Reflecting on the methodological challenges of recruiting to a United Kingdom-wide, multi-centre, randomised controlled trial in gynaecology outpatient settings

Peer reviewedPublisher PD

Individualised pelvic floor muscle training in women with pelvic organ prolapse: a multicentre randomised controlled trial

<br>Background: Pelvic organ prolapse is common and is strongly associated with childbirth and increasing age. Women with prolapsed are often advised to do pelvic floor muscle exercises, but supporting evidence is limited. Our aim was to establish if one-to-one individualised pelvic floor muscle training (PFMT) is effective in reducing prolapse symptoms.</br> <br>Methods: A parallel‐group multicentre randomised controlled trial (ISRCTN35911035) in female outpatients with newly-diagnosed, symptomatic stage I, II or III prolapse, comparing five PFMT appointments over 16 weeks (n=225) versus a lifestyle advice leaflet (n=222). Treatment allocation was by remote computer allocation using minimisation. Our primary endpoint was participants’ self-report of prolapsed symptoms at 12 months. Group assignment was masked from outcome assessors. We compared outcomes between trial groups in an intention-to-treat analysis. The cost of PFMT and savings on subsequent treatments were calculated to estimate cost-effectiveness.</br> <br>Findings: Compared to the control group, the intervention group reported fewer prolapse symptoms at 12 months (mean difference between groups in change score 1.52, 95% CI [0.46, 2.59], p=0.0053); reported their prolapse to be “better” more often (57.2% versus 44.7%, difference 12.6%, 95% CI [1.1%, 24.1%], p=0.0336); and had an increased but non-significant odds of having less severe stage of prolapse at their 6-month clinical examination, (OR 1.47, 95% CI [0.97, 2.27], p=0.07). The control group had a greater uptake of other prolapse treatment (49.6% versus 24.1%, difference 25.5%, 95% CI [14.5%, 36.0%], p <0.0001). Findings were robust to missing data. The net cost of the 25 intervention was £131.61 per woman and the cost per one-point reduction in the symptom score was £86.59, 95% CI [£50.81, £286.11]. </br&gt

Lee Silverman Voice Treatment versus NHS Speech and Language Therapy versus control for dysarthria in Parkinson’s disease (PD COMM):a UK, multicentre, pragmatic, randomised controlled trial

Objectives: We aimed to assess the clinical effectiveness of two speech and language therapy (SLT) approaches versus no speech and language therapy for dysarthria in people with Parkinson’s disease. Design: This was a pragmatic, UK-wide, multicentre, three-arm, parallel group, unblinded, randomised controlled trial. Participants were randomly assigned using minimisation in a 1:1:1 ratio to Lee Silverman Voice Treatment (LSVT LOUD®), NHS SLT, or no SLT. Analyses were based on the intention to treat principle.Setting: The speech and language therapy interventions were delivered in outpatient or home settings.Participants: Between September 2016 and March 2020, 388 people with Parkinson’s disease and dysarthria were randomised into the trial: 130 to LSVT LOUD®, 129 to NHS SLT, and 129 to no SLT.Interventions: Lee Silverman Voice Treatment (LSVT LOUD®) consisted of four, face-to-face or remote, 50-minute sessions each week delivered over 4 weeks. Home-based practice activities were set for up to 5 to 10 minutes daily on treatment days and 15 minutes twice daily on non-treatment days. NHS Speech and language therapy (NHS SLT) dosage was determined by the local therapist in response to individual participants’ needs. Prior research suggested that NHS SLT participants would receive an average of one session per week over 6 to 8 weeks. Local practices for NHS SLT were accepted, except for those within the LSVT LOUD® protocol. Main outcome measures: The primary outcome was the self-reported Voice Handicap Index (VHI) total score at 3 months.Results: People randomised to LSVT LOUD® reported lower VHI scores at 3 months post-randomisation than those who were randomised to no SLT (-8·0 points (99%CI: -13·3 to -2·6); p = 0·0001). There was no evidence of a difference in VHI scores between NHS SLT and no SLT (1·7 points; (99%Cl: -3·8 to 7·1); p = 0·43). Patients randomised to LSVT LOUD® also reported lower VHI scores than those randomised to NHS SLT (-9·6 points; (99%CI: -14·9 to -4·4); p &lt; 0.0001). There were 93 adverse events (predominately vocal strain) in the LSVT LOUD® group, 46 in the NHS SLT group, and none in the no SLT group. There were no serious adverse events. Conclusions: LSVT LOUD® was more effective at reducing the participant reported impact of voice problems than no SLT and NHS SLT. NHS SLT showed no evidence of benefit compared to no SLT. Trial registration: The completed trial registration is ISRCTN12421382. Funding: NIHR HTA Programme, project number HTA 10/135/02. <br/