Title: Multicentre randomised trial of the effectiveness and cost-effectiveness of basic versus biofeedback-mediated intensive pelvic floor muscle training for female stress or mixed urinary incontinence (OPAL-Optimal PFMT for Adherence Long-term)
Funder: Chief Scientist Office, Scotland

Abstract: DESIGN: •A multicentre, 2-arm randomised controlled trial to compare effectiveness and cost-effectiveness of basic PFMT versus biofeedback-mediated intensive PFMT, with a mixed-methods, nested process evaluation. •An interview study (two-tailed case study) with purposively selected women from both trial arms to explore experience and adherence. SETTING: UK community and outpatient settings where women are referred for the treatment of UI. To date, 14 centres we have collaborated with previously have agreed in principle to recruit. TARGET POPULATION: Women >=18 years with a new episode of stress or mixed UI. Excluded are those who: have urge UI only; have had formal PFMT or UI surgery in the last 3 years; are pregnant or <1 year postnatal; have prolapse >stage II, pelvic cancer, cognitive impairment or neurological disease; are taking antimuscarinics (unless discontinued for 6 weeks). HEALTH TECHNOLOGIES BEING ASSESSED: 1) Basic PFMT (6 appointments at weeks 0,1,3,6,10,15); 2) intensive PFMT (same appointments as basic PFMT, with use of biofeedback at all appointments, and home biofeedback during weeks 1-15). PFMT is the repetitive contraction of the pelvic floor muscles, to improve muscle function and ultimately continence. A therapist assesses the muscles and prescribes exercises; increasing repetitions, lengthening contractions and reducing rest periods over time. National guidelines recommend at least 3 month’s of PFMT as treatment for stress UI; average of 6 appointments. Basic PFMT is defined here as 6 appointments over 16 weeks, in line with recent evidence indicating that at least 15 weeks is needed to achieve muscle hypertrophy [American College of Sports Medicine 2011]. Biofeedback is the feedback of information to a woman so that she can identify and modify the action of her pelvic floor muscles. A vaginal probe measures electrical activity during muscle contraction (EMG) which is displayed on a screen. The woman uses this information as confirmation of correct contraction technique (teaching); to increase strength or timing of contractions (modulating); and to improve exercise adherence (encouraging). In the intensive PFMT arm, in addition to basic PFMT, EMG biofeedback is used at each appointment to teach, modulate and encourage; and home EMG biofeedback to reinforce and encourage effective exercising between appointments (weeks 1-15). No biofeedback equipment will be used in the basic PFMT arm. Verbal feedback during digital palpation will be permitted in both trial arms. A women's health physiotherapist or continence nurse will deliver the PFMT interventions. The proposed mechanism of action is that biofeedback will increase women’s self-efficacy for PFMT and hence their adherence, leading to improved treatment outcomes. MEASUREMENT OF COST AND OUTCOMES: • Primary outcome: UI severity at 24 months (ICIQ-UI score [Avery 2004]–leakage frequency/amount/interference, scored 0-21). • Secondary outcomes: number of episodes of UI per day (recorded by women in a leakage diary), number with cured/improved UI, other urinary symptoms (ICIQ-FLUTS), UI-specific quality of life (ICIQ-UIqol), self-efficacy for PFMT (PFME self-efficacy scale), impression of global improvement in UI (PGI-I), adherence to exercise, uptake of surgery and other UI treatment, pelvic floor muscle function. Data will be collected via participant-completed postal questionnaires at baseline, 6, 12 and 24 months. At baseline and 24 months: recording of leakage episodes and PFMT adherence by women in daily diaries. At baseline and 6 months: blinded assessment of pelvic floor muscle function to quantify muscle change. • Primary health economic outcome: incremental cost per quality adjusted life year gained at 24 months. EQ-5D responses will be used to estimate QALYs, then the incremental cost-effectiveness ratio for 24 month follow-up. Sensitivity analyses will take into account any uncertainties. Secondary economic outcomes: resource use and costs to the NHS, women and their families; EQ-5D scores over 24 months. Cost and QALY outcomes will be extrapolated over a woman’s lifetime if there are significant differences between trial arms.

Lay abstract: Accidental leakage of urine (incontinence) is a distressing problem that affects about 1 in 3 women, and the NHS spends considerable amounts of money treating it. Many women have stress incontinence: leakage that happens with coughing, sneezing and physical activity. Based on previous research, current UK guidelines recommend that these women are offered at least three months of pelvic floor muscle exercises. These exercises are taught by a specialist physiotherapist or nurse. There is evidence that these exercises can work to strengthen the muscles and decrease leakage, but it is not clear how ‘intensively’ women have to exercise to get a good result that lasts thus improving the woman's quality of life and reducing the likelihood of surgery. This research aims to find out whether the use of biofeedback can help to improve the results of the exercise teaching in both the short- and longer-term. Using a probe inserted into the vagina, biofeedback equipment shows women what their pelvic floor muscles do. Because the muscles are ‘inside’ or ‘hidden’, they cannot usually be seen, but biofeedback allows women to “see” the muscles working as they exercise. Knowing that the muscles are working correctly, through ‘seeing’ them on the biofeedback screen, is important for teaching and encouraging women to continue doing their exercises. A graph can be printed out as a record, for comparing as the treatment progresses. The women who receive biofeedback may exercise more because they are more confident about doing the exercises and the changes that the exercises can achieve. Women referred to one of the participating study centres for incontinence treatment for the first time will be asked to take part. Those who agree will be allocated at random to one of two groups, either the usual exercise programme recommended by the UK guidelines (called basic exercise) or these same exercises with the addition of biofeedback (called intensive exercise). Both groups will visit a specialist nurse or physiotherapist 6 times in 16 weeks to teach and encourage the exercises. The intensive exercise group will also have biofeedback at each appointment. At the 2nd appointment women in the intensive exercise group will also be given a simple, portable biofeedback machine so they can monitor the exercises themselves at home. We will measure the results of treatment after 6 months, 1 and 2 years. We are primarily interested in whether women in the intensive exercise group have had more improvement in their incontinence at 2 years after they start the study than the basic exercise group. We also want to find out how much urine leakage women in both groups have, how much this impacts on their lives, what other bladder problems they have, what other treatments they have had, how much exercise they did, how confident they were and how much their muscles have strengthened. We will also measure the costs of the treatments and any costs to the woman and her family, and balance these against any benefits of the intensive treatment. During the trial we will assess how well the exercise programmes were delivered by speaking with physiotherapists and nurses and listening to recordings of some treatment sessions. We will talk with women to find out how they perceive treatment and how they get on with exercising during the treatment period and once treatment is finished. We will then explore how the treatment delivery and women’s perceptions impact on the women’s incontinence. We have worked out from previous research that if 600 women take part and most complete the trial, we will have enough data to successfully compare the two treatments to find out if one is better than the other. Women's participation will be entirely voluntary and we do not believe there are any risks associated with taking part. The research team is well-placed to undertake the work. It contains both academic and clinical researchers with experience of undertaking multicentre trials of complex clinical interventions, many in the field of incontinence and exercise treatments, of undertaking research using a mix of research methods, of evaluating cost-effectiveness, and of involving consumers in research. At a cost of £1.9M this research will inform future UK spending on pelvic floor exercise interventions which currently costs around £38M per year.

Research activity codes: 6.3 MEDICAL DEVICES;6.7 PHYSICAL
Health categories: RENAL AND UROGENITAL