Influence of Sponsorship Bias on Treatment Effect Size Estimates in Randomized Trials of Oral Health Interventions: A Meta-epidemiological Study

2021 ◽  
pp. 101544
Author(s):  
Humam Saltaji ◽  
Susan Armijo-Olivo ◽  
Greta G. Cummings ◽  
Maryam Amin ◽  
Paul W. Major ◽  
...  
Keyword(s):  
Oral Health ◽  
Epidemiological Study ◽  
Treatment Effect ◽  
Effect Size ◽  
Randomized Trials ◽  
Health Interventions ◽  
Treatment Effect Size ◽  
Size Estimates

scholarly journals Phase I trial of caudate deep brain stimulation for treatment-resistant tinnitus

2020 ◽  
Vol 133 (4) ◽  
pp. 992-1001 ◽  
Author(s):  
Steven W. Cheung ◽  
Caroline A. Racine ◽  
Jennifer Henderson-Sabes ◽  
Carly Demopoulos ◽  
Annette M. Molinaro ◽  
...  
Keyword(s):  
Brain Stimulation ◽  
Treatment Effect ◽  
Effect Size ◽  
Phase I Trial ◽  
Treatment Resistant ◽  
Stimulation Parameters ◽  
Treatment Effect Size ◽  
Secondary Outcomes ◽  
Clinically Significant ◽  
Deep Brain

OBJECTIVEThe objective of this open-label, nonrandomized trial was to evaluate the efficacy and safety of bilateral caudate nucleus deep brain stimulation (DBS) for treatment-resistant tinnitus.METHODSSix participants underwent DBS electrode implantation. One participant was removed from the study for suicidality unrelated to brain stimulation. Participants underwent a stimulation optimization period that ranged from 5 to 13 months, during which the most promising stimulation parameters for tinnitus reduction for each individual were determined. These individual optimal stimulation parameters were then used during 24 weeks of continuous caudate stimulation to reach the endpoint. The primary outcome for efficacy was the Tinnitus Functional Index (TFI), and executive function (EF) safety was a composite z-score from multiple neuropsychological tests (EF score). The secondary outcome for efficacy was the Tinnitus Handicap Inventory (THI); for neuropsychiatric safety it was the Frontal Systems Behavior Scale (FrSBe), and for hearing safety it was pure tone audiometry at 0.5, 1, 2, 3, 4, and 6 kHz and word recognition score (WRS). Other monitored outcomes included surgery- and device-related adverse events (AEs). Five participants provided full analyzable data sets. Primary and secondary outcomes were based on differences in measurements between baseline and endpoint.RESULTSThe treatment effect size of caudate DBS for tinnitus was assessed by TFI [mean (SE), 23.3 (12.4)] and THI [30.8 (10.4)] scores, both of which were statistically significant (Wilcoxon signed-rank test, 1-tailed; alpha = 0.05). Based on clinically significant treatment response categorical analysis, there were 3 responders determined by TFI (≥ 13-point decrease) and 4 by THI (≥ 20-point decrease) scores. Safety outcomes according to EF score, FrSBe, audiometric thresholds, and WRS showed no significant change with continuous caudate stimulation. Surgery-related and device-related AEs were expected, transient, and reversible. There was only one serious AE, a suicide attempt unrelated to caudate neuromodulation in a participant in whom stimulation was in the off mode for 2 months prior to the event.CONCLUSIONSBilateral caudate nucleus neuromodulation by DBS for severe, refractory tinnitus in this phase I trial showed very encouraging results. Primary and secondary outcomes revealed a highly variable treatment effect size and 60%–80% treatment response rate for clinically significant benefit, and no safety concerns. The design of a phase II trial may benefit from targeting refinement for final DBS lead placement to decrease the duration of the stimulation optimization period and to increase treatment effect size uniformity.Clinical trial registration no.: NCT01988688 (clinicaltrials.gov).

scholarly journals Predictors of Treatment Response for Preschool Children With Developmental Language Disorder

2020 ◽  
Vol 29 (4) ◽  
pp. 2082-2096
Author(s):  
Leah L. Kapa ◽  
Christina Meyers-Denman ◽  
Elena Plante ◽  
Kevin Doubleday
Keyword(s):  
Treatment Effect ◽  
Effect Size ◽  
Language Disorder ◽  
Response To Treatment ◽  
Vocabulary Test ◽  
Developmental Language Disorder ◽  
Fourth Edition ◽  
Peabody Picture Vocabulary ◽  
Peabody Picture Vocabulary Test ◽  
Treatment Effect Size

