A Retrospective Analysis of Antithrombin III Replacement Therapy for the Treatment of Hepatic Sinusoidal Obstruction Syndrome in Children Following Hematopoietic Stem Cell Transplantation

J Pediatr Hematol Oncol. 2020 Mar;42(2):145-148. doi: 10.1097/MPH.0000000000001422.

Abstract

Hepatic sinusoidal obstruction syndrome (SOS) remains a serious complication of hematopoietic stem cell transplantation (HSCT). In this single institution retrospective case series, 18 children developed SOS after HSCT. Patients were treated with antithrombin III (ATIII), defibrotide, or ATIII followed by defibrotide. Twelve of 13 patients who were treated with ATIII therapy alone had complete resolution of SOS, including 4 of 5 children with severe SOS. In this limited cohort, ATIII was safe and successfully prevented progression of hepatic SOS following HSCT in the majority of children at our center.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antithrombin III / therapeutic use*
  • Child
  • Follow-Up Studies
  • Hematopoietic Stem Cell Transplantation / adverse effects*
  • Hepatic Veno-Occlusive Disease / drug therapy*
  • Hepatic Veno-Occlusive Disease / etiology
  • Hepatic Veno-Occlusive Disease / pathology
  • Humans
  • Prognosis
  • Retrospective Studies

Substances

  • Antithrombin III