Skinner M, Chai‐Adisaksopha C, O’Mahony B, Noone D, Curtis R, Frick N, Nichol M, Page D, Stonebraker J, Iorio A. Patient Reported Outcomes Burdens and Experiences Study Group. Validation of the Patient Reported Outcomes Burdens and Experiences (PROBE) Study Questionnaire. Abstract PB1412. Res Pract Thromb Haemost. 2019;3(S1):1-891. https://doi.org/10.1002/rth2.12229
Background
A substantial need exists to improve capacity to collect and interpret relevant patient-reported outcomes (PRO) data to support patient-centered research and optimal care in people with hemophilia (PWH).
Aims
Implement a structured data collection of PRO across countries to build a robust evidence base for comparative effectiveness research, evidence-based decision making, and advocacy. Three intermediate objectives were identified:
- develop a patient-led research network;
- develop a standardized questionnaire to gather PRO; and
- perform a feasibility study of implementing the PROBE questionnaire.
Additional aims were to explore the measurement properties of the PROBE questionnaire and demonstrate PROBEis valid to implement for assessing health status among PWH and participants without bleeding disorders acrossregions.
Methods
In data collection from April 2015 to February 2017, 2,101 surveys were collected through all studyphases; 1,541 met study criteria for analysis. Clinical Trial registration: NCT02439710.
Results
Outcomes of importance to PWH and metrics to consider for measurement were determined. The PROBEquestionnaire consists of four major sections (demographic data, general health problems, hemophilia-related healthproblems and health-related quality of life). PROBE questionnaire validation studies establish:
- Face validity, relevance, clarity and completeness
- Test-retest reliability (reproducibility)
- A core analytic framework (psychometric properties)
- Cross-cultural validation
Conclusions
The PROBE questionnaire is a valid and reliable tool for assessing health status among PWH and participants without bleeding disorders across regions. PROBE assesses patient-important PROs with ademonstrated short completion time. PROBE proved the feasibility to engage diverse patient communities instructured generation of real-world outcome data. The web‐based questionnaire and the paper‐based version maybe used interchangeably. The known group property of PROBE will allow its use in future clinical trials, longitudinalstudies, health technology assessment studies, routine clinical care or registries. Additional studies are planned totest responsiveness and sensitivity to change.
View Poster (English): Validation of PROBE’s Questionnaire
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