1. Eltrombopag safety and efficacy for primary chronic immune thrombocytopenia in clinical practice
- Author
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Erik de Cabo, Miguel Angel Fuertes-Palacio, Violeta Martínez-Robles, Pável E Olivera, Montserrat Cortés, Carlos Aguilar, José Ramón González-Porras, Rosa Fisac, Blanca Sanchez-Gonzalez, Marta Gómez-Nuñez, Luis Javier García-Frade, Angeles Fernández-Rodríguez, María Teresa Álvarez-Román, Susana Pérez-Crespo, Abelardo Bárez, Gloria Pérez-Rus, Marcio Andrade, Jose Angel Hernandez-Rivas, Cristina Pascual, María Jesús Peñarrubia, Isidro Jarque, Tomás José González-López, Silvia Bernat, Carmen Fernández-Miñano, and Fernando Fernández-Fuentes
- Subjects
Male ,Pediatrics ,medicine.medical_specialty ,Eltrombopag ,thrombocytopenia ,030204 cardiovascular system & hematology ,Benzoates ,TPO ,03 medical and health sciences ,chemistry.chemical_compound ,0302 clinical medicine ,Interquartile range ,medicine ,Humans ,thrombopoietin ,Adverse effect ,Thrombopoietin ,Aged ,Retrospective Studies ,Response rate (survey) ,Purpura, Thrombocytopenic, Idiopathic ,business.industry ,Hematology ,General Medicine ,Middle Aged ,Clinical trial ,Immune ,Hydrazines ,Treatment Outcome ,chemistry ,Spain ,030220 oncology & carcinogenesis ,Concomitant ,Chronic Disease ,Retreatment ,Cohort ,Pyrazoles ,ITP ,Female ,business ,Immunosuppressive Agents - Abstract
BackgroundEltrombopag is effective and safe in chronic immune thrombocytopenia (ITP). However, clinical trials may not accurately reflect what happens in clinical practice. We evaluated the efficacy and safety of eltrombopag in primary chronic ITP in a real-world setting. MethodsA total of 164 primary patients with chronic ITP from 40 Spanish centers, who had been treated with eltrombopag, were retrospectively evaluated. ResultsThe median age of our cohort (72% women) was 63 yr (interquartile range, IQR, 45-75 yr). The median time with ITP diagnosis was 81 months (IQR, 30-192 months). The median number of therapies prior to eltrombopag was 3 (IQR, 2-4). At the time of eltrombopag start, 45 patients (30%) were receiving concomitant treatment for ITP. Forty-six patients (30%) had bleeding signs/symptoms the month before the treatment started. The median platelet count at eltrombopag initiation was 22 x 10(9)/L (IQR, 8-39 x 10(9)/L). A total of 135 patients (88.8%) achieved a platelet response. The median time to platelet response was 12 d (95% CI, 9-13 d). Maintained platelet response rate during the 15-month period under examination was 75.2%. Twenty-eight patients (18.4%) experienced adverse events, mainly grades 1-2. ConclusionEltrombopag is highly effective and well tolerated in unselected patients with primary chronic ITP.
- Published
- 2016