GO-First: Real-world treatment patterns and effectiveness outcomes associated with gemtuzumab ozogamicin in first-line Acute Myeloid Leukaemia

Study type

• Study topic:
  Disease /health condition

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

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medicinal product name:
  MYLOTARG
• Study drug International non-proprietary name (INN) or common name:
  GEMTUZUMAB OZOGAMICIN
• Medical condition to be studied:
  Acute myeloid leukaemia

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:
  200

Study design details

• Main study objective:
  This study aims to describe treatment patterns and outcomes of front-line AML patients treated with Mylotarg in the real-world in Austria, Belgium and Germany.
• Outcomes:
  Co-Primary objectives: - To describe the patient demographics and clinical characteristics of patients treated with GO in intermediate and favourable cytogenetic risk, de novo AML. - To describe GO treatment patterns in the study population, including the number of doses of GO and the timing of GO doses in first line (1L) treatment. To describe first-line (1L) treatment effectiveness outcomes for the study population, including: - Time-to-next treatment - Survival (event-free survival EFS, relapse-free survival RFS, overall survival OS)
• Data analysis plan:
  Frequencies and percentages will be reported for categorical variables, including the percentage of missing/unknown data, while counts, number of missing, means, medians, standard deviations (SDs), standard errors (SEs), first and third quartiles, minimum and maximum values will be reported for continuous numeric variables. Where applicable, all estimates will be described with accompanying 95% confidence intervals (CI). Time to event analyses will be conducted, with Kaplan-Meier (KM) curves and 95% CI estimated for KM curves outputted. Finally, Sankey diagrams will be generated to aid in interpretation of the analysis of treatment patterns. Data analysis will be aligned with data extracted/collected from all data sets. Where specific variables or outcomes cannot be assessed/described subgroup analyses could be conducted for a subset of patients from certain data sources.