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Avery A Rizio,1 Kristen L McCausland,1 Michelle K White,1 Tiffany P Quock2 1QualityMetric Incorporated, LLC, Johnston, RI, USA; 2Prothena Biosciences Inc, South San Francisco, CA, USACorrespondence: Avery A RizioQualityMetric Incorporated, LLC, 1301 Atwood Avenue, Suite 216E, Johnston, RI, 02919, USATel +1 401-903-4631Email arizio@qualitymetric.comBackground: Light chain (AL) amyloidosis is a rare and progressive disease that affects myriad organs and systems. Patients with cardiac involvement have the highest risk of death. This report compiles findings across three cohorts of patients with AL amyloidosis to understand patterns of employment and work impacts.Methods: Data came from three cohorts recruited through patient advocacy organizations in the US. Patients in Cohort 1 completed the SF-36v2® Health Survey (SF-36v2), the Work Productivity and Activity Impairments – Specific Health Problem (WPAI) questionnaire, and the 12-item Kansas City Cardiomyopathy Questionnaire (KCCQ-12). The relationship between work impacts (WPAI scores) and HRQoL (SF-36v2 scores) was investigated using multivariable logistic regression and summarized according to cardiac severity using New York Heart Association (NYHA) classes estimated from KCCQ-12 scores. Changes in employment, days of missed work, and long-term disability due to AL amyloidosis were summarized for patients diagnosed in the past 24 months and stratified by NYHA class (Cohort 2). Findings were contextualized using patient interviews (Cohort 3).Results: Work-related impacts, especially reduced productivity, were common among patients with AL amyloidosis. WPAI scores were significantly related to HRQoL (p< 0.05 for all models). Among patients with cardiac involvement, the greatest degree of work impacts was observed for those in NYHA class 3 or 4. Changes in employment, missed work, and long-term disability were common among newly diagnosed patients, especially among those in NYHA class 3 or 4. Patient interviews supported the survey findings; patients described absences, reduced productivity at work, and loss of employment due to the disease and its treatment.Conclusion: Patients with AL amyloidosis, particularly those with more advanced disease, experience impacts across a range of employment-related outcomes. These findings highlight the need for more effective treatments and interventions which may improve functioning and patient outcomes, while reducing indirect costs associated with the disease.Keywords: AL amyloidosis, patient-reported outcomes, employment, work outcomes

Aim: Estimate the frequency and costs of diagnostic admissions among hospitalized patients with amyloid light chain (AL) amyloidosis. Materials & methods: This retrospective analysis used nationally representative hospital discharge data from 2017 to 2020 to report resource use and cost for hospitalizations during which AL amyloidosis was diagnosed. Results: Of 1341 admissions, 17.6% were diagnostic. Bone marrow (79.5%) and kidney (44.9%) biopsies were the most common qualifying biopsies. Diagnostic hospitalizations had longer length of stay (14.5 vs 8.4 days; p Conclusion: Diagnostic admissions are more likely to be urgent/emergent, require longer stays and have higher costs compared with hospitalizations in known AL amyloidosis patients. Improved diagnostic pathways toward early diagnosis are needed.

Purpose Amyloid transthyretin (ATTR) amyloidosis is a rare, progressive, and fatal disease. The ATTR Patient Symptom Survey (ATTR-PSS) was previously developed through literature review and concept elicitation input from clinicians and patients and revised after evaluation by a patient focus group. This study further evaluated the content validity of the ATTR-PSS through qualitative cognitive debriefing interviews with clinicians and patients. Methods Seven clinicians and 10 patients with ATTR amyloidosis were interviewed individually regarding their overall impressions, the clarity and appropriateness of the survey, relevance of concepts measured, and comprehensiveness and comprehensibility of items and response choice sets. Results Clinicians acknowledged the usefulness of the ATTR-PSS in research and clinical settings. They suggested minor modifications to the survey instructions, the addition of 3 symptoms, and the transfer of 10 conditions from the symptom list to 2 separate items. Patients found the ATTR-PSS to be easy to complete and relevant to their experiences. Their feedback resulted in modification to instruction text, edits to the description of 4 symptoms, removal of 1 symptom, and addition of 2 diagnoses. Conclusion The findings support the content validity of the ATTR-PSS as an appropriate measure of symptom frequency, severity, and impact in patients with wild-type and hereditary ATTR amyloidosis.

Purpose Cardiac dysfunction is common in amyloid light-chain (AL) amyloidosis, a rare disease caused by extracellular deposition of misfolded immunoglobulin light chains. This study aimed to examine economic/clinical disease burden in hospitalized cardiac amyloidosis patients. Patients and methods Cardiac amyloidosis patients ≥18 years old hospitalized between 2014 and 2016 were identified in claims if they had ≥1 inpatient claim consistent with amyloidosis and evidence of cardiac dysfunction. Descriptive statistics were reported. Results 3239 cardiac amyloidosis patients [1795 (55.4%) with concurrent renal disease] were identified. Mean (SD) length of stay was 8.3 (11.1) days. 25.2% were admitted to the intensive care unit. Mean overall hospitalization costs were USD$20,584. In-hospital mortality was 9.0% overall. 16.8% were readmitted within 30 days, with 11.2% dying in-hospital and a mean readmission cost of USD$18,536. Conclusion Hospitalization for cardiac amyloidosis is costly, with high rates of readmission and mortality. Opportunities exist to improve care.

Introduction Light-chain (AL) amyloidosis is a rare, progressive, and typically fatal disease. Health-related quality of life (HRQoL) has been shown to be a significant prognostic factor associated with clinical outcomes such as survival and response to treatment. A better understanding of how patterns of HRQoL may be prospectively associated with costly healthcare resource utilization, such as emergency department (ED) visits and inpatient hospitalizations, is warranted. Methods A secondary data analysis of a non-interventional, longitudinal online study of patients with AL amyloidosis (n = 224) was conducted. Negative binomial regression models were used to examine whether initial HRQoL scores (as measured by the SF-36v2® Health Survey [SF-36v2], where higher scores reflect better HRQoL) and changes in HRQoL were associated with the number of ED visits and inpatient hospitalizations during a 12-month period. Incidence rate ratios were interpreted by 5-point decrements in initial HRQoL scores and minimally important changes in HRQoL change scores. Results There were significant inverse associations between initial SF-36v2 scores and subsequent rates of ED visits and inpatient hospitalizations across all domains and summary components (p

Aims: Examine mortality and healthcare costs in Medicare beneficiaries with newly diagnosed immunoglobulin light chain (AL) amyloidosis. Patients & methods: Cases were identified in 2012–2015 Medicare 5% data with ≥1 inpatient/≥2 outpatient claims consistent with AL amyloidosis and ≥1 AL-specific treatment. Cases were matched 3:1 with disease-free controls. Descriptive statistics were reported. Results: A total of 249 (33.3%) cases were matched to 747 (66.7%) controls. A total of 19.7% of cases died within 1 year of follow-up versus 5.5% of controls; 30.6 versus 11.8% died within 2 years (p < 0.001). Mean (SD) costs in 1-year of follow-up were significantly higher among cases versus controls ($71,040 [65,766] vs $13,722 [27,493]; p Conclusion: Mortality was nearly four-times higher, and costs nearly five-times higher in beneficiaries with AL amyloidosis versus controls.

Aim: To estimate healthcare utilization and costs in amyloid light-chain (AL) amyloidosis. Patients & methods: AL amyloidosis patients were identified in 2007–2015 claims databases if they had ≥1 inpatient/≥2 outpatient claims consistent with AL amyloidosis and received ≥1 AL-specific treatment. Descriptive statistics were reported. Results: 50.1% (n = 3670) were admitted ≥1 time during the year, 11.3% (n = 827) ≥3 times. From 2007 to 2015, bortezomib use increased from 4.6 to 25.3%; melphalan use decreased from 18.9 to 2.0%; costs increased from 92,866 to $114,030. Among incident patients with at least 2 years of follow-up, healthcare utilization and costs decreased from first to second year post-diagnosis. Conclusion: AL chemotherapy-based prescribing practices changed. Total annual healthcare costs increased over time among AL amyloidosis patients.

INTRODUCTION: The amyloidoses are a group of protein-folding disorders characterized by extracellular tissue deposition of aggregated proteins as ß-pleated sheet fibrils. One of the most common and severe types is immunoglobulin light chain (AL), or "primary", amyloidosis. Prior studies of healthcare cost and resource use were hampered by the absence of an International Classification of Diseases, 9th Revision, Clinical Modification (ICD-9-CM) code specific to AL amyloidosis. Since 10/1/2017, the ICD-10-CM has included a diagnosis code for AL amyloidosis (E85.81). We believe the current study is the first to report healthcare cost and resource use using this new code. METHODS: To understand characteristics, healthcare resource utilization, costs, and clinical outcomes associated with AL amyloidosis for patients treated in US hospitals, this retrospective analysis used 2017-2020 data from the Premier Perspective® Database. The study population comprised of hospitalized patients aged ≥18 years with ≥1 inpatient claim consistent with AL amyloidosis (ICD-10-CM: E85.81) in any diagnosis field; the first qualifying hospitalization during the study period was included. Study outcomes included APR-DRG severity of illness subclass (a measure of disease burden based on the extent of organ system loss of function or physiologic decompensation), length of stay (LOS), intensive care unit (ICU) use, mortality and hospitalization costs and charges as reported in the database (inflated to 2020 USD). To provide context, we compared costs and charges to most recent national averages calculated from the 2018 National Impatient Sample (NIS). We will also tabulate NT-proBNP, troponin and differential free light chain values and report Mayo stage when possible. RESULTS: 1,341 patients were admitted to the hospital with a diagnosis of AL amyloidosis; mean (SD) age was 67.2 (11.2) years, 44.1% were female, 64.3% were White, and 62.4% had Medicare coverage. The mean (SD) Charlson Comorbidity Index was 3.9 (2.3), and 90.9% of patients had cardiac and/or renal impairment (Table 1). More than 80% of patients had either major or extreme disease according to the APR-DRG severity of illness measure. 87.6% of admissions were urgent or emergent, and 8.0% (95% CI [6.5%,9.4%]) of admitted patients died in the hospital. The mean (SD) LOS was 9.5 (9.7) days (Figure 1); during the hospital stay, 20.1% of patients were admitted to the ICU, with a mean (SD) ICU LOS of 6.5 (7.6) days (Table 2). The mean (SD) total hospitalization costs were $27,099 ($34,849) and total charges were $111,234 ($144,853) for hospitalized patients with AL amyloidosis while similar measures for all US hospital stays were $13,702 ($121) and $57,991 ($694), respectively (Figure 2). CONCLUSIONS: Disease burden and hospital costs associated with AL amyloidosis are high, particularly within this group of patients who have advanced disease as indicated by the APR-DRG classification. Mean hospitalization costs were above $27,000 per patient and many patients were admitted to the ICU. New therapies aimed at improving survival and providing clinical benefits have the potential to reduce disease burden and to yield substantial cost savings. Figure 1 Figure 1. Disclosures Quock: Prothena Biosciences, Inc: Current Employment. Chang: Dompe US, Inc.: Other: I am an employee of PHAR, LLC, which was paid by Dompe US, Inc. to conduct research; Mirum Pharmaceuticals, Inc.: Other: I am an employee of PHAR, LLC, which was paid by Mirum Pharmaceuticals, Inc. to conduct research; Greenwich Biosciences, Inc.: Other: I am an employee of PHAR, LLC, which was paid by Greenwich Biosciences to conduct research; Genentech, Inc.: Other: I am an employee of PHAR, LLC, which was paid by Genentech to conduct research; Verde Technologies: Other: I am an employee of PHAR, LLC, which was paid by Verde Technologies to conduct research; Sage: Other: I am an employee of PHAR, LLC, which was paid by Sage to conduct research; Prothena: Other: I am an employee of PHAR, LLC, which was paid by Prothena to conduct research; Pathnostics: Other: I am an employee of PHAR, LLC, which was paid by Pathnostics to conduct research; Otsuka: Other: I am an employee of PHAR, LLC, which was paid by Otsuka to conduct research; Novartis: Other: I am an employee of PHAR, LLC, which was paid by Novartis to conduct research; Kite: Other: I am an employee of PHAR, LLC, which was paid by Kite to conduct research; Jazz: Other: I am an employee of PHAR, LLC, which was paid by Jazz to conduct research; Ionis: Other: I am an employee of PHAR, LLC, which was paid by Ionis to conduct research; Illumina: Other: I am an employee of PHAR, LLC, which was paid by Illumina to conduct research; Helsinn: Other: I am an employee of PHAR, LLC, which was paid by Helsinn to conduct research; GRAIL: Other: I am an employee of PHAR, LLC, which was paid by GRAIL to conduct research; Ethicon: Other: I am an employee of PHAR, LLC, which was paid by Ethicon to conduct research; Eisai: Other: I am an employee of PHAR, LLC, which was paid by Eisai to conduct research; Celgene: Other: I am an employee of PHAR, LLC, which was paid by Celgene to conduct research; Boston Scientific Corporation: Other: I am an employee of PHAR, LLC, which was paid by Boston Scientific Corporation to conduct research; Bristol Myers Squibb: Other: I am an employee of PHAR, LLC, which was paid by BMS to conduct research; AstraZeneca: Other: I am an employee of PHAR, LLC, which was paid by AstraZeneca to conduct research; Amgen: Other: I am an employee of PHAR, LLC, which was paid by Amgen to conduct research; Akcea: Other: I am an employee of PHAR, LLC, which was paid by Akcea to conduct research; AbbVie: Other: I am an employee of PHAR, LLC, which was paid by AbbVie to conduct research; Partnership for Health Analytic Research (PHAR), LLC: Current Employment, Other; Sanofi US Services, Inc.: Other: I am an employee of PHAR, LLC, which was paid by Sanofi US Services Inc. to conduct research; Sunovion Pharmaceuticals, Inc.: Other: I am an employee of PHAR, LLC which was paid by Sunovion Pharmaceuticals, Inc. to conduct research. ; BioMarin Pharmaceuticals Inc.: Other: I am an employee of PHAR, LLC which was paid by BioMarin Pharmaceuticals, Inc. to conduct research. ; Takeda Pharmaceuticals U.S.A., Inc.: Other: I am an employee of PHAR, LLC which was paid by Takeda Pharmaceuticals U.S.A., Inc., to conduct research. ; Exact Sciences Corporation: Other: I am an employee of PHAR, LLC which was paid by Exact Sciences Corporation to conduct