。
Proposals for Revised International Working Group-European LeukemiaNet Criteria for Anemia Response in Myelofibrosis.
发表日期:2024 Aug 08
作者:
Ayalew Tefferi, Giovanni Barosi, Francesco Passamonti, Juan Carlos Hernandez-Boluda, Prithviraj Bose, Konstanze Döhner, Martin Ellis, Naseema Gangat, Jacqueline S Garcia, Heinz Gisslinger, Jason Gotlib, Paola Guglielmelli, Vikas Gupta, Claire N Harrison, Elizabeth O Hexner, Gabriela S Hobbs, Jean-Jacques Kiladjian, Steffen Koschmieder, Nicolaus Kröger, Andrew T Kuykendall, Giuseppe Gaetano Loscocco, John O Mascarenhas, Lucia Masarova, Ruben Mesa, Barbara Mora, Olatoyosi Odenike, Stephen T Oh, Animesh D Pardanani, Anand A Patel, Naveen Pemmaraju, Alessandro Rambaldi, Raajit K Rampal, Shireen Sirhan, Natasha Szuber, Moshe Talpaz, Pankit Vachhani, Alessandro M Vannucchi, Tiziano Barbui
来源:
BLOOD
摘要:
随着骨髓纤维化(MF)新药的不断涌现,用于定义贫血严重程度和治疗反应的强大而协调的框架将加强临床研究并促进研究间比较。因此,2013 年版国际工作组 - 欧洲白血病网 (IWG-ELN) MF 反应标准的主要作者被召集修改他们的文件,目的是 i) 在确定血红蛋白水平的资格时考虑性别差异标准,ii) 根据当前限制性输血实践修改输血依赖性贫血 (TDA) 的定义,以及 iii) 提供结构简单且易于应用的反应标准,该标准足够灵敏,可以检测功效信号(轻微反应),并考虑到主要回应。 2024 年 IWG-ELN 拟议标准的初稿随后在更广泛的国际专家群体中分发,并采纳了他们的反馈。拟议的文章包括 TDA 的新定义(研究入组前 12 周内≥3 个单位)和资格标准的血红蛋白阈值(女性 <10 g/dL,男性 <11 g/dL)。修订后的文件还提供了单独的(TDA 与非 TDA)和分级(主要反应与次要反应)反应标准,同时保留了 12 周筛查和治疗观察期的要求。版权所有 © 2024 美国血液学会。
With emerging new drugs in myelofibrosis (MF), a robust and harmonized framework for defining the severity of anemia and response to treatment will enhance clinical investigation and facilitate inter-study comparisons. Accordingly, the lead authors on the 2013 edition of the International Working Group-European LeukemiaNet (IWG-ELN) response criteria in MF were summoned to revise their document with the intent to i) account for gender-specific differences in determining hemoglobin levels for eligibility criteria, ii) revise definition of transfusion-dependent anemia (TDA) based on current restrictive transfusion practices, and iii) provide a structurally simple and easy to apply response criteria that are sensitive enough to detect efficacy signals (minor response) and also account for major responses. The initial draft of the 2024 IWG-ELN proposed criteria was subsequently circulated around a wider group of international experts and their feedback incorporated. The proposed articles include new definitions for TDA (≥3 units in the 12 weeks prior to study enrollment) and hemoglobin thresholds for eligibility criteria (<10 g/dL for women and <11 g/dL for men). The revised document also provides separate (TDA vs. non-TDA) and graded (major vs. minor response) response criteria while preserving the requirement for a 12-week period of screening and observation on treatment.Copyright © 2024 American Society of Hematology.