Venetoclax (ABT-199) in Patients with Chronic Lymphocytic Leukemia: Experience through the Pre-Approval Access Program in European Countries: A Medical ChartReview (ExPloReR)

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:
  Other
• Non-interventional study design, other:
  Retrospective Chart Review

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  VENETOCLAX
• Medical condition to be studied:
  Chronic lymphocytic leukaemia

Population studied

• Short description of the study population:
  The target population will be adults who participated in the Pre-Approval Access (PAA) cohort program for Venetoclax for the treatment of Chronic Lymphocytic Leukemia (CLL) in France, Germany, Netherland, Sweden and United Kingdom and received atleast one dose of the treatment.
• Age groups:
  • 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:
  Chronic lymphocytic leukemia patients
• Estimated number of subjects:
  47

Study design details

• Main study objective:
  The main objective of the study was to collect real-world evidence on the effectiveness, safety and tolerability of venetoclax treatment for CLL among patients who received venetoclax as part of PAA cohort programs in Europe
• Outcomes:
  The effectiveness of venetoclax, measured as overall response rate (ORR) according to physician assessment at week 36, ORR and complete response rate (CRR) according to physician assessment at week 52CRR according to physician assessment at week 36Overall survival at week 52Time to progression at week 52Progression-free survival at week 52Proportion of patients starting treatment in the PAA cohorts and remaining on treatment at week 36Adverse events experienced during ramp-up and throughout treatment
• Data analysis plan:
  Data were summarized using descriptive statistics, and presented as means, standard deviations (SDs), medians, and ranges for continuous variables, and numbers and percentages for categorical variables. Overall survival was summarized using Kaplan-Meier curves. Survival was measured from the time of venetoclax initiation. All summarized data are presented in aggregate, no stratifications were undertaken due to the small sample size.