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Abstract

  1. Top of page
  2. Abstract
  3. Health status and QoL questionnaires
  4. Generic instruments
  5. Disease/condition-specific QoL questionnaires
  6. Screening questionnaires
  7. Individualising expectations and goals of treatment
  8. Defining successful treatment outcome for OAB
  9. References

Overactive bladder (OAB) is a common and costly condition affecting up to 16% of the adult population in Europe and the USA. OAB results in significant impairment of the quality of life (QoL) of sufferers. In clinical trials of OAB treatments, symptom measurements remain the most important primary outcome measures. Without understanding and measuring QoL, the true impact of OAB and the benefit of treatment cannot be evaluated. Many different multidimensional QoL questionnaires have been used to assess people with OAB. Generic questionnaires lack the sensitivity to detect important improvements in QoL following OAB therapies. For this reason, disease-specific QoL measures have been developed. Disease-specific questionnaires focus on aspects of QoL impacted by lower urinary tract symptoms. Such instruments offer far greater sensitivity, specificity, and clinical relevance to patient populations with OAB. Minimal important difference assessments have improved our understanding of the relevance of improvements in questionnaire scores. Unfortunately, the apparent complexity of QoL questionnaires has restricted their widespread use in clinical practice and to some extent in clinical trials. Short transparent patient-perception measures and symptom bother scales are increasingly being used to fulfil this role. Short screening versions of these questionnaires have been developed to detect symptom bother and OAB-related QoL impairment in symptomatic but currently untreated patients. Our understanding of OAB, the impairment it causes, and the benefit of treatments both to individuals and society have greatly improved through the use of validated QoL assessment tools. The questionnaires and their usage continue to evolve.

Overactive bladder (OAB) is characterised by the storage symptoms of urgency with or without urgency incontinence, usually with frequency (>8 micturitions in 24 hours) and nocturia.1 OAB is known to adversely affect the quality of life (QoL) of sufferers, and perhaps the greatest benefits of treatment experienced by patients are improvements in QoL.2

OAB is a very common condition. The National Overactive Bladder Evaluation (NOBLE) study performed in the USA estimated the prevalence of OAB to be 16 and 16.9% in men and women, respectively,3 and a European prevalence study (SIFO) estimated that 16.6% of individuals older than 40 years are affected.4 Interestingly, neither the International Continence Society definition nor either of these large prevalence studies incorporates the assessment of symptom bother or QoL impairment as a central factor in the evaluation and diagnosis of OAB.

QoL impairment caused by OAB symptoms should, however, be central to our understanding of the impact and treatment evaluation of this condition. In the SIFO study, 60% of people with symptoms had consulted a doctor, but only 27% were currently receiving treatment. It is unknown whether symptoms were not bothersome and QoL impairment minimal for those who did not seek help. What is also unknown is whether those having sought but discontinued treatment did so because of inadequate trade-off in terms of symptoms and QoL improvement.

Understanding the true impact of a medical condition is important if we are to treat it effectively and gauge the value of different treatments. Unfortunately, we know little about the symptom complex of OAB, and while different mechanisms have been proposed to explain symptom causation even the natural history of this condition remains unclear. Without qualifying the definition of OAB by any reference to the impairment that symptoms cause, the alleviation of symptoms rather than the bother they cause becomes the major goal of treatment. The majority of studies that have investigated the treatment of OAB have for this reason concentrated on symptom alleviation as the primary outcome measure of OAB therapies. Urgency, the central defining symptom of OAB is highly subjective, and is both difficult to define and to measure. Frequency, nocturia and urgency incontinence that are easier to objectively quantify are therefore more commonly used as the primary outcome measures of studies of OAB therapy.

Assessment of burden of OAB by symptoms alone is, however, illogical and possibly misleading. Clearly, not all the participants in either the SIFO or NOBLE studies have OAB requiring intervention, and therefore it is arguable whether they should be labelled with any medical condition. The same symptoms affect people differently, and symptom impact is related to many varied and often highly individual or personal factors.

Statistically significant improvement in symptoms in clinical trials may be heralded a therapeutic success, but numerically be so small as to create doubt regarding their clinical benefit to people. In a recent meta-analytical review of antimuscarinic therapies for the treatment of OAB,5 statistically significant symptom improvement was unquestioned, but doubts were raised regarding the magnitude of improvements with treatment and therefore their clinical value for patients. A repeat meta-analysis was undertaken by Chapple and Khullar.6 They looked at data gathered from studies, including QoL and other subjective outcome data. They concluded that there were similar symptomatic improvements and also significant QoL benefit conferred on people by treatment of OAB symptoms with antimuscarinic drug therapy.

In clinical practice, we all talk to our patients and ask not only about the presence of symptoms but also how much and in what way they bother them. It would be illogical to treat non life-threatening symptoms that cause little bother with expensive and time-consuming treatments that themselves have adverse effects and treatment complications.

The importance of measuring the impact of an improvement in medical conditions from the patient’s perspective is increasingly being established in the provision of modern health care. By clinical interview, doctors do not always accurately assess the burden of conditions such as OAB. A study investigating the discrepancy in patient and physician perception of QoL impairment related to urinary symptoms found that physicians underestimated the patient’s degree of bother 25–37% of the time. Patients tend to report higher subjective distress on questionnaires than in structured interviews, and this may be related to the embarrassing nature of urinary symptoms and subsequent communication difficulties.7

Urinary symptoms and their impact on patients’ QoL can be assessed in a number of ways, but the only valid way of measuring the patient’s perspective of their predicament is through the use of psychometrically robust self-completion questionnaires.1 Questionnaires provide a valid, reproducible, rapid assessment of patient-reported disease impact, which can elicit symptom bother and are also useful for the evaluation of the efficacy of an intervention.

There are many different types of questionnaires to measure general population QoL, specific condition/disease QoL, specific symptom QoL and also questionnaires to screen for QoL impact. In addition, single global summary scales to measure urgency, bladder condition and treatment benefit are increasingly being used.

Although it is known that lower urinary tract disorders cause considerable morbidity, the adverse events and morbidity associated with treatments must not be forgotten when assessing the QoL impact of many symptoms and their management.

Health status and QoL questionnaires

  1. Top of page
  2. Abstract
  3. Health status and QoL questionnaires
  4. Generic instruments
  5. Disease/condition-specific QoL questionnaires
  6. Screening questionnaires
  7. Individualising expectations and goals of treatment
  8. Defining successful treatment outcome for OAB
  9. References

There are many different health status and QoL questionnaires to assess both men and women with lower urinary tract dysfunction; these are collectively called patient-derived outcome measures.

Questionnaires are usually completed by participants themselves or by their partner or carer on their behalf. They aim to provide an objectively quantifiable measure of subjective experiences and concerns in relation to illness–health status and QoL. The design of questionnaires is a complex and often lengthy process. Questionnaires should be psychometrically robust with established validity, reliability and responsiveness. A description of the different classes of patient-derived outcome measures is given in Table 1.

Table 1.  Type of questionnaires and different patient-derived outcome measures
Type of questionnaireProperties of questionnaire
  1. LUTS, lower urinary tract symptoms; EQ-5D, Euro Qol 5D; a standardised instrument for use as a measure of health outcome.

GenericMeasure very broad aspects of health and are suitable for a wide range of patient groups and the general population (e.g. UK SF36)8
Disease specificMeasure patients’ perception of a specific disease or health problem. These can be either specific to certain types of problem i.e. lower urinary tract dysfunction (KHQ)9 or a specific aspect of that problem i.e. OAB specific (e.g. OAB-q)10
Dimension specificAssess one particular aspect of health status–usually psychological wellbeing (e.g. Beck Depression Inventory)11
Summary itemsAsk patients to summarise diverse aspects of their health status using a single item or a very small number of items (e.g. patient perception of bladder condition)12
IndividualisedAllow patients to identify for themselves the most important aspects of their lives, which constitute their appraisal of their overall QoL—these measures have not been widely used in the assessment of incontinence and LUTS (e.g. patient generated index)13
Utility measuresIncorporate preferences or values attached to individual health states and express health states as a single index (e.g. EQ-5D)14
Screening questionnairesScreening tools do not strictly belong in the category of patient-derived outcome measures, however, there is growing interest in the development and use of self-assessed screening tools in the evaluation of urinary tract dysfunction. The aim of screening questionnaires is to identify bothersome symptoms in patients who may benefit from further advice or treatment (e.g. OAB-V8)15

Generic instruments

  1. Top of page
  2. Abstract
  3. Health status and QoL questionnaires
  4. Generic instruments
  5. Disease/condition-specific QoL questionnaires
  6. Screening questionnaires
  7. Individualising expectations and goals of treatment
  8. Defining successful treatment outcome for OAB
  9. References

Generic instruments are designed to measure very broad aspects of health, and are therefore potentially suitable for a wide range of patient groups and for general population screening. Their major drawback is that they are relatively insensitive to clinically relevant change for non life-threatening conditions such as OAB unless the population surveyed is very large. For this reason, condition or disease-specific measures of QoL have been developed.

The advantages of generic instruments are that they can be used to assess the impact of different disease states and medical interventions on general health. They are of particular value in health economic analyses where the ability to compare the morbidity of different conditions and their treatment are important.

The short form 36 questionnaire (SF36)8 is the most commonly used generic QoL questionnaire in lower urinary tract research. It consists of a single item assessing health transition, an eight-scale profile of physical (physical functioning, role limitations as a result of physical problems, bodily pain and general health perceptions) and mental health (vitality, social functioning, role limitations as a result of emotional problems and mental health).

The SF36 has been used extensively in clinical trials, including studies comparing tolterodine with oxybutinin,16 and tolterodine with placebo.17 Baseline SF36 scores were used to compare QoL of incontinent women with published values of an age and gender-matched normal population. The results for all the studies showed that almost all domains of the SF36 showed a lower QoL in incontinent women, and this effect was greater in younger women. Treatment tended to improve QoL as measured by the SF36, however, this was not statistically significant.

The Nottingham health profile (NHP) is an older generic QoL measure. It consists of 38 items covering the dimensions of mobility, pain, social isolation, emotional reactions, energy and sleep.18 Grimby et al19 used the NHP to show significantly higher levels of emotional and social impact among incontinent women, in particular, women with urge and mixed urinary symptoms compared with stress incontinent women. The study was conducted in Sweden, and assessed 120 elderly incontinent and 313 elderly continent women (control group). The incontinent group were subdivided into those with urge, stress or mixed symptoms on the basis of a bladder diary, pad test, cough provocation and history. A limitation of this study was that the control group were not assessed for incontinence using the same measures.

The sickness impact profile (SIP) is a 136 item, self- or interviewer-administered health status questionnaire.20 Twelve domains related to everyday activities are assessed: sleep and rest, emotional behaviour, body care and movement, home management, mobility, social interaction, ambulation, alertness behaviour, communication, work recreation and pastimes and eating. Participants select items that describe themselves and are related to their health. A major drawback of this questionnaire is its length.

A study conducted by Hunskaar and Vinsnes21 used the SIP to assess 70 women presenting to a self-referral centre for incontinent patients. Women were divided into subgroups based on age (40–60 and >70 years) and symptoms (predominant stress or urgency). Women were asked to complete SIP items considered attributable to their bladder symptoms. Mean questionnaire scores were low for both the groups. Younger women and those with predominantly urge symptoms scored significantly higher on the SIP. Sleep, rest, mobility, social interaction, emotions and recreational activities were most commonly affected. The study concluded that the impact of urinary incontinence on QoL was dependent on both symptoms and age.

At the time that many of these studies were conducted, generic QoL questionnaires were the only available instruments. Although they were insufficiently sensitive for use as outcome measures in treatment intervention studies, they did provide important clinical information. People with lower urinary tract dysfunction have a significantly impaired QoL, and those with predominantly urge symptoms have a greater degree of QoL impairment than those with stress symptoms. People with urgency incontinence had a poorer QoL than those with other lower urinary tract symptoms. The studies also provided information regarding how people’s lives are affected and while not ideal instruments for clinical trials these early studies provided the impetus to develop the more sensitive condition or disease-specific questionnaires.

Disease/condition-specific QoL questionnaires

  1. Top of page
  2. Abstract
  3. Health status and QoL questionnaires
  4. Generic instruments
  5. Disease/condition-specific QoL questionnaires
  6. Screening questionnaires
  7. Individualising expectations and goals of treatment
  8. Defining successful treatment outcome for OAB
  9. References

Compared with generic QoL instruments, disease-specific questionnaires offer greater sensitivity and therefore responsiveness to change. They are designed to assess the QoL of people with specific medical conditions, and therefore it is understandable that this should be the case. They are ideal for use in clinical intervention studies.

There are many different condition-specific questionnaires available, and they have been reviewed in detail in the most recent triennial report of the international consultation on incontinence (ICI).1 Questionnaires are generally either for the assessment of all people with lower urinary tract dysfunction or for those with specific lower urinary tract complaints e.g. stress incontinence/OAB/voiding difficulties.

A concern regarding questionnaires focused on very specific aspects of lower urinary tract dysfunction is that they may be ‘too specific’. It is known that the majority of patients have mixed symptoms and that over time or after treatment, the symptoms may change. A list of the available condition-specific questionnaires and their relative components is shown in Table 2, and the more commonly used questionnaires are discussed in greater detail.

Table 2.  Characteristics of validated urinary symptom and quality of life questionnaires (adapted from 3rd ICI consultation)
TypeQuestionnaireWomenMenIncontinence symptomsIncontinence QoLOAB symptomsOAB BotherGlobal assessment
  1. I-QOL, incontinence quality of life questionnaire; UDI, urogenital distress inventory; IIQ, incontinence impact questionnaire; B-SAQ, bladder control self-assessment questionnaire.

Multidimensional disease specificICIQ 
I-QOL  
KHQ
UDI   
IIQ  
OAB-q  
BFLUTS  
IUSS  
OAB-SCS  
Single itemPOSQ 
PBC 
UPS  
BSW  
PGI-S 
PGI-I 
ScreeningB-SAQ  
OAB-V8  
GenericSF36 
NHP 
SIP 

The Kings health questionnaire (KHQ) is a 33-item multidimensional disease-specific questionnaire for the assessment of men and women with lower urinary tract dysfunction.9 It has nine domains: role limitations, physical limitations, social limitations, personal relationships, emotions, sleep and energy, severity (coping) measures, incontinence impact and general health perceptions. The KHQ also includes a multi-item symptom severity scale.

The KHQ has been extensively used to evaluate people with OAB and stress urinary incontinence. It has over 30 linguistic validations (www.mapi.fr), which have made it ideal for assessment in multinational studies. The KHQ has been used in numerous clinical trials for the evaluation of tolterodine,16,22 oxybutynin17 and solifenacin.2

A further evolution of the KHQ has been the calculation of minimal important difference (MID) values for determining the clinical significance of measurable improvements in KHQ scores. MID is a measure of the amount of change in a questionnaire score that is likely to be clinically relevant to patients. MID analysis has been performed for the KHQ using data from two large-scale clinical trials comparing the efficacy and safety of tolterodine among people with OAB.23 The study findings concluded that a change of five or more points on a KHQ domain is an indication of an important effect at the patient level, although smaller changes in the symptom severity checklists are important to patients. The developers of the SF36 suggest that a five-point (5%) change is clinically meaningful, as is a two- to five-point change on the 100-point scale by the developers of the incontinence quality of life questionnaire.24

The KHQ was used in two 12-week double-blind placebo-controlled studies of solifenacin for the treatment of OAB. Pooled data showed a significant improvement in QoL for those receiving both 5 and 10 mg doses of solifenacin compared with placebo (Figure 1).25

image

Figure 1. Effect of solifenacin on domains of the KHQ after 12 weeks. PR, personal relationships; Emo, emotions; S/E, sleep/energy; SM, severity measures; SS, symptom severity. P < 0.05 vs placebo, **P= 0.005 vs placebo.

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Not only were the improvements in QoL significantly better than those receiving placebo therapy but also improvement in scores (a lower score representing an improvement in QoL) exceeded the minimal KHQ domain point scale, indicating both statistically and clinically relevant improvements for patients. The only domain that failed to show a statistically significant improvement was that measuring personal relationships. This is a common finding in such studies where participants often fail to answer very personal questions. Although sexual function is known to be impaired for those with lower urinary tract dysfunction, the ideal way to measure it has not yet been found.

Ninety-one percent of participants who completed the two 12-week studies (1637/1802) chose to participate in a 40-week open-label extension study.26 During the extension study, all participants, whether initially receiving placebo, solifenacin (5 or 10 mg), or in one study tolterodine IR 2 mg27 were commenced for the first 4 weeks of the extension study on 5 mg solifenacin. At subsequent visits during the extension study, participants were allowed to titrate up their dose of solifenacin to 10 mg if they so chose. Eighty-one percent of those patients completed the 40-week follow-up period. The KHQ was completed again at the end of the follow-up period. Additional improvements in QoL ranging from 28 to 35% of the effect seen at the end of the 12-week study were seen for those completing therapy (Figure 2).

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Figure 2. Effect of solifenacin on domains of the KHQ after 12 and 52 weeks. GHP, general health perception; Imp, incontinence impact; RL, role limitations; PL, personal limitations; SL, social limitations; PR, personal relationships; Emo, emotions; S/E, sleep/energy; SM, severity measures; SS, symptom severity.

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These are important findings with respect to QoL improvement. Firstly, compliance with therapy during the follow-up period was good, suggesting that people continued to derive benefit from treatment over time. Secondly, the improvements in QoL seen at the end of the 12-week studies continued to improve, indicating further adaptive change in QoL over time. It is possible that patients cope with many aspects of their bladder symptoms by adaptive behaviour, and this is unlikely to resolve over a short-term period such as 12 weeks. As time passes, they regain confidence and control of their bladder function, and the QoL improves further. It was important also to note in this study that symptomatic improvement was also seen and maintained for those receiving solifenacin treatment.

The Bristol female lower urinary tract symptoms (BFLUTS) questionnaire28 was developed to assess lower urinary tract symptoms in women with an emphasis on quantifying the frequency and extent of urinary incontinence. There are eight items relating to urinary incontinence, four of which quantify leakage. These items ask about the number of incontinence episodes, the type of protection and number of pads used. There are 12 items addressing symptoms of the storage and voiding phases of the micturition cycle, nine addressing QoL and four addressing sexual function. Bother associated with symptoms is assessed on a four-point scale ranging from ‘no problem’ to ‘severe problem.’ Although an excellent questionnaire to assess the impact of lower urinary tract symptoms, it has not been used widely in studies of OAB therapy.

The overactive bladder questionnaire (OAB-q) is a validated condition-specific questionnaire for the assessment of people with OAB syndrome.29 It is more commonly used in the USA where it was developed than in Europe. It consists of eight bother items assessing symptoms of frequency, nocturia, urgency and urge incontinence. There are 25 health related quality of life (HRQL) items assessing coping, sleep, concern and social interaction domains. All the items are scored on a six-point Likert scale. The OAB-q has been used as part of the clinical assessment of participants in a number of clinical trials comparing efficacies of pharmacological therapies for OAB. Higher OAB-q symptom scores indicate worse symptom bother; conversely higher HRQL scores indicate a better QoL.

The OAB-q was used in a 12-week study in people receiving tolterodine extended release (ER).30 OAB-q and perception of bladder condition scores were calculated at baseline, 4 and 12 weeks. OAB-q subscale change scores were significantly correlated with changes in urgency episodes, micturitions and incontinence episodes. The OAB-q has also been recently used in a 12-week open-label study of 5 or 10 mg solifenacin for people who had previously taken other drug therapy (VESIcare® Efficacy and Research Study US, in prep.). The study showed significant improvement in OAB symptom and HRQL scores at the end of the 12-week period (Figure 3).

image

Figure 3. Change in mean OAB-q subscale scores during the 12-week VERSUS study with open-label solifenacin 5 and 10 mg. *P= 0.0001 vs prewashout.

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The international consultation on incontinence questionnaire (ICIQ) is a questionnaire designed for the evaluation of incontinence in research and routine clinical practice.31 It has 27 linguistic translations. The questionnaire comprises three scored items; assessment of frequency, severity and perceived impact of incontinence and an unscored self-diagnostic item.

In its validation, the ICIQ short form questionnaire scores were compared with 24-hour pad weight results in 80 women with urodynamically proven stress urinary incontinence. A moderate correlation was found (r= 0.458 P= 0.000). The pad weights correlated significantly with all three components of the questionnaire (r= 0.583 P= 0.000). There was no correlation between questionnaire scores and pad test results in women with stress incontinence who had already been treated surgically.

The ICIQ questionnaire has recently been subjected to considerable psychometric testing. Although it has not yet been widely used, it has been accorded grade A recommendation (validity, reliability and responsiveness established with rigour in several data sets) from the ICI.1 The ultimate aim for the ICIQ is to develop an all encompassing modular set of questionnaires. This would have the short ICIQ as a base questionnaire, to which would be added the other questionnaires such as the KHQ, BFLUTS and OAB-q as required.

A number of other OAB-specific questionnaires have been developed for specific studies to simply address various attributes of OAB sufferers. The primary OAB symptom questionnaire (POSQ) is a two-item questionnaire, which asks participants to rate bother associated with OAB symptoms and to indicate which symptom is the most bothersome.32 The OAB symptom composite score (OAB-SCS) incorporates the Indevus urgency severity score with information from patient voiding diaries.33 The Indevus urgency severity scale (IUSS) is a single-item scale developed for use in clinical trials.34 It evaluates the intensity of urgency events associated with OAB in the context of the impact on activity at the time that the urgency sensation was experienced. In the validation of the IUSS, concerns were raised about the short test–retest period of time (14 days) and regarding the wording of the items in the scale to determine the degree of urgency. The IUSS was used in a placebo-controlled trial evaluating trospium chloride for the treatment of OAB.34 At baseline, the mean IUSS score was 1.77 (moderate severity) for both patient groups. At 12 weeks, those receiving trospium chloride experienced significant improvement and reported a mild sensation of urgency, compared with the placebo-treated group who reported no noticeable change in urgency severity.

The OAB-SCS has been used in a placebo-controlled trial to assess the effects of trospium chloride on OAB symptoms.33 A total of 1157 subjects were enrolled in the study, and of these, 581 were treated with the active drug and 509 with placebo. For each symptom, trospium was statistically superior to placebo. The OAB-SCS showed greater sensitivity than symptoms alone.

The incontinence severity index (ISI) was developed for use in epidemiological surveys to assess female incontinence.35 It consists of two questions, ‘how often do you experience urine leakage?’ and ‘how much urine do you lose?’ The responses are multiplied together and categorised into slight, moderate, severe and very severe. The ISI has been used in a large community-based epidemiological study in Norway; the aim of which was to assess the prevalence of urinary incontinence in a female population.36 The study results showed that 25% of participants had some degree of urinary incontinence and almost 7% had moderate to severe incontinence that was bothersome.

The great advantage of simple questionnaires rather than longer QoL measures is their ease of completion, interpretation and apparent transparency. For this reason, a number of short global summary questionnaires have been developed and validated for the assessment of OAB impact and treatment effects.

Short global assessments are useful in conditions such as OAB, which has multiple and varied symptoms. The underlying assumption of patient-rated global assessments is that the patient will weigh all factors related to their condition (e.g. risks and benefits of a treatment, consideration of multiple symptoms rather than one symptom), and provide a response that reflects their perspective of the construct being measured. These summaries reflect an individual’s needs, concerns and values. They are simple, direct and easy to administer and interpret.

The perception of bladder condition scale (PBC) asks people to describe ‘the degree of bladder problems they are experiencing at the moment’.32 Responses are graded on a six-point scale ranging from ‘no problems’ to ‘many severe problems’ (Table 3).

Table 3.  Perception of bladder condition questionnaire
Which of the following statements describes your bladder condition best at the moment
ScoreMy bladder condition
1Does not cause me any problems at all
2Causes me some very minor problems
3Causes me some minor problems
4Causes me (some) moderate problems
5Causes me severe problems
6Causes me many severe problems

Freeman et al.12 conducted a secondary analysis of data from a double-blind placebo-controlled study to evaluate the effect of once daily tolterodine on urinary urgency in people with OAB. Evaluation of treatment efficacy included comparison of baseline and 12-week symptom changes as measured using the PBC scale. At baseline, approximately 85% of participants in each treatment group reported that their bladder symptoms caused ‘moderate’ to ‘many severe problems’. After 12 weeks of treatment, the proportion of tolterodine-treated people reporting an improvement in their bladder symptoms was significantly greater than that observed with placebo. Forty-three percent of those treated with tolterodine rated their treatment as providing ‘much benefit’ compared with 24% of placebo recipients. Women experienced a greater improvement in urinary urgency when taking tolterodine ER compared with placebo.

