scholarly journals A randomised controlled trial to compare the safety, effectiveness and cost-effectiveness of doxycycline (200 mg/day) with that of oral prednisolone (0.5 mg/kg/day) for initial treatment of bullous pemphigoid: the Bullous Pemphigoid Steroids and Tetracyclines (BLISTER) trial

2017 ◽  
Vol 21 (10) ◽  
pp. 1-90 ◽  
Author(s):  
Joanne R Chalmers ◽  
Fenella Wojnarowska ◽  
Gudula Kirtschig ◽  
James Mason ◽  
Margaret Childs ◽  
...  
Keyword(s):  
Economic Evaluation ◽  
Bullous Pemphigoid ◽  
Controlled Trial ◽  
Oral Prednisolone ◽  
Health Technology ◽  
Short Term ◽  
Life Threatening ◽  
Secondary Outcomes ◽  
Randomised Controlled

BackgroundBullous pemphigoid (BP) is an autoimmune blistering skin disorder with increased morbidity and mortality in the elderly.ObjectivesTo evaluate the effectiveness, safety and cost-effectiveness of a strategy of initiating BP treatment with oral doxycycline or oral prednisolone. We hypothesised that starting treatment with doxycycline gives acceptable short-term blister control while conferring long-term safety advantages over starting treatment with oral prednisolone.DesignPragmatic multicentre two-armed parallel-group randomised controlled trial with an economic evaluation.SettingA total of 54 dermatology secondary care centres in the UK and seven in Germany.ParticipantsAdults with BP [three or more blisters at two sites and positive direct and/or indirect immunofluorescence (immunoglobulin G and/or complement component 3 immunofluorescence at the dermal-epidermal junction)] and able to give informed consent.InterventionsParticipants were allocated using online randomisation to initial doxycycline treatment (200 mg/day) or prednisolone (0.5 mg/kg/day). Up to 30 g/week of potent topical corticosteroids was permitted for weeks 1–3. After 6 weeks, clinicians could switch treatments or alter the prednisolone dose as per normal practice.Main outcome measuresPrimary outcomes: (1) the proportion of participants with three or fewer blisters at 6 weeks (investigator blinded) and (2) the proportion with severe, life-threatening and fatal treatment-related events at 52 weeks. A regression model was used in the analysis adjusting for baseline disease severity, age and Karnofsky score, with missing data imputed. Secondary outcomes included the effectiveness of blister control after 6 weeks, relapses, related adverse events and quality of life. The economic evaluation involved bivariate regression of costs and quality-adjusted life-years (QALYs) from a NHS perspective.ResultsIn total, 132 patients were randomised to doxycycline and 121 to prednisolone. The mean patient age was 77.7 years and baseline severity was as follows: mild 31.6% (three to nine blisters), moderate 39.1% (10–30 blisters) and severe 29.3% (> 30 blisters). For those starting on doxycycline, 83 out of 112 (74.1%) had three or fewer blisters at 6 weeks, whereas for those starting on prednisolone 92 out of 101 (91.1%) had three or fewer blisters at 6 weeks, an adjusted difference of 18.6% in favour of prednisolone [90% confidence interval (CI) 11.1% to 26.1%], using a modified intention-to-treat (mITT) analysis. Per-protocol analysis showed similar results: 74.4% compared with 92.3%, an adjusted difference of 18.7% (90% CI 9.8% to 27.6%). The rate of related severe, life-threatening and fatal events at 52 weeks was 18.2% for those started on doxycycline and 36.6% for those started on prednisolone (mITT analysis), an adjusted difference of 19.0% (95% CI 7.9% to 30.1%;p = 0.001) in favour of doxycycline. Secondary outcomes showed consistent findings. There was no significant difference in costs or QALYs per patient at 1 year between doxycycline-initiated therapy and prednisolone-initiated therapy (incremental cost of doxycycline-initiated therapy £959, 95% CI –£24 to £1941; incremental QALYs of doxycycline-initiated therapy –0.024, 95% CI –0.088 to 0.041). Using a willingness-to-pay criterion of < £20,000 per QALY gained, the net monetary benefit associated with doxycycline-initiated therapy was negative but imprecise (–£1432, 95% CI –£3094 to £230).ConclusionsA strategy of starting BP patients on doxycycline is non-inferior to standard treatment with oral prednisolone for short-term blister control and considerably safer in the long term. The limitations of the trial were the wide non-inferiority margin, the moderate dropout rate and that serious adverse event collection was unblinded. Future work might include inducing remission with topical or oral corticosteroids and then randomising to doxycycline or prednisolone for maintenance.Trial registrationCurrent Controlled Trials ISRCTN13704604.FundingThis project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full inHealth Technology Assessment; Vol. 21, No. 10. See the NIHR Journals Library website for further project information.

