Caplacizumab use for immune thrombotic thrombocytopenic purpura: the Milan thrombotic thrombocytopenic purpura registry

Pasquale Agosti; Pasqualina De Leo; Marco Capecchi; Barbara Ferrari; Ilaria Mancini; Salvatore Gattillo; Silvia Maria Trisolini; Erminia Rinaldi; Gian Marco Podda; Lucia Prezioso; Prassede Salutari; Luca Facchini; Domenica Caramazza; Giulia Tolomelli; Andrea Artoni; Flora Peyvandi

doi:10.1016/j.rpth.2023.102185

Caplacizumab use for immune thrombotic thrombocytopenic purpura: the Milan thrombotic thrombocytopenic purpura registry

Res Pract Thromb Haemost. 2023 Aug 26;7(6):102185. doi: 10.1016/j.rpth.2023.102185. eCollection 2023 Aug.

Authors

Pasquale Agosti¹, Pasqualina De Leo², Marco Capecchi³, Barbara Ferrari², Ilaria Mancini¹, Salvatore Gattillo⁴, Silvia Maria Trisolini⁵, Erminia Rinaldi⁶, Gian Marco Podda⁷, Lucia Prezioso⁸, Prassede Salutari⁹, Luca Facchini¹⁰, Domenica Caramazza¹¹, Giulia Tolomelli¹², Andrea Artoni², Flora Peyvandi^{1

2}

Affiliations

¹ Department of Pathophysiology and Transplantation, and Fondazione Luigi Villa, Università degli Studi di Milano, Milan, Italy.
² Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy.
³ Clinica Moncucco, Hematology Service, Lugano, Switzerland.
⁴ Immuno-Hematology and Transfusion Medicine Unit, San Raffaele Hospital, Milan, Italy.
⁵ Hematology, Department of Translational and Precision Medicine, "Sapienza" University of Rome, Rome, Italy.
⁶ Hematology Unit, A. Perrino Hospital, Brindisi, Italy.
⁷ U.O.C. Medicina II, ASST Santi Paolo e Carlo, Dipartimento di Scienze della Salute, Università degli Studi di Milano, Milan, Italy.
⁸ Haematology and BMT Unit, Azienda Ospedaliero-Universitaria di Parma, Parma, Italy.
⁹ Ospedale Civile "Spirito Santo", Pescara, Italy.
¹⁰ Hematology Unit, Azienda USL-IRCCS di Reggio Emilia, Viale Umberto I n, 50 42123 Reggio Emilia, Italy.
¹¹ Hematology, University Hospital "Ospedale di Circolo e Fondazione Macchi" - ASST Sette Laghi, Viale L. Borri 57, 21100, Varese, Italy.
¹² Unit of Hematology, Rimini Hospital "Infermi", Rimini, Italy.

Abstract

•Data on caplacizumab use for thrombotic thrombocytopenic purpura (TTP) in Italy are missing.•Twenty-six Italian patients were treated with caplacizumab for an acute immune TTP episode.•Caplacizumab was effective in treating acute TTP in the Italian real-world clinical setting.•Two major bleeds leading to drug discontinuation were observed.

Keywords: ADAMTS-13 protein; caplacizumab; thrombocytopenia; thrombosis; thrombotic thrombocytopenic purpura.