Epidemiology of Huntington's Disease in the United States Medicare and Medicaid Populations.

INTRODUCTION: Huntington's disease (HD) is a rare, genetic, and ultimately fatal neurodegenerative disease, with a devastating impact on individuals and families across generations. Few estimates of HD epidemiology in the United States (US) exist. METHODS: This study employed a retrospective cross-sectional design to examine the epidemiology of HD in the US Medicare and Medicaid beneficiary populations using 2016-2017 claims data from the Medicare 100% Research Identifiable Files (RIFs) and 2014 claims data from the Medicaid Analytic eXtract (MAX) files for 17 states. Medicare beneficiaries ≥65 years with a diagnosis of HD (≥1 claim with ICD-10-CM code G10) in 2017 and Medicaid beneficiaries <65 years with a diagnosis of HD (≥1 claim with ICD-9-CM code 333.4) in 2014 were identified. The study outcomes included the 2017 prevalence proportion and incidence rate of HD in the Medicare population and the 2014 prevalence proportion of HD in the Medicaid population. RESULTS: In the Medicare population, 1,941 prevalent and 819 incident cases of HD were identified in 2017, corresponding to a prevalence proportion of 13.1 per 100,000 persons and incidence rate of 6.1 per 100,000 person-years. In the Medicaid population, 353 prevalent cases of HD were identified in 2014, corresponding to a prevalence proportion of 15.2 per 100,000 persons. CONCLUSION: This study suggests that prevalence and incidence of HD in the US may be higher than previously estimated. This has important implications in raising awareness of HD among providers and payers and ensuring availability of and access to services for HD patients and care partners in the Medicare and Medicaid populations.

Direct Costs Associated with Relapsed Diffuse Large B-Cell Lymphoma Therapies.

BACKGROUND: About one third of patients with diffuse large B-cell lymphoma (DLBCL) relapse after receiving first-line (1L) treatment of rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone (R-CHOP). Relapsed patients may then be eligible for second-line (2L) therapy. The study's objective was to examine health care use and costs among treated patients with DLBCL receiving 2L therapy versus those without relapse. MATERIALS AND METHODS: We analyzed Truven Health MarketScan® claims data between 2006 and 2015. Patients (≥18 years of age) had ≥1 DLBCL claim from 1 year before to 90 days after beginning 1L therapy, and comprised those without 2L treatment for ≥2 years (cured controls) versus those who initiated non-R-CHOP chemotherapy after discontinuing 1L therapy (2L cohort). 2L patients were further subgrouped: hematopoietic stem cell transplant (HSCT [yes/no]) and time of relapse (months between 1L and 2L): early (≤3), mid (4-12), and late (>12) relapse. The primary outcome was 1- and 2-year health care costs. Hospitalization rate and length of stay were also measured. RESULTS: A total of 1,374 patients with DLBCL received R-CHOP and fulfilled all criteria: 1,157 cured controls and 217 2L patients (87 early-relapse, 66 mid-relapse, 64 late-relapse). Twenty-eight percent of 2L patients received HSCT. Charlson Comorbidity Index/mortality risk was higher for 2L patients (4.2 [SD: 3.0]) versus controls (3.8 [2.6]; p = .039), as were yearly costs (Year 1: $210,488 [$172,851] vs. $25,044 [$32,441]; p < .001 and Year 2: $267,770 [$266,536] vs. $42,272 [$49,281]; p < .001). HSCT and chemotherapy were each significant contributors of cost among 2L patients. CONCLUSION: DLBCL is resource intensive, particularly for 2L patients. Great need exists for newer, effective therapies for DLBCL that may save lives and reduce costs. IMPLICATIONS FOR PRACTICE: This study identified multiple important drivers of cost in the understudied population of patients with diffuse large B-cell lymphoma (DLBCL) receiving second-line (2L) treatment. Such drivers included hematopoietic stem cell transplant (HSCT) and chemotherapy. Even though HSCT is currently the only curative therapy for DLBCL, less than one third of patients receiving 2L and subsequent treatment underwent transplant, which indicates potential underuse. The variation in chemotherapy regimens suggested a lack of consensus for best practices. Further research focusing on newer and more effective treatment options for DLBCL has the potential to decrease mortality, in addition to reducing the extensive costs related to therapy options such as transplant.

Tolerability of central nervous system symptoms among HIV-1 infected efavirenz users: analysis of patient electronic medical record data.

Efavirenz (EFV) is a non-nucleoside reverse transcriptase inhibitor indicated for treatment of HIV-1 infection. Despite concern over EFV tolerability in clinical trials and practice, particularly related to central nervous system (CNS) adverse events, some observational studies have shown high rates of EFV continuation at one year and low rates of CNS-related EFV substitution. The objective of this study was to further examine the real-world rate of CNS-related EFV discontinuation in antiretroviral therapy naïve HIV-1 patients. This retrospective cohort study used a nationally representative electronic medical records database to identify HIV-1 patients ≥12 years old, treated with a 1st-line EFV-based regimen (single or combination antiretroviral tablet) from 1 January 2009 to 30 June 2013. Patients without prior record of EFV use during 6-month baseline (i.e., antiretroviral therapy naïve) were followed 12 months post-medication initiation. CNS-related EFV discontinuation was defined as evidence of a switch to a replacement antiretroviral coupled with record of a CNS symptom within 30 days prior, absent lab evidence of virologic failure. We identified 1742 1st-line EFV patients. Mean age was 48 years, 22.7% were female, and 8.1% had a prior report of CNS symptoms. The first year, overall discontinuation rate among new users of EFV was 16.2%. Ten percent of patients (n = 174) reported a CNS symptom and 1.1% (n = 19) discontinued EFV due to CNS symptoms: insomnia (n = 12), headache (n = 5), impaired concentration (n = 1), and somnolence (n = 1). The frequency of CNS symptoms was similar for patients who discontinued EFV compared to those who did not (10.3 vs. 9.9%; P = .86). Our study found that EFV discontinuation due to CNS symptoms was low, consistent with prior reports.

AN APPROACH TO USING DATA MINING TO SUPPORT EARLY IDENTIFICATION OF ACROMEGALY.

OBJECTIVE: Data mining using insurance claims presents an opportunity to incorporate new analytic techniques in identifying rare conditions. This study aims to identify dyads of clinical conditions associated with acromegaly that may, with further validation and testing, be used to initially identify and diagnose this rare disease more accurately and efficiently. METHODS: This case-control study used two claims databases to identify acromegaly patients (cases) (International Classification of Diseases, Ninth Revision, Clinical Modification [ICD-9-CM]: 253.0) from 2008-2013. Each case was assigned two nonacromegaly controls (same age, gender, and region). Matched patients were randomly split into development and validation datasets. With expert clinician input, we isolated common associated conditions using ICD-9-CM codes. We identified all 2-way combinations of these conditions (dyads) and calculated the rate and risk relative (RR) to controls. Dyads meeting certain criteria (case rate ≥5% [or ≥1% if RR ≥5] or observed RR > expected) were replicated in the validation dataset to confirm results. RESULTS: We identified 3,731 cases and 7,462 controls: mean age 41.8 (SD, 16.1) years, 51.8% female. A total of 32 and 38 dyads, reduced from 630, met study criteria. Among replicated dyads, case rates varied -15.9% (hypertension and metabolic disorder) to 0.6% (arthritis and menstrual abnormalities). The highest RRs (e.g., valvular insufficiency and colon polyps [RR, 13.5; rate, 0.7%]) also exceeded expected values. Replication showed similar RR direction and size. CONCLUSION: This novel analytic approach revealed several dyads that were significantly associated with an acromegaly diagnosis. Presence of high-risk condition pairs, if verified by a detailed data source (e.g., medical charts), may be incorporated into screening tools or serve as potential markers for physicians to consider an acromegaly diagnosis. ABBREVIATIONS: ICD-9-CM = International Classification of Diseases, Ninth Revision, Clinical Modification ID = identification RR = relative risk.

Impact of a high-deductible health plan on outpatient visits and associated diagnostic tests.

