Real-World Data Analysis of Patients Affected by Immune-Mediated Thrombotic Thrombocytopenic Purpura in Italy

Background: The therapeutic management of immune-mediated thrombotic thrombocytopenic purpura (iTTP) has recently benefited from the introduction of caplacizumab, an agent directed at the inhibition of platelet aggregation. This real-world analysis investigated the epidemiology and the demographic and clinical characteristics of iTTP patients in Italy before and after caplacizumab introduction in 2020. Methods: Hospitalized adults with iTTP were included using the administrative databases of healthcare entities covering 17 million residents. Epidemiological estimates of iTTP considered the 3-year period before and after caplacizumab introduction. After stratification by treatment with or without caplacizumab, iTTP patients were characterized for their baseline features. Results: The annual incidence before and after 2020 was estimated in the range of 4.3-5.8 cases/million and 3.6-4.6 cases/million, respectively. From 2018 to 2022, 393 patients with iTTP were included, and 42 of them were treated with caplacizumab. Caplacizumab-treated patients showed better clinical outcomes, with tendentially shorter hospital stays and lower mortality rates (no treated patients died at either 1 month or 3 months after caplacizumab treatment initiation, compared to 10.5% and 11.1% mortality rates at 1 and 3 months, respectively, of the untreated ones). Conclusions: These findings may suggest that caplacizumab advent provided clinical and survival benefits for patients with iTTP.

Medienart:

E-Artikel

Erscheinungsjahr:

2024

Erschienen:

2024

Enthalten in:

Zur Gesamtaufnahme - volume:13

Enthalten in:

Journal of clinical medicine - 13(2024), 5 vom: 27. Feb.

Sprache:

Englisch

Beteiligte Personen:

Angelucci, Emanuele [VerfasserIn]
Artoni, Andrea [VerfasserIn]
Fianchi, Luana [VerfasserIn]
Dovizio, Melania [VerfasserIn]
Iacolare, Biagio [VerfasserIn]
Saragoni, Stefania [VerfasserIn]
Esposti, Luca Degli [VerfasserIn]

Links:

Volltext

Themen:

Caplacizumab
Epidemiology
Immune-mediated thrombotic thrombocytopenic purpura
Journal Article
Mortality
Real-world evidence

Anmerkungen:

Date Revised 11.04.2024

published: Electronic

Citation Status PubMed-not-MEDLINE

doi:

10.3390/jcm13051342

funding:

Förderinstitution / Projekttitel:

PPN (Katalog-ID):

NLM370815351