title: The Role of Thromboelastography in Pediatric Patients with Sinusoidal Obstructive Syndrome Receiving Defibrotide.
abstract: Sinusoidal obstructive syndrome (SOS) is a potentially fatal form of hepatic injury after hematopoietic stem cell transplantation. Patients can develop liver dysfunction, portal hypertension, ascites, coagulopathies, and multisystem organ failure. The mortality rate of severe SOS has been reported as high as 98% by day 100 after transplantation. Defibrotide, which is now approved for the treatment of SOS, has significantly decreased mortality. Defibrotide is a polynucleotide with profibrinolytic, anti-ischemic, and anti-inflammatory activity. These properties can increase the risk of life-threatening bleeding in this patient population. Previous protocols have suggested maintaining international normalized ratio ≤ 1.5, platelets > 30 k/uL, and fibrinogen ≥ 150 mg/dL to minimize this risk of bleeding. However, this can be challenging in fluid-sensitive patients with SOS. Thromboelastography (TEG) is a functional assay that evaluates the balance of procoagulant and anticoagulant proteins. In this series, TEG was used to guide defibrotide therapy as well as blood product transfusions in SOS patients with abnormal coagulation studies. Each patient recovered from SOS and had no bleeding complications. A randomized clinical trial is the next step in supporting the use of TEG in SOS patients with abnormal coagulation studies receiving defibrotide therapy.
authors.affiliation: Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, Arizona. Electronic address: jwigfield@phoenixchildrens.com.
authors.forename: Joanna L
authors.initials: JL
authors.lastname: Gendreau
authors.affiliation: Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, Arizona.
authors.forename: Christine
authors.initials: C
authors.lastname: Knoll
authors.affiliation: Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, Arizona; Hematology/Oncology, Mayo Clinic Arizona, Scottsdale, Arizona.
authors.forename: Roberta H
authors.initials: RH
authors.lastname: Adams
authors.affiliation: Department of Pathology and Laboratory Medicine, Phoenix Children's Hospital, Phoenix, Arizona.
authors.forename: Leon L
authors.initials: LL
authors.lastname: Su
mesh_terms: D001778:Blood Coagulation Disorders / Q000175:diagnosis; D002648:Child; D002675:Child, Preschool; D005260:Female; D005343:Fibrinolytic Agents / Q000627:therapeutic use; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006504:Hepatic Veno-Occlusive Disease / Q000175:diagnosis / Q000188:drug therapy* / Q000209:etiology; D006801:Humans; D007223:Infant; D008297:Male; D010975:Platelet Aggregation Inhibitors / Q000627:therapeutic use; D011089:Polydeoxyribonucleotides / Q000494:pharmacology / Q000627:therapeutic use*; D013916:Thrombelastography / Q000379:methods*
chemical_list: D005343:Fibrinolytic Agents; D010975:Platelet Aggregation Inhibitors; D011089:Polydeoxyribonucleotides; C036901:defibrotide
keywords: Defibrotide; Pediatrics; Sinusoidal obstructive syndrome; Thromboelastography