An observational study of patients with Philadelphia chromosome-negative relapsed or refractory acute lymphoblastic leukemia in the US (20150253)

Study type

• Study topic:
  Human medicinal product

  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation

  Effectiveness study (incl. comparative)

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

Non-interventional study

• Non-interventional study design:
  Cohort

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 will include patients initiating treatment for Philadelphia chromosome-negative (Ph-) R/R ALL between January 2013 and March 2019 at participating clinical sites in the US.; Medical records of all patients initiating treatment for Ph- R/R ALL at participating clinical centers in the US between January 2013 and March 2019 will be eligible for inclusion.; 1. Medical records of patients participating in clinical trials will be included for purposes of comprehensively describing the treatment paths of Ph- R/R ALL patients. Other study objectives will be evaluated among these medical records up to the time the patient enrolls into a clinical trial, however patient data after enrollment in a clinical trial will not be collected.; 2. Medical records of patients with Philadelphia chromosome-positive (Ph+) disease will be excluded.; 3. If informed consent is required, medical records of patients who do not provide informed consent will be excluded.
• Age groups:
  • Paediatric Population (< 18 years)

    • Neonate

      • 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)
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 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:
  200

Study design details

• Main study objective:
  To describe treatment patterns, drug utilization, and healthcare resource utilization in patients with Ph- R/R ALL
• Outcomes:
  Drug utilization, treatment patterns, healthcare resource utilization, Best response to salvage treatment, incidence of selected adverse events, overall survival following salvage treatment, minimal residual disease status following salvage treatment, relapse-free survival, receipt of allogeneic hematopoietic stem cell transplantation
• Data analysis plan:
  Analyses of primary and secondary objectives will be descriptive in nature. Demographic, treatment, and clinical characteristics of patients will be summarized by means, standard deviations, medians and interquartile ranges, minima, and maxima for continuous variables and by counts and proportions/percentages for categorical variables. As a measure of precision, a 95% confidence interval will be calculated around point estimates (i.e. proportions). Descriptive analyses of primary and secondary objectives will be provided overall and by subpopulations of interest (eg, by treatment group, by line of salvage) when sample size permits.