Stage III melanoma incidence and impact of transitioning to the 8th AJCC staging system: a US population-based study.

AIM: To estimate incidence of stage III melanoma using the American Joint Committee on Cancer (AJCC) staging, 7th and 8th edition. PATIENTS & METHODS: The SEER US cancer registry was analyzed (2010-2014). AJCC7 stages were recorded in the data; AJCC8 stages were inferred. RESULTS: Of 106,195 melanoma patients, 7669 and 7342 had stage III melanoma by AJCC7 and AJCC8, respectively (95% overlap). Nearly 30% of patients with AJCC7 stage III melanoma were reclassified in a higher stage III group by AJCC8 versus 7% in lower stage group. Regardless of the AJCC edition, incidence of stage III melanoma has increased from 2010 to 2014 both overall and within each stage III group. CONCLUSION: Providing appropriate management to this growing population of high-risk patients is a priority.

Real-world clinical outcomes and healthcare costs in patients with Crohn's disease treated with vedolizumab versus ustekinumab in the United States.

OBJECTIVE: To compare real-world treatment persistence, dose escalation, rates of opportunistic or serious infections, and healthcare costs in patients with Crohn's disease (CD) receiving vedolizumab (VDZ) vs ustekinumab (UST) in the United States. METHODS: A retrospective observational study in adults with CD initiated on VDZ or UST on/after 26 September 2016, was performed using the IBM Truven Health MarketScan databases (1 January 2009-30 September 2018). Rates of treatment persistence, dose escalation, opportunistic or serious infection-related encounters, and healthcare costs per patient per month (PPPM) were evaluated. Entropy balancing was used to balance patient characteristics between cohorts. Event rates were assessed using weighted Kaplan-Meier analyses and compared between cohorts using log-rank tests. Healthcare costs were compared between cohorts using weighted 2-part models. RESULTS: 589 VDZ and 599 UST patients were included (172 [29.2%] and 117 [19.5%] were bio-naïve, respectively). After weighting, baseline characteristics were comparable between cohorts. No significant difference in rates of treatment persistence (12-month: VDZ, 76.5%; UST, 82.1%; p = .17), dose escalation (12-month: VDZ, 29.3%; UST, 32.7%; p = .97), or opportunistic or serious infection-related encounters were observed between VDZ and UST. Total mean healthcare costs were significantly lower for patients treated with VDZ vs UST (mean cost difference = -$5051 PPPM; p < .01). Findings were consistent in bio-naïve patients. CONCLUSIONS: In this real-world study, similar treatment persistence, dose escalation, and rates of opportunistic or serious infections were observed with VDZ- and UST-treated patients with CD. However, VDZ was associated with a significantly lower cost outlay for healthcare systems.

Crohn's disease (CD) causes inflammation in the digestive system. Vedolizumab (VDZ) and ustekinumab (UST) are therapies for patients with CD. Little is known about the clinical outcomes and healthcare costs of VDZ versus UST in the real world in the United States. We used health claims data and found that VDZ and UST had comparable real-world clinical outcomes. After 12 months of treatment, the proportions of patients with CD who stayed on treatment and those who needed to increase therapy dose were similar with VDZ and UST. The rate of infection was also similar between the two groups of patients. However, the monthly healthcare costs were $5051 less for patients treated with VDZ than with UST. This was mainly due to the lower cost of VDZ, which was almost half of that of UST. The lower treatment costs with VDZ may provide substantial savings for the healthcare system and patients specifically. Future cost-effectiveness studies on VDZ and UST are needed to aid treatment selection for patients with CD.

Mortality among US veterans with a physician-documented diagnosis of pyruvate kinase deficiency.

Real-world studies of pyruvate kinase (PK) deficiency and estimates of mortality are lacking. This retrospective observational study aimed to identify patients with PK deficiency and compare their overall survival (OS) to that of a matched cohort without PK deficiency. Patients with ≥1 diagnosis code related to PK deficiency were selected from the US Veterans Health Administration (VHA) database (01/1995-07/2019); patients with a physician-documented diagnosis were included (PK deficiency cohort; index: date of first diagnosis code related to PK deficiency). Patients in the PK deficiency cohort were matched 1:5 to patients from the general VHA population (non-PK deficiency cohort; index: random visit date during match's index year). OS from index was compared between the two cohorts. Eighteen patients in the PK deficiency cohort were matched to 90 individuals in the non-PK deficiency cohort (both cohorts: mean age 57 years, 94% males; median follow-up 6.0 and 8.0 years, respectively). At follow-up, patients in the non-PK deficiency cohort had significantly longer OS than the PK deficiency cohort (median OS: 17.1 vs. 10.9 years; hazard ratio: 2.3; p = 0.0306). During their first-year post-index, 75% and 40% of the PK deficiency cohort had laboratory-confirmed anemia and iron overload, respectively. Among patients who died, cause of death was highly heterogeneous. These results highlight the increased risk of mortality and substantial clinical burden among patients with PK deficiency. While the intrinsic characteristics of the VHA database may limit the generalizability of the results, this is the first real-world study to characterize mortality in patients with PK deficiency.

