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This multi-center cohort study by the Primary Immune Deficiency Treatment Consortium (PIDTC) collects medical record information about patients with chronic granulomatous disease (CGD), an uncommon disease in which bone marrow transplantation has been shown to be curative. Risks of bone marrow transplantation, also called hematopoietic cell transplantation (HCT), are high and not all patients with CGD may require this procedure. This study will investigate which patients benefit most from HCT and what types of transplants are optimal for patients with CGD, in the context of overall outcomes in CGD patients with and without transplant.
Subject to certain exclusion criteria, this study collects and reviews medical record and biological information of male and female patients of all ages who are being treated for CGD.
Males and Females: Ages 2 years and older
Participation entails collection of blood and tissue samples from participants and completion of several quality of life questionnaires, which can be accomplished in a single study visit arranged in connection with a regularly scheduled clinic appointment. Information collected is maintained in a non-identifiable manner in the study database.
Duration: Not applicable
National Institute of Allergy and Infectious Diseases
Primary Immune Deficiency Treatment Consortium
Rare Diseases Clinical Research Network
Study Description at National Institutes of Health
Allergy and Immunology Research
Hey Jin Chong, MD, PhD
For more information about the study or enrollment, please contact:
Linette Milkovich, RN
Clinical Research Nurse Coordinator
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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