scholarly journals A feasibility randomised controlled trial of a motivational interviewing-based intervention for weight loss maintenance in adults

2015 ◽  
Vol 19 (50) ◽  
pp. 1-378 ◽  
Author(s):  
Sharon A Simpson ◽  
Rachel McNamara ◽  
Christine Shaw ◽  
Mark Kelson ◽  
Yvonne Moriarty ◽  
...  
Keyword(s):  
Weight Loss ◽  
Cost Effectiveness ◽  
Randomised Controlled Trial ◽  
Controlled Trial ◽  
Health Technology ◽  
Face To Face ◽  
Intensive Intervention ◽  
Secondary Outcomes ◽  
Randomised Controlled

BackgroundObesity has significant health and NHS cost implications. Relatively small reductions in weight have clinically important benefits, but long-term weight loss maintenance (WLM) is challenging. Behaviour change interventions have been identified as key for WLM. Motivation is crucial to supporting behaviour change, and motivational interviewing (MI) has been identified as a successful approach to changing health behaviours. The study was designed as an adequately powered, pragmatic randomised controlled trial (RCT); however, owing to recruitment issues, the study became a feasibility trial.ObjectivesTo assess recruitment, retention, feasibility, acceptability, compliance and delivery of a 12-month intervention to support WLM. Secondary objectives were to assess the impact of the intervention on body mass index (BMI) and other secondary outcomes.DesignThree-arm individually randomised controlled trial comprising an intensive arm, a less intensive arm and a control arm.SettingCommunity setting in South Wales and the East Midlands.ParticipantsIndividuals aged 18–70 years with a current or previous BMI of ≥ 30 kg/m2who could provide evidence of at least 5% weight loss during the previous 12 months.InterventionParticipants received individually tailored MI, which included planning and self-monitoring. The intensive arm received six face-to-face sessions followed by nine telephone sessions. The less intensive arm received two face-to-face sessions followed by two telephone sessions. The control arm received a leaflet advising them on healthy lifestyle.Main outcome measuresFeasibility outcomes included numbers recruited, retention and adherence. The primary effectiveness outcome was BMI at 12 months post randomisation. Secondary outcomes included waist circumference, waist-to-hip ratio, physical activity, proportion maintaining weight loss, diet, quality of life, health service resource usage, binge eating and well-being. A process evaluation assessed intervention delivery, adherence, and participants’ and practitioners’ views. Economic analysis aimed to assess cost-effectiveness in terms of quality-adjusted life-years (QALYs).ResultsA total of 170 participants were randomised. Retention was good (84%) and adherence was excellent (intensive, 83%; less intensive, 91%). The between-group difference in mean BMI indicated the intensive arm had BMIs 1.0 kg/m2lower than the controls [95% confidence interval (CI) –2.2 kg/m2to 0.2 kg/m2]. Similarly, a potential difference was found in weight (average difference of 2.8 kg, 95% CI –6.1 kg to 0.5 kg). The intensive arm had odds of maintaining on average 43% [odds ratio(OR) 1.4, 95% CI 0.6 to 3.5] higher than controls. None of these findings were statistically significant. Further analyses controlling for level of adherence indicated that average BMI was 1.2 kg/m2lower in the intensive arm than the control arm (95% CI –2.5 kg/m2to 0.0 kg/m2). The intensive intervention led to a statistically significant difference in weight (mean –3.7 kg, 95% CI –7.1 kg to –0.3 kg). The other secondary outcomes showed limited evidence of differences between groups. The intervention was delivered as planned, and both practitioners and participants were positive about the intervention and its impact. Although not powered to assess cost-effectiveness, results of this feasibility study suggest that neither intervention as currently delivered is likely to be cost-effective in routine practice.ConclusionThis is the first trial of an intervention for WLM in the UK, the intervention is feasible and acceptable, and retention and adherence were high. The main effectiveness outcome showed a promising mean difference in the intensive arm. Owing to the small sample size, we are limited in the conclusions we can draw. However, findings suggest that the intensive intervention may facilitate long-term weight maintenance and, therefore, further testing in an effectiveness trial may be indicated. Research examining WLM is in its infancy, further research is needed to develop our understanding of WLM and to expand theory to inform the development of interventions to be tested in rigorously designed RCTs with cost-effectiveness assessed.Trial registrationCurrent Controlled Trials ISRCTN35774128.FundingThis project was funded by the National Institute for Health Research Health Technology Assessment programme and will be published in full inHealth Technology Assessment; Vol. 19, No. 50. See the NIHR Journals Library website for further project information.

