Natural History Study of Clinical and Biological Factors Determining Outcomes in Chronic Graft-Versus-Host Disease
- Conditions
- Chronic Graft vs. Host Disease
- Registration Number
- NCT00092235
- Lead Sponsor
- National Cancer Institute (NCI)
- Brief 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 8000 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
* To identify potential patients for cGVHD treatment protocols at the NCI and NIH
Eligibility:
-Patients age 1 and older 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 laboratory 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 anually as feasible
- Detailed Description
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 8000 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 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
* To identify potential patients for cGVHD treatment protocols at the NCI and NIH
Eligibility:
-Patients age 1 and older 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 laboratory multi-specialty 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 term data collection for evaluation of long-term outcomes will be conducted annually as feasible.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 650
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method To establish a multidisciplinary clinic infrastructure for study of pathogenesis and natural history of cGVHD ongoing Assessment of clinical and biological characteristics of cGVHD.
To prospectively identify candidate markers for clinical and biological prognostic factors in patients with cGVHD and develop a prognostic model 2 years + 3 months after protocol entry Patient evaluations resulting in collection of data via several medical specialties; data will be examined individually and against clinical outcomes.
To improve our current understanding of the biology of cGVHD-associated graft-versus-tumor effects (GVT). ongoing Studying mechanisms of how cGVHD exerts its anti-cancer effects via laboratory analysis.
To identify potential clinical and biological markers of cGVHD activity ongoing Assessment of risk and outcome as related to molecular markers of pathogenesis and/or stage of disease.
To identify novel biological characteristics of cGVHD and to describe them in the context of clinical history and presentation ongoing Through collection of data via several medical specialties, assess the weight of specific clinical and biological characteristics and disease severity scales for predicting major clinical outcomes.
To develop clinically relevant cGVHD grading scales ongoing Develop appropriate staging as a tool for measuring responses or outcomes in clinical studies through prospective collection and analysis of data.
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
National Institutes of Health Clinical Center
🇺🇸Bethesda, Maryland, United States