PBI55 Patient Preferences for GENE Therapy in Hemophilia: Results from the Paving Threshold Technique Survey

Overbeeke, Eline van; Hauber, AB; Michelsen, Sissel; Peerlinck, Kathelijne; Lambert, Catherine; Hermans, Cédric; Lê, Phu Quoc; Goldman, Michel; Simoens, Steven; Huys, Isabelle

doi:10.1016/j.jval.2020.08.133

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“…Further analysis of levels or subgroups with moderate vs. severe disease was not available at the time of this writing. van Overbeeke and colleagues evaluated minimal acceptable benefits required to switch from prophylaxis to gene therapy among 117 Belgian patients, predominantly with haemophilia A (84%) 24 . The qualitative research used to determine the final attributes for testing identified effect on annual bleeding rate, factor level, uncertainty of long‐term risks, impact on daily life, and probability to stop prophylaxis as the attributes of greatest concern, similar to those determined most relevant for our DCE 25 .…”

Section: Discussionmentioning

confidence: 99%

“…van Overbeeke and colleagues evaluated minimal acceptable benefits required to switch from prophylaxis to gene therapy among 117 Belgian patients, predominantly with haemophilia A (84%). 24 The qualitative research used to determine the final attributes for testing identified effect on annual bleeding rate, factor level, uncertainty of long‐term risks, impact on daily life, and probability to stop prophylaxis as the attributes of greatest concern, similar to those determined most relevant for our DCE. 25 Treatment attributes ultimately included annual bleeding rate, chance to stop prophylaxis, quality of life, and time that side effects have been studied.…”

Section: Discussionmentioning

confidence: 99%

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Patient preferences and priorities for haemophilia gene therapy in the US: A discrete choice experiment

et al. 2021

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Introduction Gene therapy has shown promise in clinical trials for patients with haemophilia, but patient preference studies have focused on factor replacement treatments. Aim We conducted a discrete choice experiment (DCE) to investigate the relative importance and differential preferences patients provide for gene therapy attributes. Methods We surveyed male adults with haemophilia in the United States recruited from patient panels including the National Hemophilia Foundation Community Voices in Research platform using an online survey over 4 months in 2020/21. Participants indicated preferences for gene therapy attributes including dosing frequency/durability, effect on annual bleeding, uncertainty related to side effects, impact on daily activities, impact on mental health, and post‐treatment requirements. The relative importance of each attribute was analysed overall and for subgroups based on haemophilia type and severity. Results A total of 183 males with haemophilia A (n = 120) or B (n = 63) were included. Half (47%) had severe haemophilia; most (75%) were White. Overall, participants gave effect on bleeding rate the greatest relative importance (31%), followed by dose frequency/durability (26%), uncertainty regarding safety issues (17%), and impact on daily activities (11%). Dose frequency/durability had the greatest importance for those with haemophilia B (35%). Conclusion People with haemophilia prioritised reduced bleeding and treatment burden; the former was more important in haemophilia A and the latter in haemophilia B, followed by safety and impact on daily life in this DCE of gene therapy attributes. These findings and differences can inform clinical and health policy decisions to improve health equity for people with haemophilia.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%