BACKGROUND: By shifting a greater share of out-of-pocket medical costs to consumers, high-deductible health plans (HDHP) might discourage use of essential outpatient services. OBJECTIVE: The objective of the study was to examine the impact of an HDHP on outpatient visits and associated laboratory and radiology tests. RESEARCH DESIGN/SUBJECTS: We used a pre-post with comparison group study design to examine the differential change in outpatient service utilization among 7953 adults who were switched from a traditional Health Maintenance Organization plan to an HDHP compared with 7953 adults remaining in traditional plans. HDHP members had full coverage of preventive laboratory tests and modest copayments for outpatient visits, similar to controls, but faced full cost sharing under the deductible for radiology tests and laboratory tests not classified as preventive. RESULTS: Compared with controls, the HDHP group experienced moderate relative decreases in overall office visits (incidence rate ratios = 0.91, or a 9% relative reduction; 95% confidence interval: 0.88, 0.94) and visits for higher-priority (0.91; 0.85, 0.97) and lower-priority (0.89; 0.81, 0.99) chronic conditions. There were no significant differences in changes in visit rates for acute higher-priority or lower-priority conditions (both 0.93; 0.86, 1.01) or preventive laboratory tests (0.97; 0.93, 1.02). HDHP members showed moderate relative reductions in the use of general laboratory tests (0.91; 0.86, 0.97) but not radiology tests (0.97; 0.91, 1.03). CONCLUSIONS: Chronic outpatient visits declined among HDHP members, although preventive laboratory tests and acute visits remained unchanged. HDHP patients with chronic illnesses who have more contact with the health care system might be more likely to reduce utilization because of increased exposure to costs associated with ambulatory visits.

Reduced mortality, complications, and economic burden among medicare beneficiaries receiving influenza antivirals.

INTRODUCTION: Antiviral therapy may be underutilized in patients at high risk for increased clinical and economic burden (e.g. older adults). We aimed to examine the benefits associated with antiviral treatment of seasonal influenza among treated and untreated Medicare beneficiaries. METHODS: This retrospective study of Medicare Claims Research Identifiable Files identified patients ≥66 years old with an influenza diagnosis in outpatient setting between October 2016-March 2019 (flu seasons 2016-2018). Index date defined as date of first claim with influenza diagnosis; baseline as the 12 months pre-index. Treated patients received antivirals ≤2 days from index. Untreated patients had no antivirals ≤6 months post-index. Treated/untreated patients were 1:1 propensity score matched. Outcomes (death, all-cause and respiratory-related healthcare resource utilization [HCRU] and costs) were assessed until death or up to 6 months post-index. Descriptive statistics were reported; Kaplan-Meier estimation was used for survival over time. RESULTS: Among 116,901 matched patient pairs, all-cause mortality within 6 months from index diagnosis was 1.6% among treated versus 4.3% among untreated patients. Rates (treated versus untreated) of all-cause inpatient hospitalizations during follow-up were 13.9% versus 22.7% and respiratory-related hospitalizations were 4.2% versus 9.0%. Mean (SD) total all-cause and respiratory-related costs were $9,830 ($18,616.0) and $900 ($4016.4) among the treated, respectively, versus $13,207 ($24,405.1) and $2,024 ($7,623.7) among untreated, respectively. All differences were statistically significant (p < 0.001). CONCLUSIONS: Lack of antiviral treatment is associated with increased mortality, HCRU, and economic burden in older Medicare beneficiaries with seasonal influenza. Future research should investigate whether the choice of antivirals affects influenza burden.

Previous studies have shown that antiviral drugs help prevent flu-related complications and lower healthcare utilization and costs. However, these previous studies have focused on working aged people with existing health problems. Our study looks at how antiviral treatment can lower the health and financial burden caused by the flu in older adults. Using a Medicare claims database from the 2016­2018 flu season, we identified 116,901 matched (treated versus untreated) patient pairs. All-cause mortality within 6 months from the index diagnosis (defined as the first claim with a flu diagnosis) was 1.6% among treated versus 4.3% among untreated patients. Rates (treated versus untreated) of all-cause inpatient hospitalizations during follow-up (defined as 6 months after the index diagnosis date) were 13.9% versus 22.7% and respiratory-related hospitalizations were 4.2% versus 9.0%. Mean total all-cause and respiratory-related costs were $9,830 and $900 among the treated, respectively, versus $13,207 and $2,024 among untreated, respectively. All differences were statistically significant (p < 0.001). This analysis of older adults with the flu found that prompt antiviral treatment is associated with lower rates of mortality and acute complications, reduced hospitalization, and lower healthcare costs. Use of antiviral treatment for patients at high risk of flu, such as older adults, is warranted.

Development and validation of a claims-based algorithm to identify patients with Neuromyelitis Optica Spectrum disorder.

