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

Journal: American Journal Of Hematology
Published:
Abstract

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 Some patients may sustain their platelet response when treatment is withdrawn but the frequency of this phenomenon is unknown.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. However, reliable markers for predicting which patients will have this response are needed.

Authors
Tomás González López, Cristina Pascual, María Álvarez Román, Fernando Fernández Fuertes, Blanca Sánchez González, Isabel Caparrós, Isidro Jarque, María Mingot Castellano, José Hernández Rivas, Mónica Martín Salces, Laura Solán, Paola Beneit, Reyes Jiménez, Silvia Bernat, Marcio Andrade, Montserrat Cortés, Maria Cortti, Susana Pérez Crespo, Marta Gómez Núñez, Pavel Olivera, Gloria Pérez Rus, Violeta Martínez Robles, Rafael Alonso, Angeles Fernández Rodríguez, María Arratibel, María Perera, Carmen Fernández Miñano, Miguel Fuertes Palacio, Juan Vázquez Paganini, Isabel Gutierrez Jomarrón, Inés Valcarce, Erik De Cabo, Adriana Sainz, Rosa Fisac, Carlos Aguilar, María Paz Martínez Badas, María Peñarrubia, María Calbacho, Carmen De Cos, Manuel González Silva, Erika Coria, Arancha Alonso, Alberto Casaus, Armando Luaña, Pilar Galán, Cristina Fernández Canal, Javier Garcia Frade, José González Porras