Effect of Default Options in Advance Directives on Hospital-Free Days and Care Choices Among Seriously Ill Patients: A Randomized Clinical Trial.

Importance: There is limited evidence regarding how patients make choices in advance directives (ADs) or whether these choices influence subsequent care. Objective: To examine whether default options in ADs influence care choices and clinical outcomes. Design, Setting, and Participants: This randomized clinical trial included 515 patients who met criteria for having serious illness and agreed to participate. Patients were enrolled at 20 outpatient clinics affiliated with the University of Pennsylvania Health System and the University of Pittsburgh Medical Center from February 2014 to April 2016 and had a median follow-up of 18 months. Data analysis was conducted from November 2018 to April 2019. Interventions: Patients were randomly assigned to complete 1 of the 3 following ADs: (1) a comfort-promoting plan of care and nonreceipt of potentially life-sustaining therapies were selected by default (comfort AD), (2) a life-extending plan of care and receipt of potentially life-sustaining therapies were selected by default (life-extending AD), or (3) no choices were preselected (standard AD). Main Outcomes and Measures: This trial was powered to rule out a reduction in hospital-free days in the intervention groups. Secondary outcomes included choices in ADs for an overall comfort-oriented approach to care, choices to forgo 4 forms of life support, patients' quality of life, decision conflict, place of death, admissions to hospitals and intensive care units, and costs of inpatient care. Results: Among 515 patients randomized, 10 withdrew consent and 13 were later found to be ineligible, leaving 492 (95.5%) in the modified intention-to-treat (mITT) sample (median [interquartile range] age, 63 [56-70] years; 279 [56.7%] men; 122 [24.8%] black; 363 [73.8%] with cancer). Of these, 264 (53.7%) returned legally valid ADs and were debriefed about their assigned intervention. Among these, patients completing comfort ADs were more likely to choose comfort care (54 of 85 [63.5%]) than those returning standard ADs (45 of 91 [49.5%]) or life-extending ADs (33 of 88 [37.5%]) (P = .001). Among 492 patients in the mITT sample, 57 of 168 patients [33.9%] who completed the comfort AD, 47 of 165 patients [28.5%] who completed the standard AD, and 35 of 159 patients [22.0%] who completed the life-extending AD chose comfort care (P = .02), with patients not returning ADs coded as not selecting comfort care. In mITT analyses, median (interquartile range) hospital-free days among 168 patients assigned to comfort ADs and 159 patients assigned to life-extending default ADs were each noninferior to those among 165 patients assigned to standard ADs (standard AD: 486 [306-717] days; comfort AD: 554 [296-833] days; rate ratio, 1.05; 95% CI, 0.90-1.23; P < .001; life-extending AD: 550 [325-783] days; rate ratio, 1.03; 95% CI, 0.88-1.20; P < .001). There were no differences among groups in other secondary outcomes. Conclusions and Relevance: In this randomized clinical trial, default options in ADs altered the choices seriously ill patients made regarding their future care without changing clinical outcomes. Trial Registration: ClinicalTrials.gov Identifier: NCT02017548.

New evidence of state variation in Medicaid payment policies for dual Medicare-Medicaid enrollees.

OBJECTIVE: To develop the first longitudinal database of state Medicaid policies for paying the cost sharing in Medicare Part B for services provided to dual Medicare-Medicaid enrollees ("duals") and an index summarizing the impact of these policies on payments for physician office services. DATA SOURCES: Medicaid policy data collected from electronic sources and inquiries with states. STUDY DESIGN: We constructed a national database of Medicaid payment policies for the period 2004-2018, consolidating information from online Medicaid policy documents, state laws, and policy data reported to us by state Medicaid programs. Using this database and state Medicaid fee schedules, we constructed a Medicaid payment index for duals. This index represented the proportion of the Medicare allowed amount that physicians would expect to be paid from Medicare and Medicaid for a subset of physician office services (evaluation and management services) based on annual state payment policies and Medicaid fee schedules. PRINCIPAL FINDINGS: In 2018, 42 states had policies to limit Medicaid payments of Medicare cost sharing when Medicaid's fee schedule was lower than Medicare's-an increase from 36 such states in 2004. In the preponderance of states with these policies, combined Medicare and Medicaid payments for evaluation and management services provided to duals averaged 78 percent of the Medicare allowed amount for these services, reflecting relatively low Medicaid fee schedules in these states. In 2013 and 2014, physicians who qualified for the Affordable Care Act's Medicaid "fee bump" were paid 100 percent of the Medicare allowed amount for these services. CONCLUSIONS: Medicaid programs vary across states and over time in their payments of cost sharing for physician office services provided to duals. Our database and index can facilitate monitoring of these policies and research on the consequences of policy changes for duals.

