ArticleClinical transplantation2024
Prognostic Factors Associated With Increased Mortality in Pediatric Veno-Occlusive Disease Following Hematopoietic Cell Transplantation.
Article in Clinical transplantation, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
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2 citing papers in PubMed.
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7 authors.
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Abstract
backgroundHepatic veno-occlusive disease (VOD) is a life-threatening complication of hematopoietic cell transplantation (HCT) and is categorized as a transplant-related, systemic endothelial disease. Severe VOD can lead to multi-organ dysfunction (MOF) and is associated with a high mortality rate.
objectiveTo evaluate the incidence of VOD in children after HCT and analyze the outcomes and risk factors associated with increased mortality. STUDY
designA retrospective cohort study of 1243 children with malignant and non-malignant diseases who underwent HCT at two large pediatric centers over 20 years.
resultsOne hundred one patients (8%) developed VOD post HCT. Most patients developed VOD post allogeneic HCT (76%) versus autologous (24%). The incidence of VOD was twice as high in children with malignant diseases compared to non-malignant (68% vs. 32%). A much higher incidence of VOD occurred in patients after a busulfan-based regimen versus total body irradiation-based and treosulfan-based, 73%, 18%, and 1%, respectively. The 100-day survival rate of HCT patients with VOD was 69%. The overall survival rate of the entire group was 50%, showing improvement over the span of the study years, from 40% between 2000 and 2009 to 63% between 2010 and 2021 (p = 0.022). Factors associated with increased mortality included infections before transplant (p = 0.013), conditioning regimen (p = 0.01), abnormal liver function (p = 0.019), presence of ascites (p = 0.008), MOF (p < 0.001), and the need for admission to a pediatric intensive care unit (p < 0.001). There was no significant difference in survival rates between children treated with defibrotide alone or with those treated with defibrotide and steroids (61% and 65%, respectively; p = 0.685).
conclusionsSevere VOD in pediatric patients following HCT remains a life-threatening complication with a high mortality rate. Early diagnosis and treatment with defibrotide are critical for managing this condition. In our cohort, the addition of steroids to defibrotide was not associated with improved outcomes.
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