Thromb Haemost 2019; 119(05): 695-704
DOI: 10.1055/s-0039-1679907
Coagulation and Fibrinolysis
Georg Thieme Verlag KG Stuttgart · New York

Clinical and Laboratory Features of Patients with Acquired Thrombotic Thrombocytopenic Purpura: Fourteen Years of the Milan TTP Registry

Ilaria Mancini
1  Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Fondazione Luigi Villa, Milan, Italy
,
Silvia Pontiggia
2  Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
,
Roberta Palla
1  Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Fondazione Luigi Villa, Milan, Italy
,
Andrea Artoni
2  Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
,
Carla Valsecchi
2  Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
,
Barbara Ferrari
2  Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
,
Danijela Mikovic
3  Haemostasis Department and Haemophilia Center, Blood Transfusion Institute of Serbia, Belgrade, Serbia
,
Flora Peyvandi
1  Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Fondazione Luigi Villa, Milan, Italy
2  Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
,
for the Italian Group of TTP Investigators› Author Affiliations
Further Information

Publication History

02 July 2018

10 January 2019

Publication Date:
12 March 2019 (eFirst)

Abstract

Acquired thrombotic thrombocytopenic purpura (TTP) is a rare thrombotic microangiopathy caused by the immune-mediated severe deficiency of ADAMTS13. We hereby report the demographic and disease-related data of acquired TTP patients recorded in the Milan TTP Registry (www.ttpdatabase.org). We performed a cross-sectional study of 302 individuals enrolled in our registry for an acute episode of acquired TTP occurred between 2002 and 2015 (female 77%; median age at onset 40 years, interquartile range: 30–50). Twenty per cent of patients had concomitant autoimmune disorders. Among potential triggers of acute episodes, infections were the most prevalent (27%), followed by estroprogestinics use and pregnancy (5 and 4% of women, respectively). At presentation, systemic (72%), bleeding (68%) and neurological (43%) symptoms were the most frequent, whereas a lower prevalence of renal (18%) and cardiovascular (10%) signs and symptoms was observed. Almost all acute events were treated by plasma exchange and steroids, and 15% by rituximab. Exacerbation of acute TTP occurred in 15% of events. The TTP-related mortality was 5%. In survivors, the median number of plasma exchange procedures to remission was 9 (interquartile range: 6–14), longer for first events than relapses (median difference 3, 95% confidence interval: 2–4). Of 251 survivors of the first TTP episode with at least a 6-month follow-up, 55% had a relapse. In conclusion, acquired TTP is a severe disease with highly variable clinical presentation, usually requiring a long hospitalization. The Milan TTP Registry represents a powerful tool to improve our knowledge and management of acquired TTP.

Authors' Contributions

I. Mancini designed the study, performed experiments, performed quality control and statistical analysis, interpreted the results and wrote the manuscript; S. Pontiggia performed experiments, collected clinical and/or experimental data, performed quality control and statistical analysis and contributed to write the manuscript; R. Palla interpreted the results, contributed to write and critically reviewed the manuscript; A. Artoni collected clinical data, interpreted the results and critically reviewed the manuscript; C. Valsecchi performed experiments, interpreted the results and critically reviewed the manuscript; B. Ferrari collected clinical data, interpreted the results and critically reviewed the manuscript; D. Mikovic, S.M. Trisolini, S. Capria, L. Facchini, K. Codeluppi, E. Rinaldi, D. Pastore, S. Campus, R.A. Podda, C. Caria, A. Caddori, D. Nicolosi, G. Giuffrida, V. Agostini, U. Roncarati, C. Mannarella, A. Fragasso, G.M. Podda, S. Birocchi, A.M. Cerbone, A. Tufano, G. Loffredo, G. Menna, M. Pizzuti, G. Re, M. Ronchi, A. De Fanti, S. Amarri, M. Defina, M. Bocchia and S. Cerù collected clinical data; F. Peyvandi designed the study, interpreted the results and critically reviewed the manuscript.


Study Group Members

The Italian Group of TTP Investigators includes: Silvia Maria Trisolini and Saveria Capria (Hematology, Department of Cellular Biotechnologies and Hematology, Policlinico Umberto 1, Sapienza University, Rome, Italy), Luca Facchini and Katia Codeluppi (Hematology Unit, Oncology Department, Azienda Unità Sanitaria Locale – IRCCS di Reggio Emilia, Reggio Emilia, Italy), Erminia Rinaldi and Domenico Pastore (Hematology Unit, A. Perrino Hospital, Brindisi, Italy), Simona Campus and Rosa Anna Podda (Pediatric Unit, Ospedale Microcitemico, Cagliari, Italy), Cinzia Caria and Aldo Caddori (Internal Medicine Unit, S.S. Trinità Hospital, Cagliari, Italy), Daniela Nicolosi and Gaetano Giuffrida (Hematology Division, Department of Clinical and Molecular Biomedicine, University of Catania, Catania, Italy), Vanessa Agostini and Umberto Roncarati (U.O. Immunoematologia e Medicina Trasfusionale/Officina Trasfusionale, Cesena e Forlì, Cesena, Italy), Clara Mannarella and Alberto Fragasso (Hematology Unit, Madonna delle Grazie Hospital, Matera, Italy), Gian Marco Podda and Simone Birocchi (Unità di Medicina III, ASST Santi Paolo e Carlo, Milano, Italy), Anna Maria Cerbone and Antonella Tufano (Department of Clinical Medicine and Surgery, AOU Federico II, Napoli, Italy), Giuseppe Loffredo and Giuseppe Menna (Department of Oncology, AORN Santobono-Pausilipon, Naples, Italy), Michele Pizzuti (Hematology Unit, AO San Carlo, Potenza, Italy), Giuseppe Re and Michela Ronchi (Internal Medicine Unit, Department of Medicine, Lugo Hospital, Ravenna, Italy), Alessandro De Fanti (Departmental Simple Unit of Pediatric Rheumatology, AUSL-IRCSS Reggio Emilia, Reggio Emilia, Italy), Sergio Amarri (Pediatrics Unit, AUSL-IRCSS Reggio Emilia, Reggio Emilia, Italy), Marzia Defina and Monica Bocchia (Università degli studi di Siena, Department of Medical, Surgery and Neuroscience, Hematology Unit, Azienda Ospedaliera Universitaria Senese, Siena, Italy) and Silvia Cerù (Hematology Unit, Santa Chiara Hospital, Trento, Italy).


* A complete list of the members of the Italian Group of TTP Investigators appears in section ”Study Group Members.”


Supplementary Material