An observational study of blinatumomab safety and effectiveness, utilisation, and treatment practices (20150136)

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  BLINATUMOMAB

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:
  279

Study design details

• Main study objective:
  The first primary objective is to characterize the safety profile of Blincyto in routine clinical practice in countries in the EU by characterising specific AEs. The second primary objective is to estimate the frequency and type of medication errors identified in patient charts.
• Outcomes:
  Incidence of specified AEs. Time to onset of first specified AEs. Summary of duration of specified AEs. Proportion of Blincyto administrations with medication errors. Incidence of AEs collected. Incidence of specified AEs and AEs collected among patient subgroups. Proportion of patient achieving CR, CR/CRh*/CRi, MRD within 2 cycles of Blincyto. Proportion of patients receiving allogeneic HSCT. 1 year and 100 day mortality proportion after HSCT. Relapse free survival time. Disease free survival. Overall survival. Healthcare resource use. Blinicyto Utilization.
• Data analysis plan:
  All analyses will be descriptive. Continuous variables will be summarised by mean, median, standard deviation, 25th percentile, 75th percentile, minimum and maximum. Categorical variables will be summarised by number and percentage. For categorical outcomes, 95% confidence intervals (CIs) will also be presented where appropriate. For time-to-event endpoints, Kaplan-Meier (KM) curves and estimates (median, 1st and 3rd quartile) of the time-to-event endpoint with 95% confidence intervals will be calculated, if estimable. Six, 12- and 24-month survival proportions and 95% confidence intervals will also be estimated. Subject incidence (and 95% CIs) for adverse events will be summarised and tabulated by system organ class and preferred term. Incidence tables will also be presented with respect to time on Blincyto at the patient level (incidence rate) and event level (event rate).

Documents

Study results

20150136 ORSR_Redacted.pdf