Efficacy and cost-effectiveness of therapist-guided internet-delivered behaviour therapy for children and adolescents with Tourette syndrome: study protocol for a single-blind randomised controlled trial

BackgroundTreatment guidelines recommend behaviour therapy (BT) for patients with Tourette syndrome (TS) and chronic tic disorder (CTD). However, BT is rarely accessible due to limited availability of trained therapists and long travel distances to specialist clinics. Internet-delivered BT has the potential of overcoming these barriers through remote delivery of treatment with minimal therapist support. In the current protocol, we outline the design and methods of a randomised controlled trial (RCT) evaluating an internet-delivered BT programme referred to as BIP TIC. The trial’s primary objective is to determine the clinical efficacy of BIP TIC for reducing tic severity in young people with TS/CTD, compared with an active control intervention. Secondary objectives are to investigate the 12-month durability of the treatment effects and to perform a health economic evaluation of the intervention.MethodsIn this single-blind superiority RCT, 220 participants (9–17 years) with TS/CTD throughout Sweden will be randomised to 10–12 weeks of either therapist-supported internet-delivered BT based on exposure with response prevention (BIP TIC) or therapist-supported internet-delivered education. Data will be collected at baseline, 3 and 5 weeks into the treatment, at post-treatment, and 3, 6, and 12 months post-treatment. The primary endpoint is the 3-month follow-up. The primary outcome is tic severity as measured by the Yale Global Tic Severity Scale – Total Tic Severity Score. Treatment response is operationalised as scores of “Very much improved” or “Much improved” on the Clinical Global Impression – Improvement scale, administered at the primary endpoint. Outcome assessors will be blind to treatment condition at all assessment points. A health economic evaluation of BIP TIC will be performed, both in the short term (primary endpoint) and the long term (12-month follow-up). There are no planned interim analyses.DiscussionParticipant recruitment started on 26 April 2019 and finished on 9 April 2021. The total number of included participants was 221. The final participant is expected to reach the primary endpoint in September 2021 and the 12-month follow-up in June 2022. Data analysis for the primary objective will commence after the last participant reaches the primary endpoint.Trial registrationClinicalTrials.gov NCT03916055. Registered on 16 April 2019

Therapist-Supported Internet-Delivered Exposure and Response Prevention for Children and Adolescents with Tourette Syndrome: A Randomized Clinical Trial

Importance: The availability of behavior therapy for individuals with Tourette syndrome (TS) and chronic tic disorder (CTD) is limited. Objective: To determine the efficacy and cost-effectiveness of internet-delivered exposure and response prevention (ERP) for children and adolescents with TS or CTD. Design, Setting, and Participants: This single-masked, parallel group, superiority randomized clinical trial with nationwide recruitment was conducted at a research clinic in Stockholm, Sweden. Out of 615 individuals assessed for eligibility, 221 participants meeting diagnostic criteria for TS or CTD and aged 9 to 17 years were included in the study. Enrollment began in April 2019 and ended in April 2021. Data were analyzed between October 2021 and March 2022. Interventions: Participants were randomized to 10 weeks of therapist-supported internet-delivered ERP for tics (111 participants) or to therapist-supported internet-delivered education for tics (comparator group, 110 participants). Main Outcomes and Measures: The primary outcome was change in tic severity from baseline to the 3-month follow-up as measured by the Total Tic Severity Score of the Yale Global Tic Severity Scale (YGTSS-TTSS). YGTSS-TTSS assessors were masked to treatment allocation. Treatment response was operationalized as a score of 1 ("Very much improved") or 2 ("Much improved") on the Clinical Global Impression-Improvement scale. Results: Data loss was minimal, with 216 of 221 participants (97.7%) providing primary outcome data. Among randomized participants (152 [68.8%] boys; mean [SD] age, 12.1 [2.3] years), tic severity improved significantly, with a mean reduction of 6.08 points on the YGTSS-TTSS in the ERP group (mean [SD] at baseline, 22.25 [5.60]; at 3-month follow-up, 16.17 [6.82]) and 5.29 in the comparator (mean [SD] at baseline, 23.01 [5.92]; at 3-month follow-up, 17.72 [7.11]). Intention-to-treat analyses showed that the 2 groups improved similarly over time (interaction effect, -0.53; 95% CI, -1.28 to 0.22; P =.17). Significantly more participants were classified as treatment responders in the ERP group (51 of 108 [47.2%]) than in the comparator group (31 of 108 [28.7%]) at the 3-month follow-up (odds ratio, 2.22; 95% CI, 1.27 to 3.90). ERP resulted in more treatment responders at little additional cost compared with structured education. The incremental cost per quality-adjusted life-year gained was below the Swedish willingness-to-pay threshold, at which ERP had a 66% to 76% probability of being cost-effective. Conclusions and Relevance: Both interventions were associated with clinically meaningful improvements in tic severity, but ERP led to higher response rates at little additional cost. Trial Registration: ClinicalTrials.gov identifier: NCT03916055

