Protocol Number: 04-C-0281

Title:

Natural History Study of Clinical and Biological Factors Determining Outcomes in Chronic Graft-Versus-Host Disease

Number:

04-C-0281

Summary:

Background:

-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 cGVHD.

-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.

Objectives:

-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 cGVHD.

-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.

Eligibility:

-Patients age 1-75 referred by the primary transplant physician for the evaluation of chronic graft-versus-host disease independent of underlying diagnosis.

Design:

-Patient undergoes initial clinical and labotory multispecialty work-up at the NCI cGVHD clinic.

-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.

Sponsoring Institute:

National Cancer Institute (NCI)

Recruitment Detail

Type: Participants currently recruited/enrolled

Gender: Male & Female

Referral Letter Required: No

Population Exclusion(s): None

Eligibility Criteria:

INCLUSION CRITERIA:

a) Any patient age 1-75 referred by the primary transplant physician for the evaluation of chronic graft-versus-host disease independently of age or underlying diagnosis;

b) Patient or the patient's legal representative is able and willing to provide consent.

EXCLUSION CRITERIA:

a) Significant medical condition or any other significant circumstance that could in the PIs assessment affect the patient's ability to tolerate, comply, or complete the study;

b) Patients who in the PIs assessment have a life expectancy less than 3 months.

Note: Because it is not always possible to make a clear clinical distinction between acute and chronic GVHD, patients with acute GVHD are not a-priori excluded until the possibility of chronic GVHD is reliably excluded on the basis of the clinical assessments in the cGVHD clinic.

Special Instructions:

Currently Not Provided

Keywords:

Natural History

Screening

Biopsies

Leukapheresis

Questionnaires

Recruitment Keyword(s):

Chronic Graft-Versus-Host Disease

cGVHD

Condition(s):

Graft vs Host Disease

Investigational Drug(s):

None

Investigational Device(s):

None

Intervention(s):

None

Supporting Site:

National Cancer Institute

Contact(s):

NCI Referral Office
National Institute of Health Clinical Center (CC), 9000 Rockville Pike, Bethesda, Maryland 20892, United States: NCI Clinical Trials Referral Office
Phone: 1-888-NCI-1937
Fax: Not Listed
Electronic Address: ncicssc@mail.nih.gov

Citation(s):

Lee SJ, Vogelsang G, Flowers ME. Chronic graft-versus-host disease. Biol Blood Marrow Transplant. 2003 Apr;9(4):215-33.

Beschorner WE, Tutschka PJ, Santos GW. Chronic graft-versus-host disease in the rat radiation chimera. I. Clinical features, hematology, histology, and immunopathology in long-term chimeras. Transplantation. 1982 Apr;33(4):393-9. No abstract available.

Tutschka PJ, Teasdall R, Beschorner WE, Santos GW. Chronic graft-versus-host disease in the rat radiation chimera. II. Immunological evaluation in long-term chimeras. Transplantation. 1982 Nov;34(5):289-94. No abstract available.

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