Preprint Article Version 1 This version is not peer-reviewed

Platelet Responses after Tapering and Discontinuation of Fostamatinib in Patients with Immune Thrombocytopenia: A Continuation of the Fostasur Study

Version 1 : Received: 14 September 2024 / Approved: 16 September 2024 / Online: 18 September 2024 (05:14:38 CEST)

How to cite: Mingot-Castellano, M.-E.; Garcia-Donas, G.; Campos-Alvarez, R. M.; Fernández-Sánchez de Mora, M. C.; Luis-Navarro, J.; Domínguez-Rodriguez, J. F.; Nieto-Hernandez, M. D. M.; Sánchez-Bazán, I.; Yera-Cobo, M.; Cardesa-Cabrera, R.; Jímenez-Gonzalo, F. J.; Caparrós-Miranda, I.; Entrena-Ureña, L.; Jurado-Herrera, S.; Fernández Jimenez, D.; Díaz-Canales, D.; Moreno-Carrasco, G.; Calderón-Cabrera, C.; Nuñez-Velazquez, R. J.; Pedrote-Amador, B.; Jiménez-Bárcenas, R. Platelet Responses after Tapering and Discontinuation of Fostamatinib in Patients with Immune Thrombocytopenia: A Continuation of the Fostasur Study. Preprints 2024, 2024091240. https://doi.org/10.20944/preprints202409.1240.v1 Mingot-Castellano, M.-E.; Garcia-Donas, G.; Campos-Alvarez, R. M.; Fernández-Sánchez de Mora, M. C.; Luis-Navarro, J.; Domínguez-Rodriguez, J. F.; Nieto-Hernandez, M. D. M.; Sánchez-Bazán, I.; Yera-Cobo, M.; Cardesa-Cabrera, R.; Jímenez-Gonzalo, F. J.; Caparrós-Miranda, I.; Entrena-Ureña, L.; Jurado-Herrera, S.; Fernández Jimenez, D.; Díaz-Canales, D.; Moreno-Carrasco, G.; Calderón-Cabrera, C.; Nuñez-Velazquez, R. J.; Pedrote-Amador, B.; Jiménez-Bárcenas, R. Platelet Responses after Tapering and Discontinuation of Fostamatinib in Patients with Immune Thrombocytopenia: A Continuation of the Fostasur Study. Preprints 2024, 2024091240. https://doi.org/10.20944/preprints202409.1240.v1

Abstract

Fostamatinib is a spleen tyrosine kinase (SYK) inhibitor approved for the treatment of adult patients with chronic immune thrombocytopenia (ITP). There is little information about dose tapering and sustained remission after discontinuation in ITP. In this retrospective, multicenter study we evaluated efficacy and safety of fostamatinib in adult patients with ITP before, during and after tapering/discontinuation (T/D). T/D was performed in subjects who achieved complete platelet response (CR) with progressive, conditional dose reduction dose every 4 weeks. Sixty-one patients were included at 14 reference centers between October 2021 and May 2023. In subjects that completed T/D (n=9), median time from treatment initiation to response was 21 days (IQR:7.5-42), time to CR 28 days (IQR:28-42), time to start tapering 116 days (IQR: 42-140), duration of tapering 112.5 days (IQR 94.5-191). Median platelet count at tapering was 232×109/L (IQR 152-345×109/L) and at discontinuation 190×109/L (IQR 142.5-316.5×109/L). With a median follow-up since discontinuation of 263 days (IQR: 247-313 days), only two patients have relapsed (at 63 and 73 days. Fostamatinib was restarted, achieving a new CR. Platelet counts higher than 100x109/L at week 12 was the only positive predictive factors for successful tapering and discontinuation.

Keywords

tapering; sustained response; ITP; fostamatinib; thrombopoietin analogs; Syk inhibitor; Syk; remission; purpura; thrombocytopenic; idiopathic; diarrhea; hemorrhage; thrombocytopenia

Subject

Medicine and Pharmacology, Hematology

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