Resumen
Introduction: GOAL-Hēm is a novel, haemophilia-specific, patient-centred outcome measure (PCOM) based on goal attainment scaling, allowing people with haemophilia (PwH) to set and monitor the attainment of individualized goals for treatment. Aim: To provide a thorough overview of the creation, validation, and development of GOAL-Hēm. Methods: Clinician workshops were held to develop a haemophilia-specific goal menu. Qualitative data from semistructured interviews with PwH and their caregivers guided further revisions to the goal menu (i.e., goal domains and descriptors). A feasibility study was performed including a 12-week, prospective, noninterventional evaluation involving clinicians and PwH at four US haemophilia treatment centres. Finally, the Patient Voice Study gathered feedback from PwH and their caregivers via an online survey, interviews, and a focus group. Results: The feasibility study validated GOAL-Hēm with successful outcomes in construct/content validity and responsiveness, including a large effect in patient- and clinician-rated goal attainments. The Patient Voice Study led to significant refinement of GOAL-Hēm goals and descriptors, resulting in a more straightforward and relatable menu for PwH and their caregivers. Overall, GOAL-Hēm captured qualitative data in areas important to PwH and employed quantitative methods to evaluate meaningful changes in those areas. The individualized tool was well equipped to handle the complex and chronic nature of haemophilia and was endorsed by PwH, their caregivers, and clinicians. Conclusion: The GOAL-Hēm development journey may serve as a roadmap for other PCOMs in a variety of settings, including clinical studies, haemophilia treatment centres for care planning, and as a tool to gather real-world evidence.
| Idioma original | English |
|---|---|
| Publicación | Haemophilia |
| DOI | |
| Estado | Accepted/In press - 2022 |
Nota bibliográfica
Funding Information:Many thanks to the PwH and caregiver participants, whose input was vital in the refinement and ongoing development of GOAL‐Hēm; and Ardea Outcomes (formerly DGI Clinical)—especially Amanda England, Sherry Fay, and Kari Knox—for their input and facilitation of the studies reviewed here. We are grateful to Dr Roberts’ staff at the Bleeding and Clotting Disorders Institute (BCDI) for their assistance with the feasibility study and the focus group in the Patient Voice Study, with special thanks to Sarah Gonzales who assisted in both studies’ successful coordination, data collection, and analysis at BCDI. Dr. Shannon Jackson and Dr. Sue Robinson at the Canadian sites assisted with study design and enrolled patients into the feasibility study. Medical writing assistance was provided by Bill Kadish, MD, Iain Patefield, MS, and Zela Keuylian, PhD, of Parexel and funded by Takeda. Development of the GOAL‐Hēm tool, the GOAL‐Hēm feasibility study, and the Patient Voice Study were funded by Takeda.
Publisher Copyright:
© 2022 The Authors. Haemophilia published by John Wiley & Sons Ltd.
ASJC Scopus Subject Areas
- Hematology
- Genetics(clinical)
PubMed: MeSH publication types
- Journal Article