Outcome Measures in Functional Urology

Abstract

Pelvic floor disorders are characterized by four domains: urinary symptoms, anorectal symptoms, pelvic organ prolapse and sexual dysfunction. The symptoms of the different domains have a significant impact on patient’s quality of life. Traditional outcome measures, such as bladder diary, urodynamics, and ultrasound, have been used for the evaluation of pelvic floor disorders and treatment efficacy. These do not take the patient’s perspective into account which has resulted in a discrepancy between patients and physicians about the impact of symptoms and effectiveness of treatment. Patient reported outcome measures (PROMs) have been developed to resolve this discordance between patients and physicians.

The aim of this thesis is to determine the position of PROMs as an outcome measure in functional urology. The first part of this thesis evaluates two additional treatment options using only traditional outcome measures and a combination of PROMs and traditional outcome measures. We have found that onabotulinum toxin-A is effective in the treatment of therapy-refractory children with dysfunctional voiding. Furthermore, the results with intermittent sacral neuromodulation suggest it to be a more cost-effective option than ‘standard’ continuous sacral neuromodulation.

The second part of this thesis focusses on the quality of PROMs used in clinical practice. During the assessment of validation studies and measurement properties using the COSMIN checklist, variable evidence was found for the quality of the PROMs to evaluate sexual function in neurological patients. An essential point of improvement is to adequately assess all measurement properties, specifically responsiveness and minimal important change, before the PROMs can be used in clinical practice.

In the third part of this thesis four Dutch versions of PROMs assessing severity of pelvic floor disorders and their impact on quality of life in children and adults are validated, thereby providing adequate Dutch versions of these PROMs. In conclusion, the findings presented in this thesis support that PROMs in functional urology sufficiently represent the patient’s perspective and PROMs could assist to manage patient expectation and improve patient satisfaction. However, it remains important to guarantee adequate quality of PROMs used in clinical practice. One of the future perspectives for the field of functional urology is to develop a prediction model based on different outcome measure to predict treatment success for individual patients.