Publications by authors named "Shelby D Reed"

168 Publications

Economic Outcomes of Rehabilitation Therapy in Older Patients With Acute Heart Failure in the REHAB-HF Trial: A Secondary Analysis of a Randomized Clinical Trial.

JAMA Cardiol 2021 Nov 24. Epub 2021 Nov 24.

Duke Clinical Research Institute, Duke University School of Medicine, Durham, North Carolina.

Importance: In the Rehabilitation Therapy in Older Acute Heart Failure Patients (REHAB-HF) trial, a novel 12-week rehabilitation intervention demonstrated significant improvements in validated measures of physical function, quality of life, and depression, but no significant reductions in rehospitalizations or mortality compared with a control condition during the 6-month follow up. The economic implications of these results are important given the increasing pressures for cost containment in health care.

Objective: To report the economic outcomes of the REHAB-HF trial and estimate the potential cost-effectiveness of the intervention.

Design, Setting, Participants: The multicenter REHAB-HF trial randomized 349 patients 60 years or older who were hospitalized for acute decompensated heart failure to rehabilitation intervention or a control group; patients were enrolled from September 17, 2014, through September 19, 2019. For this preplanned secondary analysis of the economic outcomes, data on medical resource use and quality of life (via the 5-level EuroQol 5-Dimension scores converted to health utilities) were collected. Medical resource use and medication costs were estimated using 2019 US Medicare payments and the Federal Supply Schedule, respectively. Cost-effectiveness was estimated using the validated Tools for Economic Analysis of Patient Management Interventions in Heart Failure Cost-Effectiveness Model, which uses an individual-patient simulation model informed by the prospectively collected trial data. Data were analyzed from March 24, 2019, to December 1, 2020.

Interventions: Rehabilitation intervention or control.

Main Outcomes And Measures: Costs, quality-adjusted life-years (QALYs), and the lifetime estimated cost per QALY gained (incremental cost-effectiveness ratio).

Results: Among the 349 patients included in the analysis (183 women [52.4%]; mean [SD] age, 72.7 [8.1] years; 176 non-White [50.4%] and 173 White [49.6%]), mean (SD) cumulative costs per patient were $26 421 ($38 955) in the intervention group (excluding intervention costs) and $27 650 ($30 712) in the control group (difference, -$1229; 95% CI, -$8159 to $6394; P = .80). The mean (SD) cost of the intervention was $4204 ($2059). Quality of life gains were significantly greater in the intervention vs control group during 6 months (mean utility difference, 0.074; P = .001) and sustained beyond the 12-week intervention. Incremental cost-effectiveness ratios were estimated at $58 409 and $35 600 per QALY gained for the full cohort and in patients with preserved ejection fraction, respectively.

Conclusions And Relevance: These analyses suggest that longer-term benefits of this novel rehabilitation intervention, particularly in the subgroup of patients with preserved ejection fraction, may yield good value to the health care system. However, long-term cost-effectiveness is currently uncertain and dependent on the assumption that benefits are sustained beyond study follow-up, which needs to be corroborated in future trials in this patient population.
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http://dx.doi.org/10.1001/jamacardio.2021.4836DOI Listing
November 2021

Lifetime cost-effectiveness simulation of once-weekly exenatide in type 2 diabetes: a cost-utility analysis based on the EXSCEL trial.

Diabetes Res Clin Pract 2021 Nov 20:109152. Epub 2021 Nov 20.

Diabetes Trials Unit, University of Oxford, Oxford, UK.

Aims: The Exenatide Study of Cardiovascular Event Lowering (EXSCEL) trial assessed once-weekly exenatide (EQW) vs. placebo, added to usual care in 14,752 patients with type 2 diabetes mellitus (Clinicaltrials.gov: NCT01144338). We assessed the lifetime cost-effectiveness of adding EQW vs. usual care alone from a healthcare perspective.

Methods: Medical resource use and EQ-5D utilities were collected throughout the study. Within-trial results were extrapolated to a lifetime horizon using the UK Prospective Diabetes Study Outcomes Model version 2 (UKPDS-OM2), predicting predict cardiovascular and microvascular events. Cost-effectiveness was evaluated separately for US and UK settings, with outcomes measured in quality-adjusted life-years (QALYs).

Results: EQW plus usual care gained 0.162 QALYs at an additional cost of $41,545/patient, compared with usual care in a US setting. The incremental cost-effectiveness ratio (ICER) was $259,223/QALY. In a UK setting, the QALY gain was 0.151 at an additional cost of £6357: an ICER of £42,589/QALY. Sensitivity analyses ranged between $34,369-$269,571 and £3,430-£46,560 per QALY gained.

Conclusions: In a lifetime extrapolation, adding EQW to usual care increased QALYs and costs compared with usual care alone. The base-case ICERs exceeded the commonly-cited cost-effectiveness thresholds of $100,000/QALY and £20,000/QALY. However, ICERs were considerably lower in some subgroups, and in sensitivity analyses.
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http://dx.doi.org/10.1016/j.diabres.2021.109152DOI Listing
November 2021

Quantifying Value of Hope.

Value Health 2021 Oct 12;24(10):1511-1519. Epub 2021 Aug 12.

Duke Clinical Research Institute, Preference Evaluation Research Group, Duke University, Durham, NC, USA.

Background: 'Hope' is a construct in patient-centered value frameworks, but few studies have attempted to measure the value of hope separately from treatment-related gains in quality of life and survival to support its application in economic evaluation.

Objective: To generate quantitative information on the "value of hope".

Methods: We designed a discrete-choice experiment in which treatment alternatives varied the probability of achieving 10-year survival, expected survival as the weighted sum of short-term and long-term survival, health status, and out-of-pocket cost. Two-hundred patients with cancer or history of cancer recruited by Cancer Support Community each completed 10 choice questions. We used mixed-logit and latent-class models to analyze the choice data.

Results: Relative to fixed survival periods of two, three or five years with 0% chance of 10-year survival, participants positively valued treatments with 5% and 10% chances of 10-year survival. However, participants negatively valued a 20% chance of 10-year survival that required an offsetting 80% chance of shorter survival. This finding was particularly strong when expected survival was two years. Compared to a 0% chance, dollar-equivalent values of 5% and 10% chances of long-term survival were $5,975 and $12,421, respectively, independent of health status or expected survival. The corresponding value for 20% versus 0% chance of long-term survival was negative. Latent-class analysis revealed 4 groups with distinct preference patterns.

Conclusions: Our findings affirm positive value for hope independent of expected survival and health status. However, this finding does not universally hold in all situations nor across all groups.
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http://dx.doi.org/10.1016/j.jval.2021.04.1284DOI Listing
October 2021

Factors Influential in the Selection of Radiology Residents in the Post-Step 1 World: A Discrete Choice Experiment.

J Am Coll Radiol 2021 Nov 29;18(11):1572-1580. Epub 2021 Jul 29.

Department of Radiology, Duke University Medical Center, Durham, North Carolina.

Objectives: Reporting of United States Medical Licensing Examination Step 1 results will transition from a numerical score to a pass or fail result. We sought an objective analysis to determine changes in the relative importance of resident application attributes when numerical Step 1 results are replaced.

Methods: A discrete choice experiment was designed to model radiology resident selection and determine the relative weights of various application factors when paired with a numerical or pass or fail Step 1 result. Faculty involved in resident selection at 14 US radiology programs chose between hypothetical pairs of applicant profiles between August and November 2020. A conditional logistic regression model assessed the relative weights of the attributes, and odds ratios (ORs) were calculated.

Results: There were 212 participants. When a numerical Step 1 score was provided, the most influential attributes were medical school (OR: 2.35, 95% confidence interval [CI]: 2.07-2.67), Black or Hispanic race or ethnicity (OR: 2.04, 95% CI: 1.79-2.38), and Step 1 score (OR: 1.8, 95% CI: 1.69-1.95). When Step 1 was reported as pass, the applicant's medical school grew in influence (OR: 2.78, 95% CI: 2.42-3.18), and there was a significant increase in influence of Step 2 scores (OR: 1.31, 95% CI: 1.23-1.40 versus OR 1.57, 95% CI: 1.46-1.69). There was little change in the relative influence of race or ethnicity, gender, class rank, or clerkship honors.

Discussion: When Step 1 reporting transitions to pass or fail, medical school prestige gains outsized influence and Step 2 scores partly fill the gap left by Step 1 examination as a single metric of decisive importance in application decisions.
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http://dx.doi.org/10.1016/j.jacr.2021.07.005DOI Listing
November 2021

Chemoradiation treatment patterns among United States Veteran Health Administration patients with unresectable stage III non-small cell lung cancer.

BMC Cancer 2021 Jul 16;21(1):824. Epub 2021 Jul 16.

Duke Clinical Research Institute, Duke University School of Medicine, Durham, NC, USA.

Background: The Veterans Health Administration (VHA) is the largest integrated health care system in the United States (US). Among VHA patients, the rate of use of concurrent chemoradiation therapy (CCRT) among those with unresectable, stage III non-small cell lung cancer (NSCLC) is unknown. The objective was to report recent CCRT treatment patterns in VHA patients and identify characteristics associated with receipt of CCRT.

Methods: Using Department of Veteran Affairs (VA) Cancer Registry System data linked to VA electronic medical records, we determined rates of CCRT, sequential CRT (SCRT), radiation therapy (RT) only, chemotherapy (CT) only, and neither treatment.

Results: Among 4054 VHA patients who met study criteria, CCRT rates slightly increased from 44 to 50% between 2013 and 2017. Factors associated with decreased odds of CCRT receipt compared to any other treatment included increasing age (adjusted odds ratio [aOR] per 10 years = 0.67; 95% CI: 0.60-0.76) and Charlson-Deyo comorbidity score (aOR = 0.94; 95% CI: 0.91-0.97). White race was associated with increased odds of CCRT receipt (aOR = 1.24; 95% CI: 1.004-1.53). In a chart review sample of 200 patients, less than half (n = 85) had a documented reason for not receiving CCRT. Among these, 29% declined treatment, and 71% did not receive CCRT due to "not being a candidate" for reasons related to frailty or lung nodules being too far apart for radiation therapy.

Conclusions: CCRT rates among VHA patients with unresectable, stage III NSCLC slightly increased from 2013 to 2017; however in 2017, only half were receiving CCRT. Older patients and those with multiple comorbidities were less likely to receive CCRT and even when controlling for these factors, non-white patients were less likely to receive CCRT.
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http://dx.doi.org/10.1186/s12885-021-08577-yDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8285779PMC
July 2021

Rehabilitation Intervention in Older Patients With Acute Heart Failure With Preserved Versus Reduced Ejection Fraction.

JACC Heart Fail 2021 10 7;9(10):747-757. Epub 2021 Jul 7.

Section on Cardiovascular Medicine, Department of Internal Medicine, Wake Forest School of Medicine, Winston-Salem, North Carolina, USA; Sections on Geriatrics, Department of Internal Medicine, Wake Forest School of Medicine, Winston-Salem, North Carolina, USA. Electronic address:

Objectives: This study assessed for treatment interactions by ejection fraction (EF) subgroup (≥45% [heart failure with preserved ejection fraction (HFpEF); vs <45% [heart failure with reduced ejection fraction (HFrEF)]).

Background: The REHAB-HF trial showed that an early multidomain rehabilitation intervention improved physical function, frailty, quality-of-life, and depression in older patients hospitalized with acute decompensated heart failure (ADHF).

Methods: Three-month outcomes were: Short Physical Performance Battery (SPPB), 6-min walk distance (6MWD), and Kansas City Cardiomyopathy Questionnaire (KCCQ). Six-month end points included all-cause rehospitalization and death and a global rank of death, all-cause rehospitalization, and SPPB. Prespecified significance level for interaction was P ≤ 0.1.

Results: Among 349 total participants, 185 (53%) had HFpEF and 164 (47%) had HFrEF. Compared with HFrEF, HFpEF participants were more often women (61% vs 43%) and had significantly worse baseline physical function, frailty, quality of life, and depression. Although interaction P values for 3-month outcomes were not significant, effect sizes were larger for HFpEF vs HFrEF: SPPB +1.9 (95% CI: 1.1-2.6) vs +1.1 (95% CI: 0.3-1.9); 6MWD +40 meters (95% CI: 9 meters-72 meters) vs +27 (95% CI: -6 meters to 59 meters); KCCQ +9 (2-16) vs +6 (-2 to 14). All-cause rehospitalization rate was nominally lower with intervention in HFpEF but not HFrEF [effect size 0.83 (95% CI: 0.64-1.09) vs 0.99 (95% CI: 0.74-1.33); interaction P = 0.40]. There were significantly greater treatment benefits in HFpEF vs HFrEF for all-cause death [interaction P = 0.08; intervention rate ratio 0.63 (95% CI: 0.25-1.61) vs 2.21 (95% CI: 0.78-6.25)], and the global rank end point (interaction P = 0.098) with benefit seen in HFpEF [probability index 0.59 (95% CI: 0.50-0.68)] but not HFrEF.

Conclusions: Among older patients hospitalized with ADHF, compared with HFrEF those with HFpEF had significantly worse impairments at baseline and may derive greater benefit from the intervention. (A Trial of Rehabilitation Therapy in Older Acute Heart Failure Patients [REHAB-HF]; NCT02196038).
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http://dx.doi.org/10.1016/j.jchf.2021.05.007DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8487922PMC
October 2021

Impact of financial medication assistance on medication adherence: a systematic review.

J Manag Care Spec Pharm 2021 Jul;27(7):924-935

Department of Population Health Sciences, Duke University Medical Center, Durham, North Carolina.

The prevalence of financial medication assistance (FMA), including patient assistance programs, coupons/copayment cards, vouchers, discount cards, and programs/pharmacy services that help patients apply for such programs, has increased. The impact of FMA on medication adherence and persistence has not been synthesized. The primary objective of this study was to review published studies evaluating the impact of FMA on the three phases of medication adherence (initiation [or primary adherence], implementation [or secondary adherence], and discontinuation) and persistence. Among these studies, the secondary objective was to report the impact of FMA on patient out-of-pocket costs and clinical outcomes. A systematic review was performed using MEDLINE and Web of Science. Of 656 articles identified, eight studies met all inclusion criteria. Seven studies examined FMA for medications treating cardiovascular diseases, while one study assessed FMA for cancer medications. Among included studies, FMA had a positive impact on medication adherence or persistence, and most measured this impact over one year or less. Of the three phases of medication adherence, implementation (5 of 8) was most commonly reported, followed by discontinuation (3 of 8), and then initiation (1 of 8). Regarding implementation, users of FMA had a higher mean medication possession ratio (MPR) than nonusers, ranging from 7 to 18 percentage points higher. The percentage of patients who discontinued medication was 7 percentage points lower in users of FMA versus nonusers for cardiovascular disease states. In one cancer study, FMA had a larger impact on initiation than discontinuation, ie, compared to nonusers, users of FMA were less likely to abandon an initial prescription (risk ratio= 0.12, 95% confidence interval [CI]: 0.08-0.18), and this effect was larger than the decreased likelihood of discontinuing the medication (hazard ratio = 0.76, 95% CI: 0.66-0.88). In 3 of 8 studies reporting on medication persistence, FMA increased the odds of medication persistence for one year ranged from 11% to 47%, depending on the study. In addition to adherence, half of the studies reported on FMA impacts on patient out-of-pocket costs and 3 of 8 studies reported on clinical outcomes. Impacts on patient out-of-pocket costs were mixed; two studies reported that out-of-pocket costs were higher for users of a coupon or a voucher versus nonusers, one study reported the opposite, and one study reported null effects. Impacts on clinical outcomes were either positive or null. We found that FMA has positive impacts on all phases of medication adherence as well as medication persistence over one year. Future studies should assess whether FMA has differential impacts based on phase of medication adherence and report on its longer-term (ie, beyond one year) impacts on medication adherence. This work was sponsored by a grant from Pharmaceutical Research and Manufacturers of America (PhRMA). PhRMA had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript. Hung reports past employment by Blue Cross Blue Shield Association and CVS Health and a grant from PhRMA outside of the submitted work. Zullig reports research funding from Proteus Digital Health and the PhRMA Foundation. consulting fees from Novartis. Reed reports receiving research support from Abbott Vascular, AstraZeneca, Janssen Research & Development, Monteris, PhRMA Foundation, and TESARO and consulting fees from Sanofi/Regeneron, NovoNordisk, SVC Systems, and Minomic International, Inc. Bosworth reports research grants from the PhRMA Foundation, Proteus Digital Health, Otsuka, Novo Nordisk, Sanofi, Improved Patient Outcomes, Boehinger Ingelheim, NIH, and VA, as well as consulting fees from Sanofi, Novartis, Otsuka, Abbott, Xcenda, Preventric Diagnostics, and the Medicines Company. The other authors have nothing to report. This work was presented as a poster presentation at the ESPACOMP Annual Meeting in November 2020.
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http://dx.doi.org/10.18553/jmcp.2021.27.7.924DOI Listing
July 2021

The preferences of women with ovarian cancer for oral versus intravenous recurrence regimens.

Gynecol Oncol 2021 08 28;162(2):440-446. Epub 2021 May 28.

Duke Cancer Institute, Duke University Medical Center, Durham, NC, United States of America; Duke Clinical Research Institute, Duke University School of Medicine, Durham, NC, United States of America; Department of Population Health Sciences, Duke University School of Medicine, Durham, NC, United States of America.

Objective: To assess preferences of women with ovarian cancer regarding features of available anti-cancer regimens for platinum-resistant, biomarker-positive disease, with an emphasis on oral PARP inhibitor and standard intravenous (IV) chemotherapy regimens.

Methods: A discrete-choice-experiment preferences survey was designed, tested, and administered to women with ovarian cancer, with 11 pairs of treatment profiles defined using seven attributes (levels/ranges): regimen (oral daily, IV weekly, IV monthly); probability of progression-free (PFS) at 6 months (40%-60%); probability of PFS at 2 years (10%-20%); nausea (none, moderate); peripheral neuropathy (none, mild, moderate); memory problems (none, mild); and total out-of-pocket cost ($0 to $10,000).

Results: Of 123 participants, 38% had experienced recurrence, 25% were currently receiving chemotherapy, and 18% were currently taking a PARP inhibitor. Given attributes and levels, the relative importance weights (sum 100) were: 2-year PFS, 28; cost, 27; 6-month PFS, 19; neuropathy,14; memory problems, nausea, and regimen, all ≤5. To accept moderate neuropathy, participants required a 49% (versus 40%) chance of PFS at 6 months or 14% (versus 10%) chance at 2 years. Given a 3-way choice where PFS and cost were equal, 49% preferred a monthly IV regimen causing mild memory problems, 47% preferred an oral regimen causing moderate nausea, and 4% preferred a weekly IV regimen causing mild memory and mild neuropathy.

Conclusions: These findings challenge the assumption that oral anti-cancer therapies are universally preferred by patients and demonstrate that there is no "one size fits all" regimen that is preferable to women with ovarian cancer when considering recurrence treatment regimens.
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http://dx.doi.org/10.1016/j.ygyno.2021.05.022DOI Listing
August 2021

Physical Rehabilitation for Older Patients Hospitalized for Heart Failure.

N Engl J Med 2021 07 16;385(3):203-216. Epub 2021 May 16.

From the Department of Internal Medicine, Sections of Cardiovascular Medicine (D.W.K., M.B.N., B.U.) and Gerontology and Geriatric Medicine (D.W.K., M.A.E.), and the Departments of Neurology (P.D.) and Biostatistics and Data Science (H.C., M.A.E.), Wake Forest School of Medicine, Winston-Salem, the Department of Orthopedic Surgery, Doctor of Physical Therapy Division (A.M.P.), the Department of Medicine, Division of Cardiology (R.J.M.), and the Department of Population Health Sciences (S.D.R.), Duke University School of Medicine, Durham, and Novant Health Heart and Vascular Institute, Charlotte (G.R.R.) - all in North Carolina; the Department of Medicine, Sidney Kimmel Medical College at Thomas Jefferson University (D.J.W.), and the Department of Physical Therapy, Jefferson College of Rehabilitation Sciences at Thomas Jefferson University (L.A.H.) - both in Philadelphia; and Inova Heart and Vascular Institute, Fairfax, VA (C.M.O.).

Background: Older patients who are hospitalized for acute decompensated heart failure have high rates of physical frailty, poor quality of life, delayed recovery, and frequent rehospitalizations. Interventions to address physical frailty in this population are not well established.

Methods: We conducted a multicenter, randomized, controlled trial to evaluate a transitional, tailored, progressive rehabilitation intervention that included four physical-function domains (strength, balance, mobility, and endurance). The intervention was initiated during, or early after, hospitalization for heart failure and was continued after discharge for 36 outpatient sessions. The primary outcome was the score on the Short Physical Performance Battery (total scores range from 0 to 12, with lower scores indicating more severe physical dysfunction) at 3 months. The secondary outcome was the 6-month rate of rehospitalization for any cause.

Results: A total of 349 patients underwent randomization; 175 were assigned to the rehabilitation intervention and 174 to usual care (control). At baseline, patients in each group had markedly impaired physical function, and 97% were frail or prefrail; the mean number of coexisting conditions was five in each group. Patient retention in the intervention group was 82%, and adherence to the intervention sessions was 67%. After adjustment for baseline Short Physical Performance Battery score and other baseline characteristics, the least-squares mean (±SE) score on the Short Physical Performance Battery at 3 months was 8.3±0.2 in the intervention group and 6.9±0.2 in the control group (mean between-group difference, 1.5; 95% confidence interval [CI], 0.9 to 2.0; P<0.001). At 6 months, the rates of rehospitalization for any cause were 1.18 in the intervention group and 1.28 in the control group (rate ratio, 0.93; 95% CI, 0.66 to 1.19). There were 21 deaths (15 from cardiovascular causes) in the intervention group and 16 deaths (8 from cardiovascular causes) in the control group. The rates of death from any cause were 0.13 and 0.10, respectively (rate ratio, 1.17; 95% CI, 0.61 to 2.27).

Conclusions: In a diverse population of older patients who were hospitalized for acute decompensated heart failure, an early, transitional, tailored, progressive rehabilitation intervention that included multiple physical-function domains resulted in greater improvement in physical function than usual care. (Funded by the National Institutes of Health and others; REHAB-HF ClinicalTrials.gov number, NCT02196038.).
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http://dx.doi.org/10.1056/NEJMoa2026141DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8353658PMC
July 2021

Patient Preferences for Outcomes Following DCIS Management Strategies: A Discrete Choice Experiment.

JCO Oncol Pract 2021 Nov 12;17(11):e1639-e1648. Epub 2021 Mar 12.

Department of Surgery, Duke University Medical Center, Durham, NC.

Purpose: Ductal carcinoma in situ (DCIS), a nonobligate precursor of breast cancer, is often aggressively managed with multimodal therapy. However, there is limited research on patients' preferences for trade-offs among treatment-related outcomes such as breast appearance, side effects, and future cancer risk. We sought to investigate whether women consider treatment features aside from cancer risk when making treatment choices for ductal carcinoma in situ and if so, to what degree other features influence these decisions.

Methods: A discrete choice experiment was administered to participants in a comprehensive cancer screening mammography clinic. The experimental design was used to generate constructed health profiles resulting from different management strategies. Health profiles were defined by breast appearance, severity of infection within the first year, chronic pain, hot flashes, and risk of developing or dying from breast cancer within 10 years.

Results: One hundred ninety-four women without a personal history of breast cancer completed the choice task. Across 10 choice questions, 29% always selected the health profile with a lower risk of invasive breast cancer (ie, dominated on cancer risk), regardless of the effects of treatment. For nonrisk dominators, breast cancer risk remained the most important factor but was closely followed by chronic pain (24% [95% CI, 20 to 28]) and infection (22% [95% CI, 18 to 25]). Depending on treatment outcomes, the tolerable increase in breast cancer risk was as high as 3.4%.

Conclusion: Most women were willing to make some trade-offs between invasive cancer risk and treatment-related outcomes. Our findings highlight the importance of shared decision-making weighing risks and benefits between patient and provider management of low-risk disease.
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http://dx.doi.org/10.1200/OP.20.00614DOI Listing
November 2021

EGFR mutation testing and TKI treatment patterns among veterans with stage III and IV non-small cell lung cancer.

Cancer Treat Res Commun 2021 29;27:100327. Epub 2021 Jan 29.

Duke Clinical Research Institute, Duke University School of Medicine, Durham, VA, United States; Department of Population Health Sciences, Duke University School of Medicine, Durham, VA, United States; Department of Veteran Affairs Medical Center, Durham, NC, United States.

Background: Epidermal growth factor receptor (EGFR) mutation testing is recommended in metastatic non-small cell lung cancer (NSCLC). The objective of this study was to assess changes in EGFR mutation testing patterns and tyrosine kinase inhibitor (TKI) use in US veterans with stage III-IV NSCLC between 2013 and 2017.

Patients And Methods: Retrospective study using linked data from Department of Veterans Affairs (VA) Cancer Registry System, Corporate Data Warehouse, commercial laboratories, and clinical notes. Generalized linear mixed models accounting for clustering by VA facility were used to determine factors associated with EGFR mutation testing.

Results: From 2013 to 2017, EGFR mutation testing increased from 29.5% to 38.4% among veterans with stage III-IV NSCLC and from 47.0% to 57.4% among veterans with stage IV non-squamous disease. Factors associated with increased odds of testing included being married, Medicare enrollment, and adenocarcinoma histology. Factors associated with decreased odds of testing included Medicaid eligibility, stage III disease, increasing age, being a current or former smoker, increasing Charlson-Deyo comorbidity score, and receiving cancer care in the South. Appropriate use of a TKI rose from 2013 to 2017 (17.2% to 74.1%).

Conclusion: EGFR mutation testing rates increased to almost 60% in the stage IV non-squamous NSCLC population in 2017, with residual opportunity for further increase. Several sociodemographic characteristics, comorbidities, and geographic regions were associated with EGFR mutation testing suggestive of inequitable testing decisions. Appropriate use of TKI improved drastically from 2013 to 2017 demonstrating rapidly changing practice patterns through the adoption phase of new treatment options.
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http://dx.doi.org/10.1016/j.ctarc.2021.100327DOI Listing
January 2021

Behavioral cancer pain intervention using videoconferencing and a mobile application for medically underserved patients: Rationale, design, and methods of a prospective multisite randomized controlled trial.

Contemp Clin Trials 2021 03 23;102:106287. Epub 2021 Jan 23.

Department of Psychiatry and Behavioral Sciences, Duke University Medical Center, Durham, NC, USA. Electronic address:

Background: Women with breast cancer in medically underserved areas are particularly vulnerable to persistent pain and disability. Behavioral pain interventions reduce pain and improve outcomes. Cancer patients in medically underserved areas receive limited adjunctive cancer care, as many lack access to pain therapists trained in behavioral interventions, face travel barriers to regional medical centers, and may have low literacy and limited resources. mHealth technologies have the potential to decrease barriers but must be carefully adapted for, and efficacy-tested with, medically underserved patients. We developed an mHealth behavioral pain coping skills training intervention (mPCST-Community). We now utilize a multisite randomized controlled trial to: 1) test the extent mPCST-Community reduces breast cancer patients' pain severity (primary outcome), pain interference, fatigue, physical disability, and psychological distress; 2) examine potential mediators of intervention effects; and 3) evaluate the intervention's cost and cost-effectiveness.

Methods/design: Breast cancer patients (N = 180) will be randomized to mPCST-Community or an attention control. mPCST-Community's four-session protocol will be delivered via videoconferencing at an underserved community clinic by a remote pain therapist at a major medical center. Videoconference sessions will be supplemented with a mobile application. Participants will complete self-report measures at baseline, post-intervention, and 3- and 6-month follow-ups.

Conclusions: mPCST-Community has the potential to reduce pain and disability, and decrease barriers for cancer patients in medically underserved areas. This is one of the first trials to test an mHealth behavioral cancer pain intervention developed specifically for medically underserved communities. If successful, it could lead to widespread implementation and decreased health disparities.
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http://dx.doi.org/10.1016/j.cct.2021.106287DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8009852PMC
March 2021

Is Easier Better Than Harder? An Experiment on Choice Experiments for Benefit-Risk Tradeoff Preferences.

Med Decis Making 2021 02 19;41(2):222-232. Epub 2021 Jan 19.

Duke Clinical Research Institute, Duke University, Durham, NC, USA.

Objectives: To test the convergent validity of simple and more complex study designs in a discrete-choice experiment (DCE) of multiple sclerosis (MS) treatment preferences.

Methods: Five hundred US adults with MS completed an online DCE survey. Respondents answered 8 choice questions with pairs of constructed MS treatment profiles defined by delays in problems with walking, delays in problems with cognition, thyroid disorders, and 10-y risks of kidney failure and serious brain infection (i.e., progressive multifocal leukoencephalopathy [PML]). Four hundred respondents completed choice questions using 4 levels for all attributes, except thyroid disorders with 3 levels. One hundred respondents completed choice questions using only the 2 extreme attribute levels of the 4-level version. Random-parameters logit models were used to estimate choice-model parameters.

Results: Respondents viewing the 4-level and 2-level versions agreed on the relative importance of the 3 most important attributes: cognition, walking, and PML. Respondents viewing the 4-level version indicated much stronger disutility for a 0% to 0.5% increase in kidney-failure risk than those viewing the 2-level version where the risk for kidney failure increased from 0% to 3%. Otherwise, utilities for other 4-level attributes were approximately linear but with significantly steeper slopes (except for cognition) than the 2-level estimates, indicating that attributes were perceived as more important as the number of levels increased.

Conclusions: Although the relative importance of some attributes was similar, the 2-level and 4-level versions generally failed to demonstrate convergent validity. If the study goal is attribute rankings, a 2-level version could be adequate. If goals include quantifying tradeoffs among attribute levels, more complex designs can help respondents discriminate among attribute levels. Reductions in measurement error using fewer attribute levels appear to have come at the expense of less discriminating evaluations.
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http://dx.doi.org/10.1177/0272989X20979833DOI Listing
February 2021

Surgeon Applications of Patient Preferences in Treatment Decision Making for First-Time Anterior Shoulder Dislocation.

Orthop J Sports Med 2020 Dec 4;8(12):2325967120966145. Epub 2020 Dec 4.

Duke Sport Science Institute, Division of Sports Medicine, Department of Orthopaedic Surgery, Duke University Medical Center, Durham, North Carolina, USA.

Background: Treatment of a first-time anterior shoulder dislocation (FTASD) is sensitive to patient preferences. The operative or nonoperative management debate provides an excellent opportunity to learn how surgeons apply patient preferences in treatment decisions.

Purpose: To determine how patient preferences (repeat dislocation risk, recovery difficulties, fear of surgery, treatment costs) and surgeon factors influence a surgeon's treatment plan for FTASD.

Study Design: Cross-sectional study.