INTRODUCTION: No validated algorithm exists to identify patients with neuromyelitis optica spectrum disorder (NMOSD) in healthcare claims data. We developed and tested the performance of a healthcare claims-based algorithm to identify patients with NMOSD. METHODS: Using medical record data of 101 adults with NMOSD, multiple sclerosis (MS), or myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD), we tested the sensitivity and specificity of claims-based algorithms developed through interviews with neurologists. We tested the best-performing algorithm's face validity using 2016-2019 data from IBM MarketScan Commercial and Medicare Supplemental databases. Demographics and clinical characteristics were reported. RESULTS: Algorithm inclusion criteria were age ≥ 18 years and (≥1 NMO diagnosis [or ≥ 1 transverse myelitis (TM) and ≥ 1 optic neuritis (ON) diagnosis] and ≥ 1 NMOSD drug) or (≥2 NMO diagnoses ≥90 days apart). Exclusion criteria were MS diagnosis or use of MS-specific drug after last NMO diagnosis or NMOSD drug; sarcoidosis diagnosis after last NMO diagnosis; or use of ≥1 immune checkpoint inhibitor. In medical record billing data of 50 patients with NMOSD, 30 with MS, and 21 with MOGAD, the algorithm had 82.0% sensitivity and 70.6% specificity. When applied to healthcare claims data, demographic and clinical features of the identified cohort were similar to known demographics of NMOSD. CONCLUSIONS: This clinically derived algorithm performed well in medical records. When tested in healthcare claims, demographics and clinical characteristics were consistent with previous clinical findings. This algorithm will enable a more accurate estimation of NMOSD disease burden using insurance claims datasets.

Health care payments in the Asia Pacific: validation of five survey measures of economic burden.

INTRODUCTION: Many low and middle-income countries rely on out-of-pocket payments to help finance health care. These payments can pose financial hardships for households; valid measurement of this type of economic burden is therefore critical. This study examines the validity of five survey measures of economic burden caused by health care payments. METHODS: We analyzed 2002/03 World Health Survey household-level data from four Asia Pacific countries to assess the construct validity of five measures of economic burden due to health care payments: any health expenditure, health expenditure amount, catastrophic health expenditure, indebtedness, and impoverishment. We used generalized linear models to assess the correlations between these measures and other constructs with which they have expected associations, such as health care need, wealth, and risk protection. RESULTS: Measures of impoverishment and indebtedness most often correlated with health care need, wealth, and risk protection as expected. Having any health expenditure, a large health expenditure, or even a catastrophic health expenditure did not consistently predict degree of economic burden. CONCLUSIONS: Studies that examine economic burden attributable to health care payments should include measures of impoverishment and indebtedness.

Healthcare utilization and direct medical costs of Huntington's disease among Medicaid beneficiaries in the United States.

AIMS: To provide more recent estimates of healthcare utilization and costs in Huntington's disease (HD) in the Medicaid population. MATERIALS AND METHODS: This retrospective analysis used administrative claims data for HD beneficiaries (≥1 HD claim; ICD-9-CM 333.4) from Medicaid Analytic eXtract data files from 1 January 2010-31 December 2014. The date of the first HD claim during the identification period (1 January 2011-31 December 2013) was assigned as the index date. If a beneficiary had multiple HD claims during the identification period, one was randomly chosen as the index date. Beneficiaries were required to be continuously enrolled in fee-for-service plans during the 1-year pre-index and post-index periods. Medicaid beneficiaries without HD were drawn from a 100% random sample and matched (3:1) to those with HD. Beneficiaries were classified by disease stage (early/middle/late). All-cause and HD-related (any utilization related to HD diagnosis or symptoms associated with HD) healthcare utilization and costs were reported. RESULTS: A total of 1,785 beneficiaries without HD were matched to 595 beneficiaries with HD (139 early-, 78 middle-, and 378 late-stage). The mean (SD) annual total costs were higher for beneficiaries with HD than beneficiaries without HD ($73,087 [$75,140] vs. $26,834 [$47,659], p <.001) and driven by inpatient costs ($45,190 [$48,185] vs. $13,808 [$39,596], p <.001). Total healthcare costs were highest among beneficiaries with late-stage HD (mean [SD] cost: $22,797 [$31,683] for early-stage HD vs. $55,294 [$129,290] for middle-stage HD vs. $95,251 [$60,197] for late-stage HD; p <.001). LIMITATIONS: Administrative claims are intended for billing purposes and subject to coding errors. This study did not address functional status, which may provide further insight to late-stage and end-of-life burden of HD, and indirect costs. CONCLUSIONS: Medicaid beneficiaries with HD have higher acute healthcare utilization and costs compared to beneficiaries without HD, which tend to increase with disease progression, indicating that HD beneficiaries at later disease stages have greater burden.

