Chronic graft-versus-host disease in pediatric patients: Differences and challenges

Haroun, Elio; Agrawal, Kavita; Leibovitch, Jennifer; Kassab, Joseph; Zoghbi, Marianne; Dutta, Dibyendu; Lim, Seah H.

doi:10.1016/j.blre.2023.101054

Cited by 8 publications

(7 citation statements)

References 143 publications

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“…However, the availability of the new agents for treatment and improved supportive care are associated with increased OS 8–10 . The incidence of cGVHD is between 6% to 33% of the pediatric patients with lowest incidence following umbilical cord blood transplant and highest after peripheral blood HSCT 31 . Cumulative incidence of chronic GVHD was both lower in the overall cohort and in the second period due to less use of PBSC as a stem cell source.…”

Section: Discussionmentioning

confidence: 99%

Improvement of survival after hematopoietic stem cell transplantation and trends at a pediatric transplantation center; a three‐decade journey

Sezgin,

Öner,

İnan

et al. 2024

Pediatric Transplantation

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BackgroundAdvances in stem cell transplantation have resulted in improved outcomes.MethodsThis is a retrospective study aimed to analyze changes in patient profile, transplantation, graft characteristics, and outcome among 241 pediatric patients who received stem cell transplantation in a single center between 1993 and 2019.ResultsIn the 2010–2019, compared with the 1993–2009 period, a significantly higher 5‐year overall survival (60% vs. 44%, p = .022) and an event‐free survival (53% vs. 34%, p = .025) were observed. Cumulative incidence of deaths due to relapse or progression between the 1993–2009 and 2010–2019 periods were 33% and 26% respectively (p = .66). Cumulative incidence of non‐relapse mortality was significantly higher during the 1993–2009 period compared with the 2010–2019 period for malignant diseases (57.7% vs. 28.3%, p = .007). The overall survival from acute graft‐versus‐host disease between 1993 and 2009 was 11% versus 46% between 2010 and 2019 (p = .0001). The overall survival from infection in both eras did not show any difference (p = .41).ConclusionsDevelopment in transplantation technology has led to a decrease in non‐relapse mortality and better control of graft‐versus‐host disease. However, relapse and infection remained as major causes of death. Studies evaluating institutional trends in patients undergoing HSCT and analyzing their mortality profile, can improve the management of patients, leading to a reduction in transplant‐related problems.

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Section: Discussionmentioning

confidence: 99%

Improvement of survival after hematopoietic stem cell transplantation and trends at a pediatric transplantation center; a three‐decade journey

Sezgin,

Öner,

İnan

et al. 2024

Pediatric Transplantation

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“…This key secondary end point also warrants further investigation in a larger trial of adults, where the incidence of chronic GVHD is typically higher than that observed in pediatric populations. 8 Khandelwal and colleagues should be congratulated for conducting this randomized, placebo-controlled trial in pediatric transplant recipients. Their findings are thought-provoking, hold much promise, and warrant future research to better characterize the potential impact of this simple, yet novel, approach to preventing GVHD.…”

Section: Zachariah Defilipp | Massachusetts General Hospitalmentioning

confidence: 99%

Vitamin A keeps the GVHD away?

DeFilipp

2024

Blood

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“…Chronic GVHD can involve almost every organ system although it most commonly affects the skin, eyes, mouth, liver, intestines, lungs, and the musculoskeletal system ( Haroun et al, 2023 ). 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 corticosteroids, needed to treat cGVHD manifestations ( Mitchell et al, 2021 ).…”

Section: Introductionmentioning

confidence: 99%

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

Mitchell,

Hunter,

Fry

et al. 2024

Front. Psychol.

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BackgroundChronic 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.Methods and analysisEligible 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.DiscussionThis 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.Clinical trial registrationwww.ClinicalTrials.gov, NCT04044365.

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Chronic graft-versus-host disease in pediatric patients: Differences and challenges

Cited by 8 publications

References 143 publications

Improvement of survival after hematopoietic stem cell transplantation and trends at a pediatric transplantation center; a three‐decade journey

Improvement of survival after hematopoietic stem cell transplantation and trends at a pediatric transplantation center; a three‐decade journey

Vitamin A keeps the GVHD away?

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

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