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.