ArticlePilot and feasibility studies2018
The Patient Reported Outcomes, Burdens and Experiences (PROBE) Project: development and evaluation of a questionnaire assessing patient reported outcomes in people with haemophilia.
Article in Pilot and feasibility studies, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 27 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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.
Patient Reported Outcomes Burdens and Experiences - Phase 2 - Pilot Feasibility Study
Patient Reported Outcomes Burdens and Experiences (PROBE) - Phase 3 - Longitudinal Data Collection
Who cites it
27 citing papers in PubMed.
- Normalization of Haemostasis in People with Haemophilia A: Expert Consensus on Unmet Needs and a Framework for Advancing Towards Health Equity.TH open : companion journal to thrombosis and haemostasis · 2026Article
- Comparative safety of drug therapies used in hemophilia A and B in Canada: a multi-center, retrospective study.Research and practice in thrombosis and haemostasis · 2026Article
- An analysis of attitudes toward gene therapy in people with severe hemophilia in Germany, a survey-based cross-sectional study.Therapeutic advances in hematology · 2026Article
- Feasibility of Administering the Patient Reported Outcomes, Burdens and Experiences (PROBE) Questionnaire Through the Canadian Bleeding Disorders Registry (CBDR) and Comparison of Data From the Two Sources.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Observational
- Normalization in hemophilia: conceptual foundations and clinical implications.Research and practice in thrombosis and haemostasis · 2025Review
- Emicizumab in Severe Hemophilia A: Clinical and Patient Determinants of Transition Within a Standardized Program.Journal of hematology · 2025Article
- Managing Haemophilic Arthropathy in People With Haemophilia in Japan: An Informal Consensus.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Article
- Patient-Centred Management of Well-Controlled Haemophilia: Obtaining Opinions and Definitions Through a Delphi Consensus.Journal of clinical medicine · 2025Review
- Mapping the Patient Experience in a Pediatric Hemophilia Unit: Our Patient Journey.Journal of clinical medicine · 2024Article
- The World Federation of Hemophilia World Bleeding Disorders Registry: insights from the first 10,000 patients.Research and practice in thrombosis and haemostasis · 2023Article
- 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
- Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities in health services; diversity, equity, and inclusion; and implementation science.Expert review of hematology · 2023Article
- Article
- Long-term prophylaxis: what are our options and how to define success?Hematology. American Society of Hematology. Education Program · 2022Review
- Socioeconomic participation of persons with hemophilia: Results from the sixth hemophilia in the Netherlands study.Research and practice in thrombosis and haemostasis · 2022Article
- Pain and functional disability amongst adults with moderate and severe haemophilia from the Irish personalised approach to the treatment of haemophilia (iPATH) study.European journal of haematology · 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
- 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
- Pilot and feasibility studies for pragmatic trials have unique considerations and areas of uncertainty.Journal of clinical epidemiology · 2021Article
- Living with a "hemophilia-free mind" - The new ambition of hemophilia care?Research and practice in thrombosis and haemostasis · 2021Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundThe interest of health care agencies, private payers and policy makers for patient-reported outcomes (PRO) is continuously increasing. There is a substantial need to improve capacity to collect and interpret relevant PRO data to support implementation of patient-centered research and optimal care in haemophilia. The Patient Reported Outcomes, Burdens and Experiences (PROBE) Project aims to develop a patient-led research network, to develop a standardized questionnaire to gather patient-reported outcomes and to perform a feasibility study of implementing the PROBE questionnaire.
methodsA pilot questionnaire was developed using focus group methodology. Content and face validity were assessed by a pool of persons living with haemophilia (PWH) and content experts through interactive workshops. The PROBE questionnaire was translated with the forward-backward approach. PROBE recruited national haemophilia patient non-governmental organizations (NGOs) to administer the questionnaire to people with and without haemophilia. PROBE measured the time to complete the questionnaire and gathered feedback on its content and clarity; staff time and cost required to implement the questionnaire were also collected.
resultsThe PROBE questionnaire is comprised of four major sections (demographic data, general health problems, haemophilia-related health problems and health-related quality of life using EQ-5D-5L and EQ-VAS). Seventeen NGOs participated in the pilot study of the PROBE Project, recruiting 656 participants. Of these, 71% completed the questionnaire within 15 min, and all participants completed within 30 min. The median total staff and volunteer time required for the NGOs to carry out the study within their country was 9 h (range 2 to 40 h). NGO costs ranged from $22.00 to $543.00 USD per country, with printing and postage being the most commonly reported expenditures.
conclusionsThe PROBE questionnaire assesses patient-important reported outcomes in PWH and control participants, with a demonstrated short completion time. PROBE proved the feasibility to engage diverse patient communities in the structured generation of real-world outcome research at all stages.
trial registrationTrial registration: NCT02439710.
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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.