Papers

Last updated November 2018

Test‐retest properties of the Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire and its constituent domain

Chai‐Adisaksopha C, Skinner M, Curtis R, et al. Test‐retest properties of the Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire and its constituent domain. 2018;00:1–9. doi: 10.1111/hae.13649

Abstract

Background

The Patient Reported Outcomes, Burdens and Experiences (PROBE) study aims to develop and validate questionnaire for assessing health status in patients with haemophilia and participants without bleeding disorders.

Objective

To investigate the test‐retest properties of the PROBE questionnaire.

Methods

The PROBE questionnaire covers four domains and is comprised of 29 questions. People with haemophilia (PWH) and participants without bleeding disorder were invited to participate in this study. All participants were asked to complete the PROBE questionnaire three times (paper‐based survey on two consecutive days: T1 and T2 and then a web‐based version: T3). Test‐retest properties and percentage agreement were analysed.

Results

A total of 63 participants were enrolled in this study with a median age of 50 (range: 17‐76) years. Of these, 30 (47.6%) were PWH. On the questions common to PWH and participants without bleeding disorder, Kappa coefficients ranged from 0.69 to 1.00, indicating substantial to almost perfect agreement (T1 vs T2). For haemophilia‐related questions (T1 vs T2), Kappa coefficients ranged from 0.5 to 1.0. Of these, 5 of 11 items were in perfect agreement (Kappa = 1.0). The web‐based questionnaire (T3) showed substantial to almost perfect agreement with the paper version (T1 test‐retest properties were comparable between PWH and individuals without a bleeding disorder).

Conclusions

The results suggest that PROBE is a reliable tool to assess patient‐reported outcomes for PWH and benchmark data in participants without bleeding disorder. The web‐based questionnaire and the standard paper‐based version can be used interchangeably.

 

 

Psychometric properties of the Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire

Chai-Adisaksopha C, Skinner MW, Curtis R, et al Psychometric properties of the Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire BMJ Open 2018;8:e021900. doi: 10.1136/bmjopen-2018-021900.

Abstract

Objective

To assess the psychometric properties of the Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire.

Methods

This study was a cross-sectional, multinational study. Participants were enrolled if they were more than 10 years old and people with haemophilia A or B or people without a bleeding disorder. Participants were invited through non-governmental patient organisations in 21 countries between 01/27/2016 and 02/23/2017. The following psychometric properties: missing data, floor and ceiling effects, exploratory factor analysis and internal consistency reliability were examined. A PROBE Score was derived and assessed for its convergent and known groups validity.

Results

The study analysed the data on 916 participants with median age of 37.0 (IQR 27.0 to 48.0) years, 74.8% male. In the domain assessing patient-reported outcomes (PROs), more than 15% of participants presented a ceiling effect for all items but two, and a floor effect for one item. Factor analysis identified three factors explaining the majority of the variance. Cronbach’s alpha coefficient indicated good internal consistency reliability (0.84).  PROBE items showed moderate to strong correlations with corresponding EuroQol five dimension 5-level instrument (EQ-5D-5L) domains. The PROBE Score has a strong correlation (r=0.67) with EQ-5D-5L utility index score. The PROBE Score has a known groups validity among various groups.

Conclusions

The results of this study suggest that PROBE is a valid questionnaire for evaluating PROs in people with haemophilia as well as control population. The known group property of PROBE will allow its use in future clinical trials, longitudinal studies, health technology assessment studies, routine clinical care or registries. Additional studies are needed to test responsiveness and sensitivity to change.

 

 

The Reported Outcomes, Burdens and Experiences (PROBE) Project: development and evaluation of a questionnaire assessing patient reported outcomes in people with haemophilia

Citation: Skinner, M. W., Chai-Adisaksopha, C., Curtis, R., Frick, N., Nichol M., Noone, D., O’Mahony, B., Page, P., Stonebraker, J. S. and Iorio, A. (2018). 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 4:58. doi: 10.1186/s40814-018-0253-0

Abstract

Background

The 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.

Methods

A 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.

Results

The 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.

Conclusions

The 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.