research. . Bognar: Prothena Biosciences, Inc: Other: I am an employee at Partnership for Health Analytic Research LLC which received funding from Prothena Biosciences, Inc. to conduct the research described in this abstract.; Akcea: Other: I am an employee of PHAR, LLC, which was paid by Akcea to conduct research; Amgen: Other: I am an employee of PHAR, LLC, which was paid by Amgen to conduct research; AstraZeneca: Other: I am an employee of PHAR, LLC, which was paid by AstraZeneca to conduct research; BMS: Other: I am an employee of PHAR, LLC, which was paid by BMS to conduct research; Boston Scientific Corporation: Other: I am an employee of PHAR, LLC, which was paid by Boston Scientific Corporation to conduct research; Celgene: Other: I am an employee of PHAR, LLC, which was paid by Celgene to conduct research; Eisai: Other: I am an employee of PHAR, LLC, which was paid by Eisai to conduct research; Ethicon: Other: I am an employee of PHAR, LLC, which was paid by Ethicon to conduct research; GRAIL: Other: I am an employee of PHAR, LLC, which was paid by GRAIL to conduct research; Helsinn: Other: I am an employee of PHAR, LLC, which was paid by Helsinn to conduct research; Illumina: Other: I am an employee of PHAR, LLC, which was paid by Illumina to conduct research; Ionis: Other: I am an employee of PHAR, LLC, which was paid by Ionis to conduct research; Jazz: Other: I am an employee of PHAR, LLC, which was paid by Jazz to conduct research; Kite: Other: I am an employee of PHAR, LLC, which was paid by Kite to conduct research; Novartis: Other: I am an employee of PHAR, LLC, which was paid by Novartis to conduct research; Otsuka: Other: I am an employee of PHAR, LLC, which was paid by Otsuka to conduct research; Pathnostics: Other: I am an employee of PHAR, LLC, which was paid by Pathnostics to conduct research; Sage: Other: I am an employee of PHAR, LLC, which was paid by Sage to conduct research; Verde Technologies: Other: I am an employee of PHAR, LLC, which was paid by Verde Technologies to conduct research; Genentech, Inc.: Other: I am an employee of PHAR, LLC, which was paid by Genentech to conduct research; Greenwich Biosciences, Inc.: Other: I am an employee of PHAR, LLC, which was paid by Greenwich Biosciences to conduct research; Mirum Pharmaceuticals, Inc.: Other: I am an employee of PHAR, LLC, which was paid by Mirum Pharmaceuticals to conduct research; Dompe US, Inc.: Other: I am an employee of PHAR, LLC, which was paid by Dompe US to conduct research; Sanofi US Services, Inc.: Other: I am an employee of PHAR, LLC, which was paid by Sanofi US to conduct research; Sunovion Pharmaceuticals, Inc.: Other: I am an employee of PHAR, LLC, which was paid by Sunovian to conduct research; BioMarin Pharmaceuticals Inc.: Other: I am an employee of PHAR, LLC, which was paid by BioMarin to conduct research; Takeda Pharmaceuticals U.S.A., Inc.: Other: I am an employee of PHAR, LLC, which was paid by Takeda to conduct research; Exact Sciences Corporation: Other: I am an employee of PHAR, LLC, which was paid by Exact Sciences to conduct research. D'Souza: Sanofi, Takeda, Teneobio, CAELUM, Prothena: Research Funding; Janssen, Prothena: Consultancy; Imbrium, Pfizer, BMS: Membership on an entity's Board of Directors or advisory committees. Broder: Prothena Biosciences, Inc: Other: I am an employee at Partnership for Health Analytic Research LLC which received funding from Prothena Biosciences, Inc. to conduct the research described in this abstract.; Akcea: Other: I am an employee of PHAR, LLC, which was paid by Akcea to conduct research; Amgen: Other: I am an employee of PHAR, LLC, which was paid by Amgen to conduct research; AstraZeneca: Other: I am an employee of PHAR, LLC, which was paid by AstraZeneca to conduct research; BMS: Other: I am an employee of PHAR, LLC, which was paid by BMS to conduct research; Boston Scientific Corporation: Other: I am an employee of PHAR, LLC, which was paid by Boston Scientific Corporation to conduct research; Celgene: Other: I am an employee of PHAR, LLC, which was paid by Celgene to conduct research; Eisai: Other: I am an employee of PHAR, LLC, which was paid by Eisai to conduct research; Ethicon: Other: I am an employee of PHAR, LLC, which was paid by Ethicon to conduct research; GRAIL: Other: I am an employee of PHAR, LLC, which was paid by GRAIL to conduct research; Helsinn: Other: I am an employee of PHAR, LLC, which was paid by Helsinn to conduct research; Illumina: Other: I am an employee of PHAR, LLC, which was paid by Illumina to conduct research; Ionis: Other: I am an employee of PHAR, LLC, which was paid by Ionis to conduct research; Jazz: Other: I am an employee of PHAR, LLC, which was paid by Jazz to conduct research; Kite: Other: I am an employee of PHAR, LLC, which was paid by Kite to conduct research; Novartis: Other: I am an employee of PHAR, LLC, which was paid by Novartis to conduct research; Otsuka: Other: I am an employee of PHAR, LLC, which was paid by Otsuka to conduct research; Pathnostics: Other: I am an employee of PHAR, LLC, which was paid by Pathnostics to conduct research; Prothena: Other: I am an employee of PHAR, LLC, which was paid by Prothena to conduct research; Sage: Other: I am an employee of PHAR, LLC, which was paid by Sage to conduct research; Verde Technologies: Other: I am an employee of PHAR, LLC, which was paid by Verde Technologies to conduct research; Genentech, Inc.: Other: I am an employee of PHAR, LLC, which was paid by Genentech to conduct research; Greenwich Biosciences, Inc.: Other: I am an employee of PHAR, LLC, which was paid by Greenwich Biosciences to conduct research; Mirum Pharmaceuticals, Inc.: Other: I am an employee of PHAR, LLC, which was paid by Mirum Pharmaceuticals, Inc. to conduct research; Dompe US, Inc.: Other: I am an employee of PHAR, LLC, which was paid by Dompe US, Inc. to conduct research; Sanofi US Services, Inc.: Other: I am an employee of PHAR, LLC, which was paid by Sanofi US Services Inc. to conduct research; Sunovion Pharmaceuticals, Inc.: Other: I am an employee of PHAR, LLC which was paid by Sunovion Pharmaceuticals, Inc. to conduct research. ; BioMarin Pharmaceuticals Inc.: Other: I am an employee of PHAR, LLC which was paid by BioMarin Pharmaceuticals, Inc. to conduct research. ; Takeda Pharmaceuticals U.S.A., Inc.: Other: I am an employee of PHAR, LLC which was paid by Takeda Pharmaceuticals U.S.A., Inc., to conduct research. ; Exact Sciences Corporation: Other: I am an employee of PHAR, LLC which was paid by Exact Sciences Corporation to conduct research. .

