Pediatric cGVHD Symptom Scale

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.

• 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.