Early Use of Fostamatinib in Immune Thrombocytopenia

INTRODUCTION: Fostamatinib, an oral spleen tyrosine kinase (SYK) inhibitor, prevents antibody-mediated platelet destruction and has proven efficacy and safety in immune thrombocytopenia (ITP). However, evidence from real-world use in earlier treatment lines is limited. Here the objective was to evaluate the effectiveness and safety of fostamatinib as second- or third-line therapy in adult patients with ITP in Spain. METHODS: This multicenter study included 72 adult patients treated with fostamatinib across 22 Spanish centers, assessed retrospectively and prospectively. Demographic, clinical, and laboratory data, treatment responses, and adverse events (AEs) were analyzed. RESULTS: /L. Bleeding manifestations were present in 26.4% of patients. Overall, 83.3% achieved a response and 65.3% a complete response. Response rates were 76.0% with second-line and 87.2% with third-line fostamatinib, with complete response rates of 56.0% and 70.2%, respectively. The median time to platelet response was 10.5 days, and the median time to peak platelet count was 62 days. Forty patients (55.5%) received fostamatinib as monotherapy. AEs occurred in 44.4% of patients, were predominantly grade 1-2, and most commonly consisted of diarrhea and hypertension. Treatment discontinuation due to insufficient effectiveness occurred in 27.8% of patients. CONCLUSION: Fostamatinib demonstrated high effectiveness and acceptable tolerability as second- or third-line therapy for adult ITP in real-world clinical practice.

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Journal
Advances in Therapy
Published
2026-09-01
DOI
https://doi.org/10.1007/s12325-026-03769-z
Primary Topic
Platelet Disorders and Treatments
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article
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article

Early Use of Fostamatinib in Immune Thrombocytopenia

Carmen Benet, Natalia Acedo, Gloria García-Donas Gabaldón, Roberto Hernández-Martín et al.
Advances in Therapy
Platelet Disorders and Treatments
article

Early Use of Fostamatinib in Immune Thrombocytopenia

Carmen Benet, Natalia Acedo, Gloria García-Donas Gabaldón, Roberto Hernández-Martín, Daniel Martínez‐Carballeira, Isidro Jarque, Nuria Bermejo, Tomás José González‐López, Dolores Fernandez-Jimenez, Rocio Cardesa‐Cabrera, Begoña Navas-Elorza, C. Castro, Alvaro Perona-Blázquez, Josefa Luis‐Navarro, Guillermo Enrique Haces-Tirado, Isabel González‐Gascón‐y‐Marín, Sunil Lakhwani, Ana Torres-Tienza, Silvia Bernat, Reyes Jimenez-Bárcenas, Shally Marcellini, Pilar Galán, Violeta Martínez, María Luisa Lozano
article en

Abstract

INTRODUCTION: Fostamatinib, an oral spleen tyrosine kinase (SYK) inhibitor, prevents antibody-mediated platelet destruction and has proven efficacy and safety in immune thrombocytopenia (ITP). However, evidence from real-world use in earlier treatment lines is limited. Here the objective was to evaluate the effectiveness and safety of fostamatinib as second- or third-line therapy in adult patients with ITP in Spain. METHODS: This multicenter study included 72 adult patients treated with fostamatinib across 22 Spanish centers, assessed retrospectively and prospectively. Demographic, clinical, and laboratory data, treatment responses, and adverse events (AEs) were analyzed. RESULTS: /L. Bleeding manifestations were present in 26.4% of patients. Overall, 83.3% achieved a response and 65.3% a complete response. Response rates were 76.0% with second-line and 87.2% with third-line fostamatinib, with complete response rates of 56.0% and 70.2%, respectively. The median time to platelet response was 10.5 days, and the median time to peak platelet count was 62 days. Forty patients (55.5%) received fostamatinib as monotherapy. AEs occurred in 44.4% of patients, were predominantly grade 1-2, and most commonly consisted of diarrhea and hypertension. Treatment discontinuation due to insufficient effectiveness occurred in 27.8% of patients. CONCLUSION: Fostamatinib demonstrated high effectiveness and acceptable tolerability as second- or third-line therapy for adult ITP in real-world clinical practice.

Advances in Therapy
Universidad de La Laguna (ES), Hospital Universitario de Canarias (ES), Hospital Clínico San Carlos (ES), Hospital Universitari i Politècnic La Fe (ES), Hospital General De Segovia (ES), Hospital Universitario Virgen Macarena (ES), Ayuntamiento de Castellón de la Plana (ES), Instituto de Investigación Biosanitaria de Granada (ES), Centro Regional de Hemodonación (ES), Hospital Universitario de Burgos (ES), Hospital Arnau de Vilanova (ES), Hospital General Universitari de Castelló (ES), Hospital Virgen de la Concha (ES), Hospital Riotinto (ES), Hospital Universitario de La Princesa (ES), Hospital Universitario Central de Asturias (ES), Hospital Clínico Universitario Lozano Blesa (ES), Centro de Implantología Cirugía Oral y Maxilofacial (ES), Complejo Hospitalario Universitario de Albacete (ES), Hospital Comarcal de Inca (ES), Complejo Hospitalario de Cáceres (ES), Complejo Asistencial Universitario de Palencia (ES), Hospital Universitario Infanta Leonor (ES)
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Good health and well-being
Openalex Percentile: Top 11%
Platelet Disorders and Treatments
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