Preferences and Health-Related Quality-of-Life Related to Disease and Treatment Features for Patients with Hemophilia A in a Canadian General Population Sample.

BACKGROUND

Current treatments for hemophilia A in Canada include on-demand treatment as bleeds occur and regular intravenous prophylactic factor VIII (FVIII) infusions. The subcutaneous therapy emicizumab was recently approved for treatment of hemophilia A. The objective of this study was to estimate utility values associated with hemophilia A health and treatment states from a Canadian societal perspective, including preferences related to treatment efficacy and frequency and route of administration.

METHODS

A vignette-based time trade-off (TTO) utilities elicitation was undertaken in Canadian adults to compare preferences for six hemophilia health states describing prophylactic and on-demand treatment, with varying bleed rates and frequency of treatment administration. A convenience sample was recruited via market research panels and snowball sampling. Health state/vignette definitions were informed by clinical experience, clinical trial results regarding bleed frequency, and validated by qualitative interviews of hemophilia patients and caregivers (n=10). Utilities were estimated via an online, trained interviewer-guided, vignette-based TTO exercise, where respondents valuated health states describing hemophilia patients (adults or children) receiving subcutaneous prophylaxis, intravenous prophylaxis, and on-demand treatments. Analyses included a descriptive analysis by health state; a mixed-effects analysis of utility values adjusted for subcutaneous vs infusion-based therapies and number of bleeds; and for prophylactic regimens, an analysis of utilities by frequency of infusions or injections.

RESULTS

TTO interviews were conducted with 82 respondents. Mean utilities [95% CI] were highest for subcutaneous prophylaxis (0.90 [0.87-0.93]), followed by intravenous prophylaxis (0.81 [0.78-0.85]), and on-demand treatment (0.70 [0.65-0.76]). In regression analysis, subcutaneous treatment health states were associated with a utility increment of +0.1112. Additional bleeds and more frequent infusions were associated with lower utility values (-0.0027 per bleed and -0.0003 per infusion).

CONCLUSION

Subcutaneous prophylaxis is associated with higher utility values compared to intravenous prophylactic and on-demand treatment, while increased bleeds and infusions are associated with reduced utility.

Informations de copyright

© 2021 Johnston et al.

Déclaration de conflit d'intérêts

Dr Jayson M Stoffman received consulting honorarium from Hoffman-La Roche Ltd, during the conduct of the study; and consulting honorarium from Hoffman-La Roche and Bayer Inc., outside the submitted work. Dr Robert J Klaassen reports personal fees from Amgen Inc, personal fees from Hoffman-LaRoche Ltd, personal fees from Novo Nordisk, personal fees from Octapharma EG, personal fees from Takeda Canada Inc, and personal fees from Sanofi Canada, during the conduct of the study. Ms Shade Olatunde is an employee of Hoffmann-La Roche Ltd. Dr Lina Eliasson is an employee of Sprout Behaviour Change, who were subcontracted to assist the conduct of the qualitative interviews by Broadstreet HEOR. Dr. Karissa Johnston and Alexis Mickle are employees of Broadstreet HEOR, who received funding from Hoffmann-La Roche Ltd. for the conduct of the study. The authors report no other conflicts of interest in this work.