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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

Outcome Measures

Primary Outcomes (6)

To prospectively identify candidate markers for clinical and biological prognostic factors in patients with cGVHD and develop a prognostic model
Patient evaluations resulting in collection of data via several medical specialties; data will be examined individually and against clinical outcomes.
2 years + 3 months after protocol entry
To improve our current understanding of the biology of cGVHD-associated graft-versus-tumor effects (GVT).
Studying mechanisms of how cGVHD exerts its anti-cancer effects via laboratory analysis.
ongoing
To identify potential clinical and biological markers of cGVHD activity
Assessment of risk and outcome as related to molecular markers of pathogenesis and/or stage of disease.
ongoing
To identify novel biological characteristics of cGVHD and to describe them in the context of clinical history and presentation
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.
ongoing
To establish a multidisciplinary clinic infrastructure for study of pathogenesis and natural history of cGVHD

Study Arms (4)

Cohort 1

Patients who have undergone an allogeneic stem cell transplant and are diagnosed with cGVHD

Cohort 2

Pediatric patients who have undergone an allogeneic stem cell transplant and are diagnosed with cGVHD

Cohort 3

Patients who have undergone an allogeneic stem cell transplant and choose to submit biopsy, blood and urine samples only

Cohort 4

Patients who have undergone an allogeneic stem cell transplant and are not diagnosed with cGVHD

Eligibility Criteria

Age1 Year - 120 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

Sampling MethodNon-Probability Sample

Study Population

Patients will be selected via referrals or from patients who were on companion clinical protocols at the NIH.

You may qualify if:

Any patient age 1 and older referred by the primary transplant physician for the evaluation of chronic graft-versus-host disease independently of age or underlying diagnosis

Related Publications (11)

Zhao AT, Pirsl F, Steinberg SM, Holtzman NG, Schulz E, Mina A, Mays JW, Cowen EW, Comis LE, Joe GO, Yanovski JA, Pavletic SZ. Metabolic syndrome prevalence and impact on outcomes in patients with chronic graft-versus-host disease. Bone Marrow Transplant. 2023 Dec;58(12):1377-1383. doi: 10.1038/s41409-023-02097-y. Epub 2023 Sep 8.

PMID: 37684526DERIVED

Schulz E, Pirsl F, Holtzman NG, Beshensky D, Cowen EW, Mitchell SA, Steinberg SM, Pavletic SZ. Red cell distribution width as a new prognostic biomarker in refractory chronic graft-versus-host disease. Haematologica. 2024 Jan 1;109(1):298-302. doi: 10.3324/haematol.2023.283646. No abstract available.

PMID: 37584292DERIVED

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

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Conditions

Keywords

Outcome Measures

Primary Outcomes (6)

Study Arms (4)

Cohort 1

Cohort 2

Cohort 3

Cohort 4

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

Related Publications (11)

Related Links

Study Officials

Central Study Contacts

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Conditions

Keywords

Outcome Measures

Primary Outcomes (6)

Study Arms (4)

Cohort 1

Cohort 2

Cohort 3

Cohort 4

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

Related Publications (11)

Related Links

Study Officials

Central Study Contacts

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations