A study to assess utilisation and safety of Glycopyrronium Bromide 1mg/5ml Oral Solution as licensed for symptomatic treatment of severe sialorrhoea in children and adolescents aged 3 years and older with chronic neurological disorders in the UK

Study type

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

  Safety study (incl. comparative)
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  GLYCOPYRRONIUM BROMIDE
• Medical condition to be studied:
  Salivary hypersecretion

Population studied

• Short description of the study population:
  Any patient aged <18 years prescribed Glycopyrronium Bromide 1mg/5ml Oral Solution within six months prior to enrolment, for an indication of sialorrhea, was eligible to participate.
• Age groups:
  • Children (2 to < 12 years)
  • Adolescents (12 to < 18 years)
• Estimated number of subjects:
  100

Study design details

• Study design:
  A non-interventional prospective cohort study.
• Main study objective:
  To assess utilisation and safety of Glycopyrronium Bromide 1mg/5ml Oral Solution as licensed for symptomatic treatment of severe sialorrhoea in children and adolescents aged 3 years and older with chronic neurological disorders in the UK.
• Setting:
  Patients were recruited in secondary care sites across the UK; 14 sites participated.
• Outcomes:
  To describe utilisation of Glycopyrronium Bromide 1mg/5ml Oral Solution in the UK in patients <18 years: 1. To describe off-label use in patients aged below 3 years and/or patients with mild to moderate sialorrhoea 2. To quantify the incidence of patients with the sialorrhoea indication that have a follow up consultation for the medication/indication in secondary and/or primary care, To examine safety in long-term use (as defined by >24 weeks ) for the sialorrhoea indication. This will include: 2.1. To examine the incidence of important identified and potential risks within the first 12 months after starting treatment for the sialorrhoea indication
• Data analysis plan:
  Evaluable cohort demography will be presented using summary descriptive statistics including age and gender, as reported at index date using all available information from electronic data collection forms. Duration of treatment for all patients will be presented using summary descriptive statistics. Use for longer than 24 weeks will be quantified, and adverse events reported after 24 weeks will be summarised.
• Summary results:
  Most patients enrolled in this study were prescribed Glycopyrronium Bromide according to the licensed indication: use in patients three years or older for severe drooling. Nevertheless, offlabel use was observed in approximately one-third of the cohort. Specifically, n<5 patients were prescribed the product under the age of three years, and an additional n<5 patients had a moderate drooling severity. Whilst a high proportion of patients started Glycopyrronium Bromide according to the dosing schedule, patients were also prescribed a dose which fell ; between specified Dose levels or less than the recommended Dose level 1 for the patient’s weight. Two-thirds of patients experienced at least one adverse event listed as an important identified or potential risk. Constipation was the most frequent experienced event, followed by pneumonia. Longer term safety data (>24 weeks) were available for 12 of the 17 patients. Overall, the reported adverse events are consistent with the established safety profile of the product. However, some of these adverse events were serious, either resulting in hospital admission or serious by nature of the adverse event. Deaths were reported during the study period (n<5). The confirmed number of receipt of educational materials was low for HCPs taking part in the study. Due to challenges in patient recruitment leading to a limited sample size, it is not feasible to draw definitive conclusions regarding utilisation, safety, or the ; effectiveness of the product’s additional risk minimisation measures.