Successful discontinuation of eltrombopag after complete remission in patients with primary immune thrombocytopenia

作者：Tomás José González‐López, Cristina Pascual, María Román, Fernando Fernández‐Fuertes, Blanca Sánchez‐González, Isabel Caparrós, Isidro Jarque, María Eva Mingot‐Castellano, José‐Ángel Hernández‐Rivas, Mónica Martín‐Salces, Laura Solán, Paola Beneit, Reyes Jiménez, Silvia Bernat, Marcio Andrade, Montserrat Cortés, María José Cortti, Susana Pérez‐Crespo, Marta Gómez‐Núñez, Pável Olivera, Gloria Pérez‐Rus, Violeta Martínez‐Robles, Rafael Alonso, Angeles Fernández‐Rodríguez, María Carmen Arratibel, María Perera, Carmen Fernández‐Miñano, Miguel Angel Fuertes‐Palacio, Juan Andrés Vázquez‐Paganini, Isabel Gutierrez‐Jomarrón, Inés Valcarce, Erik de Cabo, A Vera Sainz, Rosa Fisac, Carlos Aguilar, María Paz Martínez‐Badas, María Jesús Peñarrubia, María Calbacho, Carmen de Cos, Manuel González‐Silva, Erika Coria, Arancha Alonso, Alberto Casaus, Armando Luaña, Pilar Galán, Cristina Fernández‐Canal, Javier García‐Frade, José Ramón González‐Porras · 发表于：American Journal of Hematology · 年份：2014 · DOI：10.1002/ajh.23900 · 被引用次数：149 · 研究领域：Platelet Disorders and Treatments、Blood groups and transfusion、Blood properties and coagulation

Eltrombopag is effective and safe in immune thrombocytopenia (ITP). Some patients may sustain their platelet response when treatment is withdrawn but the frequency of this phenomenon is unknown. We retrospectively evaluated 260 adult primary ITP patients (165 women and 95 men; median age, 62 years) treated with eltrombopag after a median time from diagnosis of 24 months. Among the 201 patients who achieved a complete remission (platelet count >100 × 10(9) /l), eltrombopag was discontinued in 80 patients. Reasons for eltrombopag discontinuation were: persistent response despite a reduction in dose over time (n = 33), platelet count >400 × 10(9) /l (n = 29), patient's request (n = 5), elevated aspartate aminotransferase (n = 3), diarrhea (n = 3), thrombosis (n = 3), and other reasons (n = 4). Of the 49 evaluable patients, 26 patients showed sustained response after discontinuing eltrombopag without additional ITP therapy, with a median follow-up of 9 (range, 6-25) months. These patients were characterized by a median time since ITP diagnosis of 46.5 months, with 4/26 having ITP < 1 year. Eleven patients were male and their median age was 59 years. They received a median of 4 previous treatment lines and 42% were splenectomized. No predictive factors of sustained response after eltrombopag withdrawal were identified. Platelet response following eltrombopag cessation may be sustained in an important percentage of adult primary ITP patients who achieved CR with eltrombopag. Howeve...