Real-World Data on Effectiveness and Safety of First-Line Use of Caplacizumab in Italian Centers for the Treatment of Thrombotic Thrombocytopenic Purpura: The Roscapli Study

Fianchi, Luana; Bonanni, Matteo; Borchiellini, Alessandra; Valeri, Federica; Giuffrida, Gaetano; Grasso, Stephanie; Fozza, Claudio; Ponta, Michele; Tiscia, Giovanni L.; Grandone, Elvira; Vianelli, Nicola; Dedola, Alessandra; Pirozzi, Teresa; Sacco, Monica; Lancellotti, Stefano; De Cristofaro, Raimondo

doi:10.3390/jcm13216561

Open AccessArticle

Real-World Data on Effectiveness and Safety of First-Line Use of Caplacizumab in Italian Centers for the Treatment of Thrombotic Thrombocytopenic Purpura: The Roscapli Study

by

Luana Fianchi

¹,

Matteo Bonanni

¹,

Alessandra Borchiellini

²

,

Federica Valeri

²,

Gaetano Giuffrida

³

,

Stephanie Grasso

³,

Claudio Fozza

⁴,

Michele Ponta

⁴,

Giovanni L. Tiscia

⁵,

Elvira Grandone

⁵,

Nicola Vianelli

⁶,

Alessandra Dedola

⁶,

Teresa Pirozzi

⁷,

Monica Sacco

⁸

,

Stefano Lancellotti

⁷

and

Raimondo De Cristofaro

^7,8,*

¹

Hematology Unit, Fondazione Policlinico Universitario Agostino Gemelli—IRCCS, 00168 Rome, Italy

²

Regional Reference Center for Thrombotic and Haemorrhagic Disorders of Hematology, Division Department of Hematology and Oncology, A.O.U. Città della Salute e della Scienza di Torino, 10126 Torino, Italy

³

UOS e Centro di Riferimento Regionale di Malattie Ematologiche Rare, Division of Haematology, A.O.U Policlinico-S. Marco, 95123 Catania, Italy

⁴

Department of Clinical and Experimental Medicine, University of Sassari, 07100 Sassari, Italy

⁵

Thrombosis and Hemostasis Unit, Fondazione IRCCS “Casa Sollievo della Sofferenza”, S. Giovanni Rotondo, and Unità di Ostetricia e Ginecologia, Università degli Studi di Foggia, 71121 Foggia, Italy

⁶

IRCCS Azienda Ospedaliero, Istituto di Ematologia “Seràgnoli”, Universitaria di Bologna, 40121 Bologna, Italy

⁷

Service of Haemorrhagic and Thrombotic Diseases, Fondazione Policlinico Universitario Agostino Gemelli—IRCCS, 00168 Rome, Italy

⁸

Dipartimento di Medicina e Chirurgia Traslazionale, Università Cattolica S. Cuore, 00168 Rome, Italy

^*

Author to whom correspondence should be addressed.

J. Clin. Med. 2024, 13(21), 6561; https://doi.org/10.3390/jcm13216561

Submission received: 24 August 2024 / Revised: 4 October 2024 / Accepted: 29 October 2024 / Published: 31 October 2024

(This article belongs to the Special Issue Advances in Thrombosis and Haemostasis)

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Abstract

Background/Objectives: Immune thrombotic thrombocytopenic purpura (iTTP) is a thrombotic microangiopathy caused by the formation of anti-ADAMTS13 antibodies. Caplacizumab is approved for the treatment of acute episodes of iTTP in conjunction with plasma exchange (PEX) and immunosuppression. Real-world data for the use of caplacizumab in Italy have been recently published by a limited number of centers located in the northern and middle regions of the country only. Methods: A total of 38 patients with iTTP were enrolled in the study in six Italian centers spread over the entire territory of the country. The patients’ data were registered in eCRF. Results: All patients achieved normalization of platelet count (median 2.0 days, IQR: 2–4), within a time significantly shorter than in the absence of caplacizumab, as previously reported in other studies. As to the secondary aims, patients treated with caplacizumab had a few exacerbations (4/38 (10.5%)) and relapses (2/38, 5.3%). No deaths or refractoriness were observed in these patients. The total length of hospitalization was 12 days (IQR: 9–18) and only one patient required 2 days of stay in the intensive care unit. Interestingly, when caplacizumab was initiated within the first 3 days, the plasma exchange (PEX) duration was 9 days (IQR: 8–10), which was significantly lower than those reported in previous studies conducted in the absence of caplacizumab. No severe adverse event was described in the caplacizumab-treated patients. Conclusions: Caplacizumab reduced exacerbations and refractoriness compared with previously reported standard-of-care regimens. When administered in association with PEX and immunosuppressive therapy, caplacizumab provided rapid normalization of platelet count, which was responsible for lower overall hospitalization time, ICU stay, lower exacerbations and relapses compared to previously reported outcomes of studies carried out without caplacizumab.

Keywords: thrombotic thrombocytopenic purpura; von Willebrand factor; caplacizumab; ADAMTS13

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MDPI and ACS Style

Fianchi, L.; Bonanni, M.; Borchiellini, A.; Valeri, F.; Giuffrida, G.; Grasso, S.; Fozza, C.; Ponta, M.; Tiscia, G.L.; Grandone, E.; et al. Real-World Data on Effectiveness and Safety of First-Line Use of Caplacizumab in Italian Centers for the Treatment of Thrombotic Thrombocytopenic Purpura: The Roscapli Study. J. Clin. Med. 2024, 13, 6561. https://doi.org/10.3390/jcm13216561

AMA Style

Fianchi L, Bonanni M, Borchiellini A, Valeri F, Giuffrida G, Grasso S, Fozza C, Ponta M, Tiscia GL, Grandone E, et al. Real-World Data on Effectiveness and Safety of First-Line Use of Caplacizumab in Italian Centers for the Treatment of Thrombotic Thrombocytopenic Purpura: The Roscapli Study. Journal of Clinical Medicine. 2024; 13(21):6561. https://doi.org/10.3390/jcm13216561

Chicago/Turabian Style

Fianchi, Luana, Matteo Bonanni, Alessandra Borchiellini, Federica Valeri, Gaetano Giuffrida, Stephanie Grasso, Claudio Fozza, Michele Ponta, Giovanni L. Tiscia, Elvira Grandone, and et al. 2024. "Real-World Data on Effectiveness and Safety of First-Line Use of Caplacizumab in Italian Centers for the Treatment of Thrombotic Thrombocytopenic Purpura: The Roscapli Study" Journal of Clinical Medicine 13, no. 21: 6561. https://doi.org/10.3390/jcm13216561

APA Style

Fianchi, L., Bonanni, M., Borchiellini, A., Valeri, F., Giuffrida, G., Grasso, S., Fozza, C., Ponta, M., Tiscia, G. L., Grandone, E., Vianelli, N., Dedola, A., Pirozzi, T., Sacco, M., Lancellotti, S., & De Cristofaro, R. (2024). Real-World Data on Effectiveness and Safety of First-Line Use of Caplacizumab in Italian Centers for the Treatment of Thrombotic Thrombocytopenic Purpura: The Roscapli Study. Journal of Clinical Medicine, 13(21), 6561. https://doi.org/10.3390/jcm13216561

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Real-World Data on Effectiveness and Safety of First-Line Use of Caplacizumab in Italian Centers for the Treatment of Thrombotic Thrombocytopenic Purpura: The Roscapli Study

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