Purpose Enhanced Conversational Recast treatment is an effective intervention for remediating expressive grammatical deficits in preschool-age children with developmental language disorder, but not all children respond equally well. In this study, we sought to identify which child-level variables predict response to treatment of morphological deficits. Method Predictor variables of interest, including pre-intervention test scores and target morpheme production, age, and mother's level of education (proxy for socio-economic status) were included in analyses. The sample included 105 children ( M = 5;1 [years;months]) with developmental language disorder who participated in 5 weeks of daily Enhanced Conversational Recast treatment. Classification and regression tree analysis was used to identify covariates that predicted children's generalization of their trained grammatical morpheme, as measured by treatment effect size d . Results Our analysis indicates that the Structured Photographic Expressive Language Test–Preschool 2 (SPELT-P 2) scores and the Peabody Picture Vocabulary Test–Fourth Edition scores significantly predicted the degree of benefit a child derived from Enhanced Conversational Recast treatment. Specifically, a SPELT-P 2 score above 75 (but still in the impaired range, < 87) combined with a high Peabody Picture Vocabulary Test–Fourth Edition score (> 100) yielded the largest treatment effect size, whereas a SPELT-P 2 score below 75 predicted the smallest treatment effect size. Other variables included in the model did not significantly predict treatment outcomes. Conclusions Understanding individual differences in response to treatment will allow service providers to make evidence-based decisions regarding how likely a child is to benefit from Enhanced Conversational Recast treatment and the expected magnitude of the response based on the child's background characteristics.

How Much Value Would a Treatment for Alzheimer’s Disease Offer? Cost-Effectiveness Thresholds for Pricing a Disease-Modifying Therapy

2020 ◽  
Vol 17 ◽  
Author(s):  
Hankyung Jun ◽  
Sang Kyu Cho ◽  
Elmar R. Aliyev ◽  
Soeren Mattke ◽  
Sze-Chuan Suen
Keyword(s):  
Alzheimer’S Disease ◽  
Alzheimer's Disease ◽  
Cost Effectiveness ◽  
Treatment Effect ◽  
Effect Size ◽  
Cost Effective ◽  
High Price ◽  
Disease Modifying Therapy ◽  
Treatment Effect Size ◽  
Disease Modifying

Background:: Recent trials suggest that disease-modifying therapy (DMT) for Alzheimer’s disease may become available soon. With the expected high price and a large patient pool, the budget impact will be substantial. Objective: We explore combinations of effectiveness and price under which a DMT is cost-effective. Method: We used an open-source model to conduct two-way scenario analyses for both payer and societal perspectives, varying price, and treatment effect size simultaneously. The analysis generates cost-effectiveness threshold prices over a potential range of DMT effectiveness in patients aged 65+ with mild cognitive impairment due to Alzheimer’s disease in the US. Results: Under the willingness-to-pay a threshold of $150,000 per quality-adjusted life year and assuming 30% risk reduction relative to the standard of care, the maximum cost-effective price of a DMT per patient per year is ~$22,000 and ~$15,000 from societal and payer perspectives, respectively. Conclusion: Joint variation of price and treatment effect size can help assess the cost-effectiveness of a potential Alzheimer’s disease treatment.

Reducing Prison Misconducts

2006 ◽  
Vol 33 (2) ◽  
pp. 185-218 ◽  
Author(s):  
Sheila A. French ◽  
Paul Gendreau
Keyword(s):  
Treatment Effect ◽  
Effect Size ◽  
Behavioral Treatment ◽  
Meta Analysis ◽  
Effect Sizes ◽  
Treatment Programs ◽  
Correctional Treatment ◽  
Prison Programs ◽  
Treatment Effect Size ◽  
Criminogenic Needs

A meta-analysis was conducted to assess the effectiveness of correctional treatment for reducing institutional misconducts. Sixty-eight studies generated 104 effect sizes involving 21,467 offenders. Behavioral treatment programs produced the strongest effects ( r = .26, CI = .18to .34). The numbers of criminogenic needs targeted and program therapeutic integrity were found to be important moderators of effect size. Prison programs producing the greatest reductions in misconduct were also associated with larger reductions in recidivism. The magnitudes of various indices of treatment effect size with respect to misconducts were remarkably similar to results in the correctional treatment literature where community recidivism is the criterion. Recommendations are made that will assist prison authorities to manage prisons in a safe and humane manner.

Sign in / Sign up

Export Citation Format

Share Document