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

Abstract

Objectives: 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. Materials and Methods: 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. Results: 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. Conclusions: Our prophylactic regimen may have played a role in the fairly low incidence of venoocclusive disease in a pediatric population with highrisk features.

Acute Kidney Injury in Hematopoietic Stem Cell Transplantation: A Review

“…It was reported that both regimens damaged hepatic sinusoidal endothelial cells; however, the incidence of hepatic SOS was higher with busulfan-cyclophosphamide regimen compared to TBI [50]. Other risk factors include total parenteral nutrition for >1 week, young age (due to small sinusoidal lumens predisposing to easy occlusion), thalassemia major, osteopetrosis, and hemophagocytic lymphohistiocytosis in children; all have been shown to increase the risk of hepatic endothelial injury and SOS [51]. …”

The outcome and complications of total parenteral nutrition in pediatric hematopoietic stem cell transplantation

“…The incidence of sinusoidal obstruction syndrome (SOS) was statistically significant in the TPN group, and the majority of those patients received myeloablative conditioning therapy for non‐malignant diseases (hemoglobinopathy). The association between the use of TPN and the development of the hepatic SOS has been reported, especially in those who received myeloablative conditioning 16,17 . The additional analysis that was done in this study to assess whether the use of defibrotide as a prophylaxis can decrease the incidence of SOS showed that there was no statistical difference between the group that used prophylaxis and the one that did not.…”

Single Daily Busulfan Dosing for Infants with Nonmalignant Diseases Undergoing Reduced-Intensity Conditioning for Allogeneic Hematopoietic Progenitor Cell Transplantation

“…In adults, total Bu exposure above 24,000 mMol*minute was linked to a higher risk of SOS development [5,34,35]. In pediatric patients who received Bu in the setting of myeloablative conditioning, the incidence of SOS ranges between 10% and 30% [33,[36][37][38], even though the relationship between SOS and a high level of Bu exposure was less clearly established [33,38,39]. In our patients, Bu was only given for 2 days and total Bu exposure (a total AUC target of 10,000 mMol*minute) was far below this proposed threshold associated with the development of SOS.…”