Huntington's disease (HD) is a degenerative genetic disorder marked by progressive decline in cognitive and motor functions, leading to severe disability and loss of independence. The median and mean survival time after a diagnosis of HD is 15 years. Little is known about the kinds of health services used or costs associated with HD in the United States (US) in the Medicaid population. The study objective was to estimate healthcare utilization and direct medical spending among Medicaid beneficiaries with HD. The mean annual total costs were higher for beneficiaries with HD than beneficiaries without HD ($73,087 vs. $26,834). Mean total healthcare costs were highest among beneficiaries with late-stage HD ($22,797 for early-stage HD vs. $55,294 for middle-stage HD vs. $95,251 for late-stage HD). Medicaid beneficiaries with HD have higher acute healthcare utilization and costs compared to beneficiaries without HD, with utilization and costs increasing with disease progression, indicating that HD beneficiaries at later disease stages have greater burden.

Cost of cancer management by stage at diagnosis among Medicare beneficiaries.

OBJECTIVE: Estimate the annual cost of care in the 5 years following a cancer diagnosis for 17 invasive cancer types, by stage at diagnosis. METHODS: We used 2012-2016 data from the Surveillance, Epidemiology, and End Results (SEER) registry-Medicare claims database to examine cost of care among Medicare beneficiaries with a confirmed cancer diagnosis based on International Classification of Diseases for Oncology, Third Edition histology codes reported in SEER. Beneficiaries contributed to the annual cost calculations (Years 1-5) using their observed time after diagnosis. Beneficiaries were continuously enrolled in fee-for-service Medicare Parts A/B and Part D during follow-up. Total, inpatient, outpatient, and pharmacy cancer-related service costs were calculated. RESULTS: From 2012 to 2016, we identified 597,778 Medicare beneficiaries with incident cancer diagnosis within 5 years (Stage I, II, III, and IV: 32.6%, 33.4%, 15.9%, and 18.0%, respectively). In Year 1, mean (standard deviation) total costs for Stage I diagnoses varied from $7640 ($17,378) (prostate) to $94,636 ($117,636) (pancreas). Total costs increased by stage and reached $58,783 ($92,344) (prostate) to $156,982 ($175,009) (stomach) for Stage IV diagnoses in Year 1. Costs in Year 1 were significantly higher for Stage IV diagnoses than for earlier stages across all cancer types. In Years 2-5, total costs were lower than in Year 1 but continued to increase by stage. CONCLUSIONS: Beneficiaries diagnosed at later stages of cancer have higher costs of care (up to 7 times as much) than those diagnosed at earlier stages. Earlier cancer diagnosis may lead to more efficient treatment and decreased management cost.

Clinical manifestations and healthcare utilization before diagnosis of transthyretin amyloidosis.

Introduction: Initial clinical manifestations of transthyretin amyloidosis (ATTR) are not well understood, making timely diagnosis challenging. Methods: Patients aged ≥68 years newly diagnosed with ATTR were identified using Medicare Research Identifiable Files. Symptom manifestation and healthcare utilization were measured during 3 years pre-diagnosis; demographics and comorbidity index during 1-year pre-diagnosis. Controls (ATTR-free) were matched 1:1 to patients with ATTR based on age, sex and region; same index date and enrollment as match. Results: We identified 552 matched ATTR-control pairs: mean age 78.3 (standard deviation 6.3) and 64.5% male. Among patients with ATTR (vs controls), cardiovascular conditions (92.9 vs 75.9%) and hospitalization (54.0 vs 35.5%) were frequent during 3 years pre-diagnosis. Conclusion: Patients with ATTR have multiple symptoms and hospitalizations pre-diagnosis, recognition of which may facilitate earlier diagnosis and treatment.

Antifibrotic therapies reduce mortality and hospitalization among Medicare beneficiaries with idiopathic pulmonary fibrosis.

