scholarly journals The effect of delaying initiation with umeclidinium/vilanterol in patients with COPD: an observational administrative claims database analysis using marginal structural models

2018 ◽  
Vol 13 ◽  
Author(s):  
Beth Hahn ◽  
Ami R. Buikema ◽  
Lee Brekke ◽  
Amy Anderson ◽  
Eleena Koep ◽  
...  
Keyword(s):  
Structural Models ◽  
Medical Costs ◽  
Marginal Structural Models ◽  
Severe Exacerbation ◽  
Administrative Claims ◽  
Research Database ◽  
Adjusted Cost ◽  
Exacerbation Risk ◽  
The Impact

Background: Chronic obstructive pulmonary disease (COPD) is associated with high clinical and economic burden. Optimal pharmacological therapy for COPD aims to reduce symptoms and the frequency and severity of exacerbations. Umeclidinium/vilanterol (UMEC/VI) is an approved combination therapy for once-daily maintenance treatment of patients with COPD. This study evaluated the impact of delaying UMEC/VI initiation on medical costs and exacerbation risk. Methods: A retrospective analysis of patients with COPD who initiated UMEC/VI between 4/28/2014 and 7/31/2016 was conducted using the Optum Research Database. The index date was the first COPD visit after UMEC/VI available on US formulary (Commercial 4/28/2014; Medicare Advantage 1/1/2015). Patients were followed for 12 months post-index, and categorized into 12 cohorts corresponding to month (30-day period) of UMEC/VI initiation (i.e. Months 1–12) post-index. The outcomes studied during the follow up period included COPD-related and all-cause medical costs, and risk of COPD exacerbations. Marginal structural models (MSM) were used to control for time-varying confounding due to changes in treatment and severity during follow up. Results: 2,200 patients initiating UMEC/VI were included in the study sample. Patients’ average age was 69.3 years, 49.9% were female and 69.7% were Medicare insured. Following MSM analysis, 12-month adjusted COPD-related medical costs increased by 2.9% (95% confidence interval [CI]: 0.1–5.9%; p = 0.044) for each monthly delay in UMEC/VI initiation, with a 37.4% higher adjusted cost for patients initiating UMEC/VI in Month 12 versus Month 1 ($13,087 vs. $9524). The 12-month adjusted all-cause medical costs increased by 2.8% (95% CI: 0.6–5.2%; p = 0.013) for each monthly delay, with a 36.1% higher adjusted cost for patients initiating UMEC/VI at Month 12 versus Month 1 ($22,766 vs. $16,727). The monthly risk of severe exacerbation was significantly higher in patients who had not yet initiated UMEC/VI than those who had (hazard ratio: 1.74; 95% CI: 1.35–2.23; p < 0.001). Conclusions: Prompt use of UMEC/VI following a physician visit for COPD appears to result in economic and clinical benefits, with reductions in medical costs and exacerbation risk. Additional research is warranted to assess the benefits of initiating UMEC/VI as a first-line therapy compared with escalation to UMEC/VI from monotherapies.

scholarly journals The Impact of Sparse Follow-up on Marginal Structural Models for Time-to-Event Data

2015 ◽  
pp. kwv152
Author(s):  
Nassim Mojaverian ◽  
Erica E. M. Moodie ◽  
Alex Bliu ◽  
Marina B. Klein
Keyword(s):  
Structural Models ◽  
Marginal Structural Models ◽  
Event Data ◽  
Time To Event ◽  
Time To Event Data ◽  
The Impact

Estimating the Causal Effect of Some Exposure From Nonrandomized Studies: The Example of Reduced Intensity Conditioning (RIC) in Hematological Diseases.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 3365-3365
Author(s):  
Matthieu Resche-Rigon ◽  
Marie Robin ◽  
Regis Peffault de Latour ◽  
Sylvie Chevret ◽  
Gerard P Socie
Keyword(s):  
Quantitative Methods ◽  
Causal Effect ◽  
Autologous Transplantation ◽  
Structural Models ◽  
Marginal Structural Models ◽  
Reduced Intensity Conditioning ◽  
Hematological Diseases ◽  
Causal Pathway ◽  
The Impact ◽  
Reduced Intensity

