Real‐World Effectiveness and Safety of Fostamatinib in Difficult‐to‐Treat Immune Thrombocytopenia Patients. A Prospective, Multicenter Registry in France

Fostamatinib is available in France since October 2021 for the treatment of adult chronic immune thrombocytopenia (ITP). French health authorities requested a 3-year, prospective, multicenter registry to provide real-world evidence about the effectiveness and safety of fostamatinib. Patients' characteristics, treatment response (ongoing exposure to fostamatinib and a platelet count ≥ 30 × 10<sup>9</sup>/L with no rescue in the previous 4 weeks) after 3, 6, 12, and 24 months (M); bleeding; fostamatinib discontinuation; adverse drug reactions (ADRs) and other events of interest have been analyzed. In total, 164 patients were included (median age: 59 years; 55.5% women; 84.1% had previous bleeding; 30 had secondary ITP; 89.0% had chronic ITP). The median ITP duration was 7.2 years and the median number of previous ITP treatments was 6. The response rate was 44.0% (70/159) at M3, 41.9% (62/148) at M6, 32.4% (44/136) at M12 and 20.0% (21/105) at M24. Concomitant treatment (mostly TPO-RA) was used in > 60.0% of responders at each endpoint. The cumulative discontinuation rate at each endpoint was, respectively, 27.0%, 44.6%, 55.9%, and 76.2%. Seventy-one (43.3%) patients experienced at least one bleeding during fostamatinib exposure; none was fatal. One hundred adverse drug reactions (8 serious) were observed in 61 (36.7%) patients, including diarrhea in 28 (17.1%) patients, arterial hypertension in 17 (10.4%). Seven thrombosis (4.3%) and 40 infections (12 serious) were reported in 25 patients (15.2%), mostly in patients with known risk factors. In conclusion, fostamatinib in combination with TPO-RA should be considered in difficult-to-treat ITP patients. No new safety signal was observed.

Authors

Institutions

Publication Details

Journal
American Journal of Hematology
Published
2026-01-08
DOI
https://doi.org/10.1002/ajh.70194
Primary Topic
Platelet Disorders and Treatments
Type
article
Field-Weighted Citation Impact
0.00
Controls
|||
ALL TIME
JAN
FEB
MAR
APR
MAY
JUN
JUL
AUG
SEP
OCT
article

Real‐World Effectiveness and Safety of Fostamatinib in Difficult‐to‐Treat Immune Thrombocytopenia Patients. A Prospective, Multicenter Registry in France

D. Gobert, M. Ruivard, A. Sommet, T. Moulinet et al.
American Journal of Hematology
Platelet Disorders and Treatments
article

Real‐World Effectiveness and Safety of Fostamatinib in Difficult‐to‐Treat Immune Thrombocytopenia Patients. A Prospective, Multicenter Registry in France

D. Gobert, M. Ruivard, A. Sommet, T. Moulinet, B. Godeau, G. Moulis, Y. Leveneur, Mikaël Martin, Yoann Zadro, S. Audia, Helder Gil, A. Saunier, Thibault Comont, L. Terriou, Stéphane Cheze, C. Gourguechon, P. Guilpain, Miguel Carreiro, Maryse Lapeyre‐mestre, Laurie Chabbert, N. Magy‐Bertrand, Julie Graveleau, Bernard Anthony, Ailsa Robbins, Frédérique Roy‐Péaud, Marie‐Pierre Ledoux, Mikael Ebbo, François Lifermann, Jehanne Faldlallah, Bernard Bonnotte, Xavier Delbrel, et le groupe des investigateurs de CARMEN‐France, Marc Michel, Matthieu Mahévas
article en

Abstract

Fostamatinib is available in France since October 2021 for the treatment of adult chronic immune thrombocytopenia (ITP). French health authorities requested a 3-year, prospective, multicenter registry to provide real-world evidence about the effectiveness and safety of fostamatinib. Patients' characteristics, treatment response (ongoing exposure to fostamatinib and a platelet count ≥ 30 × 10<sup>9</sup>/L with no rescue in the previous 4 weeks) after 3, 6, 12, and 24 months (M); bleeding; fostamatinib discontinuation; adverse drug reactions (ADRs) and other events of interest have been analyzed. In total, 164 patients were included (median age: 59 years; 55.5% women; 84.1% had previous bleeding; 30 had secondary ITP; 89.0% had chronic ITP). The median ITP duration was 7.2 years and the median number of previous ITP treatments was 6. The response rate was 44.0% (70/159) at M3, 41.9% (62/148) at M6, 32.4% (44/136) at M12 and 20.0% (21/105) at M24. Concomitant treatment (mostly TPO-RA) was used in > 60.0% of responders at each endpoint. The cumulative discontinuation rate at each endpoint was, respectively, 27.0%, 44.6%, 55.9%, and 76.2%. Seventy-one (43.3%) patients experienced at least one bleeding during fostamatinib exposure; none was fatal. One hundred adverse drug reactions (8 serious) were observed in 61 (36.7%) patients, including diarrhea in 28 (17.1%) patients, arterial hypertension in 17 (10.4%). Seven thrombosis (4.3%) and 40 infections (12 serious) were reported in 25 patients (15.2%), mostly in patients with known risk factors. In conclusion, fostamatinib in combination with TPO-RA should be considered in difficult-to-treat ITP patients. No new safety signal was observed.

American Journal of HematologyVol. 101(4)
Jiangsu University (CN), École Nationale d'Ingénieurs de Tarbes (FR), Université de Bordeaux (FR), Université Fédérale de Toulouse Midi-Pyrénées (FR), Université Paris-Est Créteil (FR), Université de Montpellier (FR), Université de Lille (FR), Saint Louis University Hospital (US), Centre Hospitalier Universitaire de Nantes (FR), Université de Poitiers (FR), Sorbonne Université (FR), Assistance Publique Hôpitaux de Marseille (FR), Centre de Recherches en Cancérologie de Toulouse (FR), Centre Hospitalier Universitaire de Besançon (FR), Lille’s Cardiology Hospital (FR), Centre Hospitalier Universitaire de Montpellier (FR), Centre Hospitalier Universitaire Amiens-Picardie (FR), Toulouse Mathematics Institute (FR), Centre Hospitalier Régional et Universitaire de Nancy (FR), University of Clermont Auvergne (FR), Centre Hospitalier De Pau (FR), Centre Hospitalier Universitaire de Reims (FR), Centre Hospitalier de Bigorre (FR), Centre Hospitalier Universitaire de Clermont-Ferrand (FR), ATECA - Applications Technologie Avancée (FR), Centre de Recherche Saint-Antoine (FR), Yixing People's Hospital (CN), Maison des Sciences sociales et des Humanités de Dijon (FR), Centre Hospitalier de Dax –Côte d’Argent (FR), Hôpital Bichat-Claude-Bernard (FR), Lux Research (United States) (US), Centre Hospitalier Saint-Nazaire (FR), Université de Strasbourg (FR), Université de Reims Champagne-Ardenne (FR), Nantes Université (FR), Université de Caen Normandie (FR)
Good health and well-being
Openalex Percentile: Top 94%
Platelet Disorders and Treatments
DOISourceOpenAlex
AI Navigator

Ask Laika to Summarize, Analyze, and Connect papers live on the map.

Summarize Papers & Methodologies

Extract key findings, datasets, and comparative methods across publications.

Benchmark Rankings & Visual Analytics

Rank top research institutions, authors, funders, topics, and journals by Field-Weighted Citation Impact (FWCI) and paper volume with instant charts.

Connect Distant Disciplines

Bridge topological clusters on the map to find hidden collaborative intersections.