Retrospective Cohort Study Evaluating Clinical Outcomes in Patients with Granulomatosis with Polyangiitis (GPA)/Microscopic Polyangiitis (MPA) Treated with Avacopan (20230026)

Study type

• Study topic:
  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

• Name of medicine:
  TAVNEOS
• Study drug International non-proprietary name (INN) or common name:
  AVACOPAN
• Anatomical Therapeutic Chemical (ATC) code:
  (L04AJ05) avacopan

  avacopan
• Medical condition to be studied:
  Granulomatosis with polyangiitis

  Microscopic polyangiitis

Population studied

• Age groups:
  Adults (18 to < 65 years)

  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:
  305

Study design details

• Main study objective:
  To describe characteristics associated with avacopan prescription and initiation, and outcomes observed among avacopan initiators. Among avacopan initiators: (1) investigate factors associated with glucocorticoid discontinuation and relapse risk; and (2) evaluate healthcare resource utilization.
• Outcomes:
  Primary Outcomes:; • Number or participants achieving discontinuation of glucocorticoids for treatment of GPA or MPA 1 month before Month 6 and disease remission within 6 months of avacopan initiation.; • Number of participants achieving sustained remission at Month 12, defined as remission at Month 6 and remission at Month 12, with no glucocorticoid treatment for GPA/MPA 1 month before Month 12, and no relapse between Months 6 and 12.
• Data analysis plan:
  There are three primary analyses. Analysis 1 will describe the baseline characteristics of participants by avacopan prescription status (yes/no), as well as the baseline characteristics of participants prescribed avacopan by initiation status (yes/no). Analyses 2 and 3 will examine the 6- and 12-month outcomes among initiators of avacopan, respectively. Analysis 1 will be performed at least six months after the study start date when the final cohort of avacopan initiators is identified and baseline data are collected. Analyses 2 and 3 will begin once at least 6 months after the last avacopan participant is identified during the inclusion period and at least 6 months of follow-up data are collected. The primary outcomes will be analysed using a descriptive approach. Remission will be based on the Birmingham Vasculitis Activity Score version 3 with/out glucocorticoids for the treatment of GPA/MPA in the 4 weeks prior to the month 6 or 12 visits. Summary statistics for continuous variables, including the number of patients, mean, standard deviation (SD), median, Q1, Q3, minimum and maximum, will be calculated after excluding missing/unknown values.