Abstract Abstract 3365 Poster Board III-253 Introduction: Although allogeneic SCT with RIC has now gained wide acceptance, its eventual benefit again non-transplant approach is largely unknown (outside the setting of large randomized trials). When evaluating the impact on survival of reduced intensity conditioning in malignant hematological diseases, standard estimations based on Cox regression from observational databases could be biased because they ignore covariates that confound treatment decision. In this setting, we applied and compared two different statistical methods that were developed to control for confounding in estimating exposure (or treatment) effect from epidemiological studies. Patients and Methods: The statistical challenge was that allograft tended to be given when a patient was in advanced phase of his/her hematological malignancy, so that treatment was confounded by performance indicators, which in turn lie on the causal pathway between treatment and outcome. Thus, comparison of outcome first used propensity score (PS) analyses that attempt to create a comparison group of non-treated patients that closely resembles the group of treated patients by matching for the likelihood that a given patient has received the treatment. Then, we used marginal structural models (MSMs) that consist in creating, by using inverse probability of treatment weights, a pseudo-population in which the probability of treatment does no longer depend on covariates, and the effect of treatment on outcome is the same as in the original population. Result: Reduced intensity conditioning allograft was performed in 82 patients with chemotherapy-sensitive patients relapsing after autologous transplantation. Patients with myeloma (MM, 23 pts), follicular lymphoma (FL, 28 pts) or Hodgkin disease (HD, 31 pts), were compared to 276 patients who relapsed after autologous transplantation but did not underwent allogeneic stem cell transplantation (142 MM, 115 FL and 19 HD). From original datasets, 21 (91%) matched pairs could be constituted from MM patients, as compared to 19 (68%) of the FL patients, down to 15 (48%) of the HD patients. Based on these PS-matched samples, a significant benefit of reduced intensity conditioning as compared with non allografted patients was observed in MM, with estimated hazard ratio (HR) of death at 0.34 (95% confidence interval, CI: 0.14-0.88), as well as in FL (HR= 0.78, 95%CI: 0.27;2.30) and in HD (HR= 0.24; 95%CI: 0.09-0.62). MSM-based analyses that applied to the reweighted populations confirmed these trends towards survival benefits in FL, though partially erased in MM and HD. Conclusions: We reported the application of marginal structural models, a new class of causal models to estimate the effect of nonrandomized treatments as an alternative to PS based approaches in small samples. We expect that an increasing number of physicians involved in clinical cohorts become familiar with these novel and appealing quantitative methods when assessing innovative treatment effects. Disclosures: No relevant conflicts of interest to declare.

Economic benefits of adequate molecular monitoring in patients with chronic myelogenous leukemia (CML).

2013 ◽  
Vol 31 (15_suppl) ◽  
pp. 7093-7093
Author(s):  
Lei Chen ◽  
Annie Guérin ◽  
Eric Q. Wu ◽  
Katherine Dea ◽  
Stuart L. Goldberg
Keyword(s):  
Medical Service ◽  
Kinase Inhibitor ◽  
Quantitative Polymerase Chain Reaction ◽  
Chronic Phase ◽  
Medical Costs ◽  
Economic Benefits ◽  
Myelogenous Leukemia ◽  
Administrative Claims ◽  
Molecular Monitoring ◽  
The Impact

7093 Background: Molecular monitoring every 3 months using quantitative polymerase chain reaction (qPCR) of BCR-ABL mRNA transcripts on International Scale is recommended by the National Comprehensive Cancer Network and the European LeukemiaNet for patients (pts) in chronic phase of CML. A previous study has shown an underutilization of qPCR in the community setting. This study assessed the impact of the frequency of molecular monitoring on hospitalization and medical costs among CML pts receiving 1st-line tyrosine kinase inhibitor (TKI) therapies. Methods: Two U.S. administrative claims databases were combined (01/2000-06/2012) to identify adult CML pts initiated on TKIs (imatinib, dasatinib, nilotinib). Pts were followed for 12 months from their first TKI prescription and categorized into 3 cohorts based on frequency of qPCR tests (i.e., 0, 1-2, and 3-4). Number of inpatient admissions and medical service costs (measured from a US payer perspective; adjusted to 2012 U.S. dollars) were compared between cohorts. Multivariate regression models adjusted for confounding factors (e.g., age, gender, CML complexity, TKI). Results: The study included 1,205 CML pts. Over the 12-month study period, 41.0% of the pts had no qPCR test, 31.9% had 1-2 tests, and 27.1 % had 3-4 tests. Compared to pts with no qPCR monitoring, those with 3-4 tests incurred 37% fewer CML-related (i.e., a primary CML diagnosis) inpatient admissions (p=.017) during the study period, leading to a $4,000 (p=.009) reduction in CML-related inpatient costs and $5,663 (p=.005) reduction in all-cause inpatient costs, accounting for the majority of the $5,997 reduction in total medical service costs (p=.049). Pts with 1-2 tests a year showed smaller and statistically insignificant reductions from those with no test in the frequency of hospitalization and medical costs. Conclusions: Among CML pts who initiated 1st-line TKIs, pts with 3-4 qPCR tests a year incurred fewer inpatient admissions and lower medical service costs compared to pts with no test. These findings suggest that pts would benefit from regular qPCR testing and underscore the value of molecular monitoring in the delivery of quality care for Ph+ CML-CP pts on TKI therapies.

