HFA is currently inviting community members in Australia to participate in the real-world PROBE study (phase 3).
CLICK HERE TO COMPLETE THE PROBE SURVEY
The PROBE (Patient Reported Outcomes Burdens and Experiences) 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.
WHAT IS THE PROBE STUDY?
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, 2 and 3) are testing and implementing 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.
For more information about PROBE, visit the PROBE study website.
Date last reviewed: 1 August 2019
About the PROBE study
Haemophilia Foundation Australia is participating in the PROBE (Patient Reported Outcomes Burdens and Experiences) Study.
The PROBE study is a multi-national, patient-focused research study.
The PROBE study allows 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. The research will support efforts to improve treatment and comprehensive care programs in Australia and other countries around the world. All data will be kept confidential.
More details about the PROBE study, including publications, are available on the PROBE study website.
Phase 1 (2014-2015) was the very first stage of developing the questionnaire:
- Test understanding of questionnaire content and clarity (haemophilia impact only at this stage)
- Assess methodology and in-country implementation feasibility
- Establish core analytics
A small number of Australian community members with haemophilia or parents of children with haemophilia were invited to test the draft questionnaire and give feedback on the questions and instructions.
Read more about Phase 1 here.
Phase 2 (2016) was the next stage to assess the questionnaire's feasibility through a pilot study:
- Validate proof of concept
- Pilot web platform
- Assess reproducibility
- Establish analytic controls/population comparators
More than 100 Australian community members with haemophilia or who carry the gene and people who do not have a bleeding disorder tested the online and print questionnaire.
Read more about Phase 2 here.
Phase 3 (2019-beyond) is the next stage to extend the study:
- Real world roll-out - invite the wider haemophilia community to participate in the study
- Maintain the capacity to continue collecting the data
- Modify the study infrastructure to allow for longitudinal collection of PROBE data (repeating the study on the same individuals and linking their data over a number of years)
- Complement and enhance the utility of national registries or WFH Global Survey data
- Measure impact of country development initiatives.
Read more about Phase 3 here.
Who is Probe?
PROBE is a multi-national patient-focused investigator driven research project. It is led by a global team of patient and academic investigators with diverse professional credentials:
INTERNATIONAL INVESTIGATION TEAM:
Mark Skinner, JD, Institute for Policy Advancement Ltd [former WFH President] (US)
Chatree Chai-Adisaksopha MD, Division of Hematology, Department of Internal Medicine Chiang Mai University (Thailand)
Randall Curtis MBA, Factor VIII Computing (USA)
Neil Frick MS, National Hemophilia Foundation (USA)
Alfonso Iorio MD PhD FRCPC, McMaster University, Dept of Clinical Epidemiology and Biostatistics (Canada)
Michael Nichol PhD, University of Southern California, School of Policy and Planning Development (USA)
Declan Noone, European Haemophilia Consortium (Belgium)
Brian O’Mahony, Irish Haemophilia Society (Ireland)
David Page, Canadian Hemophilia Society (Canada)
Jeff Stonebraker PhD, North Carolina State University, Poole College of Management (US)
AUSTRALIAN INVESTIGATION TEAM:
Haemophilia Foundation Australia
Suzanne O’Callaghan, Policy Research and Education Manager - Leading research study implementation in Australia
Monash University, Melbourne
Dr Liz Bishop, Michael Kirby Centre for Public Health and Human Rights - Overseeing ethical process; answering any ethics concerns or queries
Mark Skinner and Alfonso Iorio's PROBE Team at McMaster University in Canada are leading the international PROBE investigation with the Australian team.
The PROBE study is voluntary and anonymous and surveys are treated confidentially. In Australia, people participating must return their print surveys in reply paid envelopes to HFA to ensure they are not identifiable. They are then uploaded securely for the international PROBE team data manager to analyze. Alternatively, if they complete their survey online, their survey results will go directly into to the secure PROBE survey database, auspiced by McMaster University, Canada. Only the PROBE teams at HFA and McMaster University will see the original print surveys and all original print surveys will be kept in locked and password protected secure storage at HFA and McMaster University. All survey data will be kept confidential and no data will be reported that could identify individuals.
The PROBE study has Human Research Ethics Approval from Monash University (MUHREC Project Number: CF15/4363 - 2015001518).
If you have any questions about the ethical process of this study, please contact:
Dr Liz Bishop. Monash University - email@example.com
If you have any concerns or complaints about the conduct of the project, please contact the Executive Officer, Monash University Human Research Ethics (MUHREC): firstname.lastname@example.org
For more information about the PROBE study, visit the PROBE study website - www.probestudy.org.
Or contact Suzanne O'Callaghan at HFA:
Date last reviewed: 3 May 2019
T: 03 9885 780
HFA participated in Phase 1 of the PROBE study.
Some of you were involved in distributing and completing the surveys for Phase 1. 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.
Our thanks to Dr Liz Bishop from the Michael Kirby Centre for Public Health and Human Rights at Monash University for her support of the ethics process.
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.
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.
An article on Phase 1 was also published in the journal Pilot and Feasibility Studies in 2018. Visit the PROBE study website to read more.
