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

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 Some patients may sustain their platelet response when treatment is withdrawn but the frequency of this phenomenon is unknown.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.
AuthorsTomás José González-López, Cristina Pascual, María Teresa Álvarez-Román, Fernando Fernández-Fuertes, Blanca Sánchez-González, Isabel Caparrós, Isidro Jarque, María Eva Mingot-Castellano, José Angel Hernández-Rivas, Mónica Martín-Salces, Laura Solán, Paola Beneit, Reyes Jiménez, Silvia Bernat, Marcio M Andrade, Montserrat Cortés, Maria José Cortti, Susana Pérez-Crespo, Marta Gómez-Núñez, Pavel E 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, Adriana 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 Garcia-Frade, José Ramón González-Porras
JournalAmerican journal of hematology (Am J Hematol) Vol. 90 Issue 3 Pg. E40-3 (Mar 2015) ISSN: 1096-8652 [Electronic] United States
PMID25400215 (Publication Type: Journal Article, Multicenter Study)
Copyright© 2014 Wiley Periodicals, Inc.
Chemical References
  • Benzoates
  • Hematinics
  • Hydrazines
  • Pyrazoles
  • Receptors, Thrombopoietin
  • eltrombopag
Topics
  • Adult
  • Aged
  • Benzoates (administration & dosage)
  • Blood Platelets (drug effects, pathology)
  • Chronic Disease
  • Drug Administration Schedule
  • Drug Monitoring
  • Erythropoiesis (drug effects)
  • Female
  • Follow-Up Studies
  • Hematinics (administration & dosage)
  • Humans
  • Hydrazines (administration & dosage)
  • Male
  • Middle Aged
  • Platelet Count
  • Purpura, Thrombocytopenic, Idiopathic (drug therapy, pathology, surgery)
  • Pyrazoles (administration & dosage)
  • Receptors, Thrombopoietin (agonists, genetics, metabolism)
  • Recurrence
  • Remission Induction
  • Retrospective Studies
  • Splenectomy
  • Treatment Outcome

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