Snoezelen: Its Long-Term and Short-Term Effects on Older People with Dementia

1997 ◽  
Vol 60 (5) ◽  
pp. 213-218 ◽  
Author(s):  
Roger Baker ◽  
Zena Dowling ◽  
Lesley Ann Wareing ◽  
Jeannette Dawson ◽  
Julian Assey
Keyword(s):  
Controlled Trial ◽  
Hospital Setting ◽  
Short Term ◽  
Home Setting ◽  
People With Dementia ◽  
Significant Difference ◽  
Post Trial ◽  
Randomised Controlled ◽  
Short Term Effects

A randomised controlled trial was conducted to investigate the long-term and short-term effects of the Snoezelen environment on the behaviour, mood and cognition of elderly patients with dementia, and to gain an understanding of the processes occurring within Snoezelen. Patients participated in either eight Snoezelen or eight activity sessions. Pre-trial, mid-trial, post-trial and follow-up assessments were carried out at home and at the day hospital. Ratings of behaviour and mood were also made before, during and after each session. In the long term, the main benefits for Snoezelen patients were in the domain of socially disturbed behaviour. In the home setting, there was a highly significant difference between the two groups in favour of Snoezelen, and in the hospital setting it was nearly significant. Short-term improvements in behaviour and mood were evident for both groups after sessions, and communication was significantly better during Snoezelen sessions in comparison with activity sessions. The processes occurring within Snoezelen sessions seemed to involve facilitation of verbal expression and memory recall.

scholarly journals Clinical effectiveness and cost-effectiveness of body psychotherapy in the treatment of negative symptoms of schizophrenia: a multicentre randomised controlled trial

2016 ◽  
Vol 20 (11) ◽  
pp. 1-100 ◽  
Author(s):  
Stefan Priebe ◽  
Mark Savill ◽  
Til Wykes ◽  
Richard Bentall ◽  
Christoph Lauber ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Negative Symptoms ◽  
Controlled Trial ◽  
Clinical Effectiveness ◽  
Health Technology ◽  
Body Psychotherapy ◽  
End Of Treatment ◽  
Secondary Outcomes ◽  
Randomised Controlled

BackgroundThe negative symptoms of schizophrenia significantly impact on quality of life and social functioning, and current treatment options are limited. In this study the clinical effectiveness and cost-effectiveness of group body psychotherapy as a treatment for negative symptoms were compared with an active control.DesignA parallel-arm, multisite randomised controlled trial. Randomisation was conducted independently of the research team, using a 1 : 1 computer-generated sequence. Assessors and statisticians were blinded to treatment allocation. Analysis was conducted following the intention-to-treat principle. In the cost-effectiveness analysis, a health and social care perspective was adopted.ParticipantsEligibility criteria: age 18–65 years; diagnosis of schizophrenia with symptoms present at > 6 months; score of ≥ 18 on Positive and Negative Syndrome Scale (PANSS) negative symptoms subscale; no change in medication type in past 6 weeks; willingness to participate; ability to give informed consent; and community outpatient. Exclusion criteria: inability to participate in the groups and insufficient command of English.SettingsParticipants were recruited from NHS mental health community services in five different Trusts. All groups took place in local community spaces.InterventionsControl intervention: a 10-week, 90-minute, 20-session group beginners’ Pilates class, run by a qualified Pilates instructor. Treatment intervention: a 10-week, 90-minute, 20-session manualised group body psychotherapy group, run by a qualified dance movement psychotherapist.OutcomesThe primary outcome was the PANSS negative symptoms subscale score at end of treatment. Secondary outcomes included measures of psychopathology, functional, social, service use and treatment satisfaction outcomes, both at treatment end and at 6-month follow-up.ResultsA total of 275 participants were randomised (140 body psychotherapy group, 135 Pilates group). At the end of treatment, 264 participants were assessed (137 body psychotherapy group, 127 Pilates group). The adjusted difference in means of the PANSS negative subscale at the end of treatment was 0.03 [95% confidence interval (CI) –1.11 to 1.17], showing no advantage of the intervention. In the secondary outcomes, the mean difference in the Clinical Assessment Interview for negative symptoms expression subscale at the end of treatment was 0.62 (95% CI –1.23 to 0.00), and in extrapyramidal movement disorder symptoms –0.65 (95% CI –1.13 to –0.16) at the end of treatment and –0.58 (95% CI –1.07 to –0.09) at 6 months’ follow-up, showing a small significant advantage of body psychotherapy. No serious adverse events related to the interventions were reported. The total costs of the intervention were comparable with the control, with no clear evidence of cost-effectiveness for either condition.LimitationsOwing to the absence of a treatment-as-usual arm, it is difficult to determine whether or not both arms are an improvement over routine care.ConclusionsIn comparison with an active control, group body psychotherapy does not have a clinically relevant beneficial effect in the treatment of patients with negative symptoms of schizophrenia. These findings conflict with the review that led to the current National Institute for Health and Care Excellence guidelines suggesting that arts therapies may be an effective treatment for negative symptoms.Future workDetermining whether or not this lack of effectiveness extends to all types of art therapies would be informative.Trial registrationCurrent Controlled Trials ISRCTN842165587.FundingThis project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full inHealth Technology Assessment; Vol. 20, No. 11. See the NIHR Journals Library website for further project information.

