OUTCOMES OF THE SPANISH COHORT OF EARLY ACCESS TO PERTUZUMAB AND TRASTUZUMAB EMTANSINE (KNOWHER STUDY)

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:
  Case-series

Study drug and medical condition

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

  TRASTUZUMAB EMTANSINE
• Medical condition to be studied:
  HER2 positive breast cancer

Population studied

• Short description of the study population:
  Adult patients (age ≥ 18 years at enrolment) from the Spain with HER2-positive metastatic or locally recurrent unresectable breast cancer and who were treated with Trastuzumab emtansine (T-DM1) and Pertuzumab under compassionate use or early access program.
• Age groups:
  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:
  Breast cancer patients
• Estimated number of subjects:
  700

Study design details

• Main study objective:
  To evaluate the effectiveness of Trastuzumab emtansine (T-DM1) and Pertuzumab in patients with HER2-positive MBC treated under compassionate use or early access program.
• Outcomes:
  Overall survival. Defined as the time between the date of start of treatment and the date of death. For subjects without documentation of death, OS will be censored on the last date the subject was known to be alive, •Progression free survival. •Best overall response rate•Duration of response •Time to treatment failure •Time to Objective Response•Time to change treatment•Time to next treatment
• Data analysis plan:
  The analysis of the present study will primarily make use of descriptive statistical methods (i.e. number, mean, median, standard deviation, rate, range, and IC95% for the estimated parameters).Where possible, and if allowed by the number of enrolled patients receiving different treatment regimens, a comparative analysis of the outcomes across various groups will also be performed.If applicable, it will be calculated IC 95% for the estimated parameters in relevant subgroups, analysis of variance (t test or F test) or non-parametric testing, such as Wilcoxon’s rank-sum test or Kruskal–Wallis test, will be used to test group differences on the continuous variables. All test performed will be two-sided and carried out with a 5% α-error rate without correction for multiplicity.Categorical variables will be summarized by numbers and proportions, and, where applicable, chi-squared testing will be used to test group differences.