Overall survival and incidence of adverse events in B-cell acute lymphoblastic leukemia (ALL) patients after allogeneic stem cell transplant: blinatumomab vs non-blinatumomab chemotherapy-- an analysis of the Center for International Blood and Marrow Transplant Research database (20170610) (Transplant outcomes among ALL in CIBMTR)

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

  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine, other:
  blyncito
• Study drug International non-proprietary name (INN) or common name:
  BLINATUMOMAB
• Anatomical Therapeutic Chemical (ATC) code:
  (L01FX07) blinatumomab

  blinatumomab
• Medical condition to be studied:
  B-cell type acute leukaemia

Population studied

• Age groups:
  Preterm newborn infants (0 – 27 days)

  Term newborn infants (0 – 27 days)

  Infants and toddlers (28 days – 23 months)

  Children (2 to < 12 years)

  Adolescents (12 to < 18 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)
• Special population of interest:
  Hepatic impaired

  Immunocompromised

  Pregnant women

  Renal impaired
• Estimated number of subjects:
  1000

Study design details

• Main study objective:
  To assess outcomes of blinatumomab and non-blinatumomab regimens as transplant enabling therapy in ALL patients.
• Outcomes:
  1. Estimate 100-day mortality; 2. Estimate the incidence of graft versus host disease (GVHD) (acute and chronic), Estimate 3-year overall survival (OS)Leukemia-free survival (LFS)Incidence of disease relapse Incidence of transplant-related mortality (TRM)Incidence of veno-occlusive disease/sinusoidal obstructive syndrome Incidence of new malignancies Incidence of GVHD by severity (acute and chronic)Incidence of early (<100 days) infections Incidence of persistent post-transplant B-cell depletion
• Data analysis plan:
  Descriptive summary of patient characteristics for all groups will be generated from all covariates specified in covariates of interest section. To estimate the incidence and risk of the outcomes of interest, the incidence proportion will be described for all outcomes. ; Time-to-event analyses will be described using KM method for OS and LFS, cumulative incidence method for competing risk outcome events for median and probabilities at fixed time points. ; Estimates will be generated for all groups. ; If at least 219 N-BL SOC patients are identified in the study, propensity scores will be estimated for the propensity of treatment with blinatumomab based on covariates listed. Adjustment method for baseline covariates between r/r BL only and r/r N-BL SOC groups will be evaluated using inverse probability of treatment weighting and the average treatment effect method. ; Weighted comparisons between groups will be made by logistic or Cox regression models estimating odds ratio and hazard ratio.