Accuracy of the Pediatric End-stage Liver Disease Score in Estimating Pretransplant Mortality Among Pediatric Liver Transplant Candidates.

Importance: Fair allocation of livers between pediatric and adult recipients is critically dependent on the accuracy of mortality estimates afforded by the Pediatric End-stage Liver Disease (PELD) and Model for End-stage Liver Disease, respectively. Widespread reliance on exceptions for pediatric recipients suggests that the 2 systems may not be comparable. Objective: To evaluate the accuracy of the PELD score in estimating 90-day pretransplant mortality among pediatric patients on the United Network for Organ Sharing (UNOS) waiting list. Design, Setting, and Participants: Patients who were listed from February 27, 2002, to March 31, 2014, for primary liver transplant were included in this retrospective analysis and were followed up for at least 2 years through June 17, 2016. The study analyzed 2 cohorts using the UNOS Standard Transplant Analysis and Research data files. The full cohort comprised 4298 patients (<18 years of age) who had chronic liver disease (excluding cancer). The reduced cohort (n = 2421) excluded patients receiving living donor transplantation or PELD exception points. Main Outcomes and Measures: Observed and expected 90-day pretransplant mortality rates evaluated at 10-point interval PELD levels. Results: Among the 4298 patients in the full cohort (mean [SD] age, 2.5 [4.2] years; 2251 [52.4%] female; 2201 [51.2%] white), PELD scores and mortality were concordant (C statistic, 0.8387 [95% CI, 0.8191-0.8584] for the full cohort and 0.8123 [95% CI, 0.7919-0.8327] for the reduced cohort). However, the estimated 90-day mortality using the PELD score underestimated the actual probability of death by as much as 17%. Conclusions and Relevance: With use of the PELD score, the ranking of risk among children was preserved, but direct comparisons between adult and pediatric candidates were not accurate. Children with chronic liver disease who are in need of transplant may be at a disadvantage compared with adults in a similar situation.

Using time-varying models to estimate post-transplant survival in pediatric liver transplant recipients.

PURPOSE: To distinguish clinical factors that have time-varying (as opposed to constant) impact upon patient and graft survival among pediatric liver transplant recipients. METHODS: Using national data from 2002 through 2013, we examined potential clinical and demographic covariates using Gray's piecewise constant time-varying coefficients (TVC) models. For both patient and graft survival, we estimated univariable and multivariable Gray's TVC, retaining significant covariates based on backward selection. We then estimated the same specification using traditional Cox proportional hazards (PH) models and compared our findings. RESULTS: For patient survival, covariates included recipient diagnosis, age, race/ethnicity, ventilator support, encephalopathy, creatinine levels, use of living donor, and donor age. Only the effects of recipient diagnosis and donor age were constant; effects of other covariates varied over time. We retained identical covariates in the graft survival model but found several differences in their impact. CONCLUSION: The flexibility afforded by Gray's TVC estimation methods identify several covariates that do not satisfy constant proportionality assumptions of the Cox PH model. Incorporating better survival estimates is critical for improving risk prediction tools used by the transplant community to inform organ allocation decisions.

Gray's time-varying coefficients model for posttransplant survival of pediatric liver transplant recipients with a diagnosis of cancer.

Transplantation is often the only viable treatment for pediatric patients with end-stage liver disease. Making well-informed decisions on when to proceed with transplantation requires accurate predictors of transplant survival. The standard Cox proportional hazards (PH) model assumes that covariate effects are time-invariant on right-censored failure time; however, this assumption may not always hold. Gray's piecewise constant time-varying coefficients (PC-TVC) model offers greater flexibility to capture the temporal changes of covariate effects without losing the mathematical simplicity of Cox PH model. In the present work, we examined the Cox PH and Gray PC-TVC models on the posttransplant survival analysis of 288 pediatric liver transplant patients diagnosed with cancer. We obtained potential predictors through univariable (P < 0.15) and multivariable models with forward selection (P < 0.05) for the Cox PH and Gray PC-TVC models, which coincide. While the Cox PH model provided reasonable average results in estimating covariate effects on posttransplant survival, the Gray model using piecewise constant penalized splines showed more details of how those effects change over time.