Barbara J Klencke, Rafe Donahue, Boris Gorsh, Catherine Ellis, Jun Kawashima, Bryan Strouse
{"title":"Anemia-related response end points in myelofibrosis clinical trials: current trends and need for renewed consensus.","authors":"Barbara J Klencke, Rafe Donahue, Boris Gorsh, Catherine Ellis, Jun Kawashima, Bryan Strouse","doi":"10.2217/fon-2023-0964","DOIUrl":null,"url":null,"abstract":"<p><p>JAK inhibitors are the current standard of care in myelofibrosis, but many do not address and may worsen anemia; thus, anemia-related responses have traditionally been overlooked as efficacy end points in pivotal clinical trials, leading to a lack of consistency and analytic detail in their reporting. Here we apply our experiences in the phase III trials of momelotinib, a JAK1/JAK2/ACVR1 inhibitor and the first therapy indicated by the US FDA for myelofibrosis patients with anemia, to highlight how application of different criteria impacts the anemia-related benefits reported for any potential treatment in myelofibrosis. We advocate for a convention of a new expert consensus panel to bring consistency and transparency to the definition of anemia-related response in myelofibrosis.</p>","PeriodicalId":12672,"journal":{"name":"Future oncology","volume":" ","pages":"703-715"},"PeriodicalIF":3.0000,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Future oncology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.2217/fon-2023-0964","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/2/6 0:00:00","PubModel":"Epub","JCR":"Q2","JCRName":"ONCOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
JAK inhibitors are the current standard of care in myelofibrosis, but many do not address and may worsen anemia; thus, anemia-related responses have traditionally been overlooked as efficacy end points in pivotal clinical trials, leading to a lack of consistency and analytic detail in their reporting. Here we apply our experiences in the phase III trials of momelotinib, a JAK1/JAK2/ACVR1 inhibitor and the first therapy indicated by the US FDA for myelofibrosis patients with anemia, to highlight how application of different criteria impacts the anemia-related benefits reported for any potential treatment in myelofibrosis. We advocate for a convention of a new expert consensus panel to bring consistency and transparency to the definition of anemia-related response in myelofibrosis.
JAK 抑制剂是骨髓纤维化目前的治疗标准，但许多抑制剂无法解决贫血问题，甚至可能加重贫血；因此，贫血相关反应历来被视为关键临床试验的疗效终点而被忽视，导致报告缺乏一致性和分析细节。莫迈罗替尼是一种 JAK1/JAK2/ACVR1 抑制剂，也是美国 FDA 批准用于骨髓纤维化贫血患者的第一种疗法，在此，我们运用自己在莫迈罗替尼 III 期临床试验中的经验，强调不同标准的应用如何影响骨髓纤维化潜在疗法的贫血相关疗效报告。我们主张召开一次新的专家共识小组会议，以实现骨髓纤维化贫血相关反应定义的一致性和透明度。
期刊介绍：
Future Oncology (ISSN 1479-6694) provides a forum for a new era of cancer care. The journal focuses on the most important advances and highlights their relevance in the clinical setting. Furthermore, Future Oncology delivers essential information in concise, at-a-glance article formats - vital in delivering information to an increasingly time-constrained community.
The journal takes a forward-looking stance toward the scientific and clinical issues, together with the economic and policy issues that confront us in this new era of cancer care. The journal includes literature awareness such as the latest developments in radiotherapy and immunotherapy, concise commentary and analysis, and full review articles all of which provide key findings, translational to the clinical setting.
京公网安备 11010802042870号
京ICP备2023020795号-1
分享
求助内容：
应助结果提醒方式：
扫码关注我们
