Transplantation and Cellular Therapy | VOL. 28
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Clinical Features, Treatment, and Outcome of Pediatric Steroid Refractory Acute Graft-Versus-Host Disease: A Multicenter Study

Publication Date Sep 1, 2022

Abstract

Steroid refractory Acute Graft-versus-Host Disease (SR-aGvHD) is a severe complication in pediatric allogeneic hematopoietic stem cell transplantation (HSCT). We aimed to assess clinical course and outcomes of pediatric SR-aGvHD. We performed a retrospective nationwide multicenter cohort study in the Netherlands. All patients aged 0-18 transplanted between 2010 and 2020 with SR-aGvHD were included. For each patient, weekly clinical aGvHD grade and stage, immunosuppressive treatment and clinical outcomes were collected. The primary study endpoint was clinical course of SR-aGvHD over time. As a secondary outcome, factors influencing overall survival and SR-aGvHD remission were identified using a multistate Cox model. 20% of transplanted children developed grade II-IV aGvHD, of which 51% (n=81) was SR-aGvHD. In these patients, second-line therapy was started at a median of 8 days after initial aGvHD-diagnosis. 49% of SR-aGvHD patients received three or more lines of therapy. One year after start of second-line therapy, 34 patients (42%) were alive and in remission of aGvHD, 14 patients (17%) had persistent GvHD and 33 patients (41%) had died. SR-aGvHD remission rate was lower in cord blood graft recipients than in bone marrow (BM) or peripheral blood stem cell (PBSC) recipients (HR 0.51, 0.27-0.94, p=0.031). Older age was associated with higher mortality (HR 2.62, 1.04-6.60, p=0.04, fourth quartile (aged 13.9-17.9) vs. first quartile (aged 0.175-3.01)). In BM/PBSC recipients older age was also associated with lower remission ra...

Concepts

Steroid Refractory Acute Graft-versus-host Disease Pediatric Hematopoietic Stem Cell Transplantation Choice Of Second Line Therapy Pediatric Allogeneic Hematopoietic Stem Cell Start Of Second-line Therapy Multicenter Study Clinical Outcomes Allogeneic Hematopoietic Stem Cell Transplantation Peripheral Blood Stem Cell Persistent GvHD

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