Treatment with fostamatinib in patients with immune thrombocytopenia: Experience from the Andalusian region in Spain—The Fostasur Study
Reyes Jiménez‐Bárcenas, Gloria García‐Donas‐Gabaldón, Rosa M. Campos-Alvarez, María Carmen Fernández‐Sánchez de Mora, Josefa Luis‐Navarro, Juan Francisco Domínguez‐Rodríguez, María del Mar Nieto‐Hernández, Irene Sánchez‐Bazán, Maria Yera‐Cobo, Rocio Cardesa‐Cabrera, Francisco José Jiménez-Gonzalo, María Antonia Ruiz‐Cobo, Isabel Socorro Caparrós-Miranda, Laura Entrena‐Ureña, Dolores Fernández Jiménez, Dana Díaz‐Canales, Gloria Moreno‐Carrasco, Cristina Calderón‐Cabrera, Ramiro Núñez, Begoña Pedrote‐Amador, María Eva Mingot‐Castellano, Grupo Andaluz de Coagulopatías (GACC)
Abstract
Summary Immune thrombocytopenia (ITP) is characterized by low platelet counts (PLTs) and an increased risk of bleeding. Fostamatinib, a spleen tyrosine kinase inhibitor, has been approved as a second‐line treatment for ITP. Real‐world data on fostamatinib are lacking. This observational, retrospective, multicentre study, conducted in the Andalusia region of Spain, evaluated 44 adult primary ITP patients (47.7% female; median age 58 years; newly diagnosed ITP 6.8%; persistent 13.6%; chronic 79.5%; median four prior treatments) after ≥ 4 weeks of fostamatinib therapy. The median PLT at the initiation of fostamatinib was 15 × 10 9 /L. Common reasons for starting fostamatinib were refractoriness or intolerance to prior therapy, oral medication preference, history of thrombosis and cardiovascular risk. Dosing was individualized based on efficacy and tolerance. After 2 weeks, global response rate was 56.8% (response and complete response). Response rates were 70.5%, 62.5% and 64% at 4 weeks, 12 weeks and at the end of the study respectively. Adverse events were mild, and no patients discontinued as a result. This real‐world study demonstrated a response rate similar to fostamatinib as seen in the pivotal clinical trials while including newly diagnosed patients and allowing for individualized dosing.