Pediatric cGVHD Symptom Scale

• ClinicalTrials.gov Identifier: NCT04044365
• Recruitment Status: Recruiting
• First Posted: August 5, 2019
• Last Update Posted: January 30, 2023
• Sponsor: National Cancer Institute (NCI)
• 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.

...

Condition or disease
Graft vs Host Disease

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 75 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

• Study Type: Observational
• Estimated Enrollment: 390 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

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=35 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 :

1. 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

• 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
• Subject must have an eligible caregiver proxy who is willing to participate in the study.
• 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.

Caregiver Proxy Inclusion Criteria

• Adult, >18 years of age, caregiver of participating subject
• Must be willing and able to provide informed consent.
• Must be able to comprehend and speak the English language

EXCLUSOIN CRITERIA:

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

Contacts

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

Locations

United States, Colorado

Children's Hospital Colorado (CHCO); Recruiting

Aurora, Colorado, United States, 80045

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, 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 (TCH)-Baylor; Recruiting

Houston, Texas, United States, 77030

Contact: Erin Doherty, M.D. 832-826-0860 eedohert@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

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

More Information

Additional Information:

NIH Clinical Center Detailed Web Page 

• Responsible Party: National Cancer Institute (NCI)
• ClinicalTrials.gov Identifier: NCT04044365
• Other Study ID Numbers: 190131; 19-C-0131
• First Posted: August 5, 2019
• Last Update Posted: January 30, 2023
• Last Verified: January 26, 2023

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.

• 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:

Graft vs Host Disease; Immune System Diseases