Coexisting or Underlying Risk Factors of Hepatic Veno-Occlusive Disease in Pediatric Hematopoietic Stem Cell Transplant Recipients Receiving Prophylaxis

To evaluate the characteristics of veno-occlusive disease in pediatric hematopoietic stem cell transplant recipients, and their effect as a prophylactic regimen on severity and outcome.This study included 204 allogeneic hematopoietic stem cell transplants performed on 187 children whose data retrospectively described the risk factors, prophylaxis, and treatment modalities of veno-occlusive disease. A prophylactic regimen composed of enoxaparin versus ursodeoxycholic acid and vitamin E was given to 167 of 204 patients.Veno-occlusive disease developed in 22 patients (10.8%). Nineteen patients experienced veno-occlusive disease despite this prophylactic regimen. The prophylaxis seemed ineffective in preventing veno-occlusive disease (P = .657). Regarding risk factors, oral busulphan use, liposomal amphotericin B vancomycin treatment, and total parenteral nutrition were associated with an increased risk of veno-occlusive disease. Conversely, renal impairment also was associated with increased mortality in patients with veno-occlusive disease. The mortality rate in the first 100 days after a hematopoietic stem cell transplant was higher in the patients with veno-occlusive disease than it was in those without the disease.Our prophylactic regimen may have played a role in the fairly low incidence of veno-occlusive disease in a pediatric population with high-risk features.