Expanded access of Blincyto® in patients with acute lymphoblastic leukaemia: a retrospective observational study (Neuf Study) (20160441)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation

  Effectiveness study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Retrospective, observational, multi-centre study

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  BLINATUMOMAB
• Medical condition to be studied:
  B precursor type acute leukaemia

Population studied

• Short description of the study population:
  The study population involved patients with B-precursor acute lymphoblastic leukemia (ALL) treated with Blincyto reported from five countries: France, Italy, Spain, UK and Russia identified from the 1st January 2014 up until 30th June 2017. ; Inclusion criteria: ; - B-precursor ALL patients who have initiated Blincyto in an expanded access setting from 1st January 2014 up until 30th June 2017.; ; Exclusion:; - Patients enrolled in Amgen expanded access protocol 20130320.; - Patients who do not provide informed consent, where required per country regulations.; - Patient’s medical chart is not available for data extraction.
• Age groups:
  Paediatric Population (< 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:
  Other
• Special population of interest, other:
  Acute lymphoblastic leukemia patients
• Estimated number of subjects:
  350

Study design details

• Main study objective:
  The primary objective is to describe the clinical characteristics and treatment patterns of patients with B precursor acute lymphoblastic leukemia, having received Blincyto in the expanded access setting and identify clinically relevant subgroups
• Outcomes:
  To describe the clinical characteristics and treatment patterns of patients with B precursor ALL, having received Blincyto in the expanded access setting and identify clinically relevant subgroups, To describe the effectiveness of Blincyto within identified subgroups as outlined in Section 9.6.2.5 of the protocol To describe Blincyto utilization within identified subgroups as outlined in Section 9.6.2.5 of the protocol
• Data analysis plan:
  All analyses will be descriptive. Continuous variables will be summarized by mean, median, standard deviation, lower and upper quartiles, and minimum and maximum values. Categorical variables will be summarized by number and percentage of patients in each category. For categorical outcomes, 95% confidence intervals (CIs) will also bepresented where appropriate. For time-to-event endpoints, Kaplan-Meier (KM) curvesand KM proportions at select time points, the number of subjects with events and thenumber of subjects censored will be used to summarise the data. Analyses will bepresented by the identified clinically relevant subgroups (listed in Section 9.7.2.5 of the protocol and identified by means of the primary endpoints), country and year of Blincyto initiation.

Documents

Study results

01.47.01.02 Observational Research Study Report Redacted