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为克隆性造血领域成功的临床试验设计绘制蓝图。

Drafting a blueprint for designing successful clinical trials in clonal haematopoiesis.

作者信息

Patel Shyam A, Weeks Lachelle D

机构信息

Department of Medicine-Division of Hematology/Oncology, Center for Clinical and Translational Science, UMass Chan Medical School, Worcester, Massachusetts, USA.

Center for Early Detection and Interception of Blood Cancers, Dana-Farber Cancer Institute, Harvard Medical School, Boston, Massachusetts, USA.

出版信息

Br J Haematol. 2025 Feb;206(2):800-802. doi: 10.1111/bjh.19948. Epub 2024 Dec 9.

Abstract

'As our understanding of the biology of clonal hematopoiesis expands, a pressing need in the field becomes the design and implementation of clinical trials to help mitigate the risk for progression to overt myeloid neoplasm. Effective clinical trial design will be informed by use of personalized genetic risk to determine eligibility, strategic endpoint selection, and identification of suitable interventions with a goldilocks balance of toxicity and reduced risk of progression. We will only reach this milestone through collaboration'. Commentary on: Haque et al. A blueprint for pursuing therapeutic interventions and early phase clinical trials in clonal haematopoiesis. Br J Haematol 2025; 206:416-427.

摘要

随着我们对克隆性造血生物学的理解不断扩展,该领域的一项紧迫需求是设计和开展临床试验,以帮助降低进展为明显髓系肿瘤的风险。有效的临床试验设计将依据个性化遗传风险来确定入选标准、战略性地选择终点指标,并识别出毒性与降低进展风险达到恰到好处平衡的合适干预措施。我们只有通过合作才能实现这一里程碑。评论文章:Haque等人。克隆性造血中寻求治疗干预和早期临床试验的蓝图。《英国血液学杂志》2025年;206:416 - 427。

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