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

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.

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Key information

Conditions

Graft vs Host Disease Immune System Diseases

Age range

5 year–18 year

Sex eligibility

All sexes

Study type

Observational

Primary location

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

Chicago, Illinois, 60611, United States

About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

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

Time frame: One Day Interview will be performed

Sponsors and collaborators

Lead sponsor

Ann & Robert H Lurie Children's Hospital of Chicago

Other

Collaborators

Dana-Farber Cancer Institute
Fred Hutchinson Cancer Center
University of Minnesota

Registry information

Official study title

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

Acronym: cGVHD

Important dates

Study start: 2008
Primary completion: 2011
Study completion: 2011
First posted: Mar 11, 2008
Registry last updated: Oct 8, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed