Evaluation of inferential methods for the net benefit and win ratio statistics.

General Pairwise Comparison (GPC) statistics, such as the net benefit and the win ratio, have been applied in clinical trial data analysis and design. In the literature, inferential methods based on re-sampling, asymptotic or exact methods have been proposed for these GPC statistics, but they have not been compared to each other. In this paper, the small sample bias of the variance estimation, Type I error control and 95% confidence interval coverage of the GPC inferential methods are evaluated using simulations.

Accuracy of Internet-Based Patient Self-Report of Postdischarge Health Care Utilization and Complications Following Orthopedic Procedures: Observational Cohort Study.

Background: The accuracy of patient self-report of health care utilization and complications has yet to be determined. If patients are accurate and engaged self-reporters, collecting this information in a manner that is temporally proximate to the health care utilization events themselves may prove valuable to health care organizations undertaking quality improvement initiatives for which such data are often unavailable.

Objective: The objective of this study was to measure the accuracy of patient self-report of health care utilization and complications in the 90 days following orthopedic procedures using an automated digital patient engagement platform.

Effectiveness of an Automated Digital Remote Guidance and Telemonitoring Platform on Costs, Readmissions, and Complications After Hip and Knee Arthroplasties.

Background: The impact of a new class of automated digital patient engagement (DPE) platforms on potentially avoidable costs, hospital admissions, and complications after discharge following hip and knee arthroplasties has not been established.

Methods: We conducted a multicenter observational cohort study comparing claims data for potentially avoidable costs, hospital admissions, and complications for 90 days after discharge following hip and knee arthroplasties at 10 practice sites in CA and NV. One hundred eighty-six patients, enrolled between 2014 and 2016 on an automated DPE platform receiving guidance and remote monitoring perioperatively, were compared with 372 patients who underwent the same procedures from the same physicians within 3 years immediately preceding platform implementation.

Rationale and design of the Transcatheter Aortic Valve Replacement to UNload the Left ventricle in patients with ADvanced heart failure (TAVR UNLOAD) trial.

Background: Coexistence of moderate aortic stenosis (AS) in patients with heart failure (HF) with reduced ejection fraction is not uncommon. Moderate AS increases afterload, whereas pharmacologic reduction of afterload is a pillar of contemporary HF management.

Hypothesis: Unloading the left ventricle by reducing the transaortic gradient with transfemoral transcatheter aortic valve replacement (TAVR) may improve clinical outcomes in patients with moderate AS and HF with reduced ejection fraction.

Transcatheter or Surgical Aortic-Valve Replacement in Intermediate-Risk Patients.

Background: Previous trials have shown that among high-risk patients with aortic stenosis, survival rates are similar with transcatheter aortic-valve replacement (TAVR) and surgical aortic-valve replacement. We evaluated the two procedures in a randomized trial involving intermediate-risk patients.

Methods: We randomly assigned 2032 intermediate-risk patients with severe aortic stenosis, at 57 centers, to undergo either TAVR or surgical replacement.

5-year outcomes of transcatheter aortic valve replacement or surgical aortic valve replacement for high surgical risk patients with aortic stenosis (PARTNER 1): a randomised controlled trial.

Background: The Placement of Aortic Transcatheter Valves (PARTNER) trial showed that mortality at 1 year, 2 years, and 3 years is much the same with transcatheter aortic valve replacement (TAVR) or surgical aortic valve replacement (SAVR) for high-risk patients with aortic stenosis. We report here the 5-year outcomes.

Methods: We did this randomised controlled trial at 25 hospitals, in Canada (two), Germany (one), and the USA (23).

5-year outcomes of transcatheter aortic valve replacement compared with standard treatment for patients with inoperable aortic stenosis (PARTNER 1): a randomised controlled trial.

Background: Based on the early results of the Placement of Aortic Transcatheter Valves (PARTNER) trial, transcatheter aortic valve replacement (TAVR) is an accepted treatment for patients with severe aortic stenosis who are not suitable for surgery. However, little information is available about the late clinical outcomes in such patients.

Methods: We did this randomised controlled trial at 21 experienced valve centres in Canada, Germany, and the USA.

Outcomes of inoperable symptomatic aortic stenosis patients not undergoing aortic valve replacement: insight into the impact of balloon aortic valvuloplasty from the PARTNER trial (Placement of AoRtic TraNscathetER Valve trial).

