Non-interventional study to assess the safety profile of idelalisib in patients with refractory follicular lymphoma (FL)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Safety study (incl. comparative)
• Data collection methods:
  Combined primary data collection and secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Multi-centre, observational, retrospective study

Study drug and medical condition

• Medical condition to be studied:
  Non-Hodgkin's lymphoma refractory

Population studied

• Short description of the study population:
  Patients aged 18 years or older treated with idelalisib for refractory follicular lymphoma (FL) in routine clinical practice.
• Age groups:
  • 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)
• Special population of interest:
  Other
• Special population of interest, other:
  Patients with refractory follicular lymphoma
• Estimated number of subjects:
  257

Study design details

• Main study objective:
  The objective of this study was to assess the overall safety profile of idelalisib monotherapy in patients with refractory FL. Serious Adverse Events (SAEs) were collected and according rates will be estimated. Focus was given to special health outcomes of interest (HOIs) as listed in the Zydelig Risk Management Plan (RMP) for the EU (Version 2.4).
• Outcomes:
  To assess the overall safety profile of idelalisib monotherapy in patients with refractory FL. To assess the effectiveness of idelalisib monotherapy in patients with refractory FL. The effectiveness of idelalisib was assessed by overall response rate (ORR), duration of response (DOR), progression-free survival (PFS), time to next treatment (TTNT), and overall survival (OS).
• Data analysis plan:
  Continuous variables were summarized by mean, standard deviation, median, lower quartile, upper quartile, minimum and maximum. Categorical variables were summarized by number and percentage of patients in each categorical definition including 95% confidence intervals. Multivariate Poisson regression analyses were used to estimate adjusted rates of ADRs, SADRs, and HOIs. Kaplan Meier were used to illustrate time-to-event analyses. Stratified analyses were used to account for potential behavioural changes of the site staff after the initiation of the study that might introduce bias between retrospective and prospective data collection.