Development and Validation of a Symptom Scale for Children With Chronic Graft-versus-Host Disease

NCT00632658 | Completed

• 1 other identifier: SCT 0208B
• Study Type: observational
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

Chronic Graft-versus-Host Disease (cGVHD) is an important cause of morbidity and mortality in patients undergoing allogeneic bone marrow transplantation. cGVHD usually occurs after 100 days following transplantation and develops in 20-60% of transplant recipients. The incidence of cGVHD varies depending on the age of the marrow recipient, the use of sibling or unrelated donor bone marrow, the use of unmanipulated T cell-depleted bone marrow, and perhaps other factors. Clinically, cGVHD is characterized by multi-system disease, which frequently mimics the clinical features of autoimmune diseases. The manifestations include skin changes (lichenoid and sclerodermatous changes, changes in pigmentation, loss of accessory structures such as hair, dystrophic nails, and rash), joint contractures, severe cramping, hepatic dysfunctions, sicca syndrome, obstructive lung disease, esophageal dysmotility, weight loss, polyserositis, immunodeficiency, and autoantibodies including anti-nuclear antibody, anti-erythrocyte antibodies, and anti-platelet antibodies.

Conditions

Graft vs Host Disease

Keywords

cGVHD

Outcome Measures

Primary Outcomes (1)

• To develop a Pediatric Chronic GVHD Symptoms Scale (PCSS) that reliably measures the disease specific burden of chronic GVHD in children

  One Day Interview will be performed

Study Arms (1)

Patients with cGVHD

Pediatric Patients with cGVHD will be asked to participate in an interview with their Physician. The interview will ask the pediatric patients questions about their cGVHD. The interview will be audio-recorded.

Eligibility Criteria

• Age: 5 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Patients with cGVHD will be identified by their local physican and asked to participate in this research study. A total of 24 children will be asked to participate in this study. The parents of all of the 24 children will also be asked to participate and give us their feed-back on what it is like to cope with a child with cGVHD.

You may qualify if:

• years of age
• Prior allogeneic Stem Cell Transplant, with any graft source, donor type, and GVHD prophylaxis allowed
• Clinical diagnosis of cGVHD
• Need for systemic treatment, defined as any medication or intervention delivered
• No evidence of primary disease relapse
• Signed, informed consent, and if applicable, adolescent assent

You may not qualify if:

• Inability to give signed informed consent

Sponsors & Collaborators

• Ann & Robert H Lurie Children's Hospital of Chicago: lead
• University of Minnesota: collaborator
• Dana-Farber Cancer Institute: collaborator
• Fred Hutchinson Cancer Center: collaborator

Study Sites (1)

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

MeSH Terms

Conditions

Graft vs Host Disease

Condition Hierarchy (Ancestors)

Immune System Diseases

Study Officials

• Reggie E Duerst, MD

  Ann & Robert H Lurie Children's Hospital of Chicago

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 4, 2008
• First Posted: March 11, 2008
• Study Start: January 1, 2008
• Primary Completion: July 1, 2011
• Study Completion: July 1, 2011
• Last Updated: October 8, 2019

Record last verified: 2019-10

Locations

US (1)