Pediatric cGVHD Symptom Scale

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ClinicalTrials.gov Identifier: NCT04044365

Recruitment Status : Recruiting

First Posted : August 5, 2019

Last Update Posted : June 24, 2022

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Sponsor:

Information provided by (Responsible Party):

National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) )

Study Description

Brief Summary:

Background:

Some children/adolescents who have had a stem cell transplant live with chronic graft-versus-host-disease (cGVHD). cGVHD is a side effect of the transplant that can cause multiple bothersome symptoms and negatively affect a child/adolescent squality of daily life. The questionnaires that measure thesymptoms caused by cGVHD are designed for adults. Children/adolescents may not describe their symptoms in the same way. The goal of this research is to improve the way we measure how bothersome these symptoms are for children/adolescents living with cGVHD.

Objective:

To develop a questionnaire (The Pediatric cGHVD Symptom Scale) for children/adolescents living with cGVHD to identify the symptoms they are experiencing and describe how bothersome those symptoms are to them. An additional goal is to design a parent/guardian companion questionnaire that can be used to capture the symptom experiences of very young children who may not be able to complete a questionnaire.

Eligibility:

Children/adolescents ages 5-17 who are receiving treatment for cGVHD after a stem cell transplant, and their parent/guardian..

Design:

This study consists of 2 projects.

Children/adolescents with cGVHD and their parent/guardianparticipants will be grouped by the child/adolescent s age: 5-7, 8-12, and 13-17.

In project 1, participants will complete an age-appropriate questionnaire about cGVHD symptoms. The questionnaire will ask about the child/adolescent s physical functioning and emotional well-being. The parent/guardian will out fill out a companion questionnaire online. The child/adolescent will then review their completed questionnaire during an interview with a researcher and will be asked whether the questions about their symptoms were difficult to understand. The parent/guardian and child/adolescent will then be interviewed together to further explore their responses to the questionnaires. Interviews will be done in person, by phone, and online. . Based on what is learned through these interviews, the wording of the questionnaire will be improved for better comprehension and ease of response.

In project 2, participants will complete this revised questionnairefor their age group along with some other questionnaires that ask about quality of life. Both the child/adolescent and parent/guardian will fill out the questionnaires online at three separate time points.

In both projects, children/adolescents with cGVHD and their parent/guardian participants will be grouped by the child/adolescent s age: 5-7, 8-12, and 13-17.

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Condition or disease
Graft vs Host Disease

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Detailed Description:

Background:

Hematopoietic stem cell transplantation (HSCT) is a curative option for many children with cancer and certain genetic disorders. However, recipients of stem cell transplant must deal with years of disfigurement and symptoms arising from an undesirable side effect of transplant, specifically chronic graft-versus-host-disease (cGVHD).

Children living with cGVHD often face many years of a disabling and painful chronic illness compounded by the side effects of prolonged immunosuppression, particularly long-term steroid use.

Chronic GVHD can involve almost every organ although it most commonly affects the skin, eyes, mouth, liver, intestines, lung and the musculoskeletal system.

There has been a large effort through the NIH cGVHD Consensus Consortium to standardize definitions, grading and response criteria for patients with cGVHD 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.

The Lee cGVHD Symptom Score has been recognized as a core cGVHD outcome measure in the adult population, however, the instrument has not been validated for use in children/adolescents younger than 18, and many of the symptom terms used in the Lee cGVHD Symptom Scale may not be well understood by younger children.

There is currently no available pediatric patient-reported outcome (PRO) measure of cGVHD symptom bother.

Primary Objective:

To develop a psychometrically valid Pediatric cGVHD Symptom Scale (PCSS) and a companion caregiver-proxy measure as counterparts to the Lee cGVHD Symptom Scale.

Eligibility:

Children of 5 to 17 years of age, who have undergone prior allogeneic stem cell transplant

Clinical diagnosis of cGVHD

Currently receiving systemic treatment for cGVHD (including phototherapies), or has tapered systemic therapy for cGVHD to discontinuation within the past 12 months

No evidence of malignant disease relapse including molecular relapse and minimal residual disease. Patients with mixed chimerism are eligible to participate

Comprehend and speak English

Design:

This multicenter non-interventional psychosocial trial will address the study aims in two projects: 1) a qualitative project using cognitive interviewing to confirm the comprehension and ease of response to the PCSS, and 2) a quantitative project to evaluate test-retest reliability, construct validity and responsiveness of the PCSS, as well as to explore concordance of symptom ratings by patients and caregiver proxies.