Methods: Eight clinical vignettes of hypothetical patients with FTASD (including age, sex, and activity level) were presented to members of the Magellan Society. A second set of matched vignettes with patient preferences and clinical variables were also presented. The vignettes represented scenarios in which evidence does not favor one treatment over another. Respondents were asked how they would manage each hypothetical case. Respondents also estimated the risk of redislocation for the nonoperative cases for comparison with the published rates. Finally, respondents completed a Likert-scale questionnaire to determine their perceptions on factors influencing their decisions.

Results: A total of 103 orthopaedic surgeons completed the survey; 48% practiced in an academic hospital; 79% were in practice for 10 years or longer; and 75% had completed a sports medicine fellowship. Patient preferences were the single most important factor influencing treatment recommendation, with activity type and age also important. Just 62% of the surgeon estimates of the risk of redislocation were consistent with the published rates. The inclusion of patient preferences to clinical variables changed treatment recommendations in 62.5% of our hypothetical cases. Respondents rated patient treatment preference as the leading factor in their treatment decision making.

Conclusion: Patient preferences were important when deciding the appropriate treatment for FTASD. Respondents were inconsistent when applying evidence in their decision making and estimates of recurrent instability. Decision support tools that deliver patient preferences and personalized evidence-based outcome estimates improve the quality of decision making at the point of care.
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http://dx.doi.org/10.1177/2325967120966145DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7720322PMC
December 2020

A randomized feasibility pilot trial of a financial incentives intervention for dietary self-monitoring and weight loss in adults with obesity.

Transl Behav Med 2021 04;11(4):954-969

School of Nursing, Duke University, Durham, NC, USA.

Financial incentives could be used to improve adherence to behavioral weight loss interventions, increasing their effectiveness. This Phase IIb randomized pilot study evaluated the feasibility and acceptability of a study protocol for providing financial incentives for dietary self-monitoring and/or weight loss. Community-dwelling adults with obesity were enrolled in a 24 week, group-based weight loss program. Participants were randomized in a 2 × 2 factorial design to receive financial incentives for both dietary self-monitoring and weekly weight loss, just one, or neither. Participants could earn up to $300, evolving from fixed weekly payments to intermittent, variable payments. The notice of reward was provided by text message. The study was conducted in three successive cohorts to evaluate study procedure changes, including dietary approach, recruitment and retention strategies, text messaging, and incentives. Descriptive statistics calculated separately for each cohort described study performance relative to predefined targets for recruitment, including minority representation; retention; adherence; and weight loss. Acceptability was assessed via postintervention qualitative interviews. In Cohort 1 (n = 34), a low-carbohydrate diet was used. Recruitment, retention, adherence, and weight loss were adequate, but minority representation was not. For Cohort 2 (n = 31), employing an additional recruitment method and switching to a reduced-calorie diet yielded adequate recruitment, minority representation, retention, and adherence but less weight loss. Returning to a low-carbohydrate diet in Cohort 3 (n = 28) yielded recruitment, minority representation, retention, adherence, and weight loss similar to Cohort 2. Participant feedback informed changes to text message timing and content and incentive amount. Through successive cohorts, we optimized recruitment and retention strategies and text messaging. An adequately powered trial is warranted to evaluate the efficacy of these incentive structures for reducing weight. The trial registration number is NCT02691260.
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http://dx.doi.org/10.1093/tbm/ibaa102DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8489416PMC
April 2021

Patient Preferences for Surgical Treatment of Knee Osteoarthritis: A Discrete-Choice Experiment Evaluating Total and Unicompartmental Knee Arthroplasty.

J Bone Joint Surg Am 2020 Dec;102(23):2022-2031

Department of Orthopaedic Surgery, Duke University Medical Center, Durham, North Carolina.

Background: Total knee arthroplasty (TKA) is a common treatment for end-stage knee osteoarthritis but is associated with increased complication rates compared with unicompartmental knee arthroplasty (UKA). UKA offers better functional outcomes but is associated with a higher risk of revision. The purpose of this study was to apply good-practice, stated-preference methods to quantify patient preferences for benefit-risk tradeoffs associated with arthroplasty treatments for end-stage knee osteoarthritis.

Methods: A discrete-choice experiment was developed with the following attributes: chance of complications, functional ability, awareness of the knee implant, and chance of needing another operation within 10 years. Patients included those aged 40 to 80 years with knee osteoarthritis. A pivot design filtered respondents into 1 of 2 surveys on the basis of self-reported functional ability (good compared with fair or poor) as measured by the Oxford Knee Score. Treatment-preference data were collected, and relative attribute-importance weights were estimated.

Results: Two hundred and fifty-eight completed survey instruments from 92 males and 164 females were analyzed, with 72 respondents in the good-function cohort and 186 in the fair/poor-function cohort. Patients placed the greatest value or relative importance on serious complications and rates of revision in both cohorts. Preference weights did not vary between cohorts for any attribute. In the good-function cohort, 42% of respondents chose TKA and 58% chose UKA. In the fair/poor-function cohort, 54% chose TKA and 46% chose UKA.

Conclusions: Patient preferences for various treatment attributes varied among patients in a knee osteoarthritis population. Complication and revision rates were the most important factors to patients, suggesting that physicians should focus on these areas when discussing treatments. The proportion of patients who chose UKA suggests that the current trend of increased UKA utilization is aligned with patient preferences.

Clinical Relevance: Systematic elicitation of patient preferences for knee arthroplasty procedures, which lays out evidence-based risks and benefits of different treatments, indicates a larger subset of the knee osteoarthritis population may prefer UKA than would be suggested by the current rates of utilization of the procedure. Arthroplasty treatment should align with patient preferences and eligibility criteria to better deliver patient-centered care.
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http://dx.doi.org/10.2106/JBJS.20.00132DOI Listing
December 2020

Benchmarking the Cost-Effectiveness of Interventions Delaying Diabetes: A Simulation Study Based on NAVIGATOR Data.

Diabetes Care 2020 10 12;43(10):2485-2492. Epub 2020 Aug 12.

Health Economics Research Centre, Nuffield Department of Population Health, University of Oxford, Oxford, U.K.

Objective: To estimate using the UK Prospective Diabetes Study Outcomes Model Version 2 (UKPDS-OM2) the impact of delaying type 2 diabetes onset on costs and quality-adjusted life expectancy using trial participants who developed diabetes in the NAVIGATOR (Nateglinide And Valsartan in Impaired Glucose Tolerance Outcomes Research) study.

Research Design And Methods: We simulated the impact of delaying diabetes onset by 1-9 years, utilizing data from the 3,058 of 9,306 NAVIGATOR trial participants who developed type 2 diabetes. Costs and utility weights associated with diabetes and diabetes-related complications were obtained for the U.S. and U.K. settings, with costs expressed in 2017 values. We estimated discounted lifetime costs and quality-adjusted life years (QALYs) with 95% CIs.

Results: Gains in QALYs increased from 0.02 (U.S. setting, 95% CI 0.01, 0.03) to 0.15 (U.S. setting, 95% CI 0.10, 0.21) as the imposed time to diabetes onset was increased from 1 to 9 years, respectively. Savings in complication costs increased from $1,388 (95% CI $1,092, $1,669) for a 1-year delay to $8,437 (95% CI $6,611, $10,197) for a delay of 9 years. Interventions costing up to $567-$2,680 and £201-£947 per year would be cost-effective at $100,000 per QALY and £20,000 per QALY thresholds in the U.S. and U.K., respectively, as the modeled delay in diabetes onset was increased from 1 to 9 years.

Conclusions: Simulating a hypothetical diabetes-delaying intervention provides guidance concerning the maximum cost and minimum delay in diabetes onset needed to be cost-effective. These results can inform the ongoing debate about diabetes prevention strategies and the design of future intervention studies.
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http://dx.doi.org/10.2337/dc20-0717DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7510029PMC
October 2020

Association of 21-Gene Assay (OncotypeDX) Testing and Receipt of Chemotherapy in the Medicare Breast Cancer Patient Population Following Initial Adoption.

