ArticleHaemophilia : the official journal of the World Federation of Hemophilia2022
The GOAL-Hēm journey: Shared decision making and patient-centred outcomes.
Article in Haemophilia : the official journal of the World Federation of Hemophilia, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers, 1 of them a synthesis that pooled it.
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
10 citing papers in PubMed, 1 synthesis or guideline pooled it, 34 citations in OpenAlex.
- A systematic review of goal attainment scaling implementation practices by caregivers in randomized controlled trials.Journal of patient-reported outcomes · 2024Pooled it
- The State of Shared Decision-Making in Immune Thrombocytopenia: A Narrative Review and Health Care Provider Perspective.Health science reports · 2026Article
- Outcome strategies for clinical trials in Neuropaediatric rare diseases.Neuroscience applied · 2026Review
- Enhancing Engagement to Support Shared Decision Making Within Haemophilia: Development of Materials for People With Haemophilia, Caregivers and Healthcare Professionals.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Article
- Clinician and Researcher Experiences with Goal Attainment Scaling: A Qualitative Study on Opportunities and Challenges for Personalized Outcome Assessments.Patient related outcome measures · 2025Article
- Mapping the Patient Experience in a Pediatric Hemophilia Unit: Our Patient Journey.Journal of clinical medicine · 2024Article
- Training healthcare professionals to administer Goal Attainment Scaling as an outcome measure.Journal of patient-reported outcomes · 2024Article
- The GOAL-Hēm journey: Shared decision making and patient-centred outcomes.Haemophilia : the official journal of the World Federation of Hemophilia · 2022Article
- Patient-advocate-led global coalition adapting fit-for-purpose outcomes measures to assure meaningful inclusion of DEEs in clinical trials.Therapeutic advances in rare diseaseReview
- Supporting Patient-Centred Decisions on Gene Therapy for Haemophilia A: A Conceptual Model.Haemophilia : the official journal of the World Federation of HemophiliaArticle
Corrections and comments
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Authors and funding
9 authors at 6 institutions in 2 countries.
Funding
Abstract
introductionGOAL-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.
aimTo provide a thorough overview of the creation, validation, and development of GOAL-Hēm.
methodsClinician 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.
resultsThe 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.
conclusionThe 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.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.