Background Light chain (AL) amyloidosis is a rare, complex disease associated with significant morbidity and mortality. Delays in diagnosis are common and may have detrimental consequences on patients’ prognosis. Too little is known regarding the patient journey to diagnosis. Objective The objective of this study was to describe the patient-reported journey to a correct diagnosis for AL amyloidosis. Methods Using a mixed-methods approach, data were collected from clinician (n = 4) and patient (n = 10) interviews and a survey of community-based patients with AL amyloidosis (n = 341). Data were used to document the patient experience between the onset of symptoms and the receipt of a diagnosis. Results Delays in diagnosis were common. Qualitative and quantitative data indicated that initial symptoms were varied and similar to other more prevalent diseases. Two themes regarding the journey to diagnosis emerged: (1) barriers to an early diagnosis; and (2) the emotional toll of the journey. Time to diagnosis was heavily influenced by how patients interpreted their initial symptoms, whether they sought early medical help, and challenges associated with making differential diagnoses. Survey results indicate that patients with primary cardiac involvement were more likely to receive a delayed diagnosis than those with primary kidney involvement. Patients described mixed emotions associated with the eventual diagnosis of AL amyloidosis. Conclusions These data support a need for better early identification and support for patients seeking a diagnosis. Increasing clinician awareness may reduce the time to diagnosis. Additional research is needed to identify optimal diagnostic testing to reduce delays in treatment initiation and subsequent severe impacts on health.