Association of Blood Pressure with Cardiovascular Outcome and Mortality: Results from the KNOW-CKD Study

2021 ◽  
Author(s):  
Jee Young Lee ◽  
Jung Tak Park ◽  
Young Su Joo ◽  
Changhyun Lee ◽  
Hae-Ryong Yun ◽  
...  
Keyword(s):  
Cardiovascular Events ◽  
Structural Models ◽  
Cardiovascular Outcome ◽  
Marginal Structural Models ◽  
Potential Hazard ◽  
Composite Outcome ◽  
Hazard Ratios ◽  
Adverse Cardiovascular Outcome ◽  
Primary Composite Outcome

Abstract Background Optimal BP control is a major therapeutic strategy to reduce adverse cardiovascular events and mortality in patients with CKD. We studied the association of BP with adverse cardiovascular outcome and all-cause death in patients with CKD. Methods Among 2,238 participants from the KoreaN cohort study for Outcome in patients With CKD, 2,226 patients with baseline BP measurements were enrolled. Main predictor was SBP categorized by 5 levels: &lt;110, 110-119, 120-129, 130-139, and ≥140 mmHg. Primary endpoint was a composite outcome of all-cause death or incident cardiovascular events. We primarily used marginal structural models using averaged and the most recent time-updated SBPs. Results During a median follow-up of 10233.79 person-years (median 4.60 years), the primary composite outcome occurred in 240 (10.8%) participants, with a corresponding incidence rate of 23.5 (95% CI, 20.7–26.6) per 1,000 patient-years. Marginal structural models with averaged SBP showed a U-shaped relationship with the primary outcome. Compared to time-updated SBP of 110–119 mmHg, hazard ratios (95% CI) for &lt;110, 120–129, 130–139, and ≥140 mmHg were 2.47 (1.48–4.11), 1.29 (0.80–2.08), 2.15 (1.26–3.69), and 2.19 (1.19–4.01), respectively. Marginal structural models with the most recent SBP also showed similar findings. Conclusions In Korean patients with CKD, there was a U-shaped association of SBP with the risk of adverse clinical outcome. Our findings highlight the importance of BP control and suggest a potential hazard of SBP &lt;110 mmHg.

scholarly journals Immunotherapy prevents long-term disability in relapsing multiple sclerosis over 15 years

2019 ◽  
Author(s):  
Tomas Kalincik ◽  
Sifat Sharmin ◽  
Charles Malpas ◽  
Tim Spelman ◽  
Dana Horakova ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Structural Models ◽  
Marginal Structural Models ◽  
Long Term Effect ◽  
Multiple Sclerosis Therapy ◽  
Relapsing Remitting ◽  
Relapsing Remitting Multiple Sclerosis ◽  
Sclerosis Therapy

ABSTRACTObjectiveWhether immunotherapy improves long-term disability in multiple sclerosis has not been satisfactorily demonstrated. This study examined the effect of immunotherapy on long-term disability outcomes in relapsing-remitting multiple sclerosis.MethodsWe studied patients from MSBase followed for ≥1 year, with ≥3 visits, ≥1 visit per year and exposed to a multiple sclerosis therapy, and a subset of patients with ≥15-year follow-up. Marginal structural models were used to compare the hazard of 12-month confirmed increase and decrease in disability, EDSS step 6 and the incidence of relapses between treated and untreated periods. Marginal structural models were continuously re-adjusted for patient age, sex, pregnancy, date, disease course, time from first symptom, prior relapse history, disability and MRI activity.Results14,717 patients were studied. During the treated periods, patients were less likely to experience relapses (hazard ratio 0.60, 95% confidence interval 0.43–0.82, p=0.0016), worsening of disability (0.56, 0.38-0.82, p=0.0026) and progress to EDSS step 6 (0.33, 0.19-0.59, p=0.00019). Among 1085 patients with ≥15-year follow-up, the treated patients were less likely to experience relapses (0.59, 0.50–0.70, p=10-9) and worsening of disability (0.81, 0.67-0.99, p=0.043).ConclusionsContinued treatment with multiple sclerosis immunotherapies reduces disability accrual (by 19-44%), the risk of need of a walking aid by 67% and the frequency of relapses (by 40-41%) over 15 years. A proof of long-term effect of immunomodulation on disability outcomes is the key to establishing its disease modifying properties.

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