If you have any questions about the PROBE survey, please contact Suzanne at HFA on 1800 807 173 or email@example.com
MORE ABOUT THE PROBE STUDY
Date last reviewed: 9 November 2018
Phase 2 of the PROBE study aimED to test:
- Whether the survey questions will capture consistent responses if they are repeated twice in the same Australian community
- The stability of the online survey.
Australia joined a number of other countries in the Phase 2 reproducibility test, including Canada, Nigeria, Poland, United States and Vietnam.
In Australia, there were two survey rounds in Phase 2 to compare results:
Round 1: 20 September - 12 December 2016
Round 2: 16 December 2016 - 22 February 2017
Survey participants were welcome to complete both rounds.
Who could participate?
Survey participants were welcome to complete both rounds. Participants lived in Australia, and were:
- adults (18 years or over) and had haemophilia or carried the gene
- or were the partner, family member or caregiver of someone with a bleeding disorder, but did not have a bleeding disorder themselves.
These two groups were also compared with each other.
Australian survey participants for phase 2:
People with haemophilia/carry the gene – 51 (required = 50)
People without a bleeding disorder – 52 (required = 50)
TOTAL – 103 (required = 100)
21 countries participated.
An important learning from this phase was that larger numbers of survey participants will be needed at a country level to provide meaningful data. Participants were grouped as controls and into haemophilia severity, eg mild/moderate/severe. When comparing the different groups of participants, the sample sizes were found to be too small for stable results at a country level, but could be demonstrated at a regional level – for Australia, this was the Western Pacific Region and include countries such as Japan, Vietnam, Australia and New Zealand.
The results validating the PROBE study at a regional level were presented at the International Society on Thrombosis & Haemophilia (ISTH) Scientific and Standardization Committee (SSC) meeting in 2018 and are available on the PROBE study website – www.probestudy.org. (1)
Feedback about the online survey and its stability has been used to fix bugs and make enhancements for the phase 3 version.
For more information, please contact Suzanne O'Callaghan at HFA:
T: 1800 807 173
1. Chai-Adisaksopha C, Noone D, Curtis R, et al. PB189: Exploring regional variations in the cross-cultural, international implementation of the Patient Reported Outcomes Burdens and Experience (PROBE) study. Abstracts of the 64th Annual Meeting of the Scientific Standardization Committee of the International Society on Thrombosis and Haemostasis, July 18–21, 2018. In Research and Practice in Thrombosis and Haemostasis July 2018;2:1-368. doi:10.1002/rth2.12125.
More about the PROBE Study
Date last reviewed: 14 March 2019
In Phase 3 of the PROBE study (real-world), the wider haemophilia community is invited to participate in the survey.
The aim is to collect information about the impact of haemophilia and current treatments from the perspective of Australian adults with haemophilia or who carry the gene. This is also compared to experiences of other adults in this community who do not have a bleeding disorder.
This is known as the ‘real-world’ study. This time community members’ questionnaire responses will be analysed and used as data about real-life experiences rather than testing whether the questionnaire works.
WHY BE INVOLVED IN PHASE 3?
For Australians this is an important and exciting opportunity to create our own database of high-quality patient data about individuals’ experience of haemophilia, treatment and quality of life. We need data like this to understand current issues for our community; and also to represent these issues to governments or treatment and service funding bodies in a credible way. Without this data HFA has not had enough strong evidence to use in our advocacy for new treatments.
WHO CAN PARTICIPATE?
All adults (18 years or over) who live in Australia are invited to participate if they
- have haemophilia or carry the gene
- do NOT have a bleeding disorder (eg, partner/wife/husband, extended family, caregiver, friend of someone with a bleeding disorder).
HOW TO PARTICIPATE?
The questionnaire can be completed:
HOW MANY RESPONSES ARE NEEDED?
For meaningful high quality data we will need several hundred responses. This is to show differences between the experiences of people with mild, moderate, and severe haemophilia or who carry the gene.
So it will be important to for as many people as possible to participate!
Please note: In May 2019 Australia was accidentally sent the test website to distribute for the PROBE real-world survey - https://stage.mcmaster.plus/PROBE/. This was corrected almost immediately and links on this website are all now to the final real-world website for PROBE - https://plus.mcmaster.ca/PROBE/
. If you completed the survey on the test website, the international team has transferred your data to the real-world website. Those visiting the test website now will receive a pop-up directing them to the real-world survey website. The data security is the same - it's just a different database.
PHASE 3 EXTENSION STUDY
In Phase 3 the international investigators are studying the capacity of the system to continue to collect country data for large numbers of participants worldwide over time.
Another aspect of Phase 3 is to investigate modifying the infrastructure of the database system and data collection process to allow for longitudinal collection of PROBE data. This is so that individuals can complete the questionnaire at regular intervals, eg every 2 years, and to link their responses each time they do the survey to see if anything has changed for them. Some countries including Canada will be testing this during Phase 3. HFA will monitor this work to see how it will affect data collection and whether it would be appropriate to do the same in the future in Australia.
For more information about the PROBE study in Australia
or for print survey packs
, please contact Suzanne O'Callaghan at HFA:
T: 1800 807 173
To find out more about PROBE international
, visit the PROBE study website – www.probestudy.org
More about the PROBE study
Date last reviewed: 1 August 2019