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

Kim, Miriam DO*; Rao, Seema MD*,†; Eickhoff, Jens C. PhD; DeSantes, Kenneth B. MD*; Capitini, Christian M. MD*

Journal of Pediatric Hematology/Oncology: January 22, 2019 - Volume Publish Ahead of Print - Issue - p
doi: 10.1097/MPH.0000000000001422
Clinical and Laboratory Observations: PDF Only

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.

Departments of *Pediatrics

Biostatistics and Medical Informatics, Carbone Cancer Center, University of Wisconsin School of Medicine and Public Health, Madison, WI

Department of Pediatrics, Texas Children’s Cancer and Hematology Center, Baylor College of Medicine, Houston, TX

M.K. is supported by a St. Baldrick’s Foundation Fellowship award. S.R. was supported by an National Institutes of Health (NIH) grant T32 CA009614. C.M.C. is supported by the NIH/National Cancer Institute grant K08 CA174750. None of these funding sources had any input in the study design, analysis, manuscript preparation, or decision to submit for publication.

C.M.C. received honorarium from Novartis for a one time advisory board, and currently receives honorarium from Nektar Therapeutics. The remaining authors declare no conflict of interest.

Reprints: Christian M. Capitini, MD, 1111 Highland Ave, Madison, WI 53705 (e-mail:

Received June 8, 2018

Accepted December 26, 2018

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