A population based cohort study to monitor the safety and effectiveness of sirolimus in patients with sporadic lymphangioleiomyomatosis (S-LAM)

Study type

• Study topic:
  Human medicinal product

  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Effectiveness study (incl. comparative)

  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:
  (L04AA10) sirolimus

  sirolimus
• Medical condition to be studied:
  Lymphangioleiomyomatosis

Population studied

• Short description of the study population:
  In this descriptive study all S-LAM patients enrolled in the UK and USA data sources who are treated or have been treated with sirolimus during the study observation period will be included.
• 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)
• Special population of interest:
  Other
• Special population of interest, other:
  Sporadic lymphangioleiomyomatosis patients
• Estimated number of subjects:
  195

Study design details

• Main study objective:
  Data within each of the UK and USA data sources will be used to: 1. Estimate the incidence proportion of adverse events among S-LAM patients treated with sirolimus. 2. Evaluate the selected effectiveness endpoints among S-LAM patients treated with sirolimus.
• Outcomes:
  incidence proportion of adverse events, and effectiveness endpoints such as change from baseline for forced expiratory volume in 1 second FEV1).
• Data analysis plan:
  All analyses will be conducted by the UK and the USA principal researchers. Patient-level data will not be provided to the MAH for analysis. There will be no hypothesis testing in this study. Data will be analysed using descriptive statistical methods only. Data will be presented separately for each country (ie, data will not be pooled).