title: A large observational study of patients with primary immune thrombocytopenia receiving romiplostim in European clinical practice.
abstract: OBJECTIVE
  Romiplostim has maintained long-term platelet counts in patients with immune thrombocytopenia (ITP) for up to 5 yr in clinical studies. This prospective observational study aimed to describe romiplostim utilisation and outcomes in European clinical practice.
  
  
  METHODS
  Adults with primary ITP who received romiplostim in routine care were eligible.
  
  
  RESULTS
  Three-hundred and forty patients were eligible for analysis, of whom 299 (88%) completed the 2-yr observation period. The median age was 62 yr, with 43% of patients aged ≥65 yr, and two-thirds of patients initiated romiplostim before splenectomy. The median average weekly dose of romiplostim was 2.8 μg/kg. The median baseline platelet count was 20 × 109/L, which increased after 2 wk of romiplostim treatment and remained >50 × 109/L thereafter. After romiplostim initiation, there was a decrease in rates of grade ≥3 bleeding events (from 12 to 2 per 100 patient-years) and ITP-related hospitalisations (from 87 to 33 per 100 patient-years). The rate of thrombotic events was 2 per 100 patient-years, and bone marrow fibrosis occurred in two patients.
  
  
  CONCLUSIONS
  Romiplostim dosing, effectiveness and safety in an unselected real-world ITP population seemed comparable with that observed in clinical studies.
authors.affiliation: Division of Haematology and Oncology, Innsbruck Medical University, Innsbruck, Austria.
authors.forename: Michael
authors.initials: M
authors.lastname: Steurer
authors.affiliation: Haematology Department, Hôpital Saint Eloi, Montpellier, France.
authors.forename: Philippe
authors.initials: P
authors.lastname: Quittet
authors.affiliation: Department of Haematology, University of Crete School of Medicine, University Hospital of Heraklion, Crete, Greece.
authors.forename: Helen A
authors.initials: HA
authors.lastname: Papadaki
authors.affiliation: Department of Haematology, A-Z Sint-Jan, Bruges-Oostende, Belgium.
authors.forename: Dominik
authors.initials: D
authors.lastname: Selleslag
authors.affiliation: Hôpital du Haut Lévèque, University of Bordeaux, Pessac, France.
authors.forename: Jean-François
authors.initials: JF
authors.lastname: Viallard
authors.affiliation: AHEPA University General Hospital, Medical Propedeutic Department of Internal Medicine, Aristotle University of Thessaloniki, Thessaloniki, Greece.
authors.forename: Georgia
authors.initials: G
authors.lastname: Kaiafa
authors.affiliation: Department of Haematology, University Hospitals Leuven, Leuven, Belgium.
authors.forename: Ann
authors.initials: A
authors.lastname: Janssens
authors.affiliation: 3rd Medical Faculty, Charles University, Prague, Czech Republic.
authors.forename: Tomas
authors.initials: T
authors.lastname: Kozak
authors.affiliation: Section of Haematology, Sahlgrenska University Hospital, Gothenburg, Sweden.
authors.forename: Hans
authors.initials: H
authors.lastname: Wadenvik
authors.affiliation: Center for Observational Research, Uxbridge, UK.
authors.forename: Marieke
authors.initials: M
authors.lastname: Schoonen
authors.affiliation: Biostatistics, LB Biostatistics, London, UK.
authors.forename: Laura
authors.initials: L
authors.lastname: Belton
authors.affiliation: International Medical Development and Research, Amgen (Europe) GmbH, Zug, Switzerland.
authors.forename: Georg
authors.initials: G
authors.lastname: Kreuzbauer
mesh_terms: D000328:Adult; D000368:Aged; D003131:Combined Modality Therapy; D005060:Europe; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D010976:Platelet Count; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000175:diagnosis / Q000188:drug therapy* / Q000601:surgery; D011961:Receptors, Fc / Q000008:administration & dosage / Q000627:therapeutic use*; D011993:Recombinant Fusion Proteins / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D013156:Splenectomy; D013926:Thrombopoietin / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D011961:Receptors, Fc; D011993:Recombinant Fusion Proteins; D013926:Thrombopoietin; C488777:romiplostim
keywords: observational research; platelet count; platelet disorders; primary immune thrombocytopenia; romiplostim; splenectomy