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

October 4, 2019 updated by: Ann & Robert H Lurie Children's Hospital of Chicago

Development and Validation of a Symptoms 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.

Study Overview

Status

Completed

Conditions

Graft vs Host Disease

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

Study Type

Observational

Enrollment (Actual)

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

United States
- Illinois
  - Chicago, Illinois, United States, 60611
    - Ann & Robert H. Lurie Children's Hospital of Chicago

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

5 years to 18 years (Child, Adult)

Accepts Healthy Volunteers

Genders Eligible for Study

All

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.

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Number of groups / cohorts

Cohorts and Interventions

Group / Cohort
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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	One Day Interview will be performed

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ann & Robert H Lurie Children's Hospital of Chicago

Collaborators

Dana-Farber Cancer Institute

Fred Hutchinson Cancer Center

University of Minnesota

Investigators

Principal Investigator: Reggie E Duerst, MD, Ann & Robert H Lurie Children's Hospital of Chicago

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2008

Primary Completion (Actual)

July 1, 2011

Study Completion (Actual)

July 1, 2011

Study Registration Dates

First Submitted

March 4, 2008

First Submitted That Met QC Criteria