Structured patient interview to assess clinical outcomes in complicated urinary tract infections in the APEKS-cUTI study: pilot investigation.
Background
The APEKS-cUTI study demonstrated the non-inferiority of cefiderocol to imipenem-cilastatin in the primary endpoint of the composite of clinical and microbiological outcome in patients with complicated urinary tract infections (cUTIs). We piloted a structured patient interview (SPI) to evaluate clinical outcomes based on patient-reported symptoms while conducting this pivotal randomized, double-blind, phase-2 study. The objectives were to assess the value of the SPI, using its performance relative to physician assessment, and also to strengthen the value of patient-reported measures in conducting clinical trials for cUTI treatment.


Methods
In addition to the protocol-defined clinical and microbiological outcomes, patients randomized in the APEKS-cUTI study were interviewed by the investigator or qualified study personnel at screening/baseline, early assessment (EA), end of treatment (EOT), test of cure (TOC), and follow-up (FUP). The 14-element questionnaire graded cUTI symptoms as absent or present, and if present, as mild, moderate, or severe. Changes in post-baseline symptoms based on patients' responses were rated by the interviewer. The overall clinical outcome was evaluated based on the responses provided by patients at each time point.


Results
Among the 371 patients in the modified intention-to-treat population, the rate of SPI completion in each treatment arm exceeded 90% at each time point. SPI-assessed clinical cure rates were 89.7% in the cefiderocol arm and 84.9% in the imipenem-cilastatin arm. There was substantial agreement between SPI evaluation and investigator global assessment of clinical outcome at TOC and FUP, with lower agreement at EA and EOT.


Conclusion
This analysis suggests that patient-reported symptoms can be effectively captured in hospitalized patients with cUTI in a clinical trial setting. Development of a validated patient-reported outcome for use in such a setting is warranted.


Registration
NCT02321800 (registered on 22 December 2014).
['Portsmouth|Simon|S|https://orcid.org/0000-0002-9853-4160', 'Echols|Roger|R|', 'Toyoizumi|Kiichiro|K|', 'Tillotson|Glenn|G|', 'Nagata|Tsutae Den|TD|']
structured patient interview clinical outcome complicated urinary tract infections cefiderocol patient reported outcomes