Pediatric cGVHD Symptom Scale

Graft vs Host Disease Clinical Trial

Official title:

Development and Psychometric Testing of a Pediatric Chronic Graft- Versus-Host Disease (GVHD) Symptom Scale (PCSS)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04044365
• Other study ID #: 190131
• Secondary ID: 19-C-0131
• Status: Recruiting
• Start date: October 2, 2019
• Est. completion date: December 31, 2025

Study information

• Verified date: June 17, 2024
• Source: National Institutes of Health Clinical Center (CC)
• Contact: Lori Wiener, Ph.D.
• Phone: (240) 760-6419
• Email: lori.wiener[@]nih.gov
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

...

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 up to 90 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.

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 up to 90 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 420
• Est. completion date: December 31, 2025
• Est. primary completion date: December 31, 2025
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 5 Years and older

Eligibility

• 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.

Related Conditions & MeSH terms

• Graft vs Host Disease

Locations

Country	Name	City	State
Canada	Children's & Women's Health Centre of British Columbia (CWHC of BC)	Vancouver BC
United States	Children's Healthcare of Atlanta - Egleston Hospital	Atlanta	Georgia
United States	Children's Hospital Colorado (CHCO)	Aurora	Colorado
United States	Johns Hopkins Hospital (JHH)	Baltimore	Maryland
United States	National Institutes of Health Clinical Center	Bethesda	Maryland
United States	Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Texas Children's Hospital (TCH)-Baylor	Houston	Texas
United States	The University of Texas MD Anderson Cancer Institute	Houston	Texas
United States	St. Jude Children s Research Hospital	Memphis	Tennessee
United States	University of Minnesota	Minneapolis	Minnesota
United States	Fred Hutchinson Cancer Center (FHCC)	Seattle	Washington
United States	Children s National Medical Center (CNMC)	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
National Cancer Institute (NCI)

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Feasability	To develop a psychometrically valid Pediatric cGVHD Symptom Scale (PCSS) and a companion parent-proxy measure as counterparts to the Lee cGVHD Symptom Scale.	3 years

External Links

•   NIH Clinical Center Detailed Web Page