Abstract
Treatment-related mortality and morbidity remain a challenge in hematopoietic stem cell transplantation (HSCT). In this retrospective, single-center study, we analyzed endothelial damage as a potential, common denominator and mechanism for the adverse effects. We evaluated the prevalence of key vascular complications and graft-versus-host disease among 122 pediatric patients with an allogeneic HSCT between 2001 and 2013. The spectrum and frequency of acute adverse events emerging ≤100 days post transplant were graded according to the CTCAE 4.03 and analyzed. We identified a total of 19/122 (15.6%) patients with vascular complications, fulfilling the criteria of capillary leak syndrome, veno-occlusive disease/sinusoidal obstruction syndrome or thrombotic microangiopathy. The patients had a poorer overall survival (77% versus 26%, p < 0.001). Nearly one half (56/122, 45.9%) had at least one, severe (grade 3 or 4) adverse event. Patients with vascular complications had more often edema/effusions (p = 0.023), thrombocytopenia (p = 0.001), gastrointestinal bleeding (p < 0.001), acute kidney injury (p < 0.001), ascites (p < 0.001) or bilirubin increase (p = 0.027). These endotheliopathy-related adverse events appeared early post HSCT, varied in their clinical phenotype and predicted a poor outcome. An unrelated donor but not previous exposure to leukemia or irradiation-based conditioning was identified as a risk factor for vascular complications and endotheliopathy.
Highlights
Allogeneic hematopoietic stem cell transplantation is a well-established therapy for hematologic and lymphoid malignancies, and for inborn or acquired disorders of the immune or hematopoietic systems and metabolism
Our aim was to identify adverse events related to endothelial dysfunction and our ability to predict vascular complications among pediatric recipients of allo-hematopoietic stem cell transplantation (HSCT) prior to day 100 post transplant
Singlecenter study shows that 75% of the patients had at least one, grade 2–4 toxic event prior to day 100 post transplant
Summary
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a well-established therapy for hematologic and lymphoid malignancies, and for inborn or acquired disorders of the immune or hematopoietic systems and metabolism. The overall survival (OS) has improved due to a decrease in treatment-related mortality (TRM) with improved HSCT practices, a decrease in the incidence of graft-versus-host disease (GVHD) and improved supportive care [1,2,3]. GVHD affects the epithelium, especially in the skin, gut mucosa and biliary tract, but when refractory, appears to involve endothelial dysfunction [4, 5]. Capillary leak syndrome (CLS), treatment-related thrombotic microangiopathy (TMA) and veno-occlusive disease (VOD) appear early on post transplant and remain associated with high mortality. The vascular complications are highly variable in their clinical course rendering early detection and timely intervention a challenge [6,7,8]
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