Amyloid light-chain (AL) amyloidosis is a rare disease caused by extracellular deposition of misfolded immunoglobulin light chains. This study aimed to provide an up-to-date estimate of prevalence and incidence of AL amyloidosis in the United States. Using claims databases from years 2007 to 2015, adults ≥18 years old with AL amyloidosis were included if they had (1) at least 1 inpatient or 2 outpatient claims consistent with AL amyloidosis and (2) received 1 AL-specific treatment. Prevalence was calculated as the number of AL patients divided by the number of enrollees on June 30th of each calendar year. Incidence was calculated as the number of patients with AL who were disease-free and enrolled with a health plan for 1 year prior, divided by the number of enrollees with enrollment from July 1st of the previous year to June 30th of each calendar year. The prevalence of AL amyloidosis increased significantly between 2007 and 2015, from 15.5 cases per million in 2007 to 40.5 in 2015, an annual percentage change (APC) of 12% ( P P = .114) with no statistically significant increase. There was an increase in AL amyloidosis prevalence over a 9-year period coupled with stable incidence rates. Although there is no diagnosis code specific to AL amyloidosis and no validated method for identifying this condition using claims data, extrapolating from our data, there are at least 12 000 adults in the United States living with AL amyloidosis, and the number seems likely to rise.

Hematopoietic stem-cell transplantation (HSCT) requires highly specialized, resource-intensive care. Myeloablative conditioning regimens used before HSCT generally require inpatient stays and are more intensive than other preparative regimens, and may therefore be more costly.To estimate the costs associated with inpatient HSCT according to the type of the conditioning regimen used and other potential contributors to the overall cost of the procedure.We used data from the Truven Health MarketScan insurance claims database to analyze healthcare costs for pediatric (age18 years) and adult (age ≥18 years) patients who had autologous or allogeneic inpatient HSCT between January 1, 2010, and September 23, 2013. We developed an algorithm to determine whether conditioning regimens were myeloablative or nonmyeloablative/reduced intensity.We identified a sample of 1562 patients who had inpatient HSCT during the study period for whom the transplant type and the conditioning regimen were determinable: 398 patients had myeloablative allogeneic HSCT; 195 patients had nonmyeloablative/reduced-intensity allogeneic HSCT; and 969 patients had myeloablative autologous HSCT. The median total healthcare cost at 100 days was $289,283 for the myeloablative allogeneic regimen cohort compared with $253,467 for the nonmyeloablative/reduced-intensity allogeneic regimen cohort, and $140,792 for the myeloablative autologous regimen cohort. The mean hospital length of stay for the index (first claim of) HSCT was 35.6 days in the myeloablative allogeneic regimen cohort, 26.6 days in the nonmyeloablative/reduced-intensity allogeneic cohort, and 21.8 days in the myeloablative autologous regimen cohort.Allogeneic HSCT was more expensive than autologous HSCT, regardless of the regimen used. Myeloablative conditioning regimens led to higher overall costs than nonmyeloablative/reduced-intensity regimens in the allogeneic HSCT cohort, indicating a greater cost burden associated with inpatient services for higher-intensity preparative conditioning regimens. Pediatric patients had higher costs than adult patients. Future research should involve validating the algorithm for identifying conditioning regimens using clinical data.

Between 2002 and 2007, the nonmedical use of prescription pain relievers grew from 11.0 million to 12.5 million people in the United States. Societal costs attributable to prescription opioid abuse were estimated at $55.7 billion in 2007. The purpose of this study was to comprehensively review the recent clinical and economic evaluations of prescription opioid abuse. A comprehensive literature search was conducted for studies published from 2002 to 2012. Articles were included if they were original research studies in English that reported the clinical and economic burden associated with prescription opioid abuse. A total of 23 studies (183 unique citations identified, 54 articles subjected to full text review) were included in this review and analysis. Findings from the review demonstrated that rates of opioid overdose-related deaths ranged from 5528 deaths in 2002 to 14,800 in 2008. Furthermore, overdose reportedly results in 830,652 years of potential life lost before age 65. Opioid abusers were generally more likely to utilize medical services, such as emergency department, physician outpatient visits, and inpatient hospital stays, relative to non-abusers. When compared to a matched control group (non-abusers), mean annual excess health care costs for opioid abusers with private insurance ranged from $14,054 to $20,546. Similarly, the mean annual excess health care costs for opioid abusers with Medicaid ranged from $5874 to $15,183. The issue of opioid abuse has significant clinical and economic consequences for patients, health care providers, commercial and government payers, and society as a whole. (Population Health Management 2014;17:372–387)

Introduction: Despite current advances in treatment, acute coronary syndromes (ACS), continues to result in high morbidity and mortality. Annually, an estimated 1.5 million hospital discharges involve patients with ACS. As such, ACS is associated with a substantial economic burden to the US healthcare system, with current estimates ranging from $75-$150 billion annually in aggregate direct medical costs. Adding anticoagulant to standard of care (SOC) may reduce the number of cardiovascular (CV) events in ACS patients. This study estimates the economic burden of mortality and CV events among ACS patients within a commercial health plan. Methods: We developed an Excel-based decision-analytic model to estimate the annual economic burden of mortality and CV events (myocardial infarction (MI), ischemic stroke (IS), stent thrombosis (ST), intracranial hemorrhage (ICH), and major bleeds) in a hypothetical commercial plan with a population of ACS patients who were candidates for rivaroxaban anticoagulation therapy. The baseline ACS population of 6,650 patients was calculated from literature estimates and included 100% on standard of care, and 0% on dual SOC and rivaroxaban anticoagulation therapy. ACS prevalence was adjusted for age and anticoagulation status. Estimates for the two-year rates for mortality and CV events were obtained from the two-year randomized double-blind, placebo-controlled rivaroxaban ATLAS clinical trial. Healthcare costs were drawn from the published literature and adjusted to 2011 USD. Results: The baseline model projected a total of 299 deaths from any cause, resulting in an estimated $9.52 million in two-year mortality-related healthcare costs to a commercial health plan. In addition, the baseline model estimated 439 MI, 67 IS, 193 ST, 13 ICH, and 27 major bleeding events that were not associated with CABG, resulting in a total of $41.3 million in two-year healthcare costs to a commercial health plan. All other factors being equal, a 10% increase in the dual SOC and rivaroxaban anticoagulation therapy from the SOC population would reduce the number of mortalities by 11, ST events by 5, and MI events by 3, while the number of ischemic stroke events would remain the same. Conversely, there would be an increase of 1 ICH event and 7 major bleeding events not associated with CABG. Two-year ACS-related mortality and CV event costs to the commercial health plan would be reduced by $48,437 for every 1,000 patients with ACS. Conclusion: Our findings suggest that a modest 10% increase in anticoagulant use among patients with ACS would reduce mortality, MI, ST and related healthcare costs by 4%, 0.7%, and 3%, respectively. Addition of anticoagulation therapy potentially reduces the incidence of ACS-related mortality, MI, ST and associated healthcare costs to a commercial health plan, and benefits from anticoagulation use should be balanced against the risk of bleeding.

