Survivors Of Pediatric Transplantation Research Articles

Development and psychometric testing of a pediatric chronic graft-versus-host disease symptom scale: protocol for a two-phase, mixed methods study.

Chronic graft-versus-host disease (cGVHD) is a debilitating late complication of hematopoietic stem cell transplantation. It is often accompanied by extensive symptom burden. No validated cGVHD patient-reported outcome (PRO) measure exists to evaluate cGVHD symptom bother in children and adolescents younger than 18 years. This paper presents the study protocol for a multi-center, two-phase protocol to develop a psychometrically valid pediatric cGVHD Symptom Scale (PCSS) and a companion caregiver-proxy measure to capture the symptom burden experienced by children with cGVHD. In the first phase of the study, our aim is to evaluate the comprehension, clarity and ease of response of the PCSS through cognitive interviewing and to iteratively refine the measure to optimize content validity. In the second phase of the study, we will quantitatively examine the measurement properties of the PCSS in children and their caregiver-proxies. Eligible participants are children/adolescents ages 5-17 with cGVHD who are receiving systemic immunosuppressive treatment or have recently tapered to discontinuation. In the first phase, we are enrolling 60 child and caregiver-proxy dyads in three child age strata (5-7, 8-12, and 13-17 years old). Semi-scripted cognitive debriefing interviews are conducted to assess comprehension, clarity, and ease of response of each PCSS item with the child alone, and then jointly with the caregiver-proxy to explore discordant ratings. In phase two, an age-stratified cohort of 120 child-caregiver dyads will be enrolled to evaluate test-retest reliability, construct validity, and responsiveness. Anchors for known-groups validity include the PedsQL module and clinical variables, including cGVHD clinician-rated severity scores. In participants ages 13-17, we will also compare responses on the PCSS with those from the Lee cGVHD Symptom Scale, to gauge the youngest age at which adolescent respondents can comprehend this adult measure. This study will yield a well-validated, counterpart measure to the Lee cGVHD Symptom Scale for use in children with cGVHD and their caregiver-proxies. This new patient-reported outcome measure can be integrated into clinical trials and care delivery for pediatric transplant survivors to improve the precision and accuracy with which their cGVHD symptom experience is captured. www.ClinicalTrials.gov, NCT04044365.

School and sports participation post-transplant.

Pediatric recipients of life-saving organ transplants are living longer, with improved graft and overall survivals. After successful transplant, children are encouraged to return to "normal life," with school attendance and participation in age-appropriate physical activities. This transition may cause stress to the recipients, parents, teachers, and other participating caregivers and staff. Planning for school reentry and assuring education for and open lines of communication with the school staff can help alleviate some of this discomfort and ease the process for the patient and the family. Cardiovascular disease has emerged as the leading cause of death in survivors of pediatric transplantation and is contributed to by modifiable risk factors such as obesity, hypertension, and the MS. Physical activity is a proven tool in decreasing surrogate markers of this risk. Sports participation is an important way to promote an enjoyment of physical activity that can ideally persist into adulthood, but conflicting advice and opinions exist regarding type and participation in physical activity. Moreover, specific recommendations are likely not applicable to all recipients, as certain degrees of rehabilitation may be needed depending on degree and length of illness. In general, a program of rehabilitation and increased physical activity has been shown to be safe and effective for most pediatric transplant recipients. Focusing on optimizing the "normal" childhood activities of going to school and participating in sports can improve the physical, social, cognitive, and mental health outcomes of this population after transplant and should be prioritized.

Quality of Life After Hematopoietic Stem Cell Transplantation in Pediatric Survivors: Comparison With Healthy Controls and Risk Factors.

