PERFORM: An epidemiological, prospective cohort study for patients with HR+/HER2- advanced breast cancer treated 1st-line with an endocrine-based palbociclib combination therapy in real world in Germany and Austria

Background CDK4/6 inhibitors, e.g. palbociclib, combined with endocrine therapy (ET) are approved and recommended 1st-line standard of care for patients (pts.) with hormone receptor-positive and human-epidermal-growth-factor-receptor 2-negative advanced breast cancer. Randomized clinical trials have presented high efficacy and a consistent safety profile. The overall goal of the non-interventional study (NIS) PERFORM is to complement clinical evidence of palbociclib/ET with new real-world insights into routine clinical decision making including genetic testing patterns over the time, effectiveness, and patient-reported quality of life (QoL). New evidence on current post-progression strategies, longitudinal effectiveness and QoL after 1st-line palbociclib/ET is generated.

Trial design The ongoing, prospective NIS seeks to enroll 1,900 pts. scheduled to receive 1st-line palbociclib/ET from 300 sites across Germany and 20 sites across Austria over a targeted recruitment period of 4 years. Total study duration is 7.5 years. PERFORM will document routine clinical practice. Primary endpoint is the progression-free survival (PFS). Subsequent therapy sequences including analysis of post-palbociclib/ET treatment strategies, in view of effectiveness (2nd-/3rd-line PFS, PFS across treatment lines) are described. Patient-relevant endpoints such as treatment satisfaction, longitudinal change and time to deterioration in QoL using the FACT-B questionnaire, as well as patient management in times of COVID-19 are documented. Another focus lies on the different routinely used genetic tests/predictors at different timepoints and changes over time. Descriptive statistics will be used to analyze data. Hazard ratios for effectiveness will be derived from Cox proportional hazard models, adjusting for baseline characteristics; propensity score matching will be performed as a sensitivity analysis.

Interessenkonflikt

Michael Patrick Lux: Honorar (self), Advisory/Consultancy, Speaker Bureau/Expert testimony, Travel/Accomodation/Expenses; Eva Amann: Shareholder/Stockholder/Stock options, Full/Part-time employment; Johanna Buncke: Shareholder/Stockholder/Stock options, Full/Part-time employment; Eva Diana Runkel: Shareholder/Stockholder/Stock options, Full/Part-time employment; Marc Thill: Honorar (self), Advisory/Consultancy, Travel/Accomodation/Expenses

Publication History

Article published online:
01 June 2021