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Standardization of its diagnosis and optimal management is challenging. This study aimed to evaluate the role of centralized, rapid testing of ADAMTS13 in patients experiencing acute TMAs requiring plasma-exchange (PEX) and to estimate the incidence of TTP in a large Italian Region.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>We perfomed a cohort study in the frame of the project \u201cSet-up of a Lombardy network for the study and treatment of patients undergoing apheresis\u201d, including 11 transfusion centers in the Region. Consecutive patients referred from 2014 to 2016 with acute TMAs requiring PEX were enrolled. Centralized ADAMTS13 activity testing was performed at the Milan Hemophilia and Thrombosis Center within 24\u00a0h.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Forty-three TMA patients (44 events) were enrolled, of whom 35 (81%) had severe ADAMTS13 deficiency. Patients with severe ADAMTS13 deficiency were younger, mainly women, with a higher prevalence of autoimmune disorders and a lower prevalence of cancer. Clinical and laboratory characteristics of patients with and without severe ADAMTS13 deficiency largely overlapped, with a lower platelet count being the only baseline marker that significantly differed between the two patient groups (ADAMTS13 activity\u2009&lt;\u200910% vs\u2009\u2265\u200910%: median difference of -27\u2009\u00d7\u200910<jats:sup>9<\/jats:sup>\/l, 95% CI \u2212\u200937 to \u2212\u20093). PEX treatment was initiated in all patients, but soon discontinued in cases without severe ADAMTS13 deficiency. In this group, the mortality rate was higher and no episode exacerbations or relapses within 6\u00a0months occured. The estimated average annual incidence of acute acquired TTP events was 1.17 [0.78\u20131.55] per million people.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Severe ADAMTS13 deficiency distinguished two groups of patients with largely overlapping clinical features but different treatment and disease course. This study provides a feasible model implemented in a large Italian region for the practical clinical approach to TMAs and underlines the importance of urgent ADAMTS13 activity testing for an accurate differential diagnosis and therapeutic approach.<\/jats:p><\/jats:sec>","DOI":"10.1186\/s13023-022-02400-y","type":"journal-article","created":{"date-parts":[[2022,6,23]],"date-time":"2022-06-23T12:15:04Z","timestamp":1655986504000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Lombardy diagnostic and therapeutic network of thrombotic 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Approval was granted by the Ethics Committee of Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (Date: 18\/12\/2013\/No: 123). Written informed consent was obtained from all subjects included in the study.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval and consent to participate"}},{"value":"Not applicable.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"Ilaria Mancini received honoraria for participating as a speaker at educational meetings organized by Instrumentation Laboratory and Sanofi-Genzyme, outside the present study. Barbara Ferrari received honoraria for participating to a Sanofi advisory board meeting, outside the present study. Roberta Palla received honoraria for participating as a speaker at educational meetings organized by Novonordisk, outside the present study. Chiara Novelli received honoraria for participating as a speaker at educational meetings and as a scientific advisor by Instrumentation Laboratory, outside the present study. Anna Falanga received honoraria for participating as a speaker at satellite symposia and educational meetings organized by Sanofi, Bayer, Roche, Leo Pharma, Stago, and Pfizer, outside the present study. Flora Peyvandi received honoraria for participating as a speaker in educational meetings\/advisory boards organized by Roche, Sobi, Sanofi and Takeda, outside the present study. The other authors do not have any conflict of interest to disclose.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"246"}}