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

Graft vs Host Disease Clinical Trial

— cGVHD  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00632658
• Other study ID #: SCT 0208B
• Status: Completed
• Start date: January 2008
• Est. completion date: July 2011

Study information

• Verified date: October 2019
• Source: Ann & Robert H Lurie Children's Hospital of Chicago
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 24
• Est. completion date: July 2011
• Est. primary completion date: July 2011
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Graft vs Host Disease

Locations

Country	Name	City	State
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois

Sponsors (4)

Lead Sponsor	Collaborator
Ann & Robert H Lurie Children's Hospital of Chicago	Dana-Farber Cancer Institute, Fred Hutchinson Cancer Research Center, University of Minnesota - Clinical and Translational Science Institute

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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