Pediatric cGVHD Symptom Scale
- - 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.
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 parent-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 parent proxies. - In Project 1, we will conduct at least 20 cognitive interviews of patient and proxy dyads (parent/guardian) 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 study sites.
Trial Phase Phase NA
Trial Type Not provided by clinicaltrials.gov
National Cancer Institute
- Primary ID 190131
- Secondary IDs NCI-2019-05449, 19-C-0131
- Clinicaltrials.gov ID NCT04044365