Who We Are

History

The Lymphangiomatosis and Gorham’s Disease Alliance (LGDA) is a patient support organization dedicated to improving understanding and treatment for the rare lymphatic malformations and osteolytic bone diseases mostly known as lymphangiomatosis and Gorham’s disease.

Lymphangiomatosis and Gorham’s disease are very rare disorders of the lymphatic system that have received little attention from the research community since their recognition more than a century ago. A key reason for this deficit is the lack of accurate, organized data regarding the incidence and diagnosis of these disorders. By establishing the International LGDA Registry for Lymphatic Malformations, the LGDA seeks to establish 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 incidence, clinical presentation(s), morbidity, and mortality of these complex diseases.

The International LGDA Registry for Lymphatic Malformations will be instrumental in accelerating the pace of basic and clinical research for the life-threatening disorders of lymphangiomatosis and Gorham’s disease and also will help generate valuable insights into bone and lymphatic biologies, an understanding of which is critical for a plethora of other diseases.
 

Objectives

The LGDA established the International LGDA Registry for Lymphatic Malformations in order to collect and store health information vital to the study of lymphangiomatosis & Gorham’s disease. The Registry seeks to:

•    document the clinical course of  lymphangiomatosis and Gorham's disease patients and their responses to therapy;    

•    elicit the natural history of lymphangiomatosis and Gorham's disease and its impact on patients and their families;

•    document and analyze the type, incidence, and outcomes of complications associated with lymphangiomatosis and Gorham's   

     disease, its diagnosis, and treatment;

•    link clinical data to biological specimens from patients with lymphangiomatosis and Gorham's disease;

•    document and analyze the type(s) and incidence of other chronic and congenital conditions in patients with

     lymphangiomatosis and Gorham's disease;

•    document and analyze the morbidity and mortality associated with lymphangiomatosis and Gorham's disease;

•    facilitate the establishment of standards for diagnosis and care of patients with lymphangiomatosis and Gorham's disease;  

•    attract scientists, researchers, and clinicians by having a trial-ready, promptly-reachable population of patients who indicate in

     their registry profile a desire and willingness to participate in clinical trials;

•    identify cohort(s) for possible genetic studies and clinical trials and keep registered patients informed about ongoing and

     upcoming clinical trials;

•    build a directory of physicians having experience caring for patients with lymphangiomatosis and Gorham's disease;        

•    generate funding for cutting edge research using de-identified statistical data; and    

•    raise awareness about lymphangiomatosis and Gorham's disease and serve as a comprehensive information resource for the

     education of patients, physicians, and families.

The International LGDA Registry for Lymphatic Malformations also seeks to contribute to basic scientific knowledge. In furtherance of this goal, de-identified registry data will be made available to research entities having Institutional Review Board approved protocols seeking to study lymphangiomatosis, Gorham’s disease, or other diseases involving the lymphatic and skeletal system, and those organs and organ systems which are frequently the location of disease involvement, such as the lungs, as well as other research projects that the LGDA Medical Advisory Council may deem will contribute to basic scientific knowledge.

Partners

The establishment of the Registry was made possible through the Global Rare Diseases Patient Registry and Data Repository (GRDR), a two-year pilot program launched in 2011 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. With the end of the pilot program in 2013, the LGDA Registry selected PatientCrossroads to remain its host.

To further its mission to advance research that will improve diagnosis and treatment of lymphangiomatosis and Gorham's disease the LGDA works closely with the Lymphatic Malformation Institute (LMI). The LMI was established in December, 2010, and is focused, as a whole, on advancing the understanding of the lymphatic system and, specifically, advancing the understanding of the rare lymphatic malformations, Lymphangiomatosis and Gorham’s Disease.

The LGDA and the LMI share the goal of finding cures and treatments for these life-threatening disorders that have received very little attention from the scientific community in the past. The LMI seeks to fill this critical gap by identifying, organizing and funding research studies by medical and research professionals, and also working closely with other foundations with similar objectives.