Alternate Title
Basic Trial Information
Objectives
Entry Criteria
Expected Enrollment
Outcomes
Outline
Published Results
Trial Contact Information
Related Information
Registry Information

Alternate Title

Clinical Evaluations and Laboratory Studies in Patients With Chronic Graft-Versus-Host Disease Who Have Undergone a Previous Donor Stem Cell Transplant

Basic Trial Information

Phase	Type	Status	Age	Sponsor	Protocol IDs
No phase specified	Biomarker/Laboratory analysis, Natural history/Epidemiology	Active	1 to 75	NCI	NCI-04-C-0281 NCT00331968

Special Category: NIH Clinical Center trial, NCI Web site featured trial

Objectives

1. Establish a multidisciplinary clinic infrastructure for studying pathogenesis and the natural history of chronic graft-vs-host disease (cGVHD).
2. Determine candidate markers for clinical and biological prognostic factors in patients with cGVHD after allogeneic hematopoietic stem cell transplantation.
3. Develop a prognostic model using patient survival and being permanently off immunosuppressive drugs for ≥ 3 months 2 years after study entry as the primary endpoint.
4. Develop clinically relevant cGVHD grading scales.
5. Determine novel biological characteristics of cGVHD and describe them in the context of clinical history and presentation.
6. Determine potential clinical and biological markers of cGVHD activity.
7. Improve current understanding of the biology of cGVHD-associated graft-vs-tumor effects.

Entry Criteria

Disease Characteristics:

• Diagnosis of chronic graft-vs-host disease (cGVHD)*, independent of underlying diagnosis, meeting the following criteria:
  • Presence of ≥ 1 clinical manifestation that is distinct for cGVHD
  • No infection, drug reaction, or recurrent malignancy
  • Tissue biopsy of ≥ 1 involved organ compatible with cGVHD, if medically possible
  • Autoimmune phenomena or entities not sufficient by itself for diagnosis of cGVHD (e.g., bronchiolitis obliterans, myositis, immune thrombocytopenic purpura, glomerulopathy, myasthenia, or serositis) need ≥ 1 other typical clinical manifestation distinct for cGVHD and/or a biopsy consistent with cGVHD

     [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 cGVHD is reliably excluded on the basis of clinical assessments in the cGVHD clinic ]

• Received prior allogeneic hematopoietic stem cell transplantation
• Prior nonmalignant diagnosis is allowed if patient has cGVHD (e.g., aplastic anemia)

Prior/Concurrent Therapy:

• See Disease Characteristics

Patient Characteristics:

• Life expectancy ≥ 3 months
• No significant medical condition or circumstance that would affect the patient's ability to tolerate, comply, or complete the study

Expected Enrollment

A total of 290 patients will be accrued for this study.

Outcomes

Overall survival as assessed by Kaplan-Meier
Graft-vs-host disease (cGVHD)-specific survival (until death due to cGVHD) as assessed by Kaplan-Meier
Time until removal of immunosuppressive drugs
Need for new form of systemic therapy
Time to discontinue systemic steroids
Therapeutic failure (need for new form of therapy or death)
Progression of underlying disease
Return to a full time job

Outline

This is a prospective, natural history, retrospective, longitudinal data collection study.

Patients referred for evaluation of chronic graft-vs-host disease (cGVHD) undergo a clinical and laboratory multispecialty diagnostic evaluation that includes blood collection and biopsies. A summary of the multidisciplinary evaluation and recommendations are conveyed to the patient and primary physician. Selected clinical outcomes data is collected from patients by telephone and mail (i.e., interviews and questionnaires) every 6 months for 3 years and then annually for up to 10 years if chronic GVHD is confirmed or cannot be ruled out.

Patients may undergo additional blood collection and tissue biopsies periodically for biomarker correlative studies.

Clark J, Yao L, Pavletic SZ, et al.: Magnetic resonance imaging in sclerotic-type chronic graft-vs-host disease. Arch Dermatol 145 (8): 918-22, 2009.[PUBMED Abstract]

Imanguli MM, Swaim WD, League SC, et al.: Increased T-bet+ cytotoxic effectors and type I interferon-mediated processes in chronic graft-versus-host disease of the oral mucosa. Blood 113 (15): 3620-30, 2009.[PUBMED Abstract]

Trial Contact Information

Trial Lead Organizations

NCI - Center for Cancer Research

Steven Pavletic, MD, Principal investigator		Ph: 301-402-4899

U.S.A.
Maryland
	Bethesda
	Warren Grant Magnuson Clinical Center - NCI Clinical Trials Referral Office
		Clinical Trials Office - Warren Grant Magnusen Clinical Center - NCI Clinical Trials Referral Office	Ph:  888-NCI-1937
New Jersey
	Hackensack
	Hackensack University Medical Center Cancer Center
		Clinical Trials Office - Hackensack University Medical Center Cancer Center	Ph:  201-996-2879

Related Information

Web site for additional information
Featured trial article

Registry Information
Official Title		Natural History Study of Clinical and Biological Factors Determining Outcomes in Chronic Graft-Versus-Host Disease
Trial Start Date		2004-09-28
Registered in ClinicalTrials.gov		NCT00331968
Date Submitted to PDQ		2004-09-28
Information Last Verified		2006-09-20

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