A Non-Interventional Registry Study of Patients with β-thalassemia to Characterise and Contextualise the Safety and Effectiveness of Betibeglogene Autotemcel

Study type

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

  Safety study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  ZYNTEGLO
• Medical condition to be studied:
  Thalassaemia beta

Population studied

• Age groups:
  Adolescents (12 to < 18 years)

  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:
  325

Study design details

• Main study objective:
  Describe the safety outcomes of patients with β-thalassaemia treated with betibeglogene autotemcel or allo-HCST in Europe
• Outcomes:
  - Describe the safety outcomes of patients with treated with betibeglogene autotemcel or allo-HCST in Europe, - Describe the effectiveness outcomes of patients with β-thalassaemia treated with betibeglogene autotemcel or allo-HCST in Europe - Describe the baseline and pretreatment characteristics of patients with β-thalassaemia treated with betibeglogene autotemcel or allo-HCST in Europe
• Data analysis plan:
  Given the study design, expected sample size, low event rates, and strong chance of unmeasured confounding, analyses will be descriptive in nature, although any known confounding will be addressed in sensitivity analyses, to the extent possible. Descriptive statistics of central tendency (medians and arithmetic or geometric means) and dispersion (standard deviation, interquartile range IQR, range) will be presented for continuous variables. For categorical variables, frequencies and percentages will be reported, where appropriate, mode, median, IQR, and range will also be reported.