Validation of the Patient Reported Outcomes Burdens and Experiences (PROBE) Study Questionnaire

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Skinner M, Chai-Adisaksopha C, Noone D, Curtis R, Frick N, Nichol M, O’Mahony B, Page D, Pastarnak A, Stonebraker J, Iorio A and the Patient Reported Outcomes Burdens and Experiences (PROBE) Study Investigator Group. Validation of the Patient Reported Outcomes Burdens and Experiences (PROBE) Study Questionnaire (Abstract). HAAB (2019).

Introduction

The interest of health care agencies, private payers and policy makers for patient-reported outcomes (PRO) is continuously increasing. There has been a substantial need to improve capacity to collect and interpret relevant PRO data to support implementation of patient-centered research and optimal care in hemophilia.

Objectives

The aims were to 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:

Data collection was initiated 8 April 2015 and completed on 23 February 2017. In total, 2,101 surveys were collected through all phases of the study.   Of these, 1,541 met study criteria for the relevant phases of research and were suitable for analysis.

Clinical Trial registration: NCT02439710.

Results

Validation studies for the PROBE questionnaire have been completed. The questionnaire was assessed for face validity, relevance, clarity and completeness(i); test-retest reliability (reproducibility) confirmed(ii); a core analytic framework (psychometric properties) established(iii); and cross-cultural validation demonstrated(iv). 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.

References

i.Skinner M, Chai-Adisaksopha C, Curtis R, Frick N, Nichol M, Noone D, et al. The Patient Reported Outcomes, Burdens and Experiences (PROBE) Project: development and evaluation of a questionnaire assessing patient reported outcomes in people with haemophilia. Pilot and Feasibility Studies. 2018;4(1):58.
ii.Chai-Adisaksopha C, Skinner MW, Curtis R, Frick N, Nichol MB, Noone D, et al. Test-retest properties of the Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire and its constituent domains. Haemophilia.0(0).
iii.Chai-Adisaksopha C, Skinner MW, Curtis R, Frick N, Nichol MB, Noone D, et al. Psychometric properties of the Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire. BmjOpen. 2018;8(8):10.
iv.Chai-Adisaksopha C, Noone D, Curtis R, Frick N, Nichol M, O’Mahony B, et al. Exploring regional variations in the cross-cultural, international implementation of the patient reported outcomes burdens and experience (PROBE) study. Research and Practice in Thrombosis and Haemostasis. 2018;2(S1):92.

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