Development and Validation of a Symptom Scale for Children With Chronic Graft-versus-Host Disease
| Status: | Completed |
|---|---|
| Conditions: | Hematology |
| Therapuetic Areas: | Hematology |
| Healthy: | No |
| Age Range: | 5 - 18 |
| Updated: | 9/28/2018 |
| Start Date: | November 25, 2009 |
| End Date: | February 6, 2018 |
Development and Validation of a Symptom Scale for Children With Chronic Graft-Versus-Host Disease
Background:
- Chronic graft-vs-host disease (GVHD) is an important cause of morbidity and mortality in
patients undergoing allogeneic bone marrow transplantation. The symptoms of chronic GVHD
are similar to those of other autoimmune diseases, and treatment for the pain often
involves steroid use that can cause severe side effects over the long term.
- At present, there is no research instrument that measures symptoms in children with
chronic GVHD. Treatment practitioners may use one of several pediatric quality of life
questionnaires, but because none of these is specific for chronic GVHD each instrument
has potential gaps in its ability to assess the full spectrum of problems experienced by
children with chronic GVHD. Researchers are interested in developing a better
understanding of the disease burden experienced by children and adolescents with chronic
GVHD.
Objectives:
- To develop a Pediatric Chronic GVHD Symptom Scale (PCSS) that reliably measures the
disease-specific burden of chronic GVHD in children.
Eligibility:
- Children and adolescents 5 to 18 years of age who have undergone prior allogeneic stem cell
transplant and have been diagnosed with chronic GVHD that requires treatment.
Design:
- There are two phases to the study; participants will enroll in phase I (question
generation) at this time.
- Researchers will interview participants and ask open-ended questions (requiring more
than a one- or two-word response) about symptoms that adults with chronic GVHD have
found problematic.
- Both parents and children will participate in the interviews, which will be
audio-recorded. Depending on the child or adolescent s age, the interviews may be
conducted together with the parents or separately.
- No treatment will be given as part of this study.
- Chronic graft-vs-host disease (GVHD) is an important cause of morbidity and mortality in
patients undergoing allogeneic bone marrow transplantation. The symptoms of chronic GVHD
are similar to those of other autoimmune diseases, and treatment for the pain often
involves steroid use that can cause severe side effects over the long term.
- At present, there is no research instrument that measures symptoms in children with
chronic GVHD. Treatment practitioners may use one of several pediatric quality of life
questionnaires, but because none of these is specific for chronic GVHD each instrument
has potential gaps in its ability to assess the full spectrum of problems experienced by
children with chronic GVHD. Researchers are interested in developing a better
understanding of the disease burden experienced by children and adolescents with chronic
GVHD.
Objectives:
- To develop a Pediatric Chronic GVHD Symptom Scale (PCSS) that reliably measures the
disease-specific burden of chronic GVHD in children.
Eligibility:
- Children and adolescents 5 to 18 years of age who have undergone prior allogeneic stem cell
transplant and have been diagnosed with chronic GVHD that requires treatment.
Design:
- There are two phases to the study; participants will enroll in phase I (question
generation) at this time.
- Researchers will interview participants and ask open-ended questions (requiring more
than a one- or two-word response) about symptoms that adults with chronic GVHD have
found problematic.
- Both parents and children will participate in the interviews, which will be
audio-recorded. Depending on the child or adolescent s age, the interviews may be
conducted together with the parents or separately.
- No treatment will be given as part of this study.
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.
- 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:
- 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.
We found this trial at
1
site
9000 Rockville Pike
Bethesda, Maryland 20892
Bethesda, Maryland 20892
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