Neuro-UrologyThe Validation of the Patient Perception of Bladder Condition (PPBC): A Single-Item Global Measure for Patients with Overactive Bladder
Introduction
For symptom-based conditions that have no physiologic or biochemical markers, patient-reported outcome measures are important tools for evaluating the effects of disease and treatment [1]. Consequently, for symptom-based conditions such as migraine [2], erectile dysfunction [3], and overactive bladder (OAB), patient outcomes are of paramount importance. OAB is a complex of symptoms characterized by urinary urgency, with or without urge incontinence, usually with frequency and nocturia [4]. Given the range and multitude of symptoms that patients with OAB experience, the effects of OAB on health-related quality of life (HRQL) are varied and substantial [5], [6]. OAB affects all facets of life from physical and social function to sexual function and sleep, work, and interpersonal relationships [7], [8], [9]. Given that the myriad of OAB symptoms have such a broad impact on HRQL, multi-item questionnaires that evaluate the impact of OAB and treatment outcomes are widely used (eg, OAB questionnaire (OAB-q; [10]), King’s Health Questionnaire (KHQ; [11])). Although such multi-item questionnaires provide a rich source of information on numerous domains of the patient’s life, such questionnaires may be difficult to score and interpret quickly, particularly in a clinical setting. As such, it can be useful to assess the patient’s overall perceived bladder condition with a single, global question.
Single-item global questions are practical because of their brevity, ease of use, and interpretation [12]. The inherent assumption of single-item global measures is that the patient will consider all facets of a disease or treatment. Then, the patient’s response will be based on consideration of all symptoms, not just one, in addition to possible treatment side effects, cost versus benefit, and other factors related to the disease or treatment. Typically, single-item, global measures are undervalued due to the lack of internal consistency reliability and carefully evaluated measurement properties; however, their simplicity in summarizing patient assessments of multifaceted concepts within a single outcome should not be underestimated.
Consequently, the Patient Perception of Bladder Condition measure (PPBC) was developed for patients with urinary problems as a global assessment of bladder condition and is recommended as a global outcome measure for urinary incontinence by the European Medicine Evaluation Association (EMEA) [13]. The purpose of this study was to evaluate and validate the PPBC as a single-item global measure among two clinical trial samples of patients with OAB. Analyses were conducted to examine the instrument’s validity and responsiveness to change in clinical symptoms.
Section snippets
Data sources
Post-hoc analyses were conducted on data from two 12-wk clinical trials of tolterodine in patients with OAB, most of whom were incontinent. Study 1 (n = 865) was a multicenter, open-label trial of tolterodine in the United States [14]. Inclusion criteria were: age ≥18 yr, diagnosis of OAB with or without urgency incontinence, and the ability to complete a micturition diary. Study 2 (n = 520) was a 12-wk, randomized, double-blind, placebo-controlled trial of tolterodine in the United States and
Demographics and descriptive statistics
Demographics and clinical characteristics of the patient samples from the two studies were fairly similar (Table 1). Both samples were primarily women and white with the mean ages of 61.0 and 58.8 yr, respectively. The majority of patients in each study were incontinent (75.3% in Study 1 and 80.4% in Study 2) with the greatest proportion of patients in both studies indicating that they had “moderate problems” (47.5% in Study 1 and 51.2% in Study 2) on the PPBC at baseline (Table 1).
Concurrent validity
In both
Discussion
The PPBC, a single-item, global measure of bladder condition, performed well in two large clinical trials of continent and incontinent patients with OAB. The PPBC had good construct validity, as demonstrated by highly significant correlations with the bladder diaries, as well as with the previously validated OAB-q and KHQ. Additionally, patients whose PPBC responses indicated relatively few problems tended to have fewer incontinent episodes and urgency episodes than patients with PPBC responses
Conclusions
The PPBC is a valid and responsive global measure of bladder condition that performs well among patients with OAB. It has been translated into 22 languages and is widely available for use (currently available on the website www.oabq.com). This single-item measure should be considered another useful tool, but not the only tool, for evaluating treatment outcomes for OAB.
Acknowledgements
Funding for this research was provided by Pfizer, Inc. The authors would also like to acknowledge Christine Thompson for her programming assistance and Fritz Hamme for his production assistance.
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