Natural Sciences
Life Sciences
Scientific Computing
Life Science

Harriet Sommer, Roche Pharma AG, Grenzach-Wyhlen

ATV-Raum des DKFZ, Im Neuenheimer Feld 242

Heidelberger Kolloquium Medizinische Biometrie, Informatik und Epidemiologie

In clinical trials for the development of antibacterial drugs, diverse primary endpoints have been used and treatment effects are usually assessed at the end of follow-up which varies between studies. A highly patient-relevant statement would be an assessment over the entire follow-up period with cure and death as co-primary endpoints. We emphasise to examine the time-dependent multistate endpoint “get cured and stay alive over time”, since this might be most relevant from the patients’ perspective and can capture different “cure patterns” over the treatment period [1,2,3]. Such time-dynamic endpoints provide valuable additional information such that potentially hidden treatment effects can be revealed that might be overlooked when only presenting incidence proportions. Based on a “cure-death” multistate model, several possibilities are introduced to evaluate a treatment difference in probabilities to be cured and alive over time. The procedures are applied to three topical data examples: An RCT by Basilea for the treatment of patients with hospital-acquired pneumonia (see also [1] and [4]), an RCT by Merck for the prevention of recurrent Clostridium difficile infection (see also [5]), and the French prospective OUTCOMEREA database to investigate the effect of early adequate treatment on extubation and discharge for patients with ventilator-associated pneumonia due to the pathogen Pseudomonas aeruginosa (see also [6]) [1] H Sommer, Pharmaceutical Statistics, 16:267–279, 2017. [2] JF Timsit, Intensive Care Medicine, 43:1002–1012, 2017. [3] MEA de Kraker, Clinical Infectious Diseases, 67(12):1922–1931, 2018. [4] H Sommer, Antimicrobial Agents and Chemotherapy, 62:e01691–17, 2018. [5] H Sommer, New England Journal of Medicine (letter to the editor), 376:1594–1595, 2017. [6] H Sommer, Critical Care Medicine, 46:1643–1648, 2018.

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