Hematopoietic stem cell transplantation has prolonged life for children with life-threatening diseases. Quality of life is an essential outcome for evaluating the long-term effects of transplantation. The aims of this study were to compare the quality of life of children posttransplantation to that of healthy peers and explore the variables associated with the quality of life of posttransplant children. A cross-sectional study was conducted with 43 pediatric transplantation survivors and 43 age- and sex-matched healthy peers. The mean age of the transplant group was 12.06 years. The mean time since transplant was 3.73 years. After covariate adjustment, there was no difference between posttransplant and healthy children in each domain and overall quality of life, except for physical functioning where the posttransplant children had lower scores than did the healthy group. Chronic graft-versus-host disease was found to be the primary factor associated with poor posttransplant overall quality of life and emotional and social functioning. Sociodemographic variables, symptom distress, and caregiver depression were not correlated with posttransplant quality of life. The quality of life of pediatric transplantation survivors was comparable to that of healthy peers. The finding that children after transplant may achieve quality of life similar to their healthy peers is important information for parents to consider as they consider treatment options. For those sick children who cannot regularly attend school, their emotional and social functioning should be closely monitored.

Child and parent perspectives of the chronic graft-versus-host disease (cGVHD) symptom experience: a concept elicitation study

Chronic graft-versus-host disease (cGVHD) is a significant cause of mortality and morbidity after allogeneic hematopoietic cell transplant and is associated with a wide range of distressing symptoms. A pediatric measure of cGVHD-related symptoms is needed to advance clinical research. Our aim was to elicit descriptions of the cGVHD symptom experience directly from children and to compare the specific language used by children to describe their symptoms and the comprehension of symptom concepts across the developmental spectrum. We used qualitative methods to identify the phrases, terms, and constructs that children (ages 5–8 [n =8], 9–12 [n =8], and 13–17 [n =8]) with cGVHD employ when describing their symptoms. The symptom experience of each participant was determined through individual interviews with each participant and parent (5–7 year olds were interviewed together with a parent). Medical practitioners with experience in evaluating cGVHD performed clinical assessments of each participant. Pediatric transplant survivors and their parents identified a wide range of bothersome cGVHD symptoms, and common concepts and terminologies to describe these experiences emerged. Overall concordance between patient and parent reports was moderate (70–75 %). No consistent pattern of child under- or over-reporting in comparison to the parent report was observed. These study results identify concepts and vocabulary to inform item generation for a new pediatric self-report measure of cGVHD symptoms for use in clinical research. The findings also confirm the prevalence and nature of symptom distress in pediatric patients with cGVHD and support implementation of systematic approaches to symptom assessment and intervention in routine clinical practice.

NCI, NHLBI/PBMTC First International Conference on Late Effects After Pediatric Hematopoietic Cell Transplantation: Persistent Immune Deficiency in Pediatric Transplant Survivors

Defective immune reconstitution is a major barrier to successful hematopoietic cell transplantation (HCT), and has important implications in the pediatric population. There are many factors that affect immune recovery, including stem cell source and graft-versus-host disease (GVHD). Complete assessment of immune recovery, including T and B lymphocyte evaluation, innate immunity, and response to neoantigens, may provide insight as to infection risk and optimal time for immunizations. The increasing use of cord blood grafts requires additional study regarding early reconstitution and impact upon survival. Immunization schedules may require modification based upon stem cell source and immune reconstitution, and this is of particular importance as many children have been incompletely immunized, or not at all, before school entry. Additional studies are needed in children post-HCT to evaluate the impact of differing stem cell sources upon immune reconstitution, infectious risks, and immunization responses.

Follow-up into adulthood is critically important for survivors of pediatric transplant

Follow-up into adulthood is critically important for survivors of pediatric transplant

Transition: Changing Our Approach to Care for Long-Term Pediatric Transplant Survivors

Transition: Changing Our Approach to Care for Long-Term Pediatric Transplant Survivors

Transition: changing our approach to care for long-term pediatric transplant survivors

Transition: changing our approach to care for long-term pediatric transplant survivors

Research agenda for pediatric gastroenterology, hepatology and nutrition: transplantation. Report of the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition for the Children's Digestive Health and Nutrition Foundation.

Research agenda for pediatric gastroenterology, hepatology and nutrition: transplantation. Report of the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition for the Children's Digestive Health and Nutrition Foundation.