Introduction: Hematopoietic stem cell transplantation (HSCT) requires specialized, resource intensive care. National hospital costs associated with HSCT were $1.3 billion in 2007 (Stranges et al., 2009). Myeloablative conditioning regimens used prior to HSCT tend to be inpatient and more intensive than other preparative regimens, and may therefore be more costly. Objective: To estimate the total 100-day and one-year costs associated with inpatient HSCT, stratified by type of conditioning regimen and other potential contributors of cost. Methods: We used a retrospective cohort study design and MarketScan database to analyze costs among autologous (auto) or allogeneic (allo) inpatient HSCT patients (pts) between 1/2/2010 and 9/23/2013. Because details of chemotherapeutic agents are not available in inpatient claims data, we relied on clinician expert input to develop an algorithm to determine whether conditioning regimens were myeloablative (MA) or nonmyeloablative/reduced intensity (NMA/RIC). The algorithm used the underlying malignancy, type of treatment (e.g. chemotherapy type, radiation), location of service, and therapy timing. Cost estimates in claims are based on amount paid by insurer. Using the date of HSCT as the index date, we examined healthcare use and costs occurring within 100 days and one year of follow-up, according to graft type, conditioning regimen, and age group (pediatric age Results: We identified 1,564 pts who had inpatient HSCT for whom graft type and conditioning regimen were determinable (MA allo: n=398; NMA/RIC allo: n=195; MA auto: n=969; NMA/RIC auto: n=2 [Figure 1]). Our sample was 92.9% adult and 61% male. The most common indications for HSCT were lymphoma (62.4%), aplastic anemia (46.3%), acute lymphocytic leukemia (21.5%), and acute myeloid leukemia (11.3%) (not mutually exclusive). In the first 100 days following transplantation, median total healthcare costs were $289,283 for MA allo ps compared to $253,467 for NMA/RIC allo pts and $140,792 for MA auto pts. Mean length of stay (LOS) for the index HSCT hospitalization was 35.6 days for MA allo pts, 26.6 days for NMA/RIC allo pts, and 21.8 days for MA auto pts. Subsequent hospitalization occurred for 42.5% of MA allo pts with a mean LOS of 9 days compared to 43.6% of NMA/RIC allo pts with 11 days and 20.8% of MA auto pts with 6.5 days. Cost of the HSCT admission made up 73-76% of 100-day costs for MA pts and 66% for NMA/RIC pts (Figure 2). 100-day costs were more than two-thirds of total median costs at one year, which were $408,876, $374,065, and $181,933 for the MA allo, NMA/RIC allo, and MA auto group, respectively. Costs for the index HSCT and for 100-day inpatient and outpatient healthcare were greater among pediatric than adult pts, for all graft and conditioning types. Median 100-day pharmacy costs were higher for adults than pediatric pts in the allo but not auto groups. Conclusions: Total per patient healthcare costs were high in all groups studied, ranging from a median of about $182K to $409K in the one-year period post HSCT.Consistent with prior reports on HSCT, our study comprised predominantly adult, male pts (Majhail et al., 2013; CIBMTR, 2014). Costs varied substantially by graft type, conditioning regimen, and age group, with highest overall 100-day and one-year costs in the MA allo group. Allo HSCT was more expensive at 100 days and one year compared to auto, regardless of regimen. MA conditioning regimen led to higher overall costs compared to NMA/RIC among allo pts, pointing to greater cost burden associated with inpatient services with higher-intensity regimens. Also, pediatric pts had larger 100-day inpatient and outpatient costs compared to adults. These observed differences may have been driven in part by disease severity, malignancy type, body weight, or treatment differences in pediatric and adult pts. Limitations of this initial analysis were that few NMA/RIC auto pts were identified because they are treated in the outpatient setting. In addition, findings were driven largely by the adult pts. Finally, we excluded many pts whose conditioning regimens were undeterminable, making our results sample-specific. Future research should target a broader patient population that includes complete conditioning regimen data and all HSCT regardless of treatment setting. This analysis was funded by Jazz Pharmaceuticals, Inc. Disclosures Quock: Jazz Pharmaceuticals: Employment, Equity Ownership. Broder:Jazz Pharmaceuticals: Other: Partnership for Health Analytic Research (PHAR) received funding from Jazz Pharmaceuticals for study design and analysis; Partnership for Health Analytic Research (PHAR): Employment. Chang:Partnership for Health Analytic Research (PHAR): Employment; Jazz Pharmaceuticals: Other: Partnership for Health Analytic Research (PHAR) received funding from Jazz Pharmaceuticals for study design and analysis. Reddy:Partnership for Health Analytic Research (PHAR): Employment; Jazz Pharmaceuticals: Other: Partnership for Health Analytic Research (PHAR) received funding from Jazz Pharmaceuticals for study design and analysis. Arai:Jazz Pharmaceuticals: Consultancy. Agarwal-Hashmi:Jazz Pharmaceuticals: Consultancy. Villa:Jazz Pharmaceuticals: Employment, Equity Ownership.

Introduction Hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS) is a potentially fatal complication of HSCT and is characterized by hepatomegaly, right upper quadrant pain, jaundice, and ascites (Gratwohl A et al JAMA 2010). Severe VOD (sVOD) is clinically characterized by multi-organ dysfunction and is estimated to have a mortality rate of over 80% (Coppell JA et al BBMT 2010). VOD and particularly sVOD pose a significant economic burden in direct medical costs by adding an estimated $53,000 to HSCT (2004 U.S. dollars; Saito et al BBMT 2008), or $78,000 in 2015 dollars. However, little research has been conducted in ascertaining the indirect costs associated with premature death due to VOD. In our study, we developed an economic model to evaluate the indirect cost associated with premature death due to sVOD among HSCT patients in the U.S. Methods An Excel-based model was developed to estimate the indirect cost associated with premature death due to sVOD among HSCT patients. Model inputs included prevalence of sVOD as well as mortality, age, salary, employment rate, and life expectancy. Inputs were obtained from the published literature, Center for International Blood & Marrow Transplant Research (CIBMTR), Centers for Disease Control and Prevention (CDC), U.S. Census Bureau, and Organisation for Economic Co-operation and Development (OECD). The model considered lost productivity as the result of premature death by calculating the number of years between the age of sVOD-related death and the assumed age of retirement (65 years old); it was assumed that the lifetime salary reflected underlying productivity of HSCT survivors who did not develop sVOD. Annual salary and life expectancy among HSCT survivors were adjusted from the general population to reflect decreased productivity in the first 2 years immediately following HSCT. Annual average salary inputs by age categories were assumed to be the same as the general population. Age of the working population was assumed to be between 18 and 65 years old. Excess mortality from sVOD was calculated as number of deaths among sVOD patients minus the number of deaths among HSCT patients who did not develop sVOD. Indirect cost was expressed as the cumulative salary for all productive years contributed by a patient and included a 3% discount for each additional year. Total lost productivity years and indirect costs by age category were obtained by multiplying each respective per patient value by the number of excess deaths in each age category. A sensitivity analysis was performed to examine the impact of changes in key model parameters on model results. Results The model estimated 361 excess deaths per year due to sVOD in the HSCT population. The excess deaths due to sVOD were associated with indirect costs of over $68 million due to reduced life expectancy and consequent productivity loss. AlloSCT patients were younger on average, resulting in greater indirect costs ($39 million) compared to AutoSCT ($29 million). AlloSCT patients lost 3,637 productive years on average compared to 2,353 lost productive years for AutoSCT patients despite fewer patients receiving AlloSCT than AutoSCT. A sensitivity analysis showed that the total indirect costs due to overall VOD (non-severe and severe) were as high as $73.3 million. Conclusion sVOD imposes a substantial economic burden on patients in terms of excess deaths, lost productivity, and indirect costs. The model underestimates indirect costs largely due to not accounting for productivity loss among children and their caregivers or those older than 65. It also does not calculate productivity loss among HSCT survivors. Future research is warranted to elicit the additional indirect costs associated with non-severe VOD. Figure 3. Deterministic Sensitivity Analysis: Total Indirect Costs due to Excess Deaths due to sVOD Compared with HSCT Survivors without VOD. Figure 3. Deterministic Sensitivity Analysis: Total Indirect Costs due to Excess Deaths due to sVOD Compared with HSCT Survivors without VOD. Disclosures Quock: Jazz Pharmaceuticals: Employment, Equity Ownership. Zhou:Jazz Pharmaceuticals: Other: Analysis Group received funding for this analysis from Jazz Pharmaceuticals, Inc.; Analysis Group: Employment. Dai:Jazz Pharmaceuticals: Other: Analysis Group received funding for this analysis from Jazz Pharmaceuticals, Inc.; Analysis Group: Employment. Tang:Jazz Pharmaceuticals: Other: Analysis Group received funding for this analysis from Jazz Pharmaceuticals, Inc.; Analysis Group: Employment. Villa:Jazz Pharmaceuticals: Employment, Equity Ownership.

Background: End-stage renal disease (ESRD) is a debilitating condition resulting in death unless treated. Treatment options include conservative care, transplantation, and dialysis. Major alternative dialysis modalities include peritoneal dialysis (PD) and in-center hemodialysis (ICHD), which have been shown to produce similar outcomes and survival. The need to provide dialysis treatment for patients with ESRD represents a significant financial challenge for global health care systems. Changes in clinically-appropriate dialysis delivery leading to more efficient use of resources would increase health systems’ ability to meet that challenge. The purpose of this paper is to evaluate the economic literature of PD and ICHD within the context of continued economic uncertainty and pressure on healthcare resource use.Methods:A systematic literature search was conducted for studies published between 2004 and 2012. Articles are included if they were original research studies in English which reported costs and/or cost effectiveness associated with PD and ICHD.Results:Twenty-four articles are included in our review, six of which are cost effectiveness studies comparing PD and ICHD.Conclusions:Our findings echo those of prior published reviews, showing that PD is significantly cost-saving compared to ICHD therapy in most developed countries and some developing countries. Increasing the use of clinically-appropriate PD would substantially reduce healthcare costs.