French Observational study on patients’ Characteristics, Utilization and Survival outcomes in Gilteritinib-treated patients (FOCUS)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology

  Drug utilisation

  Healthcare resource utilisation
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  XOSPATA 40 MG - FILM-COATED TABLET
• Study drug International non-proprietary name (INN) or common name:
  GILTERITINIB
• Anatomical Therapeutic Chemical (ATC) code:
  (L01EX13) gilteritinib

  gilteritinib
• Medical condition to be studied:
  Acute myeloid leukaemia

Population studied

• Short description of the study population:
  This study will use information from the French national health database and health insurance records in France to learn more about people with AML who use gilteritinib. This study will check what happened to people who received specialist care between 2019 and 2023.
• Age groups:
  All

  In utero

  Paediatric Population (< 18 years)

  Neonate

  Preterm newborn infants (0 – 27 days)

  Term newborn infants (0 – 27 days)

  Infants and toddlers (28 days – 23 months)

  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)

  Adult and elderly population (≥18 years)

  Adults (18 to < 65 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Elderly (≥ 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  15000

Study design details

• Study design:
  This is a longitudinal cohort study of patients with an Acute Myeloid Leukemia (AML), using the Magellan database. Magellan is a copy of the French national Health insurance claims data from Système National des Données de Santé (SNDS).
• Main study objective:
  The main aims of this study are to understand how people with AML are managed in France. This includes how often new cases of this AML occur, how many take gilteritinib and how long before they started taking it.
• Outcomes:
  Population 1 – patients with AML diagnosis over the study period; • Incidence of AML patient; • Prevalence of AML patients; • Time-to gilteritinib initiation ; Population 2 – patients with AML diagnosis and treated with; gilteritinib over the study period; • Incidence of AML patient; • Prevalence of AML patients; Population 3 – patients with AML diagnosis, treated with gilteritinib; and with HSCT over the study period ; • Overall survival from HSCT
• Data analysis plan:
  Incidence and Prevalence: Count of the annual number of patients with AML receiving secondary care each year. Count of the monthly number of patients treated with gilteritinib.; Overall Survival from HSCT: Population-based summary: Kaplan-Meier curve, censoring patients at end of study. ;