The PBC has recently been used as an outcome measure in a 12-week prospective, double-blind, double-dummy, two-arm parallel group study to compare the efficacy and safety of solifenacin 5 or 10 mg and tolterodine ER 4 mg in people with OAB (the STAR trial).36

Improvement in PBC scores was significantly better in solifenacin-treated people compared with those receiving tolterodine ER (Figure 4). Although it is not possible to make direct comparisons between studies even when the same questionnaires are used, the improvement in PBC for people taking tolterodine ER in the STAR study was similar to that seen in the previous 12-week study by Freeman et al.12

image

Figure 4. Change in patient perception of bladder condition during the 12-week STAR study. Endpoint represents the intention to treat analysis rather than those completing the study. NS, not significant.

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The urgency perception scale (UPS) is a validated scale for the assessment of urinary urgency in clinical studies of OAB treatments.38 People are asked to describe their typical experience when they feel the desire to void. The sensitivity of the UPS to detect small patient-perceived change is uncertain, and may be due to its limited response options (three-point scale). The UPS and overall treatment effect questionnaire (OTE, a 14-point scale) were administered to those taking part in the Detrol in the community study, which was an open-label, observational study of people commencing tolterodine for OAB. The responses to both questionnaires showed that 53% of participants reported no change in their condition when completing the UPS; however, 73% of them reported improvements in their condition when completing the OTE. Several people suggested increasing the number of response options of the UPS to detect small clinical changes. The UPS is being further refined to address this issue.

The benefit, satisfaction and willingness to continue (BSW) questionnaire39 is a validated questionnaire designed to capture people’s perception of the effect of their treatment in terms of the relative benefit, patient satisfaction and their intention or willingness to continue the therapy. The questionnaire consists of three items; the perception of treatment benefit question asks people if they perceived a benefit from treatment. If the person answers ‘yes’, the following question asks if the perceived benefit was of ‘little benefit’ or ‘much benefit’. The satisfaction and willingness to continue questions are also structured in the same fashion. The satisfaction question also addresses dissatisfaction with treatment. The willingness to continue item has six possible responses.

When considering benefit, satisfaction and intent to continue with an intervention, patients balance complex issues such as the degree of symptom relief, life impact, adverse effects and the costs of medication. The BSW questionnaire provides information on perceived outcome and future intent that aid in the assessment of treatment efficacy, patient impact and future intent. The BSW can help clinicians to gain an understanding of how an individual patient values their treatment.

A simple questionnaire measuring patient perception of treatment benefit was used in the STAR study.37 This was a simple three-point scale asking people to rate the benefit they perceived from therapy. The questionnaire showed greater perception of benefit for people taking solifenacin than for those taking ER tolterodine (Figure 5).

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Figure 5. Assessment of treatment benefit from the STAR study.

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The patient global impression of severity scale (PGI-S) asks people to rate ‘how their urinary tract condition is now’ on a four-point scale ranging from ‘normal’ to ‘severe’.40 The patient global impression of improvement scale (PGI-I) has been designed for use in clinical trials to assess perceived benefits of treatment. The PGI-I asks people to describe how their urinary tract condition is now, compared with how it was before they began taking medication. The responses are graded on a seven-point scale ranging from ‘very much better’ to ‘very much worse’. Both PGI-S and PGI-I have been validated for use in women with predominant stress urinary symptoms.

