Real-world outcomes in patients with first-line and second-line therapy for advanced esophageal squamous cell carcinoma.

Background: Little is known about real-world outcomes for first-line and anti-PD-1 second-line treatment for advanced/metastatic esophageal squamous cell carcinoma (ESCC). Patients & methods: Retrospective data of advanced/metastatic ESCC patients treated between 2011 and 2021 were collected from Flatiron Health. Median duration of therapy (mDoT) and median overall survival (mOS) were evaluated for patients initiating first-line and anti-PD-1 second-line therapy. Results: Among patients receiving first-line therapy (n = 948), mDoT was 1.4 months and mOS was 16.0 months, with mOS of 16.0 and 18.0 months for the non-immunotherapy and immunotherapy cohorts, respectively. Among patients receiving anti-PD-1 second-line therapy (n = 60), mDoT was 5.7 months and mOS was 10.1 months. Conclusion: Patients with advanced/metastatic ESCC have short duration of therapy, and overall survival remains limited. This real-world study underscores the need for efficacious treatments for advanced/metastatic ESCC in the first- and second-line setting. Direct comparisons of emerging therapies in the real world are urgently needed.

Trends in health care resource use and expenditures in patients with newly diagnosed paroxysmal supraventricular tachycardia in the United States.

BACKGROUND: Few data are available on the temporal patterns of health resource utilization (HRU) and expenditures around paroxysmal supraventricular tachycardia (PSVT) diagnosis. This study assessed the longitudinal trends in HRU and expenditures in the 3 years preceding and subsequent to PSVT diagnosis. METHODS: Adult patients (age 18-65 years) with newly diagnosed PSVT were identified using administrative claims from the IBM MarketScan Research Database between January 1, 2008 and December 31, 2016 and propensity-score matched (1:1) with non-PSVT controls. RESULTS: Among the 12,305 PSVT patients compared with matched controls, PSVT was associated with statistically significant higher annual rates of emergency department visits, physician office visits, inpatient hospitalizations, and diagnostic testing. HRU increased in the years preceding PSVT diagnosis, reaching its peak in the year following PSVT diagnosis. Over the 6-year follow-up period, PSVT was associated with higher mean annual per patient expenditures ($12,665) compared to matched controls ($6,004; P < .001). Upon diagnosis of PSVT, the mean expenditures per PSVT patient doubled from $11,714 in the year immediately preceding index diagnosis to $23,335 in the first postdiagnosis year. Inpatient services, diagnostic testing, and ablation procedures were the principle drivers of higher mean expenditures in the first year post-PSVT diagnosis versus the year prior to PSVT diagnosis. CONCLUSIONS: PSVT presents a substantial economic burden to health care systems. The annual expenditure per PSVT patient is within the range previously reported for atrial fibrillation. The increased HRU and expenditures in the year following diagnosis, which do not return to baseline, suggest a potential gap in non-interventional, long-term PSVT management.

Prevalence and incidence of patients with paroxysmal supraventricular tachycardia in the United States.

BACKGROUND: Paroxysmal supraventricular tachycardia (PSVT) encompasses a range of heart rhythm disorders leading to rapid heart rates. By virtue of its episodic nature, diagnosing PSVT is difficult and estimating incidence and prevalence on a population level is challenging. The objective of this study was to estimate the incidence and prevalence of PSVT in the United States (US) in contemporary practice. METHODS AND RESULTS: An observational retrospective longitudinal study using claims, enrollment, and demographic data from the IBM MarketScan® Commercial Research database (age < 65) and the Medicare Limited Data Set (age ≥ 65) from 2008 to 2016. Patients with a PSVT diagnosis code (ICD-9: 427.0; ICD-10: I47.1) on ≥2 outpatient, ≥1 emergency room, or ≥1 inpatient visit were considered as having PSVT. Patients with atrial fibrillation/atrial flutter (AF/AFL) were excluded from the initial analysis given the potential for misclassification. Incidence was estimated by assessing diagnoses made during year 5 of continuous enrollment. Finally, a sensitivity analysis was performed by including patients with both PSVT and AF/AFL diagnoses. Period prevalence and incidence rate were estimated to be 332.9 (323.2-342.9) and 57.8 (52.8-63.3) per 100 000 individuals, respectively, when excluding patients with AF/AFL. Projected to the 2018 US Census, prevalence and incidence are 1.26 million (1.21-1.30 million) and 188,981 (172,891-206,943), respectively. Including patients with AF/AFL, the prevalence may increase to 479.7 (467.9-491.8) with an incidence of 93.4 (86.9-100.5) per 100 000 individuals or a prevalence of 2.06 million (2.01-2.12 million). CONCLUSIONS: Approximately 1 in 300 people in the US had PSVT with the highest rates in older and female patients.

