Post-Authorization Long Term Safety Surveillance Study of Fostamatinib in Adult Patients with Chronic Immune Thrombocytopenia (cITP) who are Refractory to Previous Treatments

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Safety study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Prospective, non-interventional, active surveillance study

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  FOSTAMATINIB
• Medical condition to be studied:
  Immune thrombocytopenia
• Additional medical condition(s):
  Chronic Immune Thrombocytopenia (cITP)

Population studied

• 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)
• Estimated number of subjects:
  67

Study design details

• Main study objective:
  The primary objective of this PASS is to document the long-term safety and tolerability of fostamatinib in adult patients with cITP who are refractory to other treatments when used according to the current approved prescribing information per the European Summary of Product Characteristics (SmPC) in a real-world setting.
• Outcomes:
  All adverse event (AE), serious AE (SAE), suspected adverse drug reaction (ADR), complete blood count, and liver function test data will be collected, and the incidence of AEs of special interest, namely hypertension, hepatotoxicity, neutropenia and infections (including serious and opportunistic infections), osteoporosis, bone fractures and fracture healing, and diarrhoea will be calculated.
• Data analysis plan:
  Detailed methodology for summary and statistical analyses of data collected in this study will be documented in a statistical analysis plan (SAP), which will be dated, filed, and maintained by the sponsor. The SAP may modify the plans outlined in the protocol, any major modifications of primary endpoint definitions or their analyses would be reflected in a protocol amendment. For the purpose of this study, the safety analysis population will consist of all patients treated with at least 1 dose of fostamatinib. Analyses will generally be descriptive in nature and will be conducted using SAS statistical software (version 9.3 or higher). All variables will be summarized descriptively through tabular displays of number of non-missing observations, mean, median, ranges and standard deviations of continuous variables, and frequency and percentages of categorical variables. No formal hypothesis testing or comparisons is planned.