Patient preferences in the treatment of hemophilia A: A latent class analysis

Abstract

Objective To examine subgroup-specific treatment preferences and characteristics of patients with hemophilia A. Methods Best–Worst Scaling (BWS) Case 3 (four attributes: application type; bleeding frequencies/year; inhibitor development risk; thromboembolic events of hemophilia A treatment risk) conducted via online survey. Respondents chose the best and the worst option of three treatment alternatives. Data were analyzed via latent class model (LCM), allowing capture of heterogeneity in the sample. Respondents were grouped into a predefined number of classes with distinct preferences. Results The final dataset contained 57 respondents. LCM analysis segmented the sample into two classes with heterogeneous preferences. Preferences within each were homogeneous. For class 1, the most decisive factor was bleeding frequency/year. Respondents seemed to focus mainly on this in their choice decisions. With some distance, inhibitor development was the second most important. The remaining attributes were of far less importance for respondents in this class. Respondents in class 2 based their choice decisions primarily on inhibitor development, also followed, by some distance, the second most important attribute bleeding frequency/year. There was statistical significance (P < 0.05) between the number of annual bleedings and the probability of class membership. Conclusions The LCM analysis addresses heterogeneity in respondents’ choice decisions, which helps to tailor treatment alternatives to individual needs. Study results support clinical and allocative decision-making and improve the quality of interpretation of clinical data.

Haemophilia in France: Modelisation of the Clinical Pathway for Patients

“…It is 9.5 cases per 100,000 males for severe haemophilia A, 5.0 cases per 100,000 males for all severities of haemophilia B, and 1.5 cases per 100,000 males for severe haemophilia B. In 1999, the World Federation of Haemophilia Annual Global Survey identified 78,629 people with haemophilia; this number has since increased to 210,454 in 2018 [ 3 , 4 ]. Since the start of the AGS (Annual Global Survey), there has been a tripling in the number of people identified with all rare bleeding disorders reported.…”