Objectives: The aim of this report is to characterize the impact of balloon aortic valvuloplasty (BAV) in patients not undergoing aortic valve replacement in the PARTNER (Placement of AoRtic TraNscathetER Valves) trial.

Background: The PARTNER trial is the only randomized trial with independently adjudicated data of inoperable severe symptomatic aortic stenosis patients, allowing outcome analysis of unoperated-on patients.

Methods: The design and initial results of the PARTNER trial (Cohort B) were reported previously.

Long-term outcomes of inoperable patients with aortic stenosis randomly assigned to transcatheter aortic valve replacement or standard therapy.

Background: The long-term outcomes of transcatheter aortic valve replacement (TAVR) in inoperable patients with severe aortic stenosis remain unknown.

Methods And Results: In the Placement of Aortic Transcatheter Valves (PARTNER) study, 358 patients were randomly assigned to TAVR or standard therapy. We report the 3-year outcomes on these patients, and the pooled outcomes for all randomly assigned inoperable patients (n=449) in PARTNER, as well, including the randomized portion of the continued access study (n=91).

Comparison of transcatheter and surgical aortic valve replacement in severe aortic stenosis: a longitudinal study of echocardiography parameters in cohort A of the PARTNER trial (placement of aortic transcatheter valves).

Objectives: This study sought to compare echocardiographic findings in patients with critical aortic stenosis following surgical aortic valve replacement (SAVR) or transcatheter aortic valve replacement (TAVR).

Background: The PARTNER (Placement of Aortic Transcatheter Valves) trial randomized patients 1:1 to SAVR or TAVR.

Methods: Echocardiograms were obtained at baseline, discharge, 30 days, 6 months, 1 year, and 2 years after the procedure and analyzed in a core laboratory.

Determination of minimum clinically important difference (MCID) in pain, disability, and quality of life after revision fusion for symptomatic pseudoarthrosis.

Background Context: Spinal surgical outcome studies rely on patient reported outcome (PRO) measurements to assess the effect of treatment. A shortcoming of these questionnaires is that the extent of improvement in their numerical scores lacks a direct clinical meaning. As a result, the concept of minimum clinically important difference (MCID) has been used to measure the critical threshold needed to achieve clinically relevant treatment effectiveness.

Two-year outcomes after transcatheter or surgical aortic-valve replacement.

Background: The Placement of Aortic Transcatheter Valves (PARTNER) trial showed that among high-risk patients with aortic stenosis, the 1-year survival rates are similar with transcatheter aortic-valve replacement (TAVR) and surgical replacement. However, longer-term follow-up is necessary to determine whether TAVR has prolonged benefits.

Methods: At 25 centers, we randomly assigned 699 high-risk patients with severe aortic stenosis to undergo either surgical aortic-valve replacement or TAVR.

Transcatheter aortic-valve replacement for inoperable severe aortic stenosis.

Background: Transcatheter aortic-valve replacement (TAVR) is the recommended therapy for patients with severe aortic stenosis who are not suitable candidates for surgery. The outcomes beyond 1 year in such patients are not known.

Methods: We randomly assigned patients to transfemoral TAVR or to standard therapy (which often included balloon aortic valvuloplasty).

Transcatheter (TAVR) versus surgical (AVR) aortic valve replacement: occurrence, hazard, risk factors, and consequences of neurologic events in the PARTNER trial.

Background: All neurologic events in the PARTNER randomized trial comparing transcatheter aortic valve replacement (TAVR) with surgical aortic valve replacement (AVR) were analyzed.

Methods: High-risk patients with aortic stenosis were stratified into transfemoral (TF, n = 461) or transapical (TA, n = 196) strata based on their arterial anatomy and randomized: 657 received treatment assigned ("as treated"), 313 underwent AVR, and 344 TAVR. Neurologic events were prospectively adjudicated by an independent Clinical Events Committee.

Minimum clinically important difference in pain, disability, and quality of life after neural decompression and fusion for same-level recurrent lumbar stenosis: understanding clinical versus statistical significance.

Object: Spine surgery outcome studies rely on patient-reported outcome (PRO) measurements to assess treatment effect, but the extent of improvement in the numerical scores of these questionnaires lacks a direct clinical meaning. Because of this, the concept of a minimum clinically important difference (MCID) has been used to measure the critical threshold needed to achieve clinically relevant treatment effectiveness. As utilization of spinal fusion has increased over the past decade, so has the incidence of same-level recurrent stenosis following index lumbar fusion, which commonly requires revision decompression and fusion.