Real-world upper endoscopy utilization patterns among patients with gastroesophageal reflux disease, Barrett esophagus, and Barrett esophagus-related esophageal neoplasia in the United States.

This study fills a gap in literature by providing contemporary real-world evidence on the prevalence of patients with gastroesophageal reflux disease (GERD), Barrett esophagus (BE), and Barrett esophagus-related neoplasia (BERN) and their upper endoscopy utilization patterns in the United States. A retrospective cohort study design was used: adults with GERD, nondysplastic Barrett esophagus (NDBE), and BERN (indefinite for dysplasia [IND], low-grade dysplasia [LGD], high-grade dysplasia [HGD], or esophageal adenocarcinoma [EAC]) were identified from the MarketScan databases (January 01, 2015-December 31, 2019). For each disease stage, prevalence of adults in commercial claims by calendar year, annual number of upper endoscopies per patient and time between upper endoscopies were reported. In 2019, in commercial claims (N = 12,363,227), the annual prevalence rate of GERD was 13.7% and 0.70% for BE/BERN, among which, 87.1% had NDBE, 6.8% had IND, 2.3% had LGD, 1.0% had HGD, and 2.8% had EAC. From 2015-2019, the study included 3,310,385 patients with GERD, 172,481 with NDBE, 11,516 with IND, 4332 with LGD, 1549 with HGD, and 11,676 with EAC. Annual mean number of upper endoscopies was 0.20 per patient for GERD, 0.37 per patient for NDBE, 0.43 for IND, 0.58 for LGD, and 0.87 for HGD. Median time (months) to second upper endoscopy was 38.10 for NDBE, 36.63 for IND, 22.63 for LGD, and 11.90 for HGD. Upper endoscopy utilization increased from GERD to BE to BERN, and time between upper endoscopies decreased as the disease stage progressed from BE to BERN, with less frequent utilization in BERN than what would be expected from guideline recommendations for surveillance.

Healthcare Resource Utilization and Costs Among Patients With Gastroesophageal Reflux Disease, Barrett's Esophagus, and Barrett's Esophagus-Related Neoplasia in the United States.

Background: Gastroesophageal reflux disease (GERD) is a risk factor for Barrett's esophagus (BE) and BE-related neoplasia (BERN). Objectives: This study aimed to evaluate healthcare resource utilization (HRU) and costs associated with GERD, BE, and BERN in the United States. Methods: Adult patients with GERD, nondysplastic BE (NDBE), and BERN (including indefinite for dysplasia [IND], low-grade dysplasia [LGD], high-grade dysplasia [HGD] or esophageal adenocarcinoma [EAC]), were identified from a large US administrative claims database, the IBM Truven Health MarketScan® databases (Q1/2015-Q4/2019). Patients were categorized into the corresponding mutually exclusive EAC-risk/diagnosis cohorts based on the most advanced stage from GERD to EAC using diagnosis codes in medical claims. Disease-related HRU and costs (2020 USD) were calculated for each cohort. Results: Patients were categorized into the following EAC-risk/diagnosis cohorts: 3 310 385 into GERD, 172 481 into NDBE, 11 516 into IND, 4332 into LGD, 1549 into HGD, and 11 676 into EAC. Disease-related annual mean number of inpatient admissions, office visits, and emergency department visits by cohort were 0.09, 1.45, and 0.19 for GERD; 0.08, 1.55, and 0.10 for NDBE; 0.10, 1.92, and 0.13 for IND; 0.09, 2.05, and 0.10 for LGD; 0.12, 2.16, and 0.14 for HGD; and 1.43, 6.27, and 0.87 for EAC. Disease-related annual mean total healthcare costs by cohort were $6955 for GERD, $8755 for NDBE, $9675 for IND, $12 241 for LGD, $24 239 for HGD, and $146 319 for EAC. Discussion: Patients with GERD, BE, and BERN had important HRU and costs, including inpatient admissions and office visits. As patients progressed to more advanced stages, there was substantially higher disease-related resource utilization, with associated costs being 16 times higher in patients with EAC than those with NDBE. Conclusions: Findings suggest the need for early identification of high-risk individuals prior to progression to EAC to potentially improve clinical and economic outcomes in this population.

Surgical Treatment Patterns, Healthcare Resource Utilization, and Economic Burden in Patients with Tenosynovial Giant Cell Tumor Who Underwent Joint Surgery in the United States.

Background: Tenosynovial giant cell tumors (TGCT) are rare and locally aggressive neoplasms in synovium, bursae, and tendon sheaths, which cause pain, joint dysfunction, and damage to the affected joints. Objective: To evaluate the surgical patterns and economic burden among patients with TGCT who underwent joint surgery in the United States. Methods: Patients newly diagnosed with TGCT, aged 18-64 years, who underwent joint surgery post-TGCT diagnosis were identified from the OptumHealth Care Solutions, Inc database (Q1/1999-Q1/2017). Patients were required to be continuously enrolled for ≥1 year before and ≥3 years after the first TGCT diagnosis (index date). Surgical patterns were assessed post-index. Healthcare resource utilization and associated healthcare costs, and indirect costs related to work loss in year 1, year 2, and year 3 post-index, were compared with those at baseline. Results: Of 835 eligible TGCT patients, 462 (55%) patients who had ≥1 joint surgery post-index were included. During a median follow-up of 5.7 years, 78% of patients underwent their first joint surgery in year 1 and 41% had ≥1 repeat surgery. Magnetic resonance imaging utilization was highest during baseline (46%) and declined afterward (28%, 17%, and 19% in years 1, 2, and 3, respectively). Opioids and nonsteroidal anti-inflammatory drugs (NSAIDs), and physical therapy, occupational therapy, and rehabilitation services, were commonly used during baseline (45%, 40%, and 30%, respectively). More patients used opioids in year 1 vs baseline (78% vs 45%; P<0.0001), while its utilization return to baseline levels in year 2 (41%) and year 3 (42%). A similar pattern was observed for NSAIDs and physical/occupational therapy/rehabilitation services. Healthcare resource utilization and associated healthcare costs surged in year 1 and returned to baseline or lower in years 2 and 3. A similar pattern was observed for indirect costs associated with work loss. Discussion: The high proportion of patients undergoing repeat surgeries and prevalent use of opioids, NSAIDs, and physical/occupational therapy/rehabilitation services suggests an unmet medical need after surgical treatment. Conclusions: Surgical resection alone might be inadequate to control TGCT. New treatment options may complement surgery and alleviate the clinical and economic burden experienced by patients with TGCT who had received prior surgery.

Erratum: Author Correction: Surgical Treatment Patterns, Healthcare Resource Utilization, and Economic Burden in Patients with Tenosynovial Giant Cell Tumor Who Underwent Joint Surgery in the United States.

[This corrects the article DOI: 10.36469/jheor.2022.32485.].

Monitoring community pharmacist's quality of care: a feasibility study of using pharmacy claims data to assess performance.

BACKGROUND: Public pressure has increasingly emphasized the need to ensure the continuing quality of care provided by health professionals over their careers. Health profession's regulatory authorities, mandated to be publicly accountable for safe and effective care, are revising their quality assurance programs to focus on regular evaluations of practitioner performance. New methods for routine screening of performance are required and the use of administrative data for measuring performance on quality of care indicators has been suggested as one attractive option. Preliminary studies have shown that community pharmacy claims databases contain the information required to operationalize quality of care indicators. The purpose of this project was to determine the feasibility of routine use of information from these databases by regulatory authorities to screen the quality of care provided at community pharmacies. METHODS: Information from the Canadian province of Quebec's medication insurance program provided data on prescriptions dispensed in 2002 by more than 5000 pharmacists in 1799 community pharmacies. Pharmacy-specific performance rates were calculated on four quality of care indicators: two safety indicators (dispensing of contra-indicated benzodiazepines to seniors and dispensing of nonselective beta-blockers to patients with respiratory disease) and two effectiveness indicators (dispensing asthma or hypertension medications to non-compliant patients). Descriptive statistics were used to summarize performance. RESULTS: Reliable estimates of performance could be obtained for more than 90% of pharmacies. The average rate of dispensing was 4.3% (range 0 - 42.5%) for contra-indicated benzodiazepines, 15.2% (range 0 - 100%) for nonselective beta-blockers to respiratory patients, 10.7% (range 0 - 70%) for hypertension medications to noncompliant patients, and 43.3% (0 - 91.6%) for short-acting beta-agonists in over-use situations. There were modest correlations in performance across the four indicators. Nine pharmacies (0.5%) performed in the lowest quartile in all four of the indicators, and 5.3% (n = 95) performed in the lowest quartile on three of four indicators. CONCLUSIONS: Routinely collected pharmacy claims data can be used to monitor indicators of the quality of care provided in community pharmacies, and may be useful in future to identify underperforming pharmacists, measure the impact of policy changes and determine predictors of best practices.

Hospitalizations and healthcare costs associated with rifaximin versus lactulose treatment among commercially insured patients with hepatic encephalopathy in the United States.

AIMS: To assess healthcare costs and hospitalization rates associated with rifaximin therapy versus lactulose alone among patients at risk for hepatic encephalopathy (HE). METHODS AND MATERIALS: IBM Marketscan Commercial and Optum's de-identified Clinformatics Data Mart databases were used separately to identify commercially insured HE patients treated with rifaximin or lactulose alone, using an algorithm developed with clinical experts. HE-related hospitalizations were defined based on an algorithm using diagnosis codes and diagnosis-related group codes. HE-related/all-cause hospital admissions/days and healthcare costs were compared between rifaximin and lactulose episodes using incidence rate ratios and adjusted cost differences. RESULTS: In Marketscan, there were 13,515 [Optum: 5,217] rifaximin episodes and 9,946 [4,897] lactulose alone episodes included. Yearly rates of HE-related hospital admissions decreased by 33% [34%] when treated with rifaximin versus lactulose alone, and rates of HE-related hospital days similarly decreased by 43% [57%]. Yearly rates of all-cause hospital admissions decreased by 27% [27%]; rates of all-cause hospital days decreased by 33% [37%] during rifaximin episodes versus lactulose alone. This translated to $2,417 [$2,301] and $173 [$397] lower total mean medical costs and HE-related hospital costs per-patient-per-month, respectively (p < .05). Despite increased pharmacy costs associated with rifaximin, there was no change in total healthcare costs. Patients adherent to rifaximin incurred $2,891 [$2,340] lower total healthcare costs than non-adherent patients. In a simulated plan of 1 million lives, if 50% of HE patients treated with lactulose alone had rifaximin added on and were adherent to rifaximin therapy, the total cost savings would be $7.5 [$6.1] million per year ($0.62 [$0.50] per-member-per-month). CONCLUSIONS: Patients incurred significantly lower rates of HE-related and all-cause hospitalizations during rifaximin versus lactulose episodes, resulting in lower facility and professional costs. Cost savings may be possible if rifaximin adherence is improved in HE patients. LIMITATIONS: The study is subject to limitations common to claims-based analyses.

Treatment patterns and outcomes in patients with myelodysplastic syndromes treated with hypomethylating agents: a SEER-Medicare analysis.

To describe real-world treatment patterns and outcomes among adult patients with myelodysplastic syndromes (MDS) treated with hypomethylating agents (HMA), patients were identified in the SEER-Medicare database (01/2006-12/2016); 3,046 patients with MDS treated with HMA were included. An algorithm was developed to categorize patients into MDS risk groups: the majority of patients were classified as Higher-risk (70.9%), 8.0% as Intermediate-risk, and 21.1% as Unknown-risk. Overall, 77.4% of patients initiated azacitidine and 22.6% decitabine; they received an average of 5.1 index-HMA cycles, of which 90.9% were complete with a median cycle duration of 28 days. Median survival was 11.6, 18.4, and 19.1 months for the Higher-risk, Intermediate-risk, and Unknown-risk groups, respectively. Median time-to-AML transformation was 19.3 months for the Higher-risk group and 50.4 months for the Intermediate-risk group (not reached for Unknown-risk). Data highlight the unmet medical needs of patients with MDS treated with HMA, particularly for the Higher-risk MDS group.

Nusinersen for Spinal Muscular Atrophy in the United States: Findings From a Retrospective Claims Database Analysis.

INTRODUCTION: Spinal muscular atrophy (SMA) is a rare, genetic neuromuscular disorder caused by deletion/mutation of the survival motor neuron 1 gene, characterized by progressive loss of motor neurons, resulting in increasing muscular weakness, deteriorating motor function, and, in its most severe form, death before 2 years. Nusinersen, an antisense oligonucleotide that increases expression of the functional SMN protein, was approved for SMA by US and European regulatory agencies in 2016 and 2017, respectively. The indicated regimen requires intrathecal injections every 4 months, following the first four injections during the loading phase. Adherence is integral to treatment success. Adherence to nusinersen may pose particular challenges as most patients with SMA are young children who require complex multidisciplinary care (including ongoing intrathecal treatment administration and potential specialized anesthetic and surgical procedures) at specialized centers. However, real-world data on adherence to nusinersen are limited. METHODS: We conducted a retrospective claims database analysis from December 23, 2016, to November 20, 2019, to study nusinersen adherence and discontinuation/persistence in US patients with SMA types 1-3 who completed the loading phase, and to determine the impact of non-adherence or treatment discontinuation on SMA-related comorbidities, health care resource utilization (HCRU), and costs. RESULTS: We identified 23 patients with SMA type 1, 41 patients with SMA type 2, and 260 patients with SMA type 3 who had completed the loading phase. Deviations from the indicated nusinersen treatment schedule were frequent in real-world usage, with most patients receiving ≥1 dose outside the scheduled interval. Across SMA types, non-adherent patients were more likely to have had SMA-related comorbidities (e.g., feeding difficulties, dyspnea and respiratory anomalies, and muscle weakness) and greater HCRU. Persistence rates 12 months after treatment initiation for patients with SMA types 1, 2, and 3 were 55.2%, 42.4%, and 54.6%, respectively. Patients who discontinued nusinersen and those who did not had generally similar comorbidity profiles. Discontinuation was associated with greater health care costs across SMA types. CONCLUSION: Our analysis of claims data indicated that discontinuation and non-adherence to nusinersen treatment were prevalent, and associated with greater frequency of comorbidities, greater HCRU, and increased costs for patients.

Healthcare resource utilization and costs in patients with myelodysplastic syndromes treated with hypomethylating agents: a SEER-Medicare analysis.

AIMS: To describe healthcare resource utilization (HRU) and costs in patients with myelodysplastic syndromes (MDS) treated with hypomethylating agents (HMA) based on HMA-treatment response. MATERIALS AND METHODS: SEER-Medicare data (January 2006-December 2016) were used to identify adults diagnosed with MDS (SEER: January 2009-December 2015) initiated on HMA (index date). HMA-treatment success (indicators: ≥7 HMA cycles, stem cell transplantation, and transfusion independence) or failure (indicators: acute myeloid leukemia [AML], AML-like treatment, and death) was determined using a claim-based algorithm. HRU and costs were assessed from the index date to 1-year post-index, overall and stratified by HMA-treatment success or failure. Among patients with HMA-treatment failure, HRU and costs were also assessed from failure to 1-year post-failure. RESULTS: The study included 3,046 patients (mean age: 77.4 years; females: 36.8%). Rates of HMA-treatment success and failure were 44.4% and 76.2%, respectively (20.6% had HMA-treatment success then failure). Overall, patients had 15.2 inpatient admissions per-100-patients-per-month (median follow-up: 5.9 months). Patients with HMA-treatment success had 7.5 inpatient admissions per-100-patients-per-month (median follow-up: 12.0 months), while those with HMA-treatment failure had 20.4 and 35.3 admissions per-100-patients-per-month pre- and post-HMA-treatment failure, respectively (median follow-up: 4.3 and 1.8 months, pre- and post-HMA-treatment failure, respectively). Mean total healthcare costs were $12,494 per-patient-per-month overall, $8,069 per-patient-per-month among patients with HMA-treatment success, and $13,809 and $19,242 per-patient-per-month pre- and post-HMA-treatment failure, respectively. Outpatient costs (68.3%) were the main contributor of total healthcare costs overall, while inpatient costs (80.3%) were the main cost driver post-HMA-treatment failure. LIMITATIONS: Without available laboratory test results, clinical indicators observed in claims were used to assess HMA-treatment response. CONCLUSIONS: Over 75% of patients with MDS failed HMA-treatment within 6 months of initiation and were observed with more inpatient admissions than those with HMA-treatment success, translating into substantially higher healthcare costs. HMA-treatment failure results in an important economic burden in MDS patients.

Regional variations in prevalence and severity of autosomal dominant polycystic kidney disease in the United States.

OBJECTIVES: To evaluate geographic variation in the prevalence of autosomal dominant polycystic kidney disease (ADPKD) in the US, including ADPKD at risk of rapid progression. METHODS: Claims data from the IBM MarketScan Commercial and Medicare Supplemental databases (01/16/2016-12/31/2017) were used to estimate the 2017 annual and 2016-2017 two-year prevalence of diagnosed ADPKD and ADPKD at risk of rapid progression in the US overall, and stratified by census regions and states. Risk of rapid progression was identified based on either: hypertension <35 years, hematuria <30 years, albuminuria, stage 2 chronic kidney disease (CKD) <30 years, stage 3 CKD <50 years, and stage 4/5 CKD or kidney transplant <55 years. RESULTS: Annual prevalence was estimated at 2.34 and two-year prevalence at 3.61 per 10,000 in the US. Across census regions, two-year prevalence per 10,000 was highest in the Northeast (4.14) and lowest in the West (3.35). Prevalence was significantly correlated with the proportion of individuals in urban areas (r = .34, one-sided p = .026). In 2017, 37.5% of patients were identified as being at risk for rapid progression, and this proportion was larger among patients in the South (42.1%, p < .001). CONCLUSION: This estimate for ADPKD prevalence is consistent with previously reported national estimates, with regional variation suggesting that ADPKD might be under-diagnosed in rural areas with more limited access to care. More than one-third of ADPKD patients presented risk factors associated with rapid progression, highlighting the need for timely identification, as disease-modifying therapy may delay progression to end-stage renal disease.

The Economic Burden of Tenosynovial Giant Cell Tumors Among Employed Workforce in the United States.

OBJECTIVE: To assess the economic burden of tenosynovial giant cell tumor (TGCT) among US employed workforce. METHODS: Patients with TGCT medical claims (N = 1395) and matched controls (1:10) without TGCT claims (N = 13,950) were identified from the OptumHealth Care Solutions, Inc. database (January 1, 1999 to March 31, 2017). Adjusted regression models were used to compare healthcare resource utilization, time lost from work, and associated costs between cohorts. RESULTS: In patients with TGCT, the rates of inpatient admissions, emergency room visits, outpatient visits, and work loss days were 2.8, 1.5, 2.2, and 2.6 times those of matched controls, respectively (all P < 0.001). Total annual all-cause healthcare costs and work loss-related costs were $9368 and $2708 higher for TGCT patients than for matched controls, respectively (all P < 0.001). CONCLUSIONS: TGCT was associated with a significant healthcare and work loss burden on US employers.

Economic burden of relapse/recurrence in patients with major depressive disorder.

Objective: This study was conducted to determine the incremental healthcare resource utilization (HRU) and costs associated with relapse or recurrence (R/R) in patients with major depressive disorder (MDD) treated with antidepressants (AD) in US clinical practice. Methods: In this retrospective cohort study, adult patients with MDD treated with a branded AD were selected from the Truven Health Analytics MarketScan Databases (January 1, 2004-March 31, 2015). Time to first indicator of R/R was described. Characteristics, HRU, and costs were compared between patients with and without R/R. Among patients with R/R, HRU and costs were also compared between the pre- and post-R/R period. Results: From the 22,236 selected patients, 5,541 had ≥ 1 indicator of R/R and 16,695 did not. The 3-year R/R rate varied between 21.3% and 36.4% based on pattern of AD use (continuous, switch/augmentation, or early discontinuation). Patients with and without R/R presented different characteristics-notably, more intensive prior AD use and a higher comorbidity burden. HRU and costs were high in both patients with and without R/R but substantially higher among those with R/R ($20,590 vs $12,368 per-patient-per-year (PPPY); adjusted difference [aDiff] = $7,037), mainly driven by increased inpatient (IP) services (adjusted incidence rate ratio IP days = 3.95; aDiff IP costs = $3,433 PPPY). Among patients with R/R, emergency department visits, IP days, and IP admissions were over 2-times higher during the post-R/R period and total costs increased by over 50% from $19,267 to $29,419 in the post-R/R period. Conclusions: The economic burden in MDD patients is substantial, but is significantly higher among those who experience R/R.

Factors associated with more frequent diagnostic tests and procedures in patients with irritable bowel syndrome.

BACKGROUND: Irritable bowel syndrome (IBS) reduces quality of life and burdens healthcare systems. This study identified factors associated with frequent use of IBS diagnostic tests and procedures. METHODS: Using a United States claims database (2001-2012), tests and procedures in IBS patients occurring in the 2-year study period (12 months before/following the first IBS diagnosis) were analyzed: endoscopy, GI transit testing, anorectal procedures, and radiologic imaging. Patients were classified based on test/procedure frequency (3+, 1-2, or 0). Multivariate logistic regression identified factors associated with more frequent tests/procedures. RESULTS: Among 201,322 IBS patients, 41.7% had 3+ tests/procedures, 35.1% had 1-2, and 23.3% had 0. Patients with more tests/procedures were older [mean age 50.6 (3+ group), more likely to be female and had more comorbidities, including anxiety, depressive disorders, and somatization. Dyspepsia [odds ratio (95% confidence interval): 1.80 (1.72-1.87)], interstitial cystitis [1.60 (1.45-1.77)], gastroesophageal reflux disease [1.59 (1.55-1.63)], constipation [1.50 (1.45-1.54)], and dyspareunia [1.38 (1.25-1.52)] were significantly associated with more tests/procedures (3+ versus 1-2), while anxiety, depressive disorders, and somatization were not. Patients with more frequent specialist visits [emergency department (ED; 1.10 (1.09-1.11)) and gastroenterologists (1.26 (1.26-1.27))] or at least one GI-related ED visit or inpatient admission [1.95 (1.86-2.04) and 3.67 (3.48-3.87), respectively] were more likely to have more tests/procedures (all p < 0.05). CONCLUSIONS: Test frequency in patients with IBS is strongly associated with demographic and clinical characteristics, especially comorbid conditions related to IBS. Presence of common overlapping comorbid conditions should increase clinicians' confidence in making the diagnosis of IBS, thus curtailing redundant testing and reducing healthcare costs.

Second-line rituximab-bendamustine versus rituximab-gemcitabine-oxaliplatin in diffuse large B-cell lymphoma in the real world.

Aim: Despite long-term responses to first-line immunochemotherapy, many patients with diffuse large B-cell lymphoma (DLBCL) have relapsed/refractory disease. Second-line treatment options are available. However, a large proportion of patients are ineligible for transplantation/intensive therapy. Patients & methods: This observational study of 702 patients in the USA, who used second-line therapies for relapsed/refractory DLBCL, evaluated treatment patterns and overall survival (OS). The study focused on the OS outcome of patients receiving second-line rituximab-bendamustine or rituximab-gemcitabine-oxaliplatin. Results & conclusion: Rituximab-bendamustine and rituximab-gemcitabine-oxaliplatin were received by 4.6 and 1.4% of patients, respectively (N = 42/702). Median and 1-year OS rates were similar between regimens. Many of the 200 different treatment regimens observed in second line were modified versions of National Comprehensive Cancer Network regimens.

Comparative Effectiveness of Non-cisplatin First-line Therapies for Metastatic Urothelial Carcinoma: Phase 2 IMvigor210 Study Versus US Patients Treated in the Veterans Health Administration.

BACKGROUND: First-line treatments for cisplatin-ineligible patients with metastatic urothelial carcinoma (mUC) include carboplatin-based chemotherapy and checkpoint inhibitors such as atezolizumab (anti-PD-L1). OBJECTIVE: To compare overall survival (OS) among patients with mUC treated in the first-line setting with atezolizumab versus carboplatin-based chemotherapies (any carboplatin-based regimens or carboplatin-gemcitabine). DESIGN, SETTING, AND PARTICIPANTS: Cisplatin-ineligible patients with mUC from the phase 2 trial IMvigor210 (ClinicalTrials.gov NCT02951767) treated with atezolizumab and patients from the Veterans Health Administration (VHA) health care system (2006-2017, with IMvigor210 eligibility criteria applied using proxy measurements) treated according to normal clinical practice. INTERVENTIONS: IMvigor210 cohort 1 patients were treated with atezolizumab, and real-world VHA cohorts were treated with carboplatin-based regimens. OUTCOME MEASUREMENTS AND STATISTICAL ANALYSIS: Entropy-balance weighting was applied to balance prespecified baseline patient characteristics. OS was analyzed using weighted Kaplan-Meier and Cox methods. RESULTS AND LIMITATIONS: The median OS was 15.0 mo with atezolizumab (n = 110), 12.1 mo with any carboplatin-based chemotherapy (n = 282), and 8.7 mo with carboplatin-gemcitabine (n = 120). An OS benefit occurred with atezolizumab versus carboplatin-based regimens after 9 mo (hazard ratio [HR] 0.43; p = 0.004) and with atezolizumab versus carboplatin-gemcitabine after 5 mo (HR 0.52; p = 0.005). Study limitations include a predominantly male VHA cohort and ≤24-mo follow-up. Adjustment for confounding, a potential limitation of nonrandomized studies, was limited by the availability of clinical measurements in the VHA data, which allowed for replication of IMvigor210 exclusions in the VHA cohorts. CONCLUSIONS: First-line atezolizumab for cisplatin-ineligible mUC may provide an OS benefit over carboplatin-based treatments after 5-9 mo, depending on the regimen. PATIENT SUMMARY: Many patients with metastatic urothelial carcinoma are ineligible for cisplatin-based chemotherapy. This study compared patients from a clinical trial receiving the immunotherapeutic agent atezolizumab with those in Veterans Health Administration clinical practice receiving carboplatin-based chemotherapy. Atezolizumab provided a survival benefit over chemotherapy after 5-9 mo.

Severe adverse events impact overall survival and costs in elderly patients with advanced non-small cell lung cancer on second-line therapy.

OBJECTIVES: Elderly patients with advanced non-small lung cancer (aNSCLC) represent a high-risk patient population due to disease burden, comorbidities, and performance status, particularly after progressing on first-line therapy. Among elderly patients who receive second-line therapy, treatment related toxicities can have substantial impact on both clinical and economic outcomes. This study assessed the impact of severe adverse events (AEs) during second-line therapy on overall survival (OS) and all-cause heathcare costs in elderly with aNSCLC. MATERIALS AND METHODS: Patients with aNSCLC aged ≥65 years who initiated second-line chemotherapy/targeted therapy were identified in the SEER-Medicare database (2007-2011). Fifty-seven AEs were identified by literature review and consultation with two oncologists. Severe AEs were defined as AEs that required a hospitalization and were operationalized based on AE diagnosis(es) recorded during hospitalizations. OS post-second-line initiation and healthcare costs during second-line were compared between patients with and without severe AEs. RESULTS: Among 3967 patients initiating second-line therapy, 1624 (41%) had ≥1 severe AE, where hypertension (26%), anemia (24%), and pneumonia (23%) were most commonly reported. Patients with and without severe AEs had similar demographic and cancer characteristics at diagnosis and similar second-line treatment regimens, but patients with severe AEs had more comorbidities at second-line initiation. Median OS was lower in patients with versus without severe AEs (6 vs. 11 months). After multivariate adjustment, hazard of death was more than twice higher in patients with versus without severe AEs (adjusted hazard ratio [HR] 2.31, 95% CI 2.16-2.47). Healthcare costs were more than twice higher in patients with versus without severe AEs ($16,135 vs. $7559 per-patient-per-month). CONCLUSION: Severe AEs among elderly patients with aNSCLC treated with second-line chemotherapy/targeted therapy were found to be associated with decreased OS and increased healthcare costs. Results suggest a potential link between severe AEs in second-line treated aNSCLC elderly and patient survival and economic burden to the healthcare system.

Treatment patterns, healthcare resource utilization, and costs in patients with acute myeloid leukemia in commercially insured and Medicare populations.

OBJECTIVE: To describe the setting, duration, and costs of induction and consolidation chemotherapy for adults with newly-diagnosed acute myeloid leukemia (AML), who are candidates for standard induction chemotherapy, in the US. METHODS: Adults newly-diagnosed with AML who received standard induction chemotherapy in an inpatient setting were identified from the Truven Health Analytics MarketScan (2006-2015) and SEER-Medicare (2007-2011) databases. Patients were observed from induction therapy start to the first of hematopoietic stem cell transplant, 180 days after induction discharge, health plan enrollment/data availability end, or death. Induction and consolidation chemotherapy were identified using Diagnosis-Related Group codes (chemotherapy with acute leukemia) or procedure codes for AML chemotherapy administration. AML treatment episode setting (inpatient or outpatient), duration, and costs (2015 USD, payers' perspective) were described for commercially insured patients and Medicare beneficiaries. RESULTS: In total, 459 commercially insured patients and 563 Medicare beneficiaries (mean age = 54 and 66 years; 53% and 54% male; respectively) were identified. For induction therapy, mean costs were $145,189 for commercially insured patients and $85,734 for Medicare beneficiaries, and median inpatient duration was 31 days (both). Following induction, 64% of commercially insured patients and 53% of Medicare beneficiaries had ≥1 consolidation cycle; 75% and 65% of consolidation cycles were in an inpatient setting, respectively. For consolidation cycles, in the inpatient setting, mean costs were $28,137 for commercially insured patients and $28,843 for Medicare beneficiaries, median cycle duration was 6 days (both); in the outpatient setting, mean costs were $11,271 for commercially insured patients and $5,803 Medicare beneficiaries, median duration was 5 days (both). LIMITATIONS: Granular information on chemotherapy type administered was unavailable. CONCLUSIONS: This is the first exploratory study providing a complete picture of recent AML treatment patterns and management costs among commercially insured patients and Medicare beneficiaries. There is substantial heterogeneity in the management and costs of AML.