LGDA Establishes Patient Registry


Will Participate in Global Rare Diseases Patient Registry

iStock_000003384308XSmallThe Lymphangiomatosis & Gorham’s Disease Alliance (LGDA) is pleased to announce the establishment of the International LGDA Registry for Lymphatic Malformations (LGDA Registry), a major goal of the LGDA since the organization’s founding. The LGDA Registry will be instrumental in accelerating the pace of basic and clinical research for the life-threatening disorders of lymphangiomatosis and Gorham’s disease, as well as other lymphatic malformations and osteolytic bone diseases. It also will help generate valuable insights into bone and lymphatic biologies, an understanding of which is critical for a plethora of other diseases.

The International LGDA Registry for Lymphatic Malformations will serve as a centralized repository consisting of patient demographic data; clinical histories; hematological, radiological, and nuclear medicine data; and tissue specimens from known cases of lymphangiomatosis and Gorham’s disease, and related lymphatic malformations and bone diseases, in order to establish the incidence, clinical presentation(s), morbidity, and mortality of these complex diseases, while protecting patient privacy by shielding patient identities from researchers. One important benefit for lymphatic malformation patients who participate in the LGDA registry will be access to information about clinical trials and the development of new treatments from which they may benefit.

The establishment of the LGDA Registry was made possible through the Global Rare Diseases Patient Registry and Data Repository (GRDR), a two-year pilot program launched by the Office of Rare Diseases Research (ORDR), National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health, Bethesda, MD. The contract for implementation of the GRDR pilot program was awarded to PatientCrossroads, in collaboration with Childrens Hospital of Philadelphia and WebMD.

The goal of the GRDR is to create a resource of de-identified patient information from rare disease registries to help facilitate research and clinical trials; analyses of data across many disorders; and ultimately the development of drugs and therapies for treatment of individuals effected by rare diseases. Long-term goals involve development of open-science principles and a sustainable resource funded and supported by private/public partnerships.

Thirty-four organizations have been selected by the ORDR to participate in the GRDR pilot program. The LGDA is one of 15 organizations selected to establish new patient registries.

“We are very pleased to be a partner in this special pilot effort with the NIH/NCATS/ORDR and the collaborators, to help all rare diseases,”  said Jack Kelly, President of the LGDA. “Being a part of the GRDR is an exceptional opportunity for the LGDA to work with the international patient and scientific community to advance knowledge about lymphangiomatosis and Gorham’s disease and make progress towards better treatments, improved quality of life, and, ultimately, to find a cure for these extraordinarily rare diseases.”

The LGDA Registry has begun beta testing and will go ‘live’ in the next few weeks. Watch your email, check our website, and follow us on facebook for news about the coming official launch of the International LGDA Registry for Lymphatic Malformations and how you may participate.

The LGDA has named Lisa Klepper, RN, Manager, Patient Registry. She may be reached by email: lklepper@lgdalliance.org

For additional information about the Global Rare Diseases Patient Registry (GRDR) go to www.GRDR.info.