title: A novel strategy for hemolytic uremic syndrome: successful treatment with thrombomodulin α.
abstract: Hemolytic uremic syndrome (HUS) is a life-threatening infectious disease in childhood for which there is no confirmed therapeutic strategy. Endothelial inflammation leading to microthrombosis formation via complement activation is the main pathology of HUS. Thrombomodulin is an endothelial membrane protein that has anticoagulation and anti-inflammatory effects, including the suppression of complement activity. Recombinant human soluble thrombomodulin (rTM) is a novel therapeutic medicine for disseminated intravascular coagulation. We administered rTM to 3 patients with HUS for 7 days and investigated the outcomes in view of the patients' prognoses, changes in biochemical markers, complications, and adverse effects of rTM. Symptoms and laboratory data improved after initiation of rTM in all 3 patients. Abnormal activation of complements was also dramatically suppressed in 1 patient. The patients recovered without any complications or adverse effects of rTM. They were discharged having normal neurologic status and with no renal dysfunction. To our knowledge, this is the first report of rTM being used to treat HUS. These case reports show the positive effect of rTM in patients with HUS. Randomized controlled studies should be performed to assess the efficacy and safety of rTM for children with HUS.
authors: Department of Pediatrics, Kitasato University School of Medicine, Sagamihara, Japan.
authors: Takashi
authors: T
authors: Honda
authors: Shohei
authors: S
authors: Ogata
authors: Eri
authors: E
authors: Mineo
authors: Yukako
authors: Y
authors: Nagamori
authors: Shinya
authors: S
authors: Nakamura
authors: Yuki
authors: Y
authors: Bando
authors: Masahiro
authors: M
authors: Ishii
mesh_terms: D002648:Child; D002675:Child, Preschool; D005260:Female; D006463:Hemolytic-Uremic Syndrome / Q000097:blood / Q000175:diagnosis* / Q000188:drug therapy*; D006801:Humans; D007223:Infant; D008297:Male; D011994:Recombinant Proteins / Q000627:therapeutic use; D018180:Thrombomodulin / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D011994:Recombinant Proteins; C546931:THBD protein, human; D018180:Thrombomodulin