Clin Breast Cancer 2020 12 26;20(6):487-494.e1. Epub 2020 May 26.

Department of Health Management and Policy, University of Michigan School of Public Health, Ann Arbor, MI.

Background: Our objective was to investigate why early studies regarding adoption of the 21-gene recurrence score (RS) assay did not show an initial reduction in the number of patients with breast cancer receiving real-world chemotherapy.

Materials And Methods: We addressed 2 sources of confounding suspected in previous studies: (1) the early time frame during the initial adoption phase of the RS assay, and (2) suspected selective, increased administration to patients more likely to have been chemotherapy candidates. To address selective use during initial adoption, we used updated SEER-Medicare data from 2004 and 2011. To address individual selection bias, we examined whether RS test utilization was negatively associated with rates of local chemotherapy use assessed at the hospital referral region level using conventional ordinary least squares and instrumental variable approaches to adjust for selection bias.

Results: A total of 26,009 patients met inclusion criteria. Assay use was associated with a decrease in absolute percentage use of chemotherapy of 4.5% (95% confidence interval [CI], 3.2%-5.7%), which was even more pronounced in sensitivity analyses limited to later study years (2008-2011), with a decrease of 6.8% (95% CI, 5.3%-8.3%). Instrumental variable models yielded similar point estimates but were insufficiently powered to draw conclusions.

Conclusion: Receipt of the 21-gene assay was associated with decreased utilization of chemotherapy by 2008.
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http://dx.doi.org/10.1016/j.clbc.2020.05.010DOI Listing
December 2020

Cost-Effectiveness of Alirocumab in Patients With Acute Coronary Syndromes: The ODYSSEY OUTCOMES Trial.

J Am Coll Cardiol 2020 05;75(18):2297-2308

Assistance Publique-Hôpitaux de Paris, Hôpital Bichat, Université de Paris, French Alliance for Cardiovascular Trials, Institut National de la Santé et de la Recherche Médicale U1148, Paris, France; National Heart and Lung Institute, Imperial College, Royal Brompton Hospital, London, United Kingdom. Electronic address: https://twitter.com/gabrielsteg.

Background: Cholesterol reduction with proprotein convertase subtilisin-kexin type 9 inhibitors reduces ischemic events; however, the cost-effectiveness in statin-treated patients with recent acute coronary syndrome remains uncertain.

Objectives: This study sought to determine whether further cholesterol reduction with alirocumab would be cost-effective in patients with a recent acute coronary syndrome on optimal statin therapy.

Methods: A cost-effectiveness model leveraging patient-level data from ODYSSEY OUTCOMES (Evaluation of Cardiovascular Outcomes After an Acute Coronary Syndrome During Treatment With Alirocumab) was developed to estimate costs and outcomes over a lifetime horizon. Patients (n = 18,924) had a recent acute coronary syndrome and were on high-intensity or maximum-tolerated statin therapy, with a baseline low-density lipoprotein cholesterol (LDL-C) level ≥70 mg/dl, non-high-density lipoprotein cholesterol ≥100 mg/dl, or apolipoprotein B ≥80 mg/l. Alirocumab 75 mg or placebo was administered subcutaneously every 2 weeks. Alirocumab was blindly titrated to 150 mg if LDL-C remained ≥50 mg/dl or switched to placebo if 2 consecutive LDL-C levels were <15 mg/dl. Incremental cost per quality-adjusted life-year (QALY) was determined with the addition of alirocumab versus placebo and, based on clinical efficacy findings from the trial, was stratified by baseline LDL-C levels ≥100 mg/dl and <100 mg/dl.

Results: Across the overall population recruited to the ODYSSEY OUTCOMES trial, using an annual treatment cost of US$5,850, the mean overall incremental cost-effectiveness ratio was US$92,200 per QALY (base case). The cost was US$41,800 per QALY in patients with baseline LDL-C ≥100 mg/dl, whereas in those with LDL-C <100 mg/dl the cost per QALY was US$299,400. Among patients with LDL-C ≥100 mg/dl, incremental cost-effectiveness ratios remained below US$100,000 per QALY across a wide variety of sensitivity analyses.

Conclusions: In patients with a recent acute coronary syndrome on optimal statin therapy, alirocumab improves cardiovascular outcomes at costs considered intermediate value, with good value in patients with baseline LDL-C ≥100 mg/dl but less economic value with LDL-C <100 mg/dl. (Evaluation of Cardiovascular Outcomes After an Acute Coronary Syndrome During Treatment With Alirocumab [ODYSSEY OUTCOMES]; NCT01663402).
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http://dx.doi.org/10.1016/j.jacc.2020.03.029DOI Listing
May 2020

Are Videos or Text Better for Describing Attributes in Stated-Preference Surveys?

Patient 2020 08;13(4):401-408

Duke Clinical Research Institute, Duke University, 200 Morris Street, Durham, NC, 27701, USA.

Objective: In stated-preference research, the conventional approach to describing study attributes is through text, often with easy-to-understand graphics. More recently, researchers have begun to present attribute descriptions and content in videos. Some experts have expressed concern regarding internalization and retention of information conveyed via video.

Objective: Our study aimed to compare respondents' understanding of attribute information provided via text versus video.

Methods: Potential respondents were randomized to receive a text or video version of the survey. In the text version, all content was provided in text format along with still graphics. In the video version, text content was interspersed with four video clips, providing the same information as the text version. In both versions, 10 questions were embedded to assess respondents' understanding of the information presented relating to ovarian cancer treatments. Half of the questions were on treatment benefits and the other half were on treatment-related risks. Some questions asked about the decision context and definitions of treatment features, and others asked about the graphic presentation of treatment features. Preferences for ovarian cancer treatments were also compared between respondents receiving text versus video versions.

Results: Overall, 150 respondents were recruited. Of the 95 who were eligible and completed the survey, 54 respondents received the text version and 41 received the video version. Median times to completion were 24 and 30 min in the video and text arms, respectively (p < 0.01). Both groups spent an average of 35 min completing the survey. On the first comprehension question, 43% in the text arm and 61% in the video arm provided the correct response (p = 0.08). Although the mean number of correct responses was significantly higher in the video versus text arms (9.1 vs. 8.6, p = 0.02), there were no systematic differences in preferences between arms.

Conclusions: The quality of stated-preference data relies on respondents' understanding of study content. Information provided via video may better engage survey participants and improve their retention of content.
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http://dx.doi.org/10.1007/s40271-020-00416-9DOI Listing
August 2020

Moving Beyond the Tip of the Iceberg to Incorporate Patients' Perspectives and Outcomes in the Medical Device Regulatory Process.

Value Health 2020 03;23(3):298-299

RTI Health Solutions, Research Triangle Park, NC, USA. Electronic address:

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http://dx.doi.org/10.1016/j.jval.2020.01.008DOI Listing
March 2020

Patient Preferences Around Extent of Surgery in Low-Risk Thyroid Cancer: A Discrete Choice Experiment.

Thyroid 2020 07 15;30(7):1044-1052. Epub 2020 Apr 15.

Department of Surgery, University of California San Francisco-UCSF, San Francisco, California, USA.