scholarly journals The second Randomised Evaluation of the Effectiveness, cost-effectiveness and Acceptability of Computerised Therapy (REEACT-2) trial: does the provision of telephone support enhance the effectiveness of computer-delivered cognitive behaviour therapy? A randomised controlled trial

2016 ◽  
Vol 20 (89) ◽  
pp. 1-64 ◽  
Author(s):  
Sally Brabyn ◽  
Ricardo Araya ◽  
Michael Barkham ◽  
Peter Bower ◽  
Cindy Cooper ◽  
...  
Keyword(s):  
Controlled Trial ◽  
Clinical Effectiveness ◽  
Health Technology ◽  
Trial Period ◽  
Behaviour Therapy ◽  
Cognitive Behaviour ◽  
Patient Health ◽  
Secondary Outcomes ◽  
Randomised Controlled

BackgroundComputerised cognitive behaviour therapy (cCBT) is an efficient form of therapy potentially improving access to psychological care. Indirect evidence suggests that the uptake and effectiveness of cCBT can be increased if facilitated by telephone, but this is not routinely offered in the NHS.ObjectivesTo compare the clinical effectiveness and cost-effectiveness of telephone-facilitated free-to-use cCBT [e.g. MoodGYM (National Institute for Mental Health Research, Australian National University, Canberra, ACT, Australia)] with minimally supported cCBT.DesignThis study was a multisite, pragmatic, open, two-arm, parallel-group randomised controlled trial with a concurrent economic evaluation.SettingParticipants were recruited from GP practices in Bristol, Manchester, Sheffield, Hull and the north-east of England.ParticipantsPotential participants were eligible to participate in the trial if they were adults with depression scoring ≥ 10 on the Patient Health Questionnaire-9 (PHQ-9).InterventionsParticipants were randomised using a computer-generated random number sequence to receive minimally supported cCBT or telephone-facilitated cCBT. Participants continued with usual general practitioner care.Main outcome measuresThe primary outcome was self-reported symptoms of depression, as assessed by the PHQ-9 at 4 months post randomisation.Secondary outcomesSecondary outcomes were depression at 12 months and anxiety, somatoform complaints, health utility (as assessed by the European Quality of Life-5 Dimensions questionnaire) and resource use at 4 and 12 months.ResultsClinical effectiveness: 182 participants were randomised to minimally supported cCBT and 187 participants to telephone-facilitated cCBT. There was a difference in the severity of depression at 4 and 12 months, with lower levels in the telephone-facilitated group. The odds of no longer being depressed (defined as a PHQ-9 score of < 10) at 4 months were twice as high in the telephone-facilitated cCBT group [odds ratio (OR) 2.05, 95% confidence interval (CI) 1.23 to 3.42]. The benefit of telephone-facilitated cCBT was no longer significant at 12 months (OR 1.63, 95% CI 0.98 to 2.71). At 4 months the between-group difference in PHQ-9 scores was 1.9 (95% CI 0.5 to 3.3). At 12 months the results still favoured telephone-facilitated cCBT but were no longer statistically significant, with a difference in PHQ-9 score of 0.9 (95% CI –0.5 to 2.3). When considering the whole follow-up period, telephone-facilitated cCBT was asssociated with significantly lower PHQ-9 scores than minimally supported cCBT (mean difference –1.41, 95% CI –2.63 to –0.17;p = 0.025). There was a significant improvement in anxiety scores over the trial period (between-group difference 1.1, 95% CI 0.1 to 2.3;p = 0.037). In the case of somatic complaints (assessed using the Patient Health Questionnaire-15), there was a borderline statistically significant difference over the trial period (between-group difference 1.1, 95% CI 0.0 to 1.8;p = 0.051). There were gains in quality-adjusted life-years at reduced cost when telephone facilitation was added to MoodGYM. However, the results were subject to uncertainty.ConclusionsThe results showed short-term benefits from the addition of telephone facilitation to cCBT. The effect was small to moderate and comparable with that of other primary care psychological interventions. Telephone facilitation should be considered when offering cCBT for depression.LimitationsParticipants’ depression was assessed with the PHQ-9, cCBT use was quite low and there was a slightly greater than anticipated loss to follow-up.Future research recommendationsImprove the acceptability of cCBT and its capacity to address coexisting disorders. Large-scale pragmatic trials of cCBT with bibliotherapy and telephone-based interventions are required.Trial registrationCurrent Controlled Trials ISRCTN55310481.FundingThis project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full inHealth Technology Assessment; Vol. 20, No. 89. See the NIHR Journals Library website for further project information.

