A Retrospective, Non-interventional, Multi-centre Study of TEGSEDI-treated Patients to Evaluate Real-world Adherence to, and Effectiveness of the Recommendations for Platelet Monitoring, Dose Adjustment, and Steroid Initiation to Manage Risk of Thrombocytopenia (TEG4002)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Retrospective chart review study

Study drug and medical condition

• Name of medicine:
  TEGSEDI
• Name of medicine, other:
  TEGSEDI
• Anatomical Therapeutic Chemical (ATC) code:
  (N07XX15) inotersen

  inotersen
• Medical condition to be studied:
  Thrombocytopenia
• Additional medical condition(s):
  Hereditary transthyretin amyloidosis (hATTR)

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:
  100

Study design details

• Main study objective:
  To assess the real-world adherence to, and effectiveness of the proposed schedule for platelet monitoring, TEGSEDI dose adjustment, and initiation of steroids for platelet recovery in accordance with the schedule outlined in the SmPC.
• Outcomes:
  Primary outcome measure:; 1. Evaluation of real-world adherence to, and effectiveness of the recommended schedule for platelet monitoring, dose adjustment, and initiation of steroids for platelet recovery. ; ; Secondary Outcome Measures:; 2. Evaluation of real-world thrombocytopenia-related outcomes (e.g. incidence rates of thrombocytopenia, serious and non-serious bleeding events associated with thrombocytopenia) in patients who receive TEGSEDI based on level(s) of adherence with recommendations for platelet monitoring and TEGSEDI dose adjustment per the SmPC. ; 3. Evaluation of potential predictors of adherence with recommendations for platelet monitoring, TEGSEDI dose adjustment, and initiation of steroids for platelet recovery per the SmPC in patients who receive TEGSEDI.
• Data analysis plan:
  The primary analysis will include descriptive statistics to tabulate compliance with platelet monitoring and dose adjustments. Additionally, adherence will be summarized as a continuous measure as a % and will also be summarized as a dichotomous measure where adherence is defined as ≥90% compliance with the platelet monitoring and dose adjustment schedule and non-adherence defined as <90% compliance.