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. 2025 Aug 25.
doi: 10.1111/bjh.70069. Online ahead of print.

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

Elisa Lucchini  1 , Nicola Vianelli  2 , Ugo Consoli  3 , Giuseppe Carli  4 , Valentina Carrai  5 , Monica Carpenedo  6 , Valerio De Stefano  7 , Gianluca Gaidano  8 , Francesca Palandri  2 , Giuseppe Auteri  9 , Elena Rossi  7 , Andrea Patriarca  8 , Vanessa Innao  3 , Gessica Marchesini  4 , Mario Ballerini  1 , Maria Chiara Rossetti  1 , Giulia Mosini  10 , Patrizia Sciancalepore  11 , Jacopo Agnelli Giacchello  11 , Pellegrino Musto  12 , Elena Rivolti  13 , Giulia Soverini  14 , Alessandra Borchiellini  15 , Cristina Clissa  16 , Fabrizio Pane  17 , Sara Galimberti  18 , Vincenzo Pavone  19 , Antonella Poloni  20 , Francesco Buccisano  21 , Maurizio Musso  22 , Paola Fazi  23 , Francesca Paoloni  23 , Livia Gorreo Renzulli  23 , Francesco Zaja  1   24
Affiliations

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

Elisa Lucchini et al. Br J Haematol. .

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.

Keywords: ITP; fostamatinib; real world.

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