ArticleResearch and practice in thrombosis and haemostasis2021
Patient-relevant health outcomes for hemophilia care: Development of an international standard outcomes set.
Article in Research and practice in thrombosis and haemostasis, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers.
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Who cites it
26 citing papers in PubMed, 41 citations in OpenAlex.
- Assessing the real-world value of hemophilia care: how suitable are patient-centered outcomes for hospital-level comparisons?International journal for quality in health care : journal of the International Society for Quality in Health Care · 2026Article
- Integrating Clinical, Functional, and Patient-Reported Outcomes in Haemophilia Care: A Delphi-Based Consensus on a New Monitoring Tool.Journal of clinical medicine · 2026Article
- The Psychometric Properties of the Moberg Pick-Up Test (MPUT) to Assess Fine Motor Skills in Adults with Haemophilia.Healthcare (Basel, Switzerland) · 2026Article
- Patient-Centred Management of Well-Controlled Haemophilia: Obtaining Opinions and Definitions Through a Delphi Consensus.Journal of clinical medicine · 2025Review
- Economic Burden of Haemophilia from a Societal Perspective: A Scoping Review.PharmacoEconomics - open · 2025Review
- The role of public health in rare diseases: hemophilia as an example.Frontiers in public health · 2025Review
- What is a cure through gene therapy? An analysis and evaluation of the use of "cure".Medicine, health care, and philosophy · 2024Article
- Mapping the Patient Experience in a Pediatric Hemophilia Unit: Our Patient Journey.Journal of clinical medicine · 2024Article
- A decade of change towards Value-Based Health Care at a Dutch University Hospital: a complexity-informed process study.Health research policy and systems · 2024Article
- Can hemophilia be cured? It depends on the definition.Research and practice in thrombosis and haemostasis · 2024Review
- Patient-relevant health outcomes for von Willebrand disease, platelet function disorders, and rare bleeding disorders: a Delphi study.Research and practice in thrombosis and haemostasis · 2023Article
- Psychometrics of the patient-reported outcomes measurement information system measures in hemophilia: the applicability of the pediatric item banks.Research and practice in thrombosis and haemostasis · 2023Article
- Current Status and Challenges in Delivering Comprehensive Care for Patients with Hemophilia.Journal of blood medicine · 2023Review
- Self-Induced Myofascial Release in Patients with Hemophilic Ankle Arthropathy: A Pilot Observational Study.Life (Basel, Switzerland) · 2022Article
- Socioeconomic participation of persons with hemophilia: Results from the sixth hemophilia in the Netherlands study.Research and practice in thrombosis and haemostasis · 2022Article
- SYMPHONY consortium: Orchestrating personalized treatment for patients with bleeding disorders.Journal of thrombosis and haemostasis : JTH · 2022Article
- User-Centered Development and Testing of the Online Patient-Reported Outcomes, Burdens, and Experiences (PROBE) Survey and the myPROBE App and Integration With the Canadian Bleeding Disorder Registry: Mixed Methods Study.JMIR human factors · 2022Article
- Rasch Measurement and Patient Reported Value Claims: A Primer for Hemophilia.Innovations in pharmacy · 2022Article
- Generic PROMIS item banks in adults with hemophilia for patient-reported outcome assessment: Feasibility, measurement properties, and relevance.Research and practice in thrombosis and haemostasis · 2021Article
- Validation of PROMIS Profile-29 in adults with hemophilia in the Netherlands.Journal of thrombosis and haemostasis : JTH · 2021Article
Corrections and comments
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Authors and funding
19 authors at 15 institutions in 6 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundPatient-relevant health outcomes for persons with hemophilia should be identified and prioritized to optimize and individualize care for persons with hemophilia. Therefore, an international group of persons with hemophilia and multidisciplinary health care providers set out to identify a globally applicable standard set of health outcomes relevant to all individuals with hemophilia.
methodsA systematic literature search was performed to identify possible health outcomes and risk adjustment variables. Persons with hemophilia and multidisciplinary health care providers were involved in an iterative nominal consensus process to select the most important health outcomes and risk adjustment variables for persons with hemophilia. Recommendations were made for outcome measurement instruments.
resultsPersons with hemophilia were defined as all men and women with an X-linked inherited bleeding disorder caused by a deficiency of coagulation factor VIII or IX with plasma activity levels <40 IU/dL. We recommend collecting the following 10 health outcomes at least annually, if applicable: (i) cure, (ii) impact of disease on life expectancy, (iii) ability to engage in normal daily activities, (iv) severe bleeding episodes, (v) number of days lost from school or work, (vi) chronic pain, (vii) disease and treatment complications, (viii) sustainability of physical functioning, (ix) social functioning, and (x) mental health. Validated clinical as well as patient-reported outcome measurement instruments were endorsed. Demographic factors, baseline clinical factors, and treatment factors were identified as risk-adjustment variables.
conclusionA consensus-based international set of health outcomes relevant to all persons with hemophilia, and corresponding measurement instruments, was identified for use in clinical care to facilitate harmonized longitudinal monitoring and comparison of outcomes.
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