Real‐world efficacy and safety of fostamatinib in <scp>ITP</scp> patients: Italian multicentre experience. <scp>GIMEMA ITP1122</scp> study

The efficacy and safety of fostamatinib in adult patients with persistent/chronic immune thrombocytopenia (p/c ITP) were demonstrated in the FIT-1/FIT-2 trials. This retrospective, multicentre observational study evaluated real-world outcomes in consecutive p/c ITP patients treated with fostamatinib in Italy. The primary end-point, serving as a surrogate for both efficacy and safety, was the proportion of patients receiving fostamatinib for at least 6 months. Ninety-five patients were enrolled across 20 Italian centres, 59% female, median age 64 years (range 21-86). The median time from ITP diagnosis to fostamatinib initiation was 7.7 years; the median number of prior therapies was 4, including 54% of patients who received more than one thrombopoietin receptor agonist (TPO-RA) and 23% who underwent splenectomy. The overall response rate was 73%, with 32% complete responses. Most patients (74%) achieved a response within the first month. Concerning the primary end-point, 45% of patients received fostamatinib for 6 months; the median treatment duration was 7.3 months. The main cause of discontinuation was treatment failure (43%). Fifty-nine adverse events were reported in 38 patients, mostly grade 1-2, leading to fostamatinib discontinuation in 8% of patients. This real-world data confirm fostamatinib as an effective and safe option in relapsed/refractory ITP.

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Publication Details

Journal
British Journal of Haematology
Published
2025-08-25
DOI
https://doi.org/10.1111/bjh.70069
Citations
2
Primary Topic
Platelet Disorders and Treatments
Type
article
Field-Weighted Citation Impact
3.54

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article

Real‐world efficacy and safety of fostamatinib in ITP patients: Italian multicentre experience. GIMEMA ITP1122 study

Giulia Soverini, Sara Galimberti, Francesco Buccisano, Francesca Palandri et al.
2 citations
British Journal of Haematology
Platelet Disorders and Treatments
3.54
article

Real‐world efficacy and safety of fostamatinib in ITP patients: Italian multicentre experience. GIMEMA ITP1122 study

Giulia Soverini, Sara Galimberti, Francesco Buccisano, Francesca Palandri, Elena Rivolti, Francesca Paoloni, Vanessa Innao, Paola Fazi, Elisa Lucchini, Valentina Carrai, Jacopo Agnelli Giacchello, Giuseppe Auteri, Giulio Marchesini, Gianluca Gaïdano, Andrea Patriarca, Nicola Vianelli, Elena Rossi, Mario Ballerini, Valerio De Stefano, Livia Gorreo Renzulli, Giulia Mosini, Pellegrino Musto, Alessandra Borchiellini, Giuseppe Carli, Monica Carpenedo, Cristina Clissa, Antonella Poloni, Patrizia Sciancalepore, Ugo Consoli, Maurizio Musso, Vincenzo Pavone, Francesco Zaja, Fabrizio Pane, Maria Chiara Rossetti
article en
2 citations

Abstract

The efficacy and safety of fostamatinib in adult patients with persistent/chronic immune thrombocytopenia (p/c ITP) were demonstrated in the FIT-1/FIT-2 trials. This retrospective, multicentre observational study evaluated real-world outcomes in consecutive p/c ITP patients treated with fostamatinib in Italy. The primary end-point, serving as a surrogate for both efficacy and safety, was the proportion of patients receiving fostamatinib for at least 6 months. Ninety-five patients were enrolled across 20 Italian centres, 59% female, median age 64 years (range 21-86). The median time from ITP diagnosis to fostamatinib initiation was 7.7 years; the median number of prior therapies was 4, including 54% of patients who received more than one thrombopoietin receptor agonist (TPO-RA) and 23% who underwent splenectomy. The overall response rate was 73%, with 32% complete responses. Most patients (74%) achieved a response within the first month. Concerning the primary end-point, 45% of patients received fostamatinib for 6 months; the median treatment duration was 7.3 months. The main cause of discontinuation was treatment failure (43%). Fifty-nine adverse events were reported in 38 patients, mostly grade 1-2, leading to fostamatinib discontinuation in 8% of patients. This real-world data confirm fostamatinib as an effective and safe option in relapsed/refractory ITP.

British Journal of HaematologyVol. 207(6)
University of Pisa (IT), Marche Polytechnic University (IT), University of Trieste (IT), University of Ferrara (IT), Agostino Gemelli University Polyclinic (IT), Azienda Ospedaliera Citta' della Salute e della Scienza di Torino (IT), Luigi Sacco Hospital (IT), Azienda Sanitaria Unità Locale di Reggio Emilia (IT), Azienda Socio Sanitaria Territoriale Grande Ospedale Metropolitano Niguarda (IT), Azienda Ospedaliera Universitaria Integrata Verona (IT), IRCCS Azienda Ospedliero-Universitaria di Bologna Policlinico di Sant'Orsola (IT), Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia (IT), Ospedale Garibaldi (IT), Ospedali Riuniti di Ancona (IT), Fondazione Gimema Onlus (IT), Azienda Ospedaliero-Universitaria Careggi (IT), Azienda Ospedaliera S.Maria (IT), Policlinico Tor Vergata (IT), Ospedale San Bortolo (IT), ASST Fatebenefratelli Sacco (IT), Piedmont University (US), Azienda Ospedaliera Nazionale SS. Antonio e Biagio e Cesare Arrigo (IT), Gruppo Italiano per il Trapianto di Midollo Osseo (IT), La Maddalena (IT), Azienda Sanitaria Universitaria Integrata di Trieste (IT), University of Bari Aldo Moro (IT)
Grifols
Openalex Percentile: Top 7%
Platelet Disorders and Treatments
3.54
DOISourceOpenAlex
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