scholarly journals Cost-effectiveness of exercise referral schemes enhanced by self-management strategies to battle sedentary behaviour in older adults: protocol for an economic evaluation alongside the SITLESS three-armed pragmatic randomised controlled trial

BMJ Open ◽  
2018 ◽  
Vol 8 (10) ◽  
pp. e022266 ◽  
Author(s):  
Manuela Deidda ◽  
Laura Coll-Planas ◽  
Maria Giné-Garriga ◽  
Míriam Guerra-Balic ◽  
Marta Roqué i Figuls ◽  
...  
Keyword(s):  
Economic Evaluation ◽  
Cost Effectiveness ◽  
Controlled Trial ◽  
Management Strategies ◽  
Research Committee ◽  
Horizon 2020 ◽  
Reference Number ◽  
Cost Consequence ◽  
Randomised Controlled

IntroductionPromoting physical activity (PA) and reducing sedentary behaviour (SB) may exert beneficial effects on the older adult population, improving behavioural, functional, health and psychosocial outcomes in addition to reducing health, social care and personal costs. This paper describes the planned economic evaluation of SITLESS, a multicountry three-armed pragmatic randomised controlled trial (RCT) which aims to assess the short-term and long-term effectiveness and cost-effectiveness of a complex intervention on SB and PA in community-dwelling older adults, based on exercise referral schemes enhanced by a group intervention providing self-management strategies to encourage lifestyle change.Methods and analysisA within-trial economic evaluation and long-term model from both a National Health Service/personal social services perspective and a broader societal perspective will be undertaken alongside the SITLESS multinational RCT. Healthcare costs (hospitalisations, accident and emergency visits, appointment with health professionals) and social care costs (eg, community care) will be included in the economic evaluation. For the cost-utility analysis, quality-adjusted life-years will be measured using the EQ-5D-5L and capability well-being measured using the ICEpop CAPability measure for Older people (ICECAP-O) questionnaire. Other effectiveness outcomes (health related, behavioural, functional) will be incorporated into a cost-effectiveness analysis and cost-consequence analysis.The multinational nature of this RCT implies a hierarchical structure of the data and unobserved heterogeneity between clusters that needs to be adequately modelled with appropriate statistical and econometric techniques. In addition, a long-term population health economic model will be developed and will synthesise and extrapolate within-trial data with additional data extracted from the literature linking PA and SB outcomes with longer term health states.Methods guidance for population health economic evaluation will be adopted including the use of a long-time horizon, 1.5% discount rate for costs and benefits, cost consequence analysis framework and a multisector perspective.Ethics and disseminationThe study design was approved by the ethics and research committee of each intervention site: the Ethics and Research Committee of Ramon Llull University (reference number: 1314001P) (Fundació Blanquerna, Spain), the Regional Committees on Health Research Ethics for Southern Denmark (reference number: S-20150186) (University of Southern Denmark, Denmark), Office for Research Ethics Committees in Northern Ireland (ORECNI reference number: 16/NI/0185) (Queen’s University of Belfast) and the Ethical Review Board of Ulm University (reference number: 354/15) (Ulm, Germany). Participation is voluntary and all participants will be asked to sign informed consent before the start of the study.This project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement number 634 270. This article reflects only the authors' view and the Commission is not responsible for any use that may be made of the information it contains.The findings of the study will be disseminated to different target groups (academia, policymakers, end users) through different means following the national ethical guidelines and the dissemination regulation of the Horizon 2020 funding agency.Use of the EuroQol was registered with the EuroQol Group in 2016.Use of the ICECAP-O was registered with the University of Birmingham in March 2017.Trial registration numberNCT02629666; Pre-results.

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