Determination of minimum clinically important difference in pain, disability, and quality of life after extension of fusion for adjacent-segment disease.

Object: Spinal surgical outcome studies rely on patient-reported outcome (PRO) measurements to assess treatment effect. A shortcoming of these questionnaires is that the extent of improvement in their numerical scores lack a direct clinical meaning. As a result, the concept of minimum clinical important difference (MCID) has been used to measure the critical threshold needed to achieve clinically relevant treatment effectiveness.

Cerebrospinal shunt infection in patients receiving antibiotic-impregnated versus standard shunts.

Object: Cerebrospinal fluid shunt infections are associated with significant morbidity and mortality in the treatment of adult and pediatric hydrocephalus. Antibiotic-impregnated shunt (AIS) catheters have been used with the aim of reducing shunt infection. While many studies have demonstrated a reduction in shunt infection with AIS, this reported efficacy has varied within the literature.

Transcatheter versus surgical aortic-valve replacement in high-risk patients.

Background: The use of transcatheter aortic-valve replacement has been shown to reduce mortality among high-risk patients with aortic stenosis who are not candidates for surgical replacement. However, the two procedures have not been compared in a randomized trial involving high-risk patients who are still candidates for surgical replacement.

Methods: At 25 centers, we randomly assigned 699 high-risk patients with severe aortic stenosis to undergo either transcatheter aortic-valve replacement with a balloon-expandable bovine pericardial valve (either a transfemoral or a transapical approach) or surgical replacement.

Utility of minimum clinically important difference in assessing pain, disability, and health state after transforaminal lumbar interbody fusion for degenerative lumbar spondylolisthesis.

Object: Outcome studies for spine surgery rely on patient-reported outcomes (PROs) to assess treatment effects. Commonly used health-related quality-of-life questionnaires include the following scales: back pain and leg pain visual analog scale (BP-VAS and LP-VAS); the Oswestry Disability Index (ODI); and the EuroQol-5D health survey (EQ-5D). A shortcoming of these questionnaires is that their numerical scores lack a direct meaning or clinical significance.

Transcatheter aortic-valve implantation for aortic stenosis in patients who cannot undergo surgery.

Background: Many patients with severe aortic stenosis and coexisting conditions are not candidates for surgical replacement of the aortic valve. Recently, transcatheter aortic-valve implantation (TAVI) has been suggested as a less invasive treatment for high-risk patients with aortic stenosis.

Methods: We randomly assigned patients with severe aortic stenosis, whom surgeons considered not to be suitable candidates for surgery, to standard therapy (including balloon aortic valvuloplasty) or transfemoral transcatheter implantation of a balloon-expandable bovine pericardial valve.

A nonradiographic system for assessing pressure for the Codman-Hakim Programmable Valve.

Background: The Codman-Hakim Programmable Valve is widely used in shunting hydrocephalus and other conditions.

Object: To establish an accurate valve verification system for the Codman-Hakim programmable valve that does not require radiographic exposure.

Methods: Prospective clinical trial tested a new valve verification system at 9 research sites.

Thirty-day results of the SAPIEN aortic Bioprosthesis European Outcome (SOURCE) Registry: A European registry of transcatheter aortic valve implantation using the Edwards SAPIEN valve.

Background: Transcatheter aortic valve implantation was developed to mitigate the mortality and morbidity associated with high-risk traditional aortic valve replacement. The Edwards SAPIEN valve was approved for transcatheter aortic valve implantation transfemoral delivery in the European Union in November 2007 and for transapical delivery in January 2008.

Methods And Results: The SAPIEN Aortic Bioprosthesis European Outcome (SOURCE) Registry was designed to assess the initial clinical results of the Edwards SAPIEN valve in consecutive patients in Europe after commercialization.

United States feasibility study of transcatheter insertion of a stented aortic valve by the left ventricular apex.

Background: Recent US and European registries have indicated 30% to 60% of patients with critical valvular aortic stenosis (AS) are not treated surgically, usually due to advanced age and comorbidities. We report on a Food and Drug Administration approved feasibility study of a less invasive transcatheter approach to potentially treat these high-risk patients.

Methods: Between December 2006 and February 18, 2008, 40 patients underwent transcatheter insertion of a balloon expandable stainless-steel stent with an internally mounted three-leaflet equine pericardial valve (Edwards Sapien Transcatheter Heart Valve; Edwards Lifesciences, Irvine, CA) into the aortic annulus using a transapical left ventricular insertion (TA-AVI).