慢性粒细胞白血病药物开发的前景：改变范式。

IF 21 1区医学 Q1 HEMATOLOGY

Blood Pub Date : 2024-11-07 DOI:10.1182/blood.2024025648

Anthony M Hunter, Mrinal M Patnaik, Raphael Itzykson, Ruben Mesa, Chatchada Karanes, Yanxia Li, R Angelo de Claro, Kelly J Norsworthy, Marc Theoret, Elizabeth Pulte, Eric Padron

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引用次数: 0

摘要

摘要：慢性粒细胞白血病（CMML）的药物开发未能与最近在相关髓系肿瘤中观察到的成功相提并论。针对这些不足，美国食品药品管理局（FDA）于 20 年 9 月召开了 "慢性粒细胞白血病小型研讨会"：当前技术水平和试验设计 "小型研讨会。此次研讨会汇集了 FDA 主要监管人员和 CMML 药物开发方面的学术专家，共同讨论了该疾病面临的挑战，并对该疾病未来的药物开发提出了看法。专家小组探讨了迄今为止 CMML 治疗缺乏进展所面临的独特挑战，并讨论了临床试验设计、研究终点和主要监管考虑因素等相关主题。本文总结了本次研讨会的讨论要点，以促进该领域的进步。

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Perspectives on drug development in chronic myelomonocytic leukemia: changing the paradigm.

Abstract: Drug development for chronic myelomonocytic leukemia (CMML) has failed to parallel the recent success observed in related myeloid neoplasms. To address these shortcomings, the US Food and Drug Administration (FDA) held a "Mini-symposium on CMML: Current State of the Art and Trial Design" in September 2023. This symposium brought together a panel of key FDA regulators and academic experts in CMML drug development to discuss challenges and provide perspectives on future drug development for this disease. The panel explored unique challenges that underlie the lack of therapeutic advances in CMML to date and discussed relevant topics such as clinical trial design, study end points, and key regulatory considerations. This article summarizes the key points of discussion from this symposium to facilitate advancements in the field.

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来源期刊

Blood 医学-血液学

CiteScore

23.60

自引率

3.90%

发文量

955

审稿时长

1 months

期刊介绍： Blood, the official journal of the American Society of Hematology, published online and in print, provides an international forum for the publication of original articles describing basic laboratory, translational, and clinical investigations in hematology. Primary research articles will be published under the following scientific categories: Clinical Trials and Observations; Gene Therapy; Hematopoiesis and Stem Cells; Immunobiology and Immunotherapy scope; Myeloid Neoplasia; Lymphoid Neoplasia; Phagocytes, Granulocytes and Myelopoiesis; Platelets and Thrombopoiesis; Red Cells, Iron and Erythropoiesis; Thrombosis and Hemostasis; Transfusion Medicine; Transplantation; and Vascular Biology. Papers can be listed under more than one category as appropriate.