Development of a haemophilia A gene therapy shared decision-making tool for clinicians.

Haemophilia : the official journal of the World Federation of Hemophilia（2023）

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摘要

These data highlight the need for SDM tools for haemophilia A gene therapy. Key information to include in the tool are safety, efficacy, cost and detailed information on the gene therapy process. Data should be provided in an unbiased format and allow comparison to other treatments. The tool will be evaluated in clinical practice and refined as clinical trial data and real-world experience mature.

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关键词

factor VIII deficiency, gene therapy, medical decision making, patient preference, shared decision-making

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