Assessing outcomes for cost-utility analysis in mental health interventions : mapping mental health specific outcome measure GHQ-12 onto EQ-5D-3L

Background: Many intervention-based studies aiming to improve mental health do not include a multi-attribute utility instrument (MAUI) that produces quality-adjusted life-years (QALYs) and it limits the applicability of the health economic analyses. This study aims to develop 'crosswalk' transformation algorithm between a measure for psychological distress General Health Questionnaire (GHQ-12) and MAUI EuroQoL (EQ-5D-3L). Methods: The study is based on a survey questionnaire sent to a random sample in four counties in Sweden in 2012. The survey included GHQ-12 and EQ-5D instruments, as well as a question about self-rated health. The EQ-5D index was calculated using the UK and the Swedish tariff values. Two OLS models were used to estimate the EQ-5D health state values using the GHQ-12 as exposure, based on the respondents (n = 17, 101) of two counties. The algorithms were applied to the data from two other counties, (n = 15, 447) to check the predictive capacity of the models. Results: The final models included gender, age, self-rated health and GHQ-12 scores as a quantitative variable. The regression equations explained 40 % (UK tariff) and 46 % (Swedish tariff) of the variances. The model showed a satisfying predictive capacity between the observed and the predicted EQ-5D index score, with Pearson correlation = 0.65 and 0.69 for the UK and Swedish models, respectively. Conclusion: The algorithms developed in this study can be used to determine cost-effectiveness of services or interventions that use GHQ-12 as a primary outcome where the utility measures are not collected

Heterogeneity in cost-effectiveness of lifestyle counseling for metabolic syndrome risk groups -primary care patients in Sweden

Abstract Background: Clinical trials have indicated that lifestyle interventions for patients with lifestyle-related cardiovascular and diabetes risk factors (the metabolic syndrome) are cost-effective. However, patient characteristics in primary care practice vary considerably, i.e. they exhibit heterogeneity in risk factors. The cost-effectiveness of lifestyle interventions is likely to differ over heterogeneous patient groups. Methods: Patients (62 men, 80 women) in the Kalmar Metabolic Syndrome Program (KMSP) in primary care (Kalmar regional healthcare area, Sweden) were divided into three groups reflecting different profiles of metabolic risk factors (low, middle and high risk) and gender. A Markov model was used to predict future cardiovascular disease and diabetes, including complications (until age 85 years or death), with health effects measured as QALYs and costs from a societal perspective in Euro (EUR) 2012, discounted 3%. Simulations with risk factor levels at start and at 12 months follow-up were performed for each group, with an assumed 4-year sustainability of intervention effects. Results: The program was estimated cost-saving for middle and high risk men, while the incremental cost vs. do-nothing varied between EUR 3,500 -18,000 per QALY for other groups. There is heterogeneity in the cost-effectiveness over the risk groups but this does not affect the overall conclusion on the cost-effectiveness of the KMSP. Even the highest ICER (for high risk women) is considered moderately cost-effective in Sweden. The base case result was not sensitive to alternative data and methodology but considerably affected by sustainability assumptions. Alternative risk stratifications did not change the overall conclusion that KMSP is cost-effective. However, simple grouping with average risk factor levels over gender groups overestimate the cost-effectiveness. Conclusions: Lifestyle counseling to prevent metabolic diseases is cost-effective in Swedish standard primary care settings. The use of risk stratification in the cost-effectiveness analysis established that the program was cost-effective for all patient groups, even for those with very high levels of lifestyle-related risk factors for the metabolic syndrome diseases. Heterogeneity in the cost-effectiveness of lifestyle interventions in primary care patients is expected, and should be considered in health policy decisions

Heterogeneity in cost-effectiveness of lifestyle counseling for metabolic syndrome risk groups -primary care patients in Sweden

