title: Eltrombopag and high-dose dexamethasone as frontline treatment of newly diagnosed immune thrombocytopenia in adults.
abstract: Immune thrombocytopenia (ITP) results from platelet destruction and production suppression. Eltrombopag belongs to a new class of thrombopoietin-mimetic drugs that raise platelet counts in ITP patients. We performed a single-arm study to assess the response to a single course of dexamethasone (40 mg by mouth, days 1-4) in combination with eltrombopag (50 mg, days 5-32) in 12 adults with newly diagnosed ITP in an outpatient setting. Median follow-up was 12.5 months. After therapy (day 33), 100% of patients achieved at least ≥30 × 10(9)/L platelets. Four patients relapsed. Complete response at 6 months (platelets ≥100 × 10(9)/L) was achieved in 50% of patients and response at 6 months (platelets ≥30 <100 × 10(9)/L) was achieved in another 25%; relapse-free survival was 66.7% at 12 months (median response duration of 8.3 months). In conclusion, eltrombopag/dexamethasone is a feasible frontline therapy for ITP. This trial is registered at www.clinicaltrials.gov as NCT01652599.
authors.affiliation: Hematology Service, Hospital Universitario "Dr José Eleuterio González," Universidad Autónoma de Nuevo León, Monterrey, México;
authors.forename: David
authors.identifier: http://orcid.org/0000-0002-0460-6427
authors.initials: D
authors.lastname: Gómez-Almaguer
authors.affiliation: Hematology Service, Hospital Universitario "Dr José Eleuterio González," Universidad Autónoma de Nuevo León, Monterrey, México;
authors.forename: Miguel A
authors.identifier: http://orcid.org/0000-0001-8751-7050
authors.initials: MA
authors.lastname: Herrera-Rojas
authors.affiliation: Hematology Service, Hospital Universitario "Dr José Eleuterio González," Universidad Autónoma de Nuevo León, Monterrey, México;
authors.forename: José C
authors.initials: JC
authors.lastname: Jaime-Pérez
authors.affiliation: Hematology Service, Hospital Universitario "Dr José Eleuterio González," Universidad Autónoma de Nuevo León, Monterrey, México;
authors.forename: Andrés
authors.identifier: http://orcid.org/0000-0001-6904-9345
authors.initials: A
authors.lastname: Gómez-De León
authors.affiliation: Hematology Service, Hospital Universitario "Dr José Eleuterio González," Universidad Autónoma de Nuevo León, Monterrey, México;
authors.forename: Olga G
authors.initials: OG
authors.lastname: Cantú-Rodríguez
authors.affiliation: Hematology Service, Hospital Universitario "Dr José Eleuterio González," Universidad Autónoma de Nuevo León, Monterrey, México;
authors.forename: César H
authors.initials: CH
authors.lastname: Gutiérrez-Aguirre
authors.affiliation: Hematology Service, Hospital Universitario "Dr José Eleuterio González," Universidad Autónoma de Nuevo León, Monterrey, México;
authors.forename: Luz
authors.initials: L
authors.lastname: Tarín-Arzaga
authors.affiliation: Universidad del Valle de México, Villahermosa, México; and Centro de Hematología y Medicina Interna de Puebla, Clínica Ruiz, Universidad de las Américas Puebla, Universidad Popular Autónoma del Estado de Puebla, Puebla, México.
authors.forename: Jesús
authors.initials: J
authors.lastname: Hernández-Reyes
authors.affiliation: Centro de Hematología y Medicina Interna de Puebla, Clínica Ruiz, Universidad de las Américas Puebla, Universidad Popular Autónoma del Estado de Puebla, Puebla, México.
authors.forename: Guillermo J
authors.initials: GJ
authors.lastname: Ruiz-Arguelles
mesh_terms: D000328:Adult; D000368:Aged; D000893:Anti-Inflammatory Agents / Q000008:administration & dosage / Q000627:therapeutic use; D001565:Benzoates / Q000008:administration & dosage / Q000627:therapeutic use*; D003907:Dexamethasone / Q000008:administration & dosage / Q000627:therapeutic use*; D018572:Disease-Free Survival; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D004359:Drug Therapy, Combination; D005240:Feasibility Studies; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D006834:Hydrazines / Q000008:administration & dosage / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D010976:Platelet Count; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy*; D011720:Pyrazoles / Q000008:administration & dosage / Q000627:therapeutic use*; D053628:Receptors, Thrombopoietin / Q000819:agonists; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D000893:Anti-Inflammatory Agents; D001565:Benzoates; D006834:Hydrazines; D011720:Pyrazoles; D053628:Receptors, Thrombopoietin; D003907:Dexamethasone; C520809:eltrombopag