BACKGROUND: Additional real-world studies are needed to more fully elucidate the effectiveness of antifibrotic treatment in slowing the progression of idiopathic pulmonary fibrosis (IPF). OBJECTIVE: To compare mortality and hospitalization between Medicare beneficiaries with IPF who initiate antifibrotic therapy and those who did not receive treatment. METHODS: A retrospective observational study of Medicare beneficiaries using the 100% Medicare Research Identifiable File was conducted. We included patients aged 67 years and over diagnosed with IPF (≥ 1 inpatient or ≥ 2 outpatient claims with IPF diagnosis) during the study period (January 1, 2010-December 31, 2017). Patients who initiated antifibrotic treatment (pirfenidone or nintedanib) between October 15, 2014 (FDA approval date) and December 31, 2017 (ie, treated patients) were compared with those who did not receive treatment during a historical period (January 1, 2012-October 14, 2014) before the availability of antifibrotics (ie, untreated historical controls). Patients were matched by propensity score, and the outcomes, mortality, and hospitalization (all cause and respiratory related) were compared using a Cox proportional hazards model. RESULTS: We identified 4,641 treated patients and 4,641 propensity score-matched controls who met all study criteria; 352 treated patients who lacked matches were excluded from the study. Cox regression analysis of treated patients vs matched controls showed a significantly lower risk of mortality (HR = 0.62, 95% CI = 0.57-0.68); lower risk of hospitalization (HR = 0.71, 95% CI = 0.67-0.76; HR = 0.70, 95% CI = 0.64-0.76); and lower rate in number of hospitalizations per month (incident rate ratio [IRR] = 0.65, 95% CI = 0.60-0.71; IRR = 0.65, 95% CI = 0.58-0.73). CONCLUSIONS: This study suggests that treatment with antifibrotics may confer a survival benefit and protection against all-cause and respiratory-related hospitalization for IPF patients. DISCLOSURES: This work was sponsored by F. Hoffmann-La Roche/Genentech, Inc. Corral is employed by Genentech, Inc. Reddy, Chang, Broder, and Gokhale are employed by Partnership for Health Analytic Research LLC, a health services research company, which was hired by Genentech to conduct this research. Mooney has received advisory board/consulting fees and research support from Genentech, unrelated to this work. Mooney also reports advisory board/consulting fees and research support from Boehringer Ingelheim; personal fees from Imvaria; and grants from Celgene and Pliant, unrelated to this work. Through their employment with Partnership for Health Analytic Research, Reddy, Chang, Broder, and Gokhale have been compensated to conduct research for AbbVie, Akcea, ASPC, Amgen, AstraZeneca, BMS, Boston Scientific Corporation, Celgene, Eisai, Ethicon, GRAIL, Helsinn, Illumina, Innovation and Value Initiative, Ionis, Jazz, Kite, Novartis, Otsuka, Pathnostics, PhRMA, Prothena, Sage, Verde Technologies, Genentech, Inc., Greenwich Biosciences, Inc., Mirum Pharmaceuticals, Inc., Sanofi US Services, Inc., Sunovion Pharmaceuticals, Inc., and Dompe US, Inc., unrelated to this work. This research was presented as an abstract at CHEST 2020 Annual Meeting (virtual), October 18-21, 2020, and American Thoracic Society 2020 Virtual Meeting, June 2020.

The patient journey toward a diagnosis of hereditary transthyretin (ATTRv) amyloidosis.

BACKGROUND: Despite emerging treatments for hereditary transthyretin (ATTRv) amyloidosis, the disease is often misdiagnosed, with reported diagnostic delays of up to several years. Knowledge of the patient journey leading up to diagnosis may help to promote earlier intervention. The study's objective was to examine patient clinical characteristics and healthcare utilization prior to ATTRv amyloidosis diagnosis. METHODS: Patients ≥ 18 years and newly diagnosed with ATTRv amyloidosis identified in IBM® MarketScan® Commercial and Medicare Supplemental data using a claims-based algorithm as follows: diagnosis required ≥ 1 medical claim with relevant amyloidosis diagnosis code (ICD-10-CM: E85.0-.4, E85.89, E85.9; excludes light chain and wild type) during identification (ID) period (1/1/2016-12/31/2017), and ≥ 1 occurrence of qualifying criteria during 2011-2017: ≥ 15 days diflunisal use without > 30-day gap, liver transplant, or claim with specific codes E85.1 or E85.2. The index date was defined as the date of first claim with amyloidosis diagnosis code in ID period. Patients had continuous enrollment ≥ 5 years pre-index date (look-back period). Occurrence of selected comorbid conditions and symptoms and healthcare utilization (testing, emergency department visits and hospitalization) measured during the look-back period; demographics, physician specialty, and Charlson comorbidity index (CCI) measured 1 year pre-index. Patients with an ICD-9/10 amyloidosis code during the look-back period were excluded. An ATTRv-free reference cohort was created from a random sample of enrollees who lacked any diagnosis of amyloidosis and matched 3:1 to ATTRv patients on age, gender, and region to provide reference values; same index and enrollment requirement as match. RESULTS: For the 141 qualifying patients with ATTRv and 423 matched controls, mean (standard deviation) age was 62.5 (14.2) years and 53.9% were female. Mean CCI for ATTRv cohort was 2.7 (3.0) versus 1.1 (1.9) among controls. Selected comorbidities, testing, visits, and hospitalization were common among patients with ATTRv during the look-back period with higher rates versus controls. CONCLUSIONS: Patients with ATTRv amyloidosis experience multiple neurological, cardiovascular, and other clinical manifestations, testing, and hospitalization prior to diagnosis. Occurrence of potential markers of illness is most common in the year before diagnosis.

