Hepatic Sinusoidal Obstruction Syndrome in a child after chemotherapy for medulloblastoma

Abstract

Hepatic Sinusoidal Obstruction Syndrome (HSOS), the new name given to veno-occlusive disease (VOD) of the liver, is a well-known complication of high-dose chemotherapy employed with hematopoietic stem cell transplantation, but it has rarely been observed in children who receive conventional chemotherapy. HSOS following standard chemotherapy has been reported in patients receiving vincristine, actinomycin D, and cyclophosphamide for the treatment of Wilms tumor and more rarely rhabdomyosarcoma. We report a 14-year-old boy with high risk medulloblastoma treated with craniospinal radiation followed by chemotherapy, who experienced severe HSOS after only one course of chemotherapy including carboplatin, vincristine, and cyclophosphamide. To our knowledge, this is the second report of HSOS after standard dose chemotherapy for brain tumor in childhood.

Hepatic Sinusoidal Obstruction Syndrome During Chemotherapy for Childhood Medulloblastoma

“…He was treated with 1 week of oral ursodeoxycholic acid and 3 days of parenteral N -acetylcysteine, with resolution of the HSOS. To prevent recurrence, the patient subsequently received a reduction in the frequency of cyclophosphamide, with administration every second cycle 31. Our case further highlights the occurrence of HSOS outside the traditional settings discussed above.…”

Treatment of Sinusoidal Obstruction Syndrome With Defibrotide in Pediatric Cancer Patients Following Nontransplant-associated Chemotherapy: A Case Report and Review of the Literature

“…Difficulty accessing defibrotide is noted in case reports discussing the viability of other pharmaceutical agents, methylprednisone, and N-acetylcysteine, as potential therapies for VOD/SOS. 17,18 Here we present a case of nontransplant-associated SOS/VOD in a 3-year-old with recurrent Wilms tumor, as well as a literature review of 62 cases of patients with nontransplant-associated SOS/VOD successfully treated with defibrotide. Research, in the form of clinical trials, is needed to systematically evaluate the use of defibrotide in pediatric patients with nontransplant-associated SOS/VOD.…”

“…Finally, it is important to be cognizant of the financial barriers and limited market availability associated with defibrotide. Difficulty accessing defibrotide is noted in case reports discussing the viability of other pharmaceutical agents, methylprednisone, and N-acetylcysteine, as potential therapies for VOD/SOS 17,18…”