In Project 1, we will conduct at least 20 cognitive interviews of patient and proxy dyads (caregiver) within each age group (5-7; 8-12; 13-17) for a sample of 60 pediatric transplant survivors with cGVHD to refine the symptom items and response choices to ensure that they resonate across the developmental spectrum. Cognitive interviews will evaluate the clarity and comprehension of the PCSS items, the ease and accuracy of judgement of the response choices, and any errors in understanding the response choices, and will refine the PCSS in accordance with study data, before proceeding to Project 2.

In Project 2, we will evaluate test-retest reliability, construct validity, and responsiveness of the PCSS, in association with the NIH Global cGVHD Symptom Severity Score, the Peds QL, clinician ratings of organ specific cGVHD severity scores and cGVHD severity using the NIH criteria, in a sample of 120 pediatric transplant survivors with cGVHD reflecting three age groups (5-7 years; 8-12 years; 13-17 years) of equal numbers. In subjects ages 13-17, we will also compare responses on the PCSS with those from the Lee Scale, to determine the youngest age at which adolescent respondents can comprehend the Lee Scale.

This study will include CCR investigators as well as extramural investigators at national and international study sites.

Study Design

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Study Type :	Observational
Estimated Enrollment :	360 participants
Observational Model:	Cohort
Time Perspective:	Prospective
Official Title:	Development and Psychometric Testing of a Pediatric Chronic Graft- Versus-Host Disease (GVHD) Symptom Scale (PCSS)
Actual Study Start Date :	October 2, 2019
Estimated Primary Completion Date :	April 1, 2024
Estimated Study Completion Date :	June 1, 2024

Resource links provided by the National Library of Medicine

Genetic and Rare Diseases Information Center resources: Homologous Wasting Disease Chronic Graft Versus Host Disease

U.S. FDA Resources

Groups and Cohorts

Group/Cohort
1/Project 1 Child/caregiver-proxy Children age 5-7 with cGVHD and their caregiver, n=20 child/parent dyads
2/Project 1 Child/caregiver-proxy Children age 8-12 with cGVHD and their caregiver, n=20 child/parent pairs
3/Project 1 Child/caregiver-proxy Children age 13-17 with cGVHD and their caregiver, n=20 child/parent pairs
4/Project 2 Child/caregiver-proxy Children age 5-7 with cGVHD and their caregiver, n=40 child/parent pairs
5/Project 2 Child/caregiver-proxy Children age 8-12 with cGVHD and their caregiver, n=40 child/parent pairs
6/Project 2 Child/caregiver-proxy Children age 13-17 with cGVHD and their caregiver, n=40 child/parent pairs

Outcome Measures

Primary Outcome Measures :

Feasability [ Time Frame: 3 years ]
To develop a psychometrically valid Pediatric cGVHD Symptom Scale (PCSS) and a companion parent-proxy measure as counterparts to the Lee cGVHD Symptom Scale.

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:	5 Years and older (Child, Adult, Older Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	Yes
Sampling Method:	Non-Probability Sample

Study Population

Children of 5 to 17 years of age and their caregiver, who have undergone prior allogeneic stem cell transplant

Criteria

INCLUSION CRITERIA:

Pediatric Subject Inclusion Criteria:

Children aged 5 to 17 years old, who have undergone prior allogeneic stem cell transplant
Clinical diagnosis of cGVHD
Currently receiving systemic treatment for cGVHD (including phototherapies), or has had systemic therapy for cGVHD tapered to discontinuation within the past 12 months
No evidence of malignant disease relapse including molecular relapse and minimal residual disease. Patients with mixed chimerism are eligible to participate
Parent or guardian ability and willingness to sign a written informed consent document
Subjects must be able to comprehend and speak the English language
Subjects may participate in both Project 1 and Project 2 of the study. Participation in

Project 1 is not required in order to be eligible to participate in Project 2.

Parent or Guardian Proxy Inclusion Criteria
Parent or guardian of participating subject
Must be willing and able to provide informed consent. Parent or guardian (proxy) must be able to comprehend and speak the English language

EXCLUSOIN CRITERIA:

Patients who completed systemic treatment more than 3 months prior to enrollment or are receiving topical therapy only.
Patients may be excluded from this study if in the judgment of the Principal or Associate Investigator, the subject is too ill, or subject s cognitive ability would compromise their ability to participate in study related procedures.

