Sutphin J, Bartelt-Hofer J, Leach C, Hauber B, Cano V, Sussebach C, Cavanaugh Smith J, DasMahapatra P. Treatment preferences in hemophilia: results from a targeted literature review. Poster presented at the Virtual EAHAD 2021 Congress; February 3, 2021. [abstract] Haemophilia. 2021 Feb; 27(S2):134. doi: 10.1111/hae.14236.


INTRODUCTION: Previous studies have evaluated preferences for hemophilia treatment features. However, the hemophilia treatment landscape is evolving, with promising advances in prophylactic treatments and gene therapy. The study aims to measure patient, caregiver, and clinician preferences with an eye toward the future by building on previous preference elicitation research in hemophilia and eliciting preferences for features of novel treatments and potentially for health outcomes that indicate a patient with hemophilia has reached a state of health equity.

METHODS: To quantify preferences for hemophilia treatment features and outcomes (attributes), a discrete-choice experiment (DCE) will be used. DCEs are a common method for capturing tradeoffs patients are willing to make among a select set of attributes (usually between 5 and 8 attributes). A targeted literature review was conducted to compile a list of candidate attributes for use in a DCE preference study. Steps to identifying candidate attributes included a PubMed database search for previous preference studies in hemophilia, identifying publications using a previous literature review, reviewing additional documents identified by the study team, screening abstracts for suitability, reviewing full-text articles to confirm suitability, extracting example attributes, grouping similar attributes, and categorizing the attributes into domains to form a comprehensive list of potential attributes.

RESULTS: Of 110 records initially identified, 19 were retained including 10 DCEs, a conjoint rating study, a multicriteria decision analysis, a value framework, a patient-reported outcome instrument, a direct-elicitation survey, and a novel treatment model for achieving health equity in hemophilia. From these, we identified 32 potential unique attributes in 8 domains (efficacy [bleeds prevented, time to stop bleeding, function, resilience, pain recovery/avoidance], safety, administration, cost, storage, patient characteristics, patient well-being, and other [access, manufacturer, scientific evidence]). Attributes will be ranked by importance in an online survey of patients, physicians, and caregivers to inform the selection of attributes in the DCE

DISCUSSIONS/CONCLUSIONS: When completed, this study will contribute a patient-centered and clinician-informed understanding of preferences for available and emerging hemophilia treatments.

Share on: