Fostamatinib effectiveness and safety for immune thrombocytopenia in clinical practice
Tomás José González‐López, Nuria Bermejo, Rocio Cardesa‐Cabrera, Violeta Martínez‐Robles, Gerardo Aguilar-Monserrate, Gloria Pérez Segura, A Domingo, Josefa Luis‐Navarro, Sunil Lakhwani, Natalia Acedo, Marı́a Luisa Lozano, Silvia Bernat, Ana Torres-Tienza, Ana Belén García Ruano, Isidro Jarque, Pilar Galán, Carmen Benet, Shally Marcellini, Reyes Jiménez‐Bárcenas, Daniel Martínez‐Carballeira, Dunia De Miguel-Llorente, Alvaro Perona-Blázquez, Isabel González-Gascón-y-Marín, Elsa López‐Ansoar, José María Alonso-Alonso, María L. Bengochea-Casado, Francisco Javier Díaz‐Gálvez, Ana Moretó, Gemma Moreno‐Jiménez, Roberto Hernández-Martín, Erik de Cabo, Julio Dávila, Amalia Cuesta, Carmen Pastoriza, Gerardo Julio Hermida-Fernández, Covadonga García, Miguel Angel Pozas-Mañas, Carlos Aguilar-Franco, Dolores Fernandez-Jimenez, Begoña Navas-Elorza, C. Castro, Álvaro Lorenzo, Xavier Ortı́n, Marta Rizo García, Sònia Piernas, Johana Díaz‐Santa, Inmaculada Soto, Drew Provan, Gloria García-Donas Gabaldón
Abstract
ABSTRACT: Fostamatinib, a recently approved Syk inhibitor used in adult primary immune thrombocytopenia (ITP), has been shown to be safe and effective in this disorder. However, clinical trial results may not be similarly reproduced in clinical practice. Here, 138 patients with ITP (both primary and secondary) from 42 Spanish centers who had been treated with fostamatinib were evaluated prospectively and retrospectively. The median age of our cohort (55.8% women) was 66 years (interquartile range [IQR], 56-80). The median time since ITP diagnosis at fostamatinib initiation was 51 months (IQR, 10-166). The median number of therapies before fostamatinib initiation was 4 (IQR, 2-5), including eltrombopag (76.1%), romiplostim (57.2%), and IV immunoglobulins (44.2%). Fifty-eight patients (42.0%) had signs/symptoms of bleeding in the month before treatment initiation. Seventy-nine percent of patients responded to fostamatinib with 53.6% complete responses (platelet count > 100 × 109/L). Eighty-three patients (60.1%) received fostamatinib monotherapy, achieving a high response rate (85.4%). The proportion of time in response during the 27-month period examined was 83.3%. The median time to platelet response was 11 days (IQR, 7-21). Sixty-seven patients (48.5%) experienced adverse events, mainly grade 1 to 2; the commonest of which were diarrhea (n = 28) and hypertension (n = 21). One patient had deep venous thrombosis, and one patient developed acute myocardial infarction. Fostamatinib was shown to be effective with good safety profile in patients with primary and secondary ITP across a wide age spectrum in this real-world study.