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. 2023 Sep;29(5):1184-1190.
doi: 10.1111/hae.14822. Epub 2023 Jul 4.

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

Jacqueline Limjoco  1 , Courtney D Thornburg  1   2
Affiliations

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

Jacqueline Limjoco et al. Haemophilia. 2023 Sep.

Abstract

Introduction: As gene therapies are incorporated into clinical practice, shared decision-making (SDM) is recommended for implementation.

Aim: To inform development of a clinician SDM tool for haemophilia A gene therapy.

Methods: Clinicians at US Hemophilia Treatment Centers completed semi-structured interviews about their experience with SDM and provided feedback on a clinician SDM tool prototype. Interviews were transcribed verbatim for coding and thematic content analysis.

Results: Ten participants enrolled, eight physicians and two haemophilia nurses. All participants care for adults with haemophilia (1-27 years of experience) and 7 have gene therapy trials open at their institution. Confidence in having a clinical discussion about gene therapy included none (N = 1), slight (N = 3), moderate (N = 5) and high (N = 1). All participants reported familiarity with SDM and agreed that the tool would be useful for their clinical practice. Key themes in participant feedback for the tool were (1) language and presentation; (2) content; and (3) implementation. Participants highlighted the importance of providing unbiased information and having companion tools with patient-centric language.

Conclusion: 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.

Keywords: factor VIII deficiency; gene therapy; medical decision making; patient preference; shared decision-making.

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References

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