Patient and caregiver preferences for haemophilia treatments: A discrete-choice experiment.
Haemophilia
; 30(2): 375-387, 2024 Mar.
Article
en En
| MEDLINE
| ID: mdl-38198352
ABSTRACT
INTRODUCTION:
An evolving haemophilia treatment landscape provides new possibilities for previously unattainable lifestyles.AIM:
We sought to understand how people with haemophilia (PwH) and their caregivers value the potential benefits of novel prophylactic treatments. We conducted a discrete-choice experiment (DCE) to quantify preferences for features of haemophilia treatments among adults and caregivers of children with haemophilia. A best-worst scaling (BWS) exercise measured the perceived burden of treatment administration features.METHODS:
A cross-sectional, web-based survey was administered to male adults (≥18 years) and caregivers of male children (≤17 years) living with haemophilia in the United States. Respondents evaluated eight pairs of hypothetical haemophilia treatment profiles defined by six attributes in the DCE and 15 features in the BWS.RESULTS:
In the DCE, both adults with haemophilia (n = 151) and caregivers (n = 151) prioritised avoiding the risk of developing inhibitor/ anti-drug antibodies and treatments that allowed for a more active life. They placed a lower priority on reducing the number of spontaneous bleeding episodes, route and frequency of administration, and avoiding the risk of hospitalisation due to adverse events. The BWS documented the burdensomeness of IV infusions and medications that require mixing and refrigeration.CONCLUSION:
PwH and caregivers prefer treatments that enable a more active lifestyle with a lower risk of inhibitor development. Both groups valued the ability to lead an active life over reducing spontaneous bleeding, with caregivers placing the most weight on this attribute. As new treatments expand possibilities, healthcare professionals and PwH should continue to share decision-making, incorporating clinical judgment and individual preferences.Palabras clave
Texto completo:
1
Banco de datos:
MEDLINE
Asunto principal:
Hemofilia A
Tipo de estudio:
Prognostic_studies
Límite:
Adult
/
Child
/
Humans
/
Male
País/Región como asunto:
America do norte
Idioma:
En
Revista:
Haemophilia
Asunto de la revista:
HEMATOLOGIA
Año:
2024
Tipo del documento:
Article
País de afiliación:
Estados Unidos