Retrospective analysis of adolescent patients suffering from hereditary angioedema treated with berotralstat in Europe: patients’ and treatment characteristics

Study type

• Study topic:
  Human medicinal product
• Study topic, other:
  Hereditary angioedema
• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation

  Effectiveness study (incl. comparative)

  Evaluation of patient-reported outcomes

  Safety study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Anatomical Therapeutic Chemical (ATC) code:
  (B06AC06) berotralstat

  berotralstat
• Medical condition to be studied:
  Hereditary angioedema

Population studied

• Short description of the study population:
  Patients eligible for inclusion into the retrospective case series must meet all the following criteria:; • Confirmed diagnosis of HAE; • Aged 12–17 years old (inclusive) and weighing at least 40 kg at time of berotralstat initiation; • Ongoing treatment with berotralstat, started after April 2021 in line with the SmPC for at least 6 months with stable dosing and without treatment interruption being advised by the treating healthcare professional.; • Patient or parent/legal guardian willing and able to provide informed consent as applicable (informed consent signed)
• Age groups:
  • Adolescents (12 to < 18 years)
• Estimated number of subjects:
  30

Study design details

• Study design:
  Retrospective observational case series, consisting of analysis of data retrieved from medical files in adolescent patients (diagnosed 12 and 17 years old) with HAE who were started on treatment with berotralstat.
• Main study objective:
  The primary objective of this retrospective study is to describe the clinical profile of adolescent European HAE patients treated with berotralstat in order to characterize this population with particular attention to their baseline features and clinical context.
• Outcomes:
  Collected data related to effectiveness will include: ; • Patients’ demographic characteristics (age and sex) ; • History and description of HAE (date of diagnosis, HAE type, biology, familial or sporadic form) ; • Description of the disease prior to berotralstat initiation (attack frequency, triggers for HAE attacks, affected body location(s) , average duration of HAE attacks (hours), average severity of attacks (mild: no impact on daily life; moderate: moderate impact; severe: laryngeal attack/abdominal attack or attack with major impact on daily life), number of hospitalizations/hospital visits within last 6 months, acute treatment of attacks, school absenteeism related to HAE, prophylactic treatment of attacks if any ; • Rationale for prescribing berotralstat as orally administered LTP ; • Description of berotralstat treatment (date of initiation, dose, concomitant therapies, adherence to treatment, as rated by physician ; • Description of the disease within 6 months after berotralstat initiation (attack frequency, triggers for HAE attacks, affected body location(s) , average duration of HAE attacks (hours), average severity of attacks (mild: no impact on daily life; moderate: moderate impact; severe: laryngeal attack/abdominal attack or attack with major impact on daily life), number of hospitalizations/hospital visits, acute treatment of attacks, school ; absenteeism related to HAE ; • Physician’s global evaluation of disease evolution ; • Adverse events considered related to berotralstat from berotralstat initiation to the last follow-up visit at the time of case report completion, whether they were reported to local authorities, their timing relative to berotralstat initiation, severity, imputability, and management, collected in a specific form
• Data analysis plan:
  The data analysis of the case series and its presentation will be purely descriptive and exploratory in nature.