Understanding Treatment Effect Estimates When Treatment Effects Are Heterogeneous for More Than One Outcome

Brooks, John M.; Chapman, Cole G.; Schroeder, Mary C.

doi:10.1007/s40258-018-0380-z

Cited by 14 publications

(57 citation statements)

References 51 publications

(72 reference statements)

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“…Instrumental variable methods offer an approach to directly assess this question using populations of Medicare patients with PHF from across local areas. 13,[15][16][17][18][19][20] Our estimates provide evidence to suggest that current PHF surgery rates are higher than the effective surgery rate and that lowering surgery rates may be associated with lower 1-year mortality rates and adverse event risks. These results are especially pronounced for patients with PHF in highrisk patient subgroups.…”

Section: Discussionmentioning

confidence: 99%

“…21 They are contrasted with risk-adjusted regression (RAR) estimates, which provide information on surgery outcomes for the patients who underwent surgery in our data. 13,[15][16][17][18][19][20] study used deidentified Medicare data. This study follows the Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) reporting guideline.…”

Section: Introductionmentioning

confidence: 99%

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Association of Surgical Treatment With Adverse Events and Mortality Among Medicare Beneficiaries With Proximal Humerus Fracture

et al. 2020

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IMPORTANCE Meta-analyses of randomized clinical trials suggest that the advantages and risks of surgery compared with conservative management as the initial treatment for proximal humerus fracture (PHF) vary, or are heterogeneous across patients. Substantial geographic variation in surgery rates for PHF suggests that the optimal rate of surgery across the population of patients with PHF is unknown. OBJECTIVE To use geographic variation in treatment rates to assess the outcomes associated with higher rates of surgery for patients with PHF. DESIGN, SETTING, AND PARTICIPANTS This comparative effectiveness research study analyzed all fee-for-service Medicare beneficiaries with proximal humerus fracture in 2011 who were continuously enrolled in Medicare Parts A and B for the 365-day period before and immediately after their index fracture. Data analysis was performed January through June 2019. EXPOSURE Undergoing 1 of the commonly used surgical procedures in the 60 days after an index fracture diagnosis. MAIN OUTCOMES AND MEASURES Risk-adjusted area surgery ratios were created for each hospital referral region as a measure of local area practice styles. Instrumental variable approaches were used to assess the association between higher surgery rates and adverse events, mortality risk, and cost at 1 year from Medicare's perspective for patients with PHF in 2011. Instrumental variable models were stratified by age, comorbidities, and frailty. Instrumental variable estimates were compared with estimates from risk-adjusted regression models. RESULTS The final cohort included 72 823 patients (mean [SD] age, 80.0 [7.9] years; 13 958 [19.2%] men). The proportion of patients treated surgically ranged from 1.8% to 33.3% across hospital referral regions in the United States. Compared with conservatively managed patients, surgical patients were younger (mean [SD] age, 80.4 [8.1] years vs 78.0 [7.2] years; P < .001) and healthier (Charlson Comorbidity Index score of 0, 14 863 [24.4%] patients vs 3468 [29.1%] patients; Function-Related Indicator score of 0, 20 720 [34.0%] patients vs 4980 [41.8%] patients; P < .001 for both), and a larger proportion were women (49 030 [80.5%] patients vs 9835 [82.5%] patients; P < .001). Instrumental variable analysis showed that higher rates of surgery were associated with increased total costs ($8913) during the treatment period, increased adverse event rates (a 1-percentage point increase in the surgery rate was associated with a 0.19-percentage point increase in the 1-year adverse event rate; β = 0.19; 95% CI, 0.09-0.27; P < .001), and increased mortality risk (a 1-percentage point increase in the surgery rate was associated with a 0.09-percentage point increase in the 1-year mortality rate; β = 0.09; 95% CI, 0.04-0.15; P < .01). Instrumental variable mortality results were even more striking for older patients and those with higher comorbidity burdens and greater frailty. Risk-adjusted estimates suggested that surgical patients had higher costs (continued)

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Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Association of Surgical Treatment With Adverse Events and Mortality Among Medicare Beneficiaries With Proximal Humerus Fracture

et al. 2020

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“…For each subpopulation we tested the association of the measured covariates with ACEI/ARB use and for trends in each covariate across patients grouped by ATR quintiles . Linear 2‐stage least squares (2SLS) IV estimators were used (Data ) . In this study 2SLS yields estimates of the absolute average effect of ACEI/ARBs for the patients whose ACEI/ARB choice was sensitive to local area practice styles or what is known as the local average treatment effect.…”

Section: Methodsmentioning

confidence: 99%

“…Guidelines are based largely on studies of younger patients with well‐preserved renal function, that did not focus on long‐term renal, cardiovascular, or survival outcomes . Observational studies assessing ACEI/ARBs effects using risk‐adjustment approaches have shown mixed results, likely stemming from varying ability to control for confounders and heterogeneity of ACEI/ARB effects across patients …”

Section: Introductionmentioning

confidence: 99%

Use of Angiotensin‐Converting Enzyme Inhibitors and Angiotensin Receptor Blockers for Geriatric Ischemic Stroke Patients: Are the Rates Right?

