August 4, 2014
A Cardiomyopathy Patient Registry
So, what is a patient registry exactly?
To put it simply, it’s a database of medical information on a particular disease or condition. For instance, a registry might track a patients’ age, sex, symptoms, devices and medications. For years medical registries have been used to track how patients do on specific medications or with a medical device. But they can be used for so much more. By tracking lab results, clinical outcomes, genetics and family history, doctors can utilize a truly complete picture of their patient. And if we track each patient with a certain disease or condition this way, researchers can begin to see similarities and differences that can lead to new standards of treatment.
There are currently medical registries that track diabetes, congestive heart failure, depression and asthma among others. Beyond a pediatric registry, until very recently, there hasn’t been a broader registry that tracks genetic cardiomyopathies, although these conditions affect more people than ALS, Muscular Dystrophy, and Multiple Sclerosis combined.
Cardiomyopathy Registry: Why Now?
There is currently no standard of care for cardiomyopathies. Treatment is done hospital by hospital, and there are limited options: invasive surgery, heart transplant or off-label use of heart drugs developed for other diseases. Not a whole lot of choice. A cardiomyopathy registry could help identify how patients fare on different treatments and could also support the development of novel therapies.
More and more is understood about the underlying genetics of Hypertrophic Cardiomyopathy and Dilated Cardiomyopathy.. The Sarcomeric Human Cardiomyopathy Registry (SHaRe) has been set up to accelerate this understanding. Pooling information about many patients – including their clinical symptoms and their underlying genetics can give doctors tremendous insight into the commonalities between certain gene mutations and patient outcomes. It can help identify in which patients a drug might likely work, and in which it will not. And even more importantly, it could help direct new drug discoveries that target the condition on the genetic level.
This new registry is finally offering hope where there has been very little. Hope for a future free of invasive surgery or transplant. Hope for freedom from a hodgepodge of drug therapies that treat only symptoms.
Hope for a better, longer, healthier life.