Natural History Study of Clinical and Biological Factors Determining Outcomes in Chronic Graft-Versus-Host Disease
- Chronic graft-versus-host disease (cGVHD) is a multi-organ alloimmune and autoimmune
disorder that occurs following allogeneic hematopoietic stem cell transplantation
(alloHSCT). It is characterized by immune dysregulation, immunodeficiency, impaired
organ function, and decreased survival.
- Each year about 7000 patients receive allogeneic hematopoietic stem cell transplant
(alloHSCT) in North America and about 50% of patients who are transplanted develop
- Chronic GVHD is also a disorder that simultaneously affects many organ systems in
highly variable fashion and requires complex and coordinated medical management by
multiple medical specialties. There is an urgent need for progress in understanding
and effective treatments for cGVHD as it is one of the most serious complications of
cancer therapy and hematopoietic stem cell transplantation.
- To establish a multidisciplinary clinic infrastructure for study of the pathogenesis
and natural history of cGVHD.
- To prospectively identify clinical and biological prognostic markers in patients with
- To develop clinically relevant cGVHD grading scales.
- To identify novel biological characteristics of cGVHD and to describe them in the
context of clinical history and presentation.
- To identify potential clinical and biological markers of cGVHD activity.
- To improve understanding of the biology of cGVHD-associated graft-versus-tumor effects.
-Patients age 1-75 referred by the primary transplant physician for the evaluation of
chronic graft-versus-host disease independent of underlying diagnosis.
- Patient undergoes initial clinical and laboratory multispecialty work-up at the NCI
- Minimally invasive biopsies and rarely, deep tissue biopsy may be obtained to confirm
the diagnosis and/or rule-out other pathologic process (in adults only).
- Long tem data collection for evaluation of long-term outcomes will be conducted every
six months during the first three years of the study and then yearly.
Steven Z Pavletic, M.D.
National Cancer Institute (NCI)
United States: Federal Government
|National Institutes of Health Clinical Center, 9000 Rockville Pike||Bethesda, Maryland 20892|