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

NCT ID: NCT00632658

Last Updated: 2019-10-08

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 24 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2008-01-31
• Study Completion Date: 2011-07-31

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.

Detailed Description

A large number of children with cGVHD have to deal with many years of a disfiguring and painful chronic illness with the side effects of long term steroid use. The number of stem cell transplants done in children is only growing given that we are now transplanting patients with a variety of nonmalignant disorders and given the use of alternative donor sources. The broad categories of limited and extensive cGVHD are recognized by clinicians, but are not particularly useful in clinical practice. Since cGVHD may involve almost every organ system adn since cGVHD constitutes a waxing and waning nature, cGVHD makes clinical management very difficult and complicated. Currently, there is a symptoms scale used in the adult population for measuring symptom burden for adults with cGVHD. This scale is called the "Lee Symptoms Scale". The purpose of this project is to develop a scale that is similar in design to the Lee Scale, but it is specifically designed to measure the burden of cGVHD in the pediatric population

Conditions

Graft vs Host Disease

Study Design

• Observational Model Type: CASE_ONLY
• Study Time Perspective: PROSPECTIVE

Study Groups

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.

No interventions assigned to this group

Eligibility Criteria

Inclusion Criteria

• 5-18 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

Exclusion Criteria

• Inability to give signed informed consent

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Minnesota

OTHER

Sponsor Role: collaborator

Dana-Farber Cancer Institute

OTHER

Sponsor Role: collaborator

Fred Hutchinson Cancer Center

OTHER

Sponsor Role: collaborator

Ann & Robert H Lurie Children's Hospital of Chicago

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Reggie E Duerst, MD

Role: PRINCIPAL_INVESTIGATOR

Ann & Robert H Lurie Children's Hospital of Chicago

Locations

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

Chicago, Illinois, United States

Countries

United States

Other Identifiers

SCT 0208B

Identifier Type: -

Identifier Source: org_study_id