PROBE study

How can HFA and haemophilia organisations around the world have access to good quality data about the treatment and health experiences of people with bleeding disorders? The multi-national PROBE (Patient Reported Outcomes Burdens and Experiences) Study aims to do precisely that. Haemophilia Foundation Australia has joined the PROBE investigation team to be part of this important international study on the impact of living with a bleeding disorder.

PROBE is a patient-focused research project led by a global team of patient and academic investigators, including Mark Skinner, former WFH President, and Assoc Prof Alfonso Iorio from McMaster University, Canada, who have worked closely with HFA on the Australian arm of the study. The research will support efforts to improve treatment and comprehensive care programs in Australia and other countries around the world.

In the future the PROBE study will cover the range of bleeding disorders. The initial stages of the survey (Phases 1 and 2) are testing the research methodology in haemophilia and allow people with haemophilia to report their haemophilia severity, treatment history and the impact of haemophilia on their daily life. This data will be used to analyse the perspectives of people with haemophilia on outcomes that affect their own life and care. It is particularly important data to have when HFA tries to understand what the issues are for our community, and will enable us to quantify and represent these issues in a robust and credible way to our Council and others, such as governments or treatment and service funding bodies.

PHASE 1

Some of you have been involved in distributing and completing the surveys for Phase 1 of the PROBE study. Thank you for your support! We needed 35-50 completed surveys by the end of 2015 to have enough data to be analysed, and by Christmas had received 39 surveys, which was an amazing effort in a very short few weeks!

What did Phase 1 involve?

• Seeking ethics approval via Monash University, Melbourne
• Testing how clear the questions are
• Testing the methodology and the statistics that could be gained from the results.

Phase 1 tested a print survey on haemophilia only. Some Australian community members with haemophilia and carers or parents of children with haemophilia completed the draft questionnaire and gave feedback on the questions and instructions. The completed surveys and question feedback sheets were then scanned and transferred electronically to the PROBE research team at McMaster University for analysis.

RESULTS

HFA received a report on the Australian results in January 2016 and was invited to participate in an international meeting where the worldwide results were discussed:

• More than 700 surveys were completed by 15 countries
• Results demonstrated validity and that the research methodology was robust
• Phase 1 data is useful for understanding how to use the potential results but cannot be used for representation on health issues at this point – we need to wait for phase 3
• Australian feedback on questions was put in a spreadsheet and each comment was addressed by the research team leaders
• As a result of global feedback, new questions will be added in Phase 2 for women who carry the gene
• Some other small changes were made to the Australian version of the survey
• For good comparable data, in future Australia needs to recruit more people with moderate haemophilia to complete the survey.

A poster about Phase 1 was presented at the European Association for Haemophilia and Allied Disorders (EAHAD) Congress in Sweden in February 2016 and very well received.

PHASE 2

The next phase involves reproducibility – testing an online version of the survey as well, and retesting the same community (ie, Australia) twice in several months to see whether the results remain consistent. There will be more information about this soon. Stay tuned!

If you have any questions about the PROBE survey, please contact Suzanne at HFA on 1800 807 173 or socallaghan@haemophilia.org.au; or visit the PROBE section on the HFA website.