Brooks

Chapman

Suneja

et al. 2018

JAHA

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BackgroundOur objective is to estimate the effects associated with higher rates of renin‐angiotensin system antagonists, angiotensin‐converting enzyme inhibitors and angiotensin receptor blockers (ACEI/ARBs), in secondary prevention for geriatric (aged >65 years) patients with new ischemic strokes by chronic kidney disease (CKD) status.Methods and ResultsThe effects of ACEI/ARBs on survival and renal risk were estimated by CKD status using an instrumental variable (IV) estimator. Instruments were based on local area variation in ACEI/ARB use. Data abstracted from charts were used to assess the assumptions underlying the instrumental estimator. ACEI/ARBs were used after stroke by 45.9% and 45.2% of CKD and non‐CKD patients, respectively. ACEI/ARB rate differences across local areas grouped by practice styles were nearly identical for CKD and non‐CKD patients. Higher ACEI/ARB use rates for non‐CKD patients were associated with higher 2‐year survival rates, whereas higher ACEI/ARB use rates for patients with CKD were associated with lower 2‐year survival rates. While the negative survival estimates for patients with CKD were not statistically different from zero, they were statistically lower than the estimates for non‐CKD patients. Confounders abstracted from charts were not associated with the instrumental variable used.ConclusionsHigher ACEI/ARB use rates had different survival implications for older ischemic stroke patients with and without CKD. ACEI/ARBs appear underused in ischemic stroke patients without CKD as higher use rates were associated with higher 2‐year survival rates. This conclusion is not generalizable to the ischemic stroke patients with CKD, as higher ACEI/ARBS use rates were associated with lower 2‐year survival rates that were statistically lower than the estimates for non‐CKD patients.

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“…pre-conditions affecting the outcomes) closely related to "real world" are excluded from those studies. 24,25 On the other hand, pragmatic trials are designed to take place in real-world clinical practice settings, where typical patients receive the interventions by clinicians who sometimes did not have any research background. 26 In order to achieve this purpose, pragmatic trials must enroll participants from heterogeneous practice settings, including a broad range of demographic features, to assess several significant outcomes from comparisons involving clinically relevant alternative interventions.…”

Section: Experimental Studies -From Explanatory Rct's To Pragmatic Trmentioning

confidence: 99%

Practice based research in dentistry: an alternative to deal with clinical questions

et al. 2020

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Clinical interventions in dental practice should be determined based on the best scientific evidence available. Well-designed randomized clinical trials (RCTs) provide important evidence supporting the efficacy of interventions and are usually considered as the best primary evidence. However, the strict criteria adopted by most RCTs reduce their external validity since some findings from these studies might not work under usual conditions. On the other hand, practice-based research (PBR) studies have been designed to better define the effectiveness of clinical interventions under settings closer to "real-world" conditions. Therefore, this review aimed to describe different PBR designs discussing some advantages and limitations of such studies. The stimulus to organization PBR networks is discussed since the studies performed by these networks involve large number of clinicians and important conclusions can be drawn. Designs of observational studies including surveys and cohort studies based on practice are presented. Survey methods are important to know the behavior of practitioners regarding diagnostic and decision of treatment. Cohorts allow assessing different cofounders contributing to some outcome since large sample sizes and long followup periods can be observed in some of these studies. Pragmatic trials designed to take place in real-world clinical practice settings are also discussed as a useful design to assess the effectiveness of clinical interventions. In conclusion, this review sought to present PBR studies as alternative designs to answer clinical questions, but not replacing randomized clinical trials.

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Understanding Treatment Effect Estimates When Treatment Effects Are Heterogeneous for More Than One Outcome

Cited by 14 publications

References 51 publications

Association of Surgical Treatment With Adverse Events and Mortality Among Medicare Beneficiaries With Proximal Humerus Fracture

Association of Surgical Treatment With Adverse Events and Mortality Among Medicare Beneficiaries With Proximal Humerus Fracture

Use of Angiotensin‐Converting Enzyme Inhibitors and Angiotensin Receptor Blockers for Geriatric Ischemic Stroke Patients: Are the Rates Right?

Practice based research in dentistry: an alternative to deal with clinical questions

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