Connective Issues Fall 2018

RESEARCH

CHANGE THE FUTURE Participate in Research on Marfan and Related Conditions

Have you ever wondered what types of orthotics work best for Marfan feet or whether physical therapy works for Marfan back pain or which migraine medication works best? These are some of the many questions that face those with Marfan syndrome and and related conditions and those who take care of them. So how does your doctor know the answers? He/she may have read some careful randomized study, but there are only a small number of these studies and they frequently don’t address the issues that affect day-to-day quality of life. Most of what he/she knows comes from his/her experience with you, but each doctor, even Marfan specialists, see only a small number of patients. What if he/she and you could learn from not only his/her experience, but also from the experiences of many other doctors and patients. You can imagine how much better the answers would be. Backpack Health has created a tool that allows us to learn from all of our shared experiences. By gathering the health experience of many people with Marfan syndrome and related conditions, like Loeys Dietz syndrome and Ehlers-Danlos syndrome, in one place, we have the opportunity to learn much more quickly and effectively about the issues that affect daily life. By keeping your health records on the Backpack Health app, you can easily allow that information to be shared with researchers to help study Marfan syndrome and related conditions. It also allows researchers and you—through The Marfan Foundation—to quickly ask new questions without having to reenter data you previously answered. Of course privacy is an important issue with something as personal as our health records. Information in Backpack Health will only be made available for research when you TIP: YOUR MEDICAL RECORDS ARE NEVER SHARED, EVEN THOUGH YOU ARE UPLOADING OR ATTACHING THEM TO YOUR HEALTH ENTRIES. IN ORDER FOR THE DATA THAT YOU ARE AGREEING TO SHARE WITH THE REGISTRY TO BE INCLUDED IN FUTURE RESEARCH USE, IT’S IMPORTANT TO CREATE A HEALTH ENTRY NAMED APPROPRIATELY THAT INCLUDES THE KEY POINTS. FOR EXAMPLE, IF YOU HAVE AN ECHOCARDIOGRAM REPORT, YOU SHOULD NOTE THE DATE OF THE PROCEDURE ON THE “PROCEDURE” TAB. THEN, THE MEASUREMENTS FROM THE REPORT, SUCH AS HEIGHT, WEIGHT AND Z-SCORE, GO ON THE “MEASUREMENTS” TAB. DROP DOWN LISTS ARE AVAILABLE FOR COMMON PROCEDURES.

BACKPACK HEALTH IS FREE, EASY TO USE, AND SECURE. BY JOINING, YOU CAN HELP ADVANCE RESEARCH.

consent (give permission). Even then, the information included in the registry will be de-identified. This means that your name and other identifying details are removed so there is no way it can be traced back to you. Then, when approved scientists access registry data to conduct a study, they will have the benefit of including your experience in their research. “The detailed data from the app health entries and the surveys that will be conducted among participants are essential to allow scientists to compile enough data to complete studies that are sufficiently powered to show true results. Having a registry that has meaningful data will also help spark the interest of the pharmaceutical industry for drug development for these rare conditions,” said Dr. Josephine Grima, chief science officer for The Marfan Foundation. “Patient participation and willingness to take the time and effort to do this will help ensure the success of this registry.” Joining Backpack Health and participating in the registry is free. Visit our website, marfan.org, and click on the Back- pack Health icon on the top to join. If you have any questions about the registry, please contact Dr. Grima, jgrima@marfan.org.

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