Targeted Literature Review to Describe Safety Outcomes in Immune Thrombocytopenia Patients Treated With Thrombopoietin-Receptor Agonists

Study type

• Study topic:
  Human medicinal product

  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Systematic review and meta-analysis

Study drug and medical condition

• Medical condition to be studied:
  Immune thrombocytopenia

Population studied

• Short description of the study population:
  Studies that include information on treated adult Immune Thrombocytopenia Patients (ITP) patients from all countries/regions will be included.
• 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 Thrombocytopenia Patients
• Estimated number of subjects:
  0

Study design details

• Main study objective:
  1. To estimate the incidence of bone marrow fibrosis/reticulin formation in adults with immune thrombocytopenia (ITP) who have been treated with romiplostim therapy, other Thrombopoietin-Receptor Agonists (TPO-RAs), and other ITP therapies.2. To estimate the incidence of hematologic malignancy in adults with ITP who have been treated with each of the therapies listed above.
• Outcomes:
  bone marrow fibrosis/reticulin formation, hematologic malignancy, thrombotic/thromboembolic events, pre-malignant stages (acute myelogenous leukemia and myelodysplastic syndromes), leukocytosis, anemia, acute renal failure
• Data analysis plan:
  A narrative synthesis of the studies included in the systematic literature review will be presented that will describe the overall strength of the collective evidence to address the research questions. If sufficient data are available for a given study outcome (e.g. incidence of bone marrow fibrosis among adult ITP patients treated with romiplostim) from the literature review, the data may be quantitatively summarized using a meta-analysis. Sufficient data is defined as at least 3 studies within each patient group (romiplostim, other TPO-RAs, or specific therapies within the other ITP therapies group) using the same outcome definition.