Myopic and Forward Looking Behavior in Branded Oral Anti-Diabetic Medication Consumption: An Example from Medicare Part D.

We evaluate consumption responses to the non-linear Medicare Part D prescription drug benefit. We compare propensity-matched older patients with diabetes and Part D Standard or low-income-subsidy (LIS) coverage. We evaluate monthly adherence to branded oral anti-diabetics, with high end-of-year donut hole prices (>$200) for Standard patients and consistent, low (≤$6) prices for LIS. As an additional control, we examine adherence to generic anti-diabetics, with relatively low, consistent prices for Standard patients. If Standard patients are forward looking, they will reduce branded adherence in January, and LIS-Standard differences will be constant through the year. Contrary to this expectation, branded adherence is lower for Standard patients in January and diverges from LIS as the coverage year progresses. Standard-LIS generic adherence differences are minimal. Our findings suggest that seniors with chronic conditions respond myopically to the nonlinear Part D benefit, reducing consumption in response to high deductible, initial coverage and gap prices. Thus, when the gap is fully phased out in 2020, cost-related nonadherence will likely remain in the face of higher spot prices for more costly branded medications. These results contribute to studies of Part D plan choice and medication adherence that suggest that seniors may not make optimal healthcare decisions. Copyright © 2016 John Wiley & Sons, Ltd.

Trends in acute myocardial infarction hospitalizations: Are we seeing the whole picture?

BACKGROUND: Payers and policy makers rely on studies of trends in acute myocardial infarction (AMI) hospitalizations and spending that count only hospitalizations where the AMI is the principal discharge diagnosis. Hospitalizations with AMI coded as a secondary diagnosis are ignored. The effects of excluding these hospitalizations on estimates of trends are unknown. METHODS: Observational study of all AMI hospitalizations in Fee-for-Service Medicare beneficiaries 65 years and older, from 2002 through 2011. RESULTS: We studied 3,663,137 hospitalizations with any AMI discharge diagnosis over 288,873,509 beneficiary-years. Of these, 66% had AMI coded as principal (versus secondary). From 2002 to 2011, AMI hospitalization rates declined 24.5% (from 1,485 per 100,000 beneficiary-years in 2002 to 1,122 in 2011). Meanwhile, the proportion of these hospitalizations with a secondary AMI diagnosis increased from 28% to 40%; by 2011 these secondary AMI hospitalizations accounted for 43% of all expenditures for hospitalizations with AMI, or $2.8 billion. Major changes in comorbidities, principal diagnoses and mean costs for hospitalizations with a non-principal AMI diagnosis occurred in the 2006-2008 timeframe. CONCLUSIONS: Current estimates of the burden of AMI ignore an increasingly large proportion of overall AMI hospitalizations and spending. Changes in the characteristics of hospitalizations that coincided with major payment and policy changes suggest that non-clinical factors affect AMI coding. Failing to consider all AMIs could inflate estimates of population health improvements, overestimate the value of AMI prevention and treatment and underestimate current and future AMI burden and expenditures.

Clinical, economic, and health-related quality of life outcomes in patients with overweight or obesity in the United States: 2016-2018.

Objectives: This study aimed to estimate clinical, economic (including productivity), and health-related quality of life (HRQoL) outcomes and associated individual characteristics among adults with overweight (OW) or obesity in the United States. Methods: This study included adult respondents with body mass index (BMI) ≥18.5 kg/m2 in the 2017-2018 National Health and Nutrition Examination Survey (NHANES) and 2016 Medical Expenditure Panel Survey. Respondents were classified according to BMI. Individual characteristics were described by BMI categories. Multivariable regression models estimated the association between BMI categories and outcomes, adjusting for individual characteristics. Results: Nearly three-quarters (73.7%) of NHANES participants were OW or obese. Relative to Normal weight (NW), respondents with Class 3 obesity had more obesity-related complications (2.07 vs. 4.62, p < 0.001). Higher BMI was associated with significantly lower HRQoL, lower productivity, and higher healthcare expenditures as well as more frequent weight loss attempts in the previous 12 months. Weight loss surgery and prescription anti-obesity medications (AOMs) were used only by a very small proportion of individuals. Despite frequent weight loss attempts, most respondents did not achieve clinically meaningful weight loss. Conclusions: Adults with OW or obesity experienced worse clinical, economic and HRQoL outcomes than those with NW. Better use of evidence-based obesity treatments, including prescription AOMs, should be considered to achieve more clinically meaningful weight reduction and improved outcomes in individuals with OW or obesity.

Catheter ablation for supraventricular tachycardia and health resource utilization and expenditures: A propensity-matched cohort study.

BACKGROUND: Few data are available regarding temporal patterns of health resource utilization (HRU) and expenditures among patients undergoing catheter ablation for paroxysmal supraventricular tachycardia (PSVT). This study aimed to describe expenditures and HRU in patients with PSVT who underwent catheter ablation compared to a matched cohort of patients on medical therapy alone. METHODS: Using a large US administrative database, we identified adult patients (age 18 to 65 years) with a new PSVT diagnosis between 2008 and 2016. Propensity-score matching was used to assemble a PSVT cohort treated with ablation or medical therapy alone (N = 2556). Longitudinal trends in HRU and expenditures in the 3-years preceding and following PSVT diagnosis were compared. RESULTS: There were no significant differences in expenditures between groups except within the first year after PSVT diagnosis: $48,004 ablation vs. $17,560 medical therapy (p < 0.001). This difference was driven by procedural expenditures, where the mean cost of catheter ablation was $32,057 ± SD 26,737. In Years 2 and 3 post-ablation, HRU and expenditures decreased to the levels associated with the medical therapy group, although fewer ablation patients required any prescription for beta-blockers, calcium channel blockers, or anti-arrhythmic drugs (32% ablation vs. 42% medical therapy group, p < 0.001). CONCLUSION: Catheter ablation reduces medication burden in PSVT, yet health resource use and expenditures were similar beyond 2 years post-ablation when compared to PSVT patients on medical therapy alone. Additional studies are required to better understand drivers of these sustained health expenditures, and barriers to achieving cost-savings for a potentially curative procedure.

Burden of illness and mortality in men with Adrenomyeloneuropathy: a retrospective cohort study.

BACKGROUND: Adrenomyeloneuropathy (AMN) is a neurodegenerative disease phenotype of X-linked adrenoleukodystrophy (ALD), resulting in progressive myeloneuropathy causing spastic paraparesis, sensory ataxia, and bowel/bladder symptoms. We conducted a retrospective cohort study using two large administrative databases to characterize mortality and the burden of illness in adult men with AMN in the US. RESULTS: Healthcare resource use was assessed using a national commercial insurance claims database (2006-2021). Males with AMN ages 18-64 years and no evidence of cerebral ALD or other peroxisomal disorders were included and 1:4 matched on demographic characteristics to individuals without AMN. All study participants were followed for as long as observable. Patients with AMN were also identified in the Medicare Limited Dataset (2017-2022); mortality and age at death were compared with all Medicare enrollees. We identified 303 commercially insured men with AMN. Compared with non-AMN, individuals with AMN had significantly more inpatient hospital admissions (0.44 vs. 0.04 admissions/patient/year), outpatient clinic (8.88 vs. 4.1 visits/patient/year), outpatient hospital (5.33 vs. 0.99 visits/patient/year), and home healthcare visits (4.66 vs. 0.2 visits/patient/year), durable medical equipment claims (0.7 vs. 0.1 claims/patient/year), and prescription medication fills (18.1 vs. 5.4 fills/patient/year) (all p < 0.001). Average length-of-stay per hospitalization was also longer in AMN (8.88 vs. 4.3 days; p < 0.001). Rates of comorbidities were significantly more common in AMN compared to controls, including peripheral vascular disease (4.6% vs. 0.99%), chronic pulmonary disease (6.3% vs. 2.6%), and liver disease (5.6% vs. 0.88%), all p < 0.001. Among individuals age < 65 with Medicare disability coverage, mortality rates were 5.3x higher for adult AMN males (39.3% vs. 7.4%) and the age at death significantly younger (47.0 ± 11.3 vs. 56.5 ± 7.8 years), both p < 0.001. Among Medicare beneficiaries ages ≥ 65 mortality rates were 2.2x higher for men with AMN vs. those without AMN (48.6% vs. 22.4%), p < 0.001. CONCLUSION: AMN imposes a substantial and underrecognized health burden on men, with higher healthcare utilization, greater medical comorbidity, higher mortality rates, and younger age at death.

Hospitalizations and Mortality From Myasthenia Gravis: Trends From 2 US National Datasets

BACKGROUND AND OBJECTIVES: Myasthenia gravis (MG) is a rare neuromuscular disorder where IgG antibodies damage the communication between nerves and muscles, leading to muscle weakness that can be severe and have a significant impact on patients' lives. MG exacerbations include myasthenic crisis with respiratory failure, the most serious manifestation of MG. Recent studies have found MG prevalence increasing, especially in older patients. This study examined trends in hospital admissions and in-hospital mortality for adult patients with MG and readmissions and postdischarge mortality in older (65 years or older) adults with MG. METHODS: Data from the Nationwide Inpatient Sample (NIS), an all-payer national database of hospital discharges, were used to characterize trends in hospitalizations and in-hospital mortality related to MG exacerbations and MG crisis among adult patients aged 18 years or older. The Medicare Limited Data Set, a deidentified, longitudinal research database with demographic, enrollment, and claims data was used to assess hospitalizations, length of stay (LOS), readmissions, and 30-day postdischarge mortality among fee-for-service Medicare beneficiaries aged 65 years or older. The study period was 2010-2019. Multinomial logit models and Poisson regression were used to test for significance of trends. RESULTS: Hospitalization rates for 19,715 unique adult patients and 56,822 admissions increased from 2010 to 2019 at an average annualized rate of 4.9% (MG noncrisis: 4.4%; MG crisis: 6.8%; all p < 0.001). Readmission rates were approximately 20% in each study year for both crisis and noncrisis hospitalizations; the in-hospital mortality rate averaged 1.8%. Among patients aged 65 years or older, annualized increases in hospitalizations were estimated at 5.2%, 4.2%, and 7.7% for all, noncrisis, and crisis hospitalizations, respectively (all p < 0.001). The average LOS was stable over the study period, ranging from 11.3 to 13.1 days, but was consistently longer for MG crisis admissions. Mortality among patients aged 65 years or older was higher compared with that in all patients, averaging 5.0% across each of the study years. DISCUSSION: Increasing hospitalization rates suggest a growing burden associated with MG, especially among older adults. While readmission and mortality rates have remained stable, the increasing hospitalization rates indicate that the raw numbers of readmissions-and deaths-are also increasing. Mortality rates are considerably higher in older patients hospitalized with MG.

Healthcare Utilization Among Infants Covered by Medicaid and Newly Diagnosed With Respiratory Syncytial Virus.

Background: Infants covered by Medicaid have higher respiratory syncytial virus (RSV) hospitalization rates than those with commercial insurance, but findings are limited to the inpatient setting. This birth cohort study describes healthcare encounters for RSV across all settings among infants covered by Medicaid and the Children's Health Insurance Program. Methods: Medicaid claims for infants born and residing in Arizona (AZ), California (CA), Florida (FL), Michigan (MI), North Carolina (NC), New York (NY), and Texas (TX) were analyzed for first diagnosis of RSV in 2016-2018 using International Classification of Diseases, Tenth Revision codes. Encounters on the day of first diagnosis were examined by setting in 7 states and by setting and race in CA, FL, and NC. Results: A total of 80 945 infants were diagnosed with RSV in 7 states in 2016-2018. The highest encounter rates for first RSV diagnosis were in the emergency department (ED) in 5 states (11.0-33.4 per 1000 in AZ, CA, FL, MI, and NY) and outpatient setting in 2 states (54.8 and 68.5 per 1000 in TX and NC). Significantly higher outpatient encounter rates were found in CA and NC for White infants compared to non-White infants. In NC, ED encounter rates were significantly higher for non-White infants than White infants, whereas in CA, the rates were comparable. In these 2 states, hospitalization rates were similar across groups. In FL, compared with White infants, non-White infants had significantly higher encounter rates in each setting on the day of first RSV diagnosis. Conclusions: This is the first study to describe the burden of RSV by setting and race. Medicaid infants who are newly diagnosed with RSV have the highest burden in ED and outpatient settings.

Cost sharing and decreased branded oral anti-diabetic medication adherence among elderly Part D Medicare beneficiaries.

BACKGROUND: Although the Medicare Part D coverage gap phase-out should reduce cost-related nonadherence (CRN) among seniors with diabetes, preferential generic prescribing may have already decreased CRN, while smaller numbers of patients using more costly branded oral anti-diabetic (OAD) medications remain vulnerable to CRN. OBJECTIVE: To estimate the effects of cost sharing in the Part D standard (non-LIS) benefit on adherence to different OAD classes, comparing two classes dominated by inexpensive generic medications and two by more costly branded medications. DESIGN AND PATIENTS: Retrospective cohort study using dispensed prescription data for elderly non-LIS (N=81,047) and LIS (low-income subsidy) (N=150,359) beneficiaries using same class OAD(s) in 2008 and 2009. Logistic regression modeled non-LIS likelihood; LIS and non-LIS patients matched using propensity outcome (N=38,054). Logistic regression, controlling for demographic and health status characteristics, modeled effects of non-LIS coverage on 2009 OAD class adherence. MAIN MEASURES: Main outcome measures were within-class OAD coverage year adherence, with patients considered adherent when days supplied to calendar days ratio at least 0.8. KEY RESULTS: Non-LIS patients had 0.52 and 0.57 times the odds of branded-only DPP-4 Inhibitor (N=1,812; 95 % CI: 0.43, 0.63; P<0.001) and Thiazolidinedione (TZD) (N=6,290; 95 % CI: 0.52, 0.63; P<0.001) adherence. Most patients (N=32,510; 82 %) used OADs in primarily generic classes, where we found no significant (Biguanides; N=21,377) or small differences (Sulfonylureas/Glinides [N=19,240; OR: 0.91; 95 % CI: 0.86, 0.97; P=0.002]) in adherence odds. Crude adherence rates were sub-optimal when CRN was not a factor (Non-LIS/LIS: Biguanides: 65 %/65 %; Sulfonylureas/Glinides: 66 %/68 %; LIS: DPP-4 Inhibitors: 66 %; TZDs: 67 %). CONCLUSIONS: Gap elimination would not affect generic, but should reduce branded OAD CRN. Branded copayments may continue to lead to CRN. Policy initiatives and benefit changes targeting both cost deterrents for patients with more complex disease and non-cost generic OAD underuse are recommended.

Evaluating the financial impact of utilizing recombinant porcine factor VIII or recombinant FVIIa for patients with acquired hemophilia A.

OBJECTIVE: To evaluate the financial impact of utilizing rpFVIII or rFVIIa during a hospital admission for the diagnosis of acquired hemophilia A (AHA) by reviewing the margin between the cost to the hospital for providing care and the amount the hospital is reimbursed by the Centers for Medicare & Medicaid Services (CMS) in the US. METHODS: Data source was the Medicare Limited Data Set, which contains claims for hospitalizations, charges, and amounts reimbursed by CMS. Study patients were hospitalized with AHA and treated with rpFVIII and/or rFVIIa between 1/1/2015 and 12/31/2019. CMS Fiscal Year 2020 Impact Files, with hospital-level cost-to-charge ratios (CCRs), were used to estimate hospital costs. Sensitivity analyses were conducted to estimate margins at different CCRs. RESULTS: Hospital margins were, on average, positive with use of either rpFVIII or rFVIIa (rpFVIII: $51,548.89; rFVIIa: $35,943.80). Sensitivity analysis results suggest that the use of rpFVIII is similiar, compared with rFVIIa for a large majority of hospitals. CONCLUSIONS: While there may be higher reimbursement for rpFVIII hospitalizations, this analysis suggests that the use of rpFVIII, compared to rFVIIa, may have no impact on hospital finances for the majority of hospitals, despite rpFVIII's higher per unit cost.

Health care resource utilization and costs in patients with painful diabetic neuropathy treated with 10 kHz spinal cord stimulation therapy.

BACKGROUND: Diabetic peripheral neuropathy, a common comorbidity of diabetes, is a neurodegenerative disorder that targets sensory, autonomic, and motor nerves frequently associated with painful diabetic neuropathy (PDN). PDN carries an economic burden as the result of reduced work and productivity. A recent multicenter randomized controlled trial, SENZA-PDN (NCT03228420), assessed the impact of high-frequency (10 kHz) spinal cord stimulation (SCS) on pain relief. The effects of high-frequency SCS on health care resource utilization and medical costs are not known. OBJECTIVE: To evaluate the effect of high-frequency (10 kHz) SCS on health care resource utilization (HRU) and medical costs in patients with PDN using data from the SENZA-PDN trial. METHODS: Participants with PDN were randomly assigned 1:1 to receive either 10 kHz SCS plus conventional medical management (CMM) (SCS treatment group) or CMM alone (CMM treatment group). Patient outcomes and HRU up to the 6-month follow-up are reported here. Costs (2020 USD) for each service was estimated based on publicly available Medicare fee schedules, Medicare claims data, and literature. HRU metrics of inpatient and outpatient contacts and costs are reported as means and SDs. Univariate and bivariate analyses were used to compare SCS and CMM treatment groups at 6 months. RESULTS: At 6-month follow up, the SCS arm experienced approximately half the mean rate of hospitalizations per patient compared with the CMM treatment group (0.08 vs 0.15; P = 0.066). The CMM treatment group's total health care costs per patient were approximately 51% higher compared with the SCS treatment group (equivalent to mean annual cost per patient of $9,532 vs $6,300). CONCLUSIONS: Our analysis of the SENZA-PDN trial indicates that the addition of 10 kHz SCS therapy results in lower rates of hospitalization and consequently lower health care costs among patients with PDN compared with those receiving conventional management alone.

The healthcare resource utilization and costs of chronic lung allograft dysfunction following lung transplantation in patients with commercial insurance in the United States.

AIMS: Chronic lung allograft dysfunction (CLAD), a common complication of lung transplantation, is the leading cause of death for lung transplant recipients. While data on lung transplant costs are available, the impact of CLAD on healthcare resource use (HRU) and cost is not well understood. The primary objective was to quantify the HRU and costs of CLAD in the US using real-world data. METHODS: A longitudinal retrospective analysis was performed of commercial claims data from the IQVIA PharMetrics Plus database for patients aged 18-64 who underwent lung transplantation between January 1, 2006 and September 30, 2018. Lung transplantation was identified using International Classification of Disease and Common Procedure Terminology procedure codes. Patients studied were observable for at least 12 months before and after transplantation. Patients who developed CLAD were identified using novel, diagnosis codes for incident lung disease at least one year following transplantation. Descriptive analyses were conducted to assess the study's outcomes prior to and following a CLAD diagnosis. All-cause HRU and costs, the study's primary outcomes, leading up to and following CLAD diagnosis were calculated. RESULTS: Among 129 transplant patients who developed CLAD, healthcare costs were substantially higher in the year following diagnosis ($198,113), compared to the year leading to diagnosis ($85,276). Inpatient admissions were responsible for most costs in years 1 and 2 following diagnosis ($99,372 and $83,348 respectively). Drug costs were higher in the 12 months post-index, compared to the 12 months pre-index ($3,600 vs $2,527). LIMITATIONS: Claims data do not include clinical data, have limits determining loss of follow-up, and do not provide granularity to determine disease severity. Also, there is no ICD-10-CM code specific to CLAD or BOS. CONCLUSIONS: CLAD after lung transplant is associated with substantial HRU and costs. Further work is needed to develop interventions that reduce this impact.

Lung Function Monitoring After Lung Transplantation and Allogeneic Hematopoietic Stem Cell Transplantation.

PURPOSE: Bronchiolitis obliterans syndrome (BOS) is a major cause of morbidity and mortality in lung transplantation and allogeneic hematopoietic stem cell transplantation (allo-HSCT) recipients. Clinical guidelines recommend lung function monitoring to aid early identification of BOS, but real-world rates of pulmonary function testing (PFT) have not been studied. The purpose of this study was to quantify PFT rates in lung transplantation and allo-HSCT recipients. METHODS: This longitudinal retrospective study used US data from the IQVIA PharMetrics Plus commercial claims database (January 1, 2006-September 30, 2018) and the Medicare Limited Data Set (January 1, 2010-December 31, 2018). Study recipients had no evidence of transplantation 12 months before transplantation, which was identified by using diagnosis and procedure codes. PFTs were identified by using procedure codes. Outcomes were percentage of recipients who received ≥1 PFT in each follow-up year, including spirometry, lung diffusion capacity, lung function volume test, and plethysmography, including the average number of total and specific tests per recipient. FINDINGS: The study identified 367 commercially insured and 1776 Medicare recipients who underwent lung transplantation; 92% and 86% received ≥1 lung function test in the first year after transplantation, respectively. Among recipients observable 3 years after transplant, 85% and 83% received ≥1 PFT. Among 2187 commercially insured and 1864 Medicare recipients who underwent allo-HSCT, 44% and 36% received ≥1 lung function test in the first posttransplant year. In the third year after transplant, only 31% and 26% of observable allo-HSCT recipients underwent any PFT. IMPLICATIONS: Morbidity and mortality from BOS remain high in lung transplant and allo-HSCT recipients, but lung function testing in the first posttransplant year is not universal, with substantially lower rates among allo-HSCT recipients. Furthermore, testing rates in all cohorts declined over time. Increased and sustained monitoring could lead to earlier detection of BOS and earlier intervention and treatment.

The economic burden of NIPC and BOS following allogeneic HSCT in patients with commercial insurance in the United States.

Noninfectious pulmonary complications (NIPC) after allogeneic hematopoietic stem cell transplantation (alloHSCT), including bronchiolitis obliterans syndrome (BOS), cause significant morbidity and mortality, but their impact on health care resource utilization (HRU) and costs is unknown. This longitudinal retrospective study quantified the economic burden of NIPC and BOS in alloHSCT patients using commercial claims data from the IQVIA PharMetrics Plus database. Study patients were aged 0 to 64 years and underwent alloHSCT between 1 January 2006 and 30 September 2018, and were observable 12 months before and up to 5 years after index alloHSCT. NIPC patients were identified using International Classification of Disease (ICD) diagnosis codes. Outcomes were mean per patient HRU (inpatient admissions, outpatient office, hospital visits, and prescription medications) and costs paid by insurers in each post-transplant year. Among 2162 alloHSCT patients, 254 developed NIPCs, and 155 were propensity score (PS)-matched to non-NIPC patients. The year following transplantation, NIPC patients had significantly higher inpatient admission rates (3.8 ± 3.2 vs non-NIPC: 2.6 ± 2.4; P < .001) and higher total costs ($567 870 vs $412 400; P = .07), reflecting higher costs for inpatient admissions ($452 475 vs $300 202; P = .06). Among those observable for more years, costs remained higher for NIPC patients, reflecting significantly higher inpatient admission rates in the first 3 years following transplant. Subanalysis of patients with diagnoses likely reflective of BOS were consistent with these findings. AlloHSCT patients who developed NIPC had higher health care resource utilization and incurred higher costs compared with alloHSCT patients who did not develop NIPC following transplant.

Insights from Real-World Analysis of Treatment Patterns in Patients with Newly Diagnosed Knee Osteoarthritis.

BACKGROUND: Several nonpharmacologic and pharmacologic treatments are available for the management of knee osteoarthritis (OA)-related pain and for improving functionality; however, clinical guideline recommendations vary on their use. OBJECTIVE: To compare the treatment patterns in a real-world setting versus the guideline recommendations for the treatment of newly diagnosed patients with knee OA. METHODS: This retrospective analysis used data from the electronic health records of the Geisinger Health System between January 1, 2010, and December 2018 to identify adults with newly diagnosed knee OA who had not received previous therapy with intra-articular corticosteroids, opioids, intra-articular hyaluronic acid, or prescription nonsteroidal anti-inflammatory drugs (NSAIDs). Eligible patients were evaluated for the mutually exclusive treatment categories after diagnosis, including prescription NSAIDs, intra-articular corticosteroids, intra-articular hyaluronic acid (specifically an intra-articular bioengineered hyaluronic acid), opioids, physical therapy, bracing, and total knee arthroplasty. These 7 treatment categories were evaluated for utilization patterns in the real-world setting. RESULTS: A total of 8776 patients with a new diagnosis of knee OA were identified; 88.2% of them received 1 of the 7 evaluated treatments. The most frequently prescribed first treatment was intra-articular corticosteroids (26%), followed by opioids (17.6%), and intra-articular bioengineered hyaluronic acid (14.9%). The most often prescribed second treatment was opioids (15.8%), followed by physical therapy (14%), NSAIDs (11.8%), and intra-articular bioengineered hyaluronic acid (9.6%). Of note, 22.9% of the patients received only 1 evaluated therapy during the study period and did not receive a second treatment. CONCLUSIONS: Real-world treatment patterns in patients with newly diagnosed knee OA indicate that prescribers are using the spectrum of the available therapies that, at times, are different from the current treatment guideline recommendations.

Costs and health resource use in patients with X-linked myotubular myopathy: insights from US commercial claims.

BACKGROUND: In X-linked myotubular myopathy (XLMTM), mutations in the MTM1 gene result in absence or dysfunction of myotubularin, a protein required for normal development, maintenance, and function of skeletal muscle. Extreme muscle weakness results in severe respiratory failure that is fatal for approximately half of XLMTM-affected children by age 18 months. Most surviving patients require invasive mechanical ventilation, feeding tubes, and wheelchairs for mobility, due to profoundly impaired motor function. Little is known about the costs of care for this rare disease. Currently, there are no approved therapies for XLMTM. OBJECTIVE: To quantify the direct medical costs and health care resource utilization (HRU) incurred by XLMTM patients and paid by commercial insurers. METHODS: A retrospective, longitudinal study was conducted using the IQVIA PharMetrics Plus commercial database of adjudicated claims for more than 140 million individuals with commercial insurance coverage in the United States. An algorithm based on demographic information, diagnosis and procedure codes, and medications was used to identify XLMTM patients younger than aged 2 years during the study period from January 1, 2006, through September 30, 2018. All-cause direct medical costs and HRU during each month were calculated. Costs were grouped as inpatient hospital admissions (including the intensive care unit or neonatal intensive care unit [NICU]); emergency department visits; outpatient services (outpatient hospital visits, office visits, physician/provider office visits, ambulatory surgeries and procedures, laboratory tests, and imaging tests); and prescription medications. Monthly costs and HRU over time were stratified by age and use of mechanical ventilation. RESULTS: 49 patients met the study criteria. All had at least 1 inpatient hospital admission, and 36 (73%) had at least 1 NICU stay. All patients received ventilation at some time during the study period, including 40 (82%) treated with invasive ventilation. Mean monthly per patient direct medical costs were highest in the first year of life ($74,831), including costs for inpatient admissions ($69,025), outpatient services ($5,266), and prescription medication ($540). Mean monthly costs were lower in the second, third, and fourth years of life ($23,207, $13,044, and $9,440, respectively). When annualized, these all-cause monthly medical costs totaled $897,978 per patient in the first year of life and nearly $1.5 million total for patients who survived the first 4 years of life. Costs were consistently highest when patients were receiving invasive ventilation and lowest when they were not receiving ventilation (i.e., before they started on ventilator support). CONCLUSIONS: This direct health care cost and HRU analysis demonstrates the substantial economic burden associated with XLMTM. Costs are highest in the first year of life and are particularly significant for patients receiving invasive ventilation. DISCLOSURES: This study was funded by Audentes Therapeutics, an Astellas Company, and was conducted by PRECISIONheor with funding from Audentes Therapeutics, an Astellas Company. Slocomb is an employee of Audentes Therapeutics, an Astellas Company; James was an employee at the time of the study. Sacks, Healey, and Cyr are employees of PRECISIONheor. Graham participated in the medical/scientific advisory board for Audentes as part of a clinical trial design for XLMTM but declares no vested interest or holdings that would represent a conflict of interest. Beggs received consulting fees from Audentes Therapeutics, for work on this study, and has received grants from Alexion Pharmaceuticals, Audentes Therapeutics, Dynacure SAS, Pfizer Pharmaceuticals, along with personal fees from Asklepios Biopharmaceutical, Inc., Ballard Biologics, Biogen, F. Hoffmann-La Roche AG, GLG, Guidepoint Global, and Kate Therapeutics, unrelated to this study. In addition, Beggs has a patent (Patent number: 10736945) for systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM) licensed to Audentes Therapeutics.

Clinical complications in patients with primary and recurrent Clostridioides difficile infection: A real-world data analysis.

OBJECTIVE: Clostridioides difficile infection and recurrent C. difficile infection result in substantial economic burden and healthcare resource use. Sepsis and bowel surgery are known to be serious complications of C. difficile infection. This study evaluated clinical complications in patients with C. difficile infection and recurrent C. difficile infection during a 12-month period following the primary C. difficile infection. METHODS: A retrospective analysis of commercial claims data from the IQVIA PharMetrics Plus™ database was conducted for patients aged 18-64 years with an index C. difficile infection episode requiring inpatient stay or an outpatient visit for C. difficile infection followed by a C. difficile infection treatment. Each C. difficile infection episode ended after a 14-day C. difficile infection-claim-free period was observed. Recurrent C. difficile infection was defined as a further C. difficile infection episode within an 8-week window following the claim-free period. Clinical complications were documented over 12 months of follow-up and stratified by the number of recurrent C. difficile infection episodes (0 rCDI, 1 rCDI, 2 rCDI, and 3+ rCDI). RESULTS: In total, 46,571 patients with index C. difficile infection episode were included. During the 6-month pre-index, the mean (standard deviation) baseline Charlson comorbidity index score, by increasing the recurrent C. difficile infection group, was 1.2 (1.9), 1.5 (2.2), 1.8 (2.3), and 2.3 (2.5). During the 12-month follow-up, sepsis occurred in 16.5%, 27.3%, 33.1%, and 43.3% of patients, and subtotal colectomy or diverting loop ileostomy was performed in 4.6%, 7.3%, 8.9%, and 10.5% of patients, respectively, by increasing the recurrent C. difficile infection group. CONCLUSIONS: Reduction in recurrent C. difficile infection is an important step to reduce the burden of serious clinical complications, and new treatments are needed to reduce C. difficile infection recurrence.