Treatment with fostamatinib in patients with immune thrombocytopenia: Experience from the Andalusian region in Spain-The Fostasur Study.
СтатьяМногоцентровое исследованиеНаблюдательное исследованиеНазвание и аннотация: Перевод пока не подготовлен
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Jiménez-Bárcenas Reyes, García-Donas-Gabaldón Gloria, Campos-Álvarez Rosa María, Fernández-Sánchez de Mora María Carmen, Luis-Navarro Josefa, Domínguez-Rodríguez Juan Francisco, Nieto-Hernández María Del Mar, Sánchez-Bazán Irene, Yera-Cobo Maria, Cardesa-Cabrera Rocio, Jiménez-Gonzalo Francisco José, Ruiz-Cobo María Antonia, Caparrós-Miranda Isabel, Entrena-Ureña Laura, Fernández Jiménez Dolores, Díaz-Canales Dana, Moreno-Carrasco Gloria, Calderón-Cabrera Cristina, Núñez-Vázquez Ramiro José, Pedrote-Amador Begoña, Mingot-Castellano María Eva, Grupo Andaluz de Coagulopatías (GACC)
Аннотация
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 × 109/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.