title: The Oklahoma Thrombotic Thrombocytopenic Purpura-Hemolytic Uremic Syndrome (TTP-HUS) Registry: a community perspective of patients with clinically diagnosed TTP-HUS.
abstract: The Oklahoma Thrombotic Thrombocytopenic Purpura-Hemolytic Uremic Syndrome (TTP-HUS) Registry enrolls all consecutive patients for whom plasma exchange treatment is requested for clinically diagnosed TTP-HUS within a defined geographic region. During 14.5 years, from January 1, 1989 until June 30, 2003, 301 patients have been enrolled; follow-up is complete on 300 patients. Clinical categories have been designated based on associated conditions and potential etiologies; presenting features and clinical outcomes have been defined to allow comparisons between groups. ADAMTS-13 activity was measured on 142 (88%) of 161 consecutive patients enrolled from 1995 to 2001. Only 13% of all patients, and 33% of patients with idiopathic TTP-HUS, had severe ADAMTS-13 deficiency (<5% activity). The presenting features and clinical outcomes of patients with severe ADAMTS-13 deficiency were heterogeneous and not distinct from patients without severe ADAMTS-13 deficiency. These data suggest that severe ADAMTS-13 deficiency does not detect all patients who may be appropriately diagnosed with TTP-HUS and who may respond to plasma exchange treatment. Prospective data from consecutive patients are essential to translate new observations on pathogenesis into improved patient care.
authors.affiliation: Department of Medicine, College of Medicine, The University of Oklahoma Health Sciences Center, Oklahoma City, 73190, USA.
authors.forename: James N
authors.initials: JN
authors.lastname: George
authors.forename: Sara K
authors.initials: SK
authors.lastname: Vesely
authors.forename: Deirdra R
authors.initials: DR
authors.lastname: Terrell
mesh_terms: D051722:ADAM Proteins; D000071120:ADAMTS13 Protein; D000293:Adolescent; D000328:Adult; D000368:Aged; D003967:Diarrhea / Q000150:complications; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation; D006463:Hemolytic-Uremic Syndrome / Q000150:complications* / Q000175:diagnosis* / Q000453:epidemiology / Q000628:therapy; D006801:Humans; D008297:Male; D008666:Metalloendopeptidases / Q000032:analysis / Q000172:deficiency / Q000378:metabolism; D008875:Middle Aged; D009825:Oklahoma / Q000453:epidemiology; D010951:Plasma Exchange; D011247:Pregnancy; D011697:Purpura, Thrombotic Thrombocytopenic / Q000150:complications* / Q000175:diagnosis* / Q000453:epidemiology / Q000628:therapy; D012042:Registries; D016896:Treatment Outcome; D014841:von Willebrand Factor / Q000378:metabolism
chemical_list: D014841:von Willebrand Factor; D051722:ADAM Proteins; D008666:Metalloendopeptidases; D000071120:ADAMTS13 Protein; C099604:ADAMTS13 protein, human