PB0290 - Caplacizumab for the management of acute immune-mediated thrombotic thrombocytopenic purpura: real world data from the Milan TTP Registry

Sunday, July 10, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Presenting Author(s)

Pasquale Agosti, MD

MD, PhD
Università degli Studi di Milano, Department of Pathophysiology and Transplantation and Fondazione Luigi Villa, Milan, Italy
Milano, Lombardia, Italy

Co-Author(s)

PD

Pasqualina De Leo

Research fellow
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy.
Milano, Lombardia, Italy
MC

Marco Capecchi

Medical Doctor
Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Università degli Studi di Milano, Department of Biomedical Sciences for Health, Milan, Italy
Milan, Lombardia, Italy
Andrea Artoni, MD

MD
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
Milano, Lombardia, Italy
Ilaria Mancini, PhD

Post-Doc Research Fellow
Università degli Studi di Milano, Department of Pathophysiology and Transplantation and Fondazione Luigi Villa, Milan, Italy
Milan, Lombardia, Italy
MB

Marina Biganzoli

Lab Technician
former Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
Milan, Lombardia, Italy
BF

Barbara Ferrari

MD
Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
Milan, Lombardia, Italy
SG

Salvatore Gattillo

Medical Doctor
Immuno-Hematology and Transfusion Medicine Unit, San Raffaele Hospital, 20132 Milan, Italy.
Milano, Lombardia, Italy
ST

Silvia Maria Trisolini

Medical Doctor
Hematology, Department of Translational and Precision Medicine, "Sapienza" University of Rome, Rome, Italy
Roma, Lazio, Italy
ER

Erminia Rinaldi

MD
Haematology, A. Perrino Hospital, Brindisi, Italy
Brindisi, Puglia, Italy
GP

Gianmarco Podda

Head
Medicina 3, ASST Santi Paolo e Carlo, San Paolo
Milano, Lombardia, Italy
LP

Lucia Prezioso

MD
U.O. Ematologia E Centro Trapianti Midollo Osseo, Ospedale Maggiore, Parma, Italy
Parma, Emilia-Romagna, Italy
SP

Salutari Prassede

Medical Doctor
Ospedale Civile "Spirito Santo," Pescara
Pescara, Abruzzi, Italy
LF

Luca Facchini

Medical Doctor
Hematology Unit, Azienda Unità Sanitaria Locale-IRCCS di Reggio Emilia, Reggio Emilia, Italy
Reggio Emilia, Emilia-Romagna, Italy
DC

Domenica Caramazza

Medical Doctor
Hematology, University Hospital "Ospedale di Circolo e Fondazione Macchi" - ASST Sette Laghi, Viale L. Borri 57, 21100, Varese, Italy
Varese, Lombardia, Italy
GT

Giulia Tolomelli

Medical Doctor
Unit of Hematology, Rimini Hospital "Infermi", Rimini, Italy
Rimini, Emilia-Romagna, Italy
Flora Peyvandi, MD, PhD

Professor of Internal Medicine; Director of the Angelo Bianchi Bonomi Hemophilia & Thrombosis Center
Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy; Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy
Milan, Lombardia, Italy

Background: Acute immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare but life-threatening thrombotic microangiopathy. In 2018 caplacizumab, an anti-von Willebrand factor nanobody, was approved by the European Medicines Agency for the treatment of acute iTTP.
To date, data stemming from real-world experience are limited.

Aims: To describe the efficacy and safety endpoints of caplacizumab treatment in Italian iTTP patients from a real-world clinical setting.

Methods: In this observational retrospective study, 26 patients from the Milan TTP Registry receiving caplacizumab treatment for an acute iTTP episode between 2018 and August 2021 were enrolled.

Results: The main patient characteristics and acute event features are shown in table 1.
All patients were treated with plasma-exchange (PEX), corticosteroids and caplacizumab, except for one patient with mild thrombocytopenia (113000/µL) treated with steroids and caplacizumab. Rituximab was used in 16/24 patients (66.7%).
Caplacizumab was started a median of 1 day after PEX initiation (table 2). Thirty percent of patients started caplacizumab 7 days or more after PEX start to treat refractory TTP or exacerbation. All patients achieved clinical remission. In patients treated with caplacizumab within 24 hours after PEX start, the median time to platelet count normalization was 3.5 days (3.0-4.3), with a median of 6 days of PEX (4.3-7.8) to achieve clinical response. One patient experienced acute TTP exacerbation 11 days after stopping caplacizumab.
Two caplacizumab-related adverse events leading to drug discontinuation were observed, an episode of hemoptysis treated with a plasma-derived von Willebrand factor/factor VIII-containing concentrate and artery embolization, and a gastrointestinal bleeding requiring blood transfusion in a patient with several conditions predisposing to bleeding.

Conclusion(s): With a median of 4 days to achieve platelet count normalization, caplacizumab proved effective and safe for the treatment of acute iTTP in the real-world clinical setting of the Milan TTP Registry, consistently with the results of randomized clinical trials.