Patient preferences pertaining to surgical options for thyroid cancer management are not well studied. Our aim was to conduct a discrete choice experiment (DCE) to characterize participants' views on the relative importance of various risks and benefits associated with lobectomy versus total thyroidectomy for low-risk thyroid cancer. Adult participants with low-risk thyroid cancer or a thyroid nodule requiring surgery were asked to choose between experimentally designed surgical options with varying levels of risk of nerve damage (1%, 9%, 14%), hypocalcemia (0%, 3%, 8%), risk of needing a second surgery (0%, 40%), cancer recurrence (1%, 3%, 5%), and need for daily thyroid hormone supplementation (yes, no). Their choices were analyzed using random-parameters logit regression. One hundred fifty participants completed an online DCE survey. Median age was 58 years; 82% were female. Twenty-four participants (16%) had a diagnosis of thyroid cancer at the time of completing the survey, and 126 (84%) had a thyroid nodule necessitating surgery. On average, 35% of participants' choices were explained by differences in the risk of cancer recurrence; 28% by the chance of needing a second surgery; 19% by the risk of nerve damage; and 9% by differences in risks of hypocalcemia and the need for thyroid hormone supplementation. When accounting for differences in postoperative risks, the average patient favored lobectomy over total thyroidectomy as long as the chance of needing a second (i.e., completion) surgery after initial lobectomy remained below 30%. Participants would accept a 4.1% risk of cancer recurrence if the risk of a second surgery could be reduced from 40% to 10%. While patients with thyroid cancer may have clear preferences for extent of surgery, common themes moderating preferences for surgical interventions were identified in the DCE. Adequate preoperative evaluation to decrease the chance of a second surgery and providing patients with a good understanding of risks and benefits associated with extent of surgery can lead to better treatment decision-making.
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http://dx.doi.org/10.1089/thy.2019.0590DOI Listing
July 2020

Patient preferences for maintenance PARP inhibitor therapy in ovarian cancer treatment.

Gynecol Oncol 2020 03 22;156(3):561-567. Epub 2020 Jan 22.

Duke Cancer Institute, Duke University Medical Center, Durham, NC, United States of America; Duke Clinical Research Institute, Duke University School of Medicine, Durham, NC, United States of America; Department of Population Health Sciences, Duke University School of Medicine, Durham, NC, United States of America.

Objective: To measure preferences of women with ovarian cancer regarding risks, side effects, costs and benefits afforded by maintenance therapy (MT) with a poly ADP ribose polymerase (PARP) inhibitor.

Methods: A discrete-choice experiment elicited preferences of women with ovarian cancer regarding 6 attributes (levels in parentheses) relevant to decisions for MT versus treatment break: (1) overall survival (OS; 36, 38, 42 months); (2) progression-free survival (PFS; 15, 17, 21 months); (3) nausea (none, mild, moderate); (4) fatigue (none, mild, moderate); (5) probability of death from myelodysplastic syndrome/acute myelogenous leukemia (MDS/AML; 0% to 10%); (6) monthly out-of-pocket cost ($0 to $1000). Participants chose between 2 variable MT scenarios and a static scenario representing treatment break, with multiple iterations. Random-parameters logit regression was applied to model choices as a function of attribute levels.

Results: 95 eligible participants completed the survey; mean age was 62, 48% had recurrence, and 17% were ever-PARP inhibitor users. Participants valued OS (average importance weight 24.5 out of 100) and monthly costs (24.6) most highly, followed by risk of death from MDS/AML (17.9), nausea (14.7), PFS (10.5) and fatigue (7.8). Participants would accept 5% risk of MDS/AML if treatment provided 2.2 months additional OS or 4.8 months PFS. Participants would require gains of 2.6 months PFS to accept mild treatment-related fatigue and 4.4 months to accept mild nausea.

Conclusions: When considering MT, women with ovarian cancer are most motivated by gains in OS. Women expect at least 3-4 months of PFS benefit to bear mild side effects of treatment.
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http://dx.doi.org/10.1016/j.ygyno.2020.01.026DOI Listing
March 2020

Within-Trial Evaluation of Medical Resources, Costs, and Quality of Life Among Patients With Type 2 Diabetes Participating in the Exenatide Study of Cardiovascular Event Lowering (EXSCEL).

Diabetes Care 2020 02 5;43(2):374-381. Epub 2019 Dec 5.

Diabetes Trials Unit, University of Oxford, Oxford, U.K.

Objective: To compare medical resource use, costs, and health utilities for 14,752 patients with type 2 diabetes who were randomized to once-weekly exenatide (EQW) or placebo in addition to usual diabetes care in the Exenatide Study of Cardiovascular Event Lowering (EXSCEL).

Research Design And Methods: Medical resource use data and responses to the EuroQol 5-Dimension (EQ-5D) instrument were collected at baseline and throughout the trial. Medical resources and medications were assigned values by using U.S. Medicare payments and wholesale acquisition costs, respectively. Secondary analyses used English costs.

Results: Patients were followed for an average of 3.3 years, during which time those randomized to EQW experienced 0.41 fewer inpatient days (7.05 vs. 7.46 days; relative rate ratio 0.91; = 0.05). Rates of outpatient medical visits were similar, as were total inpatient and outpatient costs. Mean costs for nonstudy diabetes medications over the study period were ∼$1,600 lower with EQW than with placebo ( = 0.01). Total within-study costs, excluding study medication, were lower in the EQW arm than in the placebo arm ($28,907 vs. $30,914; ≤ 0.01). When including the estimated cost of EQW, total mean costs were significantly higher in the EQW group than in the placebo group ($42,697 vs. $30,914; < 0.01). With English costs applied, mean total costs, including exenatide costs, were £1,670 higher in the EQW group than the placebo group (£10,874 vs. £9,204; < 0.01). There were no significant differences in EQ-5D health utilities between arms over time.

Conclusions: Medical costs were lower in the EQW arm than the placebo arm, but total costs were significantly higher once the cost of branded exenatide was incorporated.
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http://dx.doi.org/10.2337/dc19-0950DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7210004PMC
February 2020

Patients' Willingness to Accept Mitral Valve Procedure-Associated Risks Varies Across Severity of Heart Failure Symptoms.

Circ Cardiovasc Interv 2019 12 22;12(12):e008051. Epub 2019 Nov 22.

Duke Clinical Research Institute (S.D.R., A.O.F., F.R.J., J.M.G., R.J.M., M.W.K., S.V.), Duke University School of Medicine, Durham, NC.

Background: The Food and Drug Administration's Center for Drugs and Radiological Health issued Guidance in 2016 on generating patient preference information to aid evaluation of medical devices. Consistent with this guidance, we aimed to provide quantitative patient preference evidence on benefit-risk tradeoffs relevant to transcatheter mitral valve repair versus medical therapy for patients with heart failure and symptomatic secondary mitral regurgitation.

Methods: A discrete-choice experiment survey was designed to quantify patients' tolerance for 30-day mortality or serious bleeding risks to achieve improvements in physical functioning or reductions in heart failure hospitalizations. Two samples were recruited: an online US panel of individuals reporting a diagnosis of heart failure (n=244) and patients with heart failure treated at Duke University Health System (n=175). Random-effects logit regression was used to model treatment choices as a function of benefit and risk levels.

Results: Across both samples, approximately one-quarter (23.5%) consistently chose device profiles offering the higher level of physical functioning despite mortality and bleeding risks as high as 10%. Among respondents who at least once chose a device profile offering a lower level of functioning, improvement in physical functioning equivalent to a change from New York Heart Association class IV to III was ≈6 times more preferred than a change from New York Heart Association class III to II. Estimated discrete-choice experiment utility gains and losses revealed that respondents would accept up to a 9.7 percentage-point (95% CI, 8.2%-13.3%) increase in risk of 30-day mortality with devices that could improve functioning from New York Heart Association class IV to III, or up to 2.0% (95% CI, 1.4%-2.7%) for an improvement from New York Heart Association class III to II.

Conclusions: Severity of heart failure symptoms influences patients' willingness to accept risks associated with mitral valve medical devices. These findings can inform shared decision-making discussions with patients who are being evaluated for transcatheter mitral valve repair.
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http://dx.doi.org/10.1161/CIRCINTERVENTIONS.119.008051DOI Listing
December 2019

Cytomegalovirus in Allogeneic Hematopoietic Transplantation: Impact on Costs and Clinical Outcomes Using a Preemptive Strategy.

Biol Blood Marrow Transplant 2020 03 9;26(3):568-580. Epub 2019 Nov 9.

