Non-interventional retrospective study evaluating the real-life idelalisib use in the population enrolled in the French cohort Temporary Authorization for Use (ATUc) in Chronic Lymphocytic Leukemia (CLL) and indolent Non-Hodgkin Lymphoma (iNHL) (REALIST)

Study type

• Study topic:
  Disease /health condition

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

• Name of medicine:
  ZYDELIG
• Medical condition to be studied:
  Chronic lymphocytic leukaemia

  Non-Hodgkin's lymphoma unspecified histology indolent stage IV

Population studied

• Short description of the study population:
  Adult patients who have been enrolled in the ATUc for either CLL or iNHL according to the ATUc indications and who initiated treatment with idelalisib alone, or in combination with rituximab.
• 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)
• Estimated number of subjects:
  110

Study design details

• Main study objective:
  The primary objective of this study is to describe the effectiveness of idelalisib ± rituximab 6 months after the idelalisib initiation following the enrollment of patients in the ATUc program Overall Response Rate (ORR)
• Outcomes:
  This non-interventional study is designed to generate long-term (up to 12 months) real-life effectiveness and safety data in the patients enrolled in this ATUc. From the initiation of idelalisib treatment, the retrospective duration of follow-up will be up to 12 months for each patient which will allow collecting information about therapeutic strategies prescribed after idelalisib discontinuation, -Effectiveness outcome,-Outcome of adverse drug reactions (ADRs),serious adverse drug reactions (SADRs), fatal adverse events (fatal AEs),-Health Economics and Outcomes Research (HEOR) data including iNHL and CLL resource utilization and costs in clinical practice and treatment options
• Data analysis plan:
  The statistical analysis will be essentially descriptive, using SAS in the version 9.4 or higher. The risk is fixed to 5% in two-sided situation for all the analyses. A statistical analysis plan will be finalized and approved before the database lock.Summary statistics will be presented per patients’ group (CLL, iNHL) and include:• Nominal variables: number of missing values, frequencies and percentages.• Ordinal variables: number of missing values, frequencies, percentages, median, minimum and maximum.• Continuous variables: number (N) of observations, number of missing values, mean, standard deviation, 25th percentile, median, 75th percentile, minimum and maximumWhen applicable, p-values will be presented as two-sided p-values and the level of significance is set to 5% (two-sided). Additionally, corresponding 95%-CI will be provided, when applicable.Methods for comparative analysis will be used, as appropriate.

Documents

Study results

GS-FR-312-2033-csr-body_f-redact