Clinical trial NCT04044365: Pediatric cGVHD Symptom Scale
Official title: Development and Psychometric Testing of a Pediatric Chronic Graft- Versus-Host Disease (GVHD) Symptom Scale (PCSS)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Graft vs Host Disease
Keywords: Hematopoietic Stem Cell Transplantation (HSCT)
Allogeneic Stem Cell Transplant
Phototherapies
Graft Vs Host Disease
Natural History
Lead sponsor: National Institutes of Health Clinical Center (CC)
Sponsor class: NIH
First posted: 2019-08-05
Enrollment: 420
Sex: ALL
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
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

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.

...

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 5 to 17 years of age for Project 1 or 8-17 years of age for Project 2, 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
* Caregivers of children 5-17 years of age with cGVHD enrolled as a proxies

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 (COMPLETE), 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 100 pediatric transplant survivors with cGVHD reflecting two age groups (8-12 years; 13-17 years) of equal numbers and 20 caregivers of children age 5-7 who will serve as proxies. In participants 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.
* The overall accrual ceiling is 420, which includes pediatric and caregiver participants and accounts for inevaluable participants
* This study will include CCR investigators as well as extramural investigators at national and international study sites.

Eligibility criteria: * INCLUSION CRITERIA:

Pediatric Participant Inclusion Criteria:

* Children aged 5 to 17 years old (Project 1) or children aged 8 to 17 years old (Project 2)
* Clinical diagnosis of cGVHD following allogeneic stem cell transplant
* Currently receiving systemic treatment for GVHD (including phototherapies), or has had systemic therapy for GVHD 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
* 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
* Must be able to comprehend and speak the English language
* Participants may enroll 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 old) caregiver of a child participating in Project 1 or Project 2; OR Adult (\>=18 years old) caregiver of a child aged 5 to 7 years old who meets all Pediatric Participant Inclusion Criteria except Project 2 criteria noted above in the section Pediatric Participant Inclusion Criteria
* Must be willing and able to provide informed consent
* Must be able to comprehend and speak the English language

EXCLUSOIN CRITERIA:

Children may be excluded from this study if in the judgment of the Principal or Associate Investigator, the child is too ill, or the child s cognitive ability would compromise their ability to participate in study related procedures.

Primary outcomes:
measure: Feasability
timeFrame: 3 years
Secondary outcomes:
Other outcomes:
Locations:
city: Aurora
country: United States
facility: Children's Hospital Colorado (CHCO)
state: Colorado
city: Washington
country: United States
facility: Children s National Medical Center (CNMC)
state: District of Columbia
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta - Egleston Hospital
state: Georgia
city: Chicago
country: United States
facility: Lurie Children's Hospital of Chicago
state: Illinois
city: Baltimore
country: United States
facility: Johns Hopkins Hospital (JHH)
state: Maryland
city: Bethesda
country: United States
facility: National Institutes of Health Clinical Center
state: Maryland
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Memphis
country: United States
facility: St. Jude Children s Research Hospital
state: Tennessee
city: Houston
country: United States
facility: Texas Children's Hospital (TCH)-Baylor
state: Texas
city: Houston
country: United States
facility: The University of Texas MD Anderson Cancer Institute
state: Texas
city: Seattle
country: United States
facility: Fred Hutchinson Cancer Center (FHCC)
state: Washington
city: Vancouver BC
country: Canada
facility: Children's &amp; Women's Health Centre of British Columbia (CWHC of BC)
Study officials:
affiliation: National Cancer Institute (NCI)
name: Lori S Wiener, Ph.D.
role: PRINCIPAL_INVESTIGATOR
References: