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Consensus on the standardization of terminology in thrombotic thrombocytopenic purpura and related thrombotic microangiopathies. Accepted ArticleThis article is protected by copyright. All rights reserved. Running title: Terminology in TTP and TMAsKey words: ADAMTS-13 protein, human; thrombocytopenia; diagnosis, differential; thrombotic microangiopathy; thrombotic thrombocytopenic purpura Essentials• An international collaboration provides a consensus for clinical definitions.• This concerns thrombotic microangiopathies and thrombotic thrombocytopenic purpura (TTP).• The consensus defines diagnosis, disease monitoring and response to treatment.• Requirements for ADAMTS-13 are given. Abstract:Background Thrombotic thrombocytopenic purpura (TTP) and hemolytic uremic syndrome (HUS) are two important acute conditions to diagnose. Thrombotic Microangiopathy is a broad pathophysiological process that leads to microangiopathic hemolytic anemia, thrombocytopenia and involves capillary and small vessel platelet aggregates. The most common cause being disseminated intravascular coagulation (DIC), which may be differentiated by abnormal Accepted ArticleThis article is protected by copyright. All rights reserved. IntroductionThe elucidation of the pathophysiology of TTP and HUS, in the past 20 years, has transformed our understanding of the phenotypes, genotypes and therapies for these life-threatening conditions. Work on standardization has been addressed [1], but this document aims to develop robust criteria for future clinical use, studies and trials.Clinical and pathophysiologic features of TTP, atypical Hemolytic Uremic Syndrome (aHUS) and disorders with similar presentations, their investigation and subsequent management vary. This consensus document aims to rationalize and standardize definitions. Conditions often included in the initial differential diagnosis of TTP are discussed. Definitions for remission, refractory and relapsing disease are defined.ADAMTS-13 (a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13) assays are central to diagnosis and are discussed. We therefore also describe the minimum requirements for validation of current and future assays. Methods:The development of this document involved key international, primarily clinical, experts in TTP and related TMAs. Accepted ArticleThis article is protected by copyright. All rights reserved.Hemostasis (ISTH) and American Society of Hematology (ASH) and all versions of the document have been reviewed and edited by the authors. Articles were identified by a computer-assisted search of the literature published in English using the National Library of Medicine PubMed database. The authors also undertook a focused review of the available literature. Where there was a lack of robust evidence, the international working group concluded a consensus-based approach was preferable. The conclusions are relevant to both children and adults. Thrombotic MicroangiopathyThe term TMA is a pathological term used to describe occlusive micro...

Results highlight the benefits of eculizumab in adult patients with aHUS: improvement in hematologic, renal, and quality-of-life parameters; dialysis discontinuation; and transplant protection.