Romiplostim effectiveness stratified on duration of immune thrombocytopenia at initiation

Study type

• Study topic:
  Human medicinal product

  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation

  Effectiveness study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Secondary analysis of data collected through a European, multi-center, observational cohort study (Amgen Study 20070225)

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  ROMIPLOSTIM
• Medical condition to be studied:
  Immune thrombocytopenia

Population studied

• Short description of the study population:
  The previously conducted cohort study (Amgen Study 20070225) enrolled 340 eligible participants. Patients were ≥ 18 years old, diagnosed with primary ITP, and had received at least one dose of romiplostim. Exclusion criteria included receipt (or planned receipt) of platelet related products (eg, recombinant human thrombopoietin [rHuTPO], thrombopoietin receptor agonists, pegylated recombinant human megakaryocyte growth and development factor [PEG-rHuMGDF]), participation in any interventional clinical study, or initiation of romiplostim prior to commercial availability of the product
• Age groups:
  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Special population of interest:
  Other
• Special population of interest, other:
  Immune thrombocytopenic purpura patients
• Estimated number of subjects:
  340

Study design details

• Main study objective:
  Among patients who received romiplostim in routine care in one of seven countries (Austria, Belgium, Czech Republic, France, Greece, Portugal and Sweden), describe the following overall and within strata of duration of ITP: patient profile at romiplostim initiation, patterns of romiplostim use and overall platelet count trends, effectiveness of romiplostim via platelet-based and other endpoints.
• Outcomes:
  Durable platelet response, median overall platelet count, overall platelet response, time to first platelet response, Discontinuation of concurrent medications, bleeding, splenectomy, adverse drug reaction, thrombotic events, bone marrow fibrosis
• Data analysis plan:
  Descriptive statistics of the clinical and demographic characteristics of the cohort will be provided, as will patterns of romiplostim use and platelet count trends. Estimates of medians, proportions, probabilities, and rates will be calculated for the endpoints of interest and presented with appropriate measures of variability. No hypotheses will be tested.