BACKGROUND: Clinical trials have indicated that lifestyle interventions for patients with lifestyle-related cardiovascular and diabetes risk factors (the metabolic syndrome) are cost-effective. However, patient characteristics in primary care practice vary considerably, i.e. they exhibit heterogeneity in risk factors. The cost-effectiveness of lifestyle interventions is likely to differ over heterogeneous patient groups. METHODS: Patients (62 men, 80 women) in the Kalmar Metabolic Syndrome Program (KMSP) in primary care (Kalmar regional healthcare area, Sweden) were divided into three groups reflecting different profiles of metabolic risk factors (low, middle and high risk) and gender. A Markov model was used to predict future cardiovascular disease and diabetes, including complications (until age 85 years or death), with health effects measured as QALYs and costs from a societal perspective in Euro (EUR) 2012, discounted 3%. Simulations with risk factor levels at start and at 12 months follow-up were performed for each group, with an assumed 4-year sustainability of intervention effects. RESULTS: The program was estimated cost-saving for middle and high risk men, while the incremental cost vs. do-nothing varied between EUR 3,500 -- 18,000 per QALY for other groups. There is heterogeneity in the cost-effectiveness over the risk groups but this does not affect the overall conclusion on the cost-effectiveness of the KMSP. Even the highest ICER (for high risk women) is considered moderately cost-effective in Sweden. The base case result was not sensitive to alternative data and methodology but considerably affected by sustainability assumptions. Alternative risk stratifications did not change the overall conclusion that KMSP is cost-effective. However, simple grouping with average risk factor levels over gender groups overestimate the cost-effectiveness. CONCLUSIONS: Lifestyle counseling to prevent metabolic diseases is cost-effective in Swedish standard primary care settings. The use of risk stratification in the cost-effectiveness analysis established that the program was cost-effective for all patient groups, even for those with very high levels of lifestyle-related risk factors for the metabolic syndrome diseases. Heterogeneity in the cost-effectiveness of lifestyle interventions in primary care patients is expected, and should be considered in health policy decisions

Att sätta värde på tidiga insatser för barn och unga: Ett evidensbaserat beslutstöd för sociala investeringar

Abstrakt svenska Psykisk ohälsa bland barn och unga ökar risken för socialt och ekonomiskt utanförskap i vuxen ålder, vilket är kostsamt för individen och samhället. Denna studie presenterar en samhällsekonomisk modell för beräkning av hälso- och ekonomiska vinster av tidiga insatser för barn och unga. Modellen bygger på tre grundstenar – evidens kring förebyggande insatser, koppling mellan riskfaktorer bland barn och negativa utfall i vuxen ålder samt monetär värdering av dessa. Kartläggande översikter användes för att sammanställa evidensen kring effekter av förebyggande insatser och länkar mellan riskfaktorer och långsiktiga utfall. Den samhällsekonomiska modellen skattar kostnader, potentiella besparingar och vinster i form av avkastning per investerad krona. Modellen kan användas som beslutsunderlag för tidigare preventiva insatser inom folkhälsa. Abstrakt engelskaMental ill-health among children and youth increase the risk of adverse social and economic outcomes later in life, resulting in high individual and societal costs. This paper presents a model to estimate the potential health and economic gains from early prevention. The model is based on three pillars – evidence of preventive interventions, associations between risk factors among children and adverse adult outcomes and monetary valuation of these. Scoping reviews were used to synthesize the evidence of intervention effects and links between risk factors and long-term outcomes. The model presents intervention effects and costs, potential savings and return on investment. The model can be used for decision-making purposes within early prevention and public health. 

Heterogeneity in cost-effectiveness of lifestyle counseling for metabolic syndrome risk groups -primary care patients in Sweden

BACKGROUND: Clinical trials have indicated that lifestyle interventions for patients with lifestyle-related cardiovascular and diabetes risk factors (the metabolic syndrome) are cost-effective. However, patient characteristics in primary care practice vary considerably, i.e. they exhibit heterogeneity in risk factors. The cost-effectiveness of lifestyle interventions is likely to differ over heterogeneous patient groups. METHODS: Patients (62 men, 80 women) in the Kalmar Metabolic Syndrome Program (KMSP) in primary care (Kalmar regional healthcare area, Sweden) were divided into three groups reflecting different profiles of metabolic risk factors (low, middle and high risk) and gender. A Markov model was used to predict future cardiovascular disease and diabetes, including complications (until age 85 years or death), with health effects measured as QALYs and costs from a societal perspective in Euro (EUR) 2012, discounted 3%. Simulations with risk factor levels at start and at 12 months follow-up were performed for each group, with an assumed 4-year sustainability of intervention effects. RESULTS: The program was estimated cost-saving for middle and high risk men, while the incremental cost vs. do-nothing varied between EUR 3,500 -- 18,000 per QALY for other groups. There is heterogeneity in the cost-effectiveness over the risk groups but this does not affect the overall conclusion on the cost-effectiveness of the KMSP. Even the highest ICER (for high risk women) is considered moderately cost-effective in Sweden. The base case result was not sensitive to alternative data and methodology but considerably affected by sustainability assumptions. Alternative risk stratifications did not change the overall conclusion that KMSP is cost-effective. However, simple grouping with average risk factor levels over gender groups overestimate the cost-effectiveness. CONCLUSIONS: Lifestyle counseling to prevent metabolic diseases is cost-effective in Swedish standard primary care settings. The use of risk stratification in the cost-effectiveness analysis established that the program was cost-effective for all patient groups, even for those with very high levels of lifestyle-related risk factors for the metabolic syndrome diseases. Heterogeneity in the cost-effectiveness of lifestyle interventions in primary care patients is expected, and should be considered in health policy decisions