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


N/A
5 Years
18 Years
Not Enrolling
Both
Chronic Graft-Versus-Host-Disease

Thank you

Trial Information

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


Background:

- Currently, a large number of children with chronic GVHD have to deal with many years of
a disfiguring and painful chronic illness with the side effects of long term steroid
use.

- The broad categories of limited and extensive chronic GVHD are recognized by
clinicians, but are not particularly useful in clinical practice. Chronic GVHD may
involve almost every organ although it most commonly affects skin, eyes, mouth, liver,
intestines, lung and musculoskeletal system.

- Recently, there has been a large effort through the NIH Consensus for chronic GVHD to
standardize response criteria for patients with chronic GVHD, on clinical trials.
Because of the absence of evidence suggesting which response criteria truly correlates
with improvement, the NIH Consensus Panel has recommended following not only physical
signs of chronic GVHD, but also symptoms of GVHD.

Primary Objective:

- Develop a Pediatric Chronic GVHD Symptom Scale (PCSS) that reliably measures the disease
specific burden of chronic GVHD in children.

Secondary Objectives:

- Correlate high and low scores on the PCSS with standard quality of life measures.

- Determine using the Rasch measurement model whether the PCSS has sufficient sensitivity
to change for the scale to be useful in clinical intervention trials.

Eligibility:

- Children of 5 to 18 years of age, who have undergone prior allogeneic stem cell
transplant

- Clinical diagnosis of chronic GVHD with need for systemic treatment

- No evidence of primary disease relapse

- Must be willing to sign informed consent, or if applicable, child assent

Design:

- With the final goal of developing a scale that is similar in design to the Lee Scale
but which measures the symptom burden more specifically for children with chronic GVHD,
the study will be conducted in two phases: item generation and psychometric validation.

- In phase I, the local team at each institution will interview participants using a
script to identify symptom concerns for pediatric chronic GVHD patients. Based on
interviews, we will decide whether to test different scales for the different age
groups, or a single scale for all.

- Data from Phase II will be used to finalize and validate the pediatric symptom scale,
through the assessment of test-retest characteristics, use of item reduction,
examination of construct validity, internal consistency, convergent and divergent
validity, and evaluation of sensitivity to change.

Inclusion Criteria


- INCLUSION CRITERIA:

- Children of 5 to 18 years of age, who have undergone prior allogeneic stem cell
transplant.

- Clinical diagnosis of chronic GVHD with need for systemic treatment.

- No evidence of primary disease relapse.

- Must be willing to sign informed consent, or if applicable, child assent.

Type of Study:

Observational

Study Design:

Time Perspective: Prospective

Principal Investigator

Lori Wiener, Ph.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

National Cancer Institute (NCI)

Authority:

United States: Federal Government

Study ID:

100019

NCT ID:

NCT01036958

Start Date:

November 2009

Completion Date:

Related Keywords:

  • Chronic Graft-Versus-Host-Disease
  • Pediatric cGVHD
  • Response Criteria
  • Symptoms
  • Quality of Life
  • Graft-Versus-Host Disease
  • GVHD
  • Pediatric GVHD
  • Graft vs Host Disease

Name

Location

National Institutes of Health Clinical Center, 9000 Rockville PikeBethesda, Maryland  20892