Division of Hematologic Malignancies and Cellular Therapy, Department of Medicine, Duke University Medical Center, Durham, North Carolina. Electronic address:

Cytomegalovirus (CMV) results in significant morbidity and mortality following hematopoietic cell transplantation (HCT). Establishing the cost and clinical impact is imperative to the selection of appropriate CMV preventative strategies. This is a retrospective cohort study of consecutive patients undergoing their first allogeneic HCT between January 1, 2009, and December 31, 2013. Detailed clinical and institutional cost data were obtained from the start of conditioning through 1-year post-transplantation. Baseline characteristics, resource utilization, costs, and outcomes were compared between patients with and without clinically significant CMV infection (csCMVi). One hundred seventy out of 388 patients (44%) developed csCMVi within 1 year after HCT. Within the first year post-HCT, patients with csCMVi had a significantly longer transplantation-related length of stay (mean, 91.7 days versus 78.3 days; P < .0001) and more frequent and prolonged hospitalizations (mean, 2.4 versus 1.7 admissions [P < .0001]; mean, 39.1 versus 31.5 inpatient days [P = .001]) without significantly more admissions to the intensive care unit (28.2% versus 21.6%; P = .408). The use of granulocyte colony-stimulating factor was greater in patients with csCMVi (73.5% versus 54.1%; P = .0001), although no significant differences were demonstrated in mean platelet or red blood cell (RBC) transfusions. Total costs were also higher in patients with csCMVi (mean cost difference, $45,811; 95% CI, $26,385 to $67,544). However, the incidence of graft-versus-host disease (GVHD) and selected infectious complications was not significantly different between the 2 groups. There were no significant differences in 1-year and 5-year post-transplantation overall survival (OS) or nonrelapse mortality (NRM) between those with and those without csCMVi, although relapse of underlying disease was significantly lower in the csCMVi group. Overall, our data show that allogeneic HCT recipients with csCMVi had significantly greater medical resource utilization and costs than those without csCMVi. However, clinical outcomes, including GVHD, infections, and mortality, were similar in the 2 groups. Further study is needed to determine the cost-effectiveness of CMV preventive modalities.
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http://dx.doi.org/10.1016/j.bbmt.2019.11.005DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7418947PMC
March 2020

Something Is Better Than Nothing: The Value of Active Intervention in Stated Preferences for Treatments to Delay Onset of Alzheimer's Disease Symptoms.

Value Health 2019 09 20;22(9):1063-1069. Epub 2019 Jul 20.

Janssen R&D, LLC, Department of Epidemiology, Titusville, NJ, USA.

Background: The objective of the study was to understand respondents' willingness to accept hypothetical treatment-related risks in return for the benefit of additional time with normal memory from potential Alzheimer's disease interception therapies.

Methods: A US web-based discrete-choice survey was administered to respondents ages 60 to 85 years with no Alzheimer's disease diagnosis and no cognitive symptoms. Choice questions required respondents to indicate whether they preferred a constant, no-treatment condition described as 4 years of normal memory followed by 3 years of cognitive impairment and 5 years of dementia or an interception treatment with chosen risks of disabling stroke and death, but with increased duration of normal memory. The study design included internal validity tests to verify data quality.

Results: On average, respondents were willing to accept a 5% to 13% risk of stroke or death in the first year for treatments that could provide 1 or more additional years with normal memory. Nevertheless, 30% of respondents failed a simple internal-validity test question where the treatment alternative offered no improvement in disease progression but had significant side effects. These respondents also were more likely to choose active treatment in the subsequent series of choice questions. This unexpected finding is consistent with hopeful attitudes of patients with debilitating and potentially fatal conditions.

Conclusion: Pro-treatment attitudes are clinically relevant and can affect the analysis and interpretation of stated-preference data. Internal-validity tests generally are underutilized in preference research. This study demonstrated how analysis of apparent validity failures can yield important insights about patient preferences.
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http://dx.doi.org/10.1016/j.jval.2019.03.022DOI Listing
September 2019

Patient preferences for attributes of primary surgical debulking versus neoadjuvant chemotherapy for treatment of newly diagnosed ovarian cancer.

Cancer 2019 12 27;125(24):4399-4406. Epub 2019 Aug 27.

Duke Cancer Institute, Duke University Medical Center, Durham, North Carolina.

Background: Randomized trials have reported conflicting findings on survival for advanced-stage ovarian cancer treated with primary debulking surgery (PDS) versus neoadjuvant chemotherapy with interval debulking; surgical complications and mortality are higher with PDS. We assessed women's preferences for tradeoffs related to this important clinical decision.

Methods: Ovarian cancer patients were recruited to complete a discrete-choice experiment (DCE) consisting of 8 choice tasks presenting experimentally designed treatment alternatives in terms of treatment order, extent of surgery including risk of ostomy, chance of death from surgical complications (1%-10%), readmission for surgical complications (5%-50%), progression-free survival (1-3 years), and overall survival (3-5 years). Random-parameters logit regression was applied to model participants' choices as a function of attribute levels.

Results: A total of 101 ovarian cancer survivors completed the DCE survey; of these participants, 30% were receiving chemotherapy at the time, and 33% had prior recurrence. Overall survival was of greatest importance to participants (36/100), followed by risk of readmission due to complications (23/100), progression-free survival (19/100), surgical mortality (16/100), extent of surgery (4/100), and order of surgery and chemotherapy (2/100). Overall, the participants would tolerate a 15-percentage point increase in risk of major complications (95% confidence interval [CI], 3%-29%) or a 4-percentage point increase in the risk of surgical mortality (95% CI, 2%-13%) in order to increase their expected overall survival from 3 to 3.5 years.

Conclusions: Patients would accept a moderately higher risk of perioperative complications and surgical mortality in exchange for substantial gains in survival. These quantitative findings provide clinicians with a framework to discuss preferences with patients and to incorporate preferences into clinical trial design.
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http://dx.doi.org/10.1002/cncr.32447DOI Listing
December 2019

Comparing the Noncomparable: The Need for Equivalence Measures That Make Sense in Health-Economic Evaluations.

Value Health 2019 06 17;22(6):684-692. Epub 2019 May 17.

Division of Gastroenterology, Department of Biostatistics and Epidemiology, University of Pennsylvania, PA, USA.

Background: The popularity of quality-adjusted life years (QALYs) has been resistant to concerns about validity and reliability. Utility-theoretic outcome equivalents are widely used in other areas of applied economics. Equivalence values can be derived for time, money, risk, and other metrics. These equivalence measures preserve all available information about individual preferences and are valid measures of individual welfare changes.

Objective: The objective of this study was to derive alternative generalized equivalence measures from first principles and illustrate their application in an empirical comparative-effectiveness example.

Methods: We specify a general-equilibrium model incorporating neoclassical utility functions, health production function, severity-duration preferences, and labor-market tradeoff function. The empirical implementation takes advantage of discrete-choice experiment methods that are widely accepted in other areas of applied economics and increasingly in health economics. We illustrate the practical significance of restrictive QALY assumptions using comparative-effectiveness results based on both QALYs and estimates of welfare-theoretic time-equivalent values for anti-tumor necrosis factor and prolonged corticosteroid treatments for Crohn's disease in three distinct preference classes.

Results: The QALY difference between the two treatments is 0.2 months, while time-equivalent values range between 0.5 and 1.3 months for aggregate and class-specific differences. Thus, the QALY-based analysis understates welfare-theoretic values by 60%-85%.

Conclusion: These results suggest that using disease-specific equivalence values offer a meaningful alternative to QALYs to compare global outcomes across treatments. The equivalence values approach is consistent with principles of welfare economics and offers several features not represented in QALYs, including accounting for preference nonlinearities in disease severity and duration, inclusion of preference-relevant nonclinical healthcare factors, representing preferences of clinically-relevant patient subpopulations, and including utility losses related to risk aversion.
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http://dx.doi.org/10.1016/j.jval.2019.03.011DOI Listing
June 2019
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