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04044365

Contacts

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Contact: Lori Wiener, Ph.D.	(240) 760-6419	lori.wiener@nih.gov

Locations

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United States, Colorado
Children's Hospital Colorado (CHCO)	Recruiting
Aurora, Colorado, United States, 80129
Contact: Amy Keating, M.D. 720-777-0166 Amy.Keating@UCDenver.edu
United States, District of Columbia
Children s National Medical Center (CNMC)	Recruiting
Washington, District of Columbia, United States, 20010
Contact: Alexa Yarish 202-476-6250 ajyarish@childrensnational.org
United States, Georgia
Children's Healthcare of Atlanta - Egleston Hospital	Recruiting
Atlanta, Georgia, United States, 30322
Contact: Katherine K. Garrett, MPH, ELS 404-785-3644 Katherine.Garrett@choa.org
United States, Illinois
Lurie Children's Hospital of Chicago	Recruiting
Chicago, Illinois, United States, 60611
Contact: Anne Lakes 312-227-1876 alakes@luriechildrens.org
United States, Maryland
Johns Hopkins Hospital (JHH)	Recruiting
Baltimore, Maryland, United States, 21287
Contact: Kenneth R. Cook, M.D. 410-955-8751 kcooke5@jhmi.edu
National Institutes of Health Clinical Center	Recruiting
Bethesda, Maryland, United States, 20892
Contact: For more information at the NIH Clinical Center contact National Cancer Institute Referral Office 888-624-1937
United States, Minnesota
University of Minnesota	Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Margaret MacMillan, M.D. 612-626-2961 macmi002@umn.edu
United States, Ohio
Cincinnati Children's Hospital Medical Center (CCHMC)	Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Pooja Khandelwal, M.D. 513-803-9063 pooja.khandelwal@cchmc.org
United States, Tennessee
St. Jude Children s Research Hospital	Recruiting
Memphis, Tennessee, United States, 38105
Contact: Marian Shaw 901-595-8149 Marian.Shaw@stjude.org
United States, Texas
Texas Children's Hospital - Baylor College of Medicine	Recruiting
Houston, Texas, United States, 77030
Contact: Josalind Randall 832-824-6835 jxrandal@texaschildrens.org
The University of Texas MD Anderson Cancer Institute	Recruiting
Houston, Texas, United States, 77030
Contact: Alba Rubi Banegas 713-792-9857 aarubi@mdanderson.org
United States, Washington
Fred Hutchinson Cancer Center (FHCC)	Recruiting
Seattle, Washington, United States, 98109
Contact: Sheri Ballard 206-667-5160 sballard@fredhutch.org
Canada
Children's & Women's Health Centre of British Columbia (CWHC of BC)	Recruiting
Vancouver BC, Canada
Contact: Colleen Fitzgerald (604) 875-2345 cfitzgerald@cw.bc.ca

Sponsors and Collaborators

National Cancer Institute (NCI)

Investigators

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Principal Investigator:	Lori Wiener, Ph.D.	National Cancer Institute (NCI)

More Information

Additional Information:

NIH Clinical Center Detailed Web Page This link exits the ClinicalTrials.gov site

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Responsible Party:	National Cancer Institute (NCI)
ClinicalTrials.gov Identifier:	NCT04044365
Other Study ID Numbers:	190131 19-C-0131
First Posted:	August 5, 2019 Key Record Dates
Last Update Posted:	June 24, 2022
Last Verified:	June 13, 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD:	Yes
Plan Description:	.All IPD recorded in the medical record will be shared with intramural investigators upon request.
Supporting Materials:	Study Protocol Statistical Analysis Plan (SAP) Informed Consent Form (ICF)
Time Frame:	Clinical data available during the study and indefinitely.
Access Criteria:	Clinical data will be made available via subscription to BTRIS and with the permission of the study PI.

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Studies a U.S. FDA-regulated Drug Product:	No
Studies a U.S. FDA-regulated Device Product:	No

Keywords provided by National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) ):


Hematopoietic Stem Cell Transplantation (HSCT) Allogeneic Stem Cell Transplant Phototherapies Graft Vs Host Disease Natural History

Additional relevant MeSH terms:

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Graft vs Host Disease Immune System Diseases

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