title: Eltrombopag safety and efficacy for primary chronic immune thrombocytopenia in clinical practice.
abstract: BACKGROUND
  Eltrombopag 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.
  
  
  METHODS
  A total of 164 primary patients with chronic ITP from 40 Spanish centers, who had been treated with eltrombopag, were retrospectively evaluated.
  
  
  RESULTS
  The 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 × 10(9) /L (IQR, 8-39 × 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.
  
  
  CONCLUSIONS
  Eltrombopag is highly effective and well tolerated in unselected patients with primary chronic ITP.
authors.affiliation: Department of Hematology, Hospital Universitario de Burgos, Burgos, Spain.
authors.forename: Tomás José
authors.initials: TJ
authors.lastname: González-López
authors.affiliation: Department of Hematology, Hospital Universitario La Paz, Madrid, Spain.
authors.forename: María Teresa
authors.initials: MT
authors.lastname: Alvarez-Román
authors.affiliation: Department of Hematology, Hospital General Universitario Gregorio Marañón, Madrid, Spain.
authors.forename: Cristina
authors.initials: C
authors.lastname: Pascual
authors.affiliation: Department of Hematology, Hospital del Mar, Barcelona, Spain.
authors.forename: Blanca
authors.initials: B
authors.lastname: Sánchez-González
authors.affiliation: Department of Hematology, Hospital Universitario Insular de Gran Canaria, Las Palmas de Gran Canaria, Spain.
authors.forename: Fernando
authors.initials: F
authors.lastname: Fernández-Fuentes
authors.affiliation: Department of Hematology, Hospital Universitario La Fé, Valencia, Spain.
authors.forename: Isidro
authors.initials: I
authors.lastname: Jarque
authors.affiliation: Department of Hematology, Hospital General Universitario Gregorio Marañón, Madrid, Spain.
authors.forename: Gloria
authors.initials: G
authors.lastname: Pérez-Rus
authors.affiliation: Department of Hematology, Hospital de Santiago de Compostela, Santiago de Compostela (La Coruña), Spain.
authors.forename: Susana
authors.initials: S
authors.lastname: Pérez-Crespo
authors.affiliation: Department of Hematology, Hospital de La Plana, Castellón, Spain.
authors.forename: Silvia
authors.initials: S
authors.lastname: Bernat
authors.affiliation: Department of Hematology, Hospital Infanta Leonor, Madrid, Spain.
authors.forename: José Angel
authors.initials: JA
authors.lastname: Hernández-Rivas
authors.affiliation: Department of Hematology, Hospital Universitario Miguel Servet, Zaragoza, Spain.
authors.forename: Marcio M
authors.initials: MM
authors.lastname: Andrade
authors.affiliation: Department of Hematology, Fundació Hospital Asil de Granollers, Granollers (Barcelona), Spain.
authors.forename: Montserrat
authors.initials: M
authors.lastname: Cortés
authors.affiliation: Department of Hematology, Hospital Parc Taulí, Sabadell (Barcelona), Spain.
authors.forename: Marta
authors.initials: M
authors.lastname: Gómez-Nuñez
authors.affiliation: Department of Hematology, Hospital Universitario Vall de Hebron, Barcelona, Spain.
authors.forename: Pavel
authors.initials: P
authors.lastname: Olivera
authors.affiliation: Department of Hematology, Hospital de León, León, Spain.
authors.forename: Violeta
authors.initials: V
authors.lastname: Martínez-Robles
authors.affiliation: Department of Hematology, Hospital Universitario Central de Asturias, Oviedo (Asturias), Spain.
authors.forename: Angeles
authors.initials: A
authors.lastname: Fernández-Rodríguez
authors.affiliation: Department of Hematology, Hospital Clínico Lozano Blesa, Zaragoza, Spain.
authors.forename: Miguel Angel
authors.initials: MA
authors.lastname: Fuertes-Palacio
authors.affiliation: Department of Hematology, Hospital Vega Baja, Orihuela (Alicante), Spain.
authors.forename: Carmen
authors.initials: C
authors.lastname: Fernández-Miñano
authors.affiliation: Department of Hematology, Hospital del Bierzo, Ponferrada (León), Spain.
authors.forename: Erik
authors.initials: E
authors.lastname: de Cabo
authors.affiliation: Department of Hematology, Hospital de Segovia, Segovia, Spain.
authors.forename: Rosa
authors.initials: R
authors.lastname: Fisac
authors.affiliation: Department of Hematology, Hospital de Soria, Soria, Spain.
authors.forename: Carlos
authors.initials: C
authors.lastname: Aguilar
authors.affiliation: Department of Hematology, Hospital de Avila, Avila, Spain.
authors.forename: Abelardo
authors.initials: A
authors.lastname: Bárez
authors.affiliation: Department of Hematology, Hospital Clínico de Valladolid, Valladolid, Spain.
authors.forename: María Jesús
authors.initials: MJ
authors.lastname: Peñarrubia
authors.affiliation: Department of Hematology, Hospital Universitario Río Hortega, Valladolid, Spain.
authors.forename: Luis Javier
authors.initials: LJ
authors.lastname: García-Frade
authors.affiliation: Department of Hematology, IBSAL-Hospital Universitario de Salamanca, Salamanca, Spain.
authors.forename: José Ramón
authors.initials: JR
authors.lastname: González-Porras
mesh_terms: D000368:Aged; D001565:Benzoates / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D002908:Chronic Disease; D005260:Female; D006801:Humans; D006834:Hydrazines / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D007166:Immunosuppressive Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D008297:Male; D008875:Middle Aged; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000175:diagnosis / Q000188:drug therapy* / Q000276:immunology; D011720:Pyrazoles / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D019233:Retreatment; D012189:Retrospective Studies; D013030:Spain; D016896:Treatment Outcome
chemical_list: D001565:Benzoates; D006834:Hydrazines; D007166:Immunosuppressive Agents; D011720:Pyrazoles; C520809:eltrombopag
keywords: ITP; Immune; TPO; thrombocytopenia; thrombopoietin