Healthcare utilization and cost burden of Huntington's disease among Medicare beneficiaries in the United States.

AIMS: To examine healthcare utilization and costs in a US Medicare population diagnosed with Huntington's disease (HD). METHODS: This was a retrospective matched cohort study using Medicare fee-for-service (FFS) claims data using 2013-2017 Research Identifiable Files. Medicare beneficiaries diagnosed with HD based on the presence of at least one medical claim with an International Classification of Diseases, Ninth or Tenth Revision, Clinical Modification (ICD-9/10-CM) diagnosis code for HD (ICD-9-CM: 333.4; ICD-10-CM: G10) during the identification period (2014-2016). Beneficiaries without HD were drawn from a 5% random sample of Medicare beneficiaries and 1:1 matched to those with HD for comparison. All-cause and HD-related (any utilization related to HD diagnosis or symptoms associated with HD) healthcare utilization and costs were reported. RESULTS: We identified 3,688 matched pairs of beneficiaries with and without HD. Of those with HD, 1,922 (52.1%) were late-stage, 916 (24.8%) were middle-stage, and 850 (23.1%) were early-stage. Mean [SD] annual total healthcare costs were higher for HD beneficiaries than beneficiaries without HD ($41,631 [57,393] vs. $17,222 [31,218], p < .001) and were primarily driven by outpatient pharmacy costs ($19,182 [45,469] vs. $4,318 [11,553], p < .001). In the stratified analysis, total healthcare costs were highest among beneficiaries with late-stage HD (mean [SD] cost: $20,475 [$41,122] for early-stage vs. $29,733 [$44,977] for middle-stage vs. $56,657 [$64,185] for late-stage; p < .001). LIMITATIONS: Results are not generalizable to beneficiaries enrolled in other non-FFS Medicare plans. Administrative claims are intended for billing purposes, not research, and may not capture all symptoms, comorbidities, and other adverse events. CONCLUSIONS: This original, comprehensive analysis of healthcare utilization and economic burden among Medicare beneficiaries with HD found that healthcare needs and associated costs are substantially higher among Medicare beneficiaries who are diagnosed with HD compared to beneficiaries without HD.

Healthcare use and costs among Medicare enrollees on pirfenidone versus nintedanib for idiopathic pulmonary fibrosis.

Aim: Compare healthcare utilization and costs between Medicare beneficiaries with idiopathic pulmonary fibrosis (IPF) receiving pirfenidone or nintedanib. Methods: Retrospective cohort study of Medicare beneficiaries (100% Research Identifiable Files) with IPF who initiated pirfenidone or nintedanib between 15 October 2014 and 31 December 2015. Inverse probability of treatment weighting using propensity scores adjusted for baseline covariates. Outcomes: hospitalization and monthly costs. Results: Hazard and incidence rate ratios (95% CI) for all-cause (0.79 [0.68-0.91]; 0.69 [0.59-0.82]) and respiratory-related (0.80 [0.65-0.97]; 0.71 [0.57-0.90]) hospitalizations favored pirfenidone versus nintedanib. Monthly inpatient costs were lower for pirfenidone versus nintedanib patients; outpatient and pharmacy costs were similar. Conclusion: In patients with IPF, pirfenidone compared with nintedanib has a moderate but significant protective effect on hospitalization, corresponding to lower inpatient costs.

Burden of Nasal Polyps in the United States.

OBJECTIVE: To investigate the clinical and health care burden of chronic rhinosinusitis (CRS) with nasal polyps (CRSwNP) in the United States. STUDY DESIGN: Retrospective, cross-sectional design with analyses of patient visits from 2 