Development of a digital patient-reported outcome measure (PROM) for real-time assessment of overactive bladder syndrome.

Herrewegh A1, Vork L2, Leue C3, Kruimel J2, van Koeveringe G1, Vrijens D1

Research Type

Clinical

Abstract Category

Male Lower Urinary Tract Symptoms (LUTS) / Voiding Dysfunction

Abstract 299
Bladder Outlet Obstruction
Scientific Podium Short Oral Session 18
Thursday 30th August 2018
12:12 - 12:20
Hall A
Overactive Bladder Incontinence Questionnaire Quality of Life (QoL) Outcomes Research Methods
1. Maastricht University Medical Center+, Department of Urology, Pelvic Care Center, 2. Maastricht University Medical Center+, Division of Gastroenterology-Hepatology, Department of Internal Medicine, 3. Maastricht University Medical Center+, Department of Psychiatry and Medical Psychology
Presenter
Links

Abstract

Hypothesis / aims of study
In the current diagnostic process for overactive bladder syndrome (OAB), biased retrospective questionnaires are frequently used. There is a need for a new assessment tool that embraces the heterogeneity of the OAB complex and overcomes the limitations of retrospective questionnaires, including contextual and recall bias. A momentary assessment tool, the digital Experience Sampling Method (ESM), is promising. ESM captures random repetitive measurements during the day, in the context of daily life, and is capable of measuring potential contextual triggers and psychological aspects. The aim of this study is to develop a new patient-reported outcome measure (PROM) for OAB, a smartphone app named ‘Uromate’, following the FDA guidelines on PROM development (1).
Study design, materials and methods
‘Uromate’ was created based on the ESM literature. The development of ‘Uromate’ consisted of three steps: the development of an initial draft list of questions, a focus group study, and an expert meeting to evaluate which items should be implemented in an urological ESM tool. The initial draft list consisted of questions regarding different domains: somatic items (urological and otherwise), nutrition and drug use, psychological items, and situation and company. Items concerning social factors, contextual and environmental factors and psychological status following the ESM specific construct, were derived from questions used in previous ESM validation studies in the field of Psychiatry and Gastroenterology  (2)(3). Urological questions were derived from validated questionnaires (i.e., OAB-q, ICIQ-FLUTS/MLUTS, ICIQ-OAB, KHQoL, BFLUTS, SF-36) and were rephrased to suit momentary assessment. The possible end-points for all items were ranged by use of an 11-points Numeric Rating Scale (NRS), if applicable. The 11-points NRS was chosen based on FDA recommendations (1). Focus group interviews and an expert meeting were conducted to broadly explore the knowledge and experience from patients and experts for item selection, in agreement with FDA guidelines on PROM development (1). The experts in the meeting were specialists in the field of functional urology or other functional disorders including two urologists, a gynaecologist, a gastroenterologist and a hospital psychiatrist.
Results
Thirteen female subjects were present during the focus group meetings, of whom 9 suffered from OAB and 4 from mixed urinary incontinence (MUI) (Table 1). Saturation of input, the point during the study where no new input was brought in, was reached after two meetings.

The majority of patients experienced urgency as an intensified sensation of the normal urge. One patient experienced no urgency but only pain in the lower abdomen prior to urinary leakage. Another patient experienced leakage without prodromal sensations. Urgency to defecate was also incorporated in ‘Uromate’, since some patients stated that both urinary and faecal urgency occurred simultaneously. 

Patients noted that liquid intake, the amount rather than the nature of the liquid, was a very important item. Most patients adjusted the amount of fluid intake when going outside. Coffee or tea were not mentioned as voiding triggers. 

Several somatic complaints were included in the proposed list of questions for ‘Uromate’, such as palpitations, sweating, shortness of breath, dizziness, muscle pain and painful joints. A few patients experienced the whole range of proposed somatic complaints. However, focus group participants could not mention one specific non-urological somatic complaint associated with OAB. Additionally, patients advised to add vaginal pain as a somatic symptom to the list of questions.

Furthermore, patients were asked to point out the least relevant psychological items, leading to a substantially shortened list of items. ‘Energetic’, ‘enthusiastic’, ‘happy’, ‘strong’, ‘worried’, ‘inspired’, ‘disappointed’, ‘insecure’ and ‘guilty’ were removed, because patients found those items not to be associated with OAB. 

Situation and company, were very important factors, influencing the psyche and severity of complaints. Patients stated that they felt uncomfortable in situations where people did not show understanding of their urological complaints. They felt that people do not take their complaints seriously. 

A morning questionnaire was developed to evaluate the symptom pattern during the night. Participants considered the frequency of awakening and whether awakening was due to urological symptoms the most important. 

Initially, sexuality questions were not incorporated, because repeated assessment was not considered useful. Nevertheless, patients missed questions about sexuality in the list of ‘Uromate’ items. To them, sexuality was an important item, because their sexual functioning was impaired due to OAB complaints. Hence, integration in the morning questionnaire was proposed.

The next step in the development of ‘Uromate’ was the expert meeting. During this meeting the list of questions was shortened by making sub-questions in the case of positive answers. A validated icon of the Visual Prostate Symptom Score (VPSS) depicting the urinary stream was added. The experts decided to use a validated 4-points urgency scale. Additionally, it was decided to evaluate the degree of untenability as well, using a 11-points NRS. Moreover, there was agreement between experts and focus group participants on merging muscle- and joint complaints together, in order to identify a possible link between OAB and fibromyalgia. Experts decided to add scrotal pain and prolapse sensation as gender-specific questions. Most of the initial psychological items, considering positive affect, were removed during the focus group interviews. However, experts agreed that a couple of positive options must be present in the ESM-questionnaire, whereas otherwise a negative response bias might be introduced. Therefore, the items ‘cheerful’ and ‘relaxed’ were kept as positive affect items to maintain an overall neutral question tone. The items ‘anxious’, ‘lonely’ and ‘nervous’ were removed to prevent response fatigue. Carbonated drinks were not incorporated in the questions, because there is little evidence on the association with urinary complaints. Morning questions about sexuality were added, such as pain and urinary urgency and incontinence during intercourse.
Interpretation of results
According to patients with OAB, their complaints are influenced by social, contextual, environmental factors and psychological status. This was confirmed in the expert meeting.
Concluding message
This is the first report of the development of an urological digital ESM tool, the ‘Uromate’, which is a newly developed PROM to measure real-time symptoms in the context of daily life. ‘Uromate’ meets the needs for a modern assessment tool for OAB that overcomes the limitations of today’s retrospective questionnaires and captures the heterogeneous, multifactorial character of OAB.
Figure 1
References
  1. U.S. Department of Health and Human Services FaDA, Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER), Center for Devices and Radiological Health (CDRH). Guidance for Industry Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. 2009.
  2. Vork L, Keszthelyi D, Mujagic Z, et al. Development, content validity, and cross-cultural adaptation of a patient-reported outcome measure for real-time symptom assessment in irritable bowel syndrome. Neurogastroenterology & Motility. 2017.
  3. Os J, Verhagen S, Marsman A, et al. The experience sampling method as an mHealth tool to support self-monitoring, self-insight, and personalized health care in clinical practice. Depression and Anxiety. 2017.
Disclosures
Funding None Clinical Trial Yes Public Registry No RCT No Subjects Human Ethics Committee Maastricht Medical Ethics Committee (METC 16-4-189) Helsinki Yes Informed Consent Yes
20/11/2024 19:19:38