Screening questionnaires

  1. Top of page
  2. Abstract
  3. Health status and QoL questionnaires
  4. Generic instruments
  5. Disease/condition-specific QoL questionnaires
  6. Screening questionnaires
  7. Individualising expectations and goals of treatment
  8. Defining successful treatment outcome for OAB
  9. References

Screening is an effective method of raising awareness of important and prevalent health conditions. Embarrassment and uncertainty as to whether lower urinary tract symptoms are important or treatable can prevent people from seeking help. Often people may not present to a healthcare professional until their symptoms become very severe, or a trigger [urinary tract infection (UTI), immobility, long distance travelling etc.] prevents established coping strategies from controlling their symptoms. The goal of screening for lower urinary tract symptoms is early intervention and treatment. Identification of people with early or less troublesome symptoms may help us to understand the aetiology and natural history of the condition and improve the efficacy of subsequent treatment.

The overactive bladder awareness tool (OAB-V8)15 is a validated instrument, which consists of the first eight items of the OABq. The eight items ask people to report bother associated with the following symptoms: urgency, incontinence, nocturia and frequency. A commonly reported finding of users of this questionnaire is the apparent similarity of many of the questions, and uncertainty as to whether the same symptom is being measured repeatedly. This problem is a result of the difficulty in defining urgency and therefore expressing the symptom to people in an easily interpretable manner.

Screening questionnaires will, however, have an important future role to identify people with OAB who would benefit from treatment but who for many reasons may be reluctant to seek help.

Individualising expectations and goals of treatment

  1. Top of page
  2. Abstract
  3. Health status and QoL questionnaires
  4. Generic instruments
  5. Disease/condition-specific QoL questionnaires
  6. Screening questionnaires
  7. Individualising expectations and goals of treatment
  8. Defining successful treatment outcome for OAB
  9. References

A very clear message from all of the studies that have assessed the QoL impact and benefit of treatment for people with OAB is the very personal nature of this condition. The same symptoms affect different people in diverse ways, and while the aim of treatment may be the same, the individual expectations and treatment goals will differ. Individualising treatment is something we intuitively strive for in clinical practice. Ultimately, the success of interventions will always be measured by what people expect them to do. For this reason, it is important to offer realistic goals when treating people with OAB so that their expectations are achieved, and treatment will be successful. For many people, the ultimate aim may be to control symptoms rather than cure them and long-term therapy may be required to achieve this. Measuring goals and expectations in clinical trials is at present complex and cumbersome. A study by Elkadry et al.41 showed that many people undergoing pelvic floor and continence surgery did not fulfil their goals of treatment even when the surgery appeared to be successful. In their study, 90% of participants had an objective cure of their prolapse and 74% an objective cure of their urinary symptoms. Over 50% of them had at least one unmet goal of their treatment, and there was a relatively poor correlation between objective surgical cure and goal fulfilment.

Defining successful treatment outcome for OAB

  1. Top of page
  2. Abstract
  3. Health status and QoL questionnaires
  4. Generic instruments
  5. Disease/condition-specific QoL questionnaires
  6. Screening questionnaires
  7. Individualising expectations and goals of treatment
  8. Defining successful treatment outcome for OAB
  9. References

At present, the best measure of treatment outcome for OAB is unclear. OAB is defined by the presence of urgency, and measuring improvement in urgency is therefore crucial. Urgency is, however, difficult to define and to measure due to its highly subjective nature. Various ways of measuring urgency have been used but none is perfect. Frequency nocturia and urgency incontinence episodes are easier to measure, but it is not certain what magnitude of improvement is important to patients.

QoL assessment clearly has a major role to play in the treatment evaluation of OAB, but questionnaires are complex and not easy to apply in clinical practice. Simple global measures of bladder condition and treatment benefit are transparent, simple to complete and to interpret, but fail to offer an in depth understanding of the multidimensional impairment that OAB causes.

Ultimately, it is likely that the best approach for the present time will be to use a combination of all the available tools. Only by this means can we be certain that a patient’s symptoms and perception of their OAB are improved, their QoL is better and they feel that treatment has been beneficial. Although complex, this is undoubtedly at present the best way to understand the impact of OAB and gauge the success or otherwise of therapy.

References

  1. Top of page
  2. Abstract
  3. Health status and QoL questionnaires
  4. Generic instruments
  5. Disease/condition-specific QoL questionnaires
  6. Screening questionnaires
  7. Individualising expectations and goals of treatment
  8. Defining successful treatment outcome for OAB
  9. References