Long-term, Non-interventional Study of Recipients of Yescarta® for Treatment of Relapsed or Refractory Diffuse Large B-Cell Lymphoma, Primary Mediastinal Large B-Cell Lymphoma, and Follicular Lymphoma

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Safety study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Secondary use of EBMT (European Society for Blood and Marrow Transplantation) and CIBMTR (Center for International Blood and Marrow Transplant Research) data)

Study drug and medical condition

• Medicinal product name:
  YESCARTA
• Study drug International non-proprietary name (INN) or common name:
  AXICABTAGENE CILOLEUCEL
• Medical condition to be studied:
  Diffuse large B-cell lymphoma

  Primary mediastinal large B-cell lymphoma

  Follicular lymphoma

Population studied

• 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:
  2550

Study design details

• Main study objective:
  The primary objective is to evaluate the incidence rate and severity of adverse drug reactions (ADRs) in patients treated with YESCARTA (pooled and by indication), including secondary malignancies, cytokine release syndrome (CRS), neurologic events, serious infections, prolonged cytopenias, hypogammaglobulinemia, and pregnancy outcomes in female patients of childbearing potential or partners of male patients.; ;
• Outcomes:
  Incidence rates of secondary malignancies, CRS, neurologic events, prolonged cytopenias, serious infections, hypogammaglobulinemia and pregnancies, and as applicable severity, time to onset, type and location, management and resolution of these events. Use of replacement immunoglobulin therapy and pregnancy outcome among females with childbearing potential. Overall survival, time to next treatment, time to relapse or progression of the primary disease, primary and secondary endpoints on subgroups by gender, age, and in special populations. Incidence rates, resolution and time to onset of TLS (also severity) and aggravation of GvHD (chronic and acute, for acute also severity and relationship to cell therapy), frequency of detection of RCR.
• Data analysis plan:
  Analysis of endpoints for this study will include all eligible Yescarta patients within the EBMT Registry and CIBMTR Registry. The following will be summarized descriptively: Categorical variables by number and percentage of patients, including 95% confidence intervals, Continuous variables by mean, standard deviation, median, lower quartile, upper quartile, minimum and maximum. The following analyses will be provided: patient incidence of endpoint events, Multivariate Poisson regression analyses for cumulative incidence rates adjusted for follow-up period, specified subgroups and other potential confounders (demographics and baseline characteristics), Kaplan-Meier curves for all time-to-event data, Competing risk analysis method for time to onset and duration of endpoint events, time to relapse or progression, time to next treatment, and cumulative incidence at specified time points, Cox-proportional Hazard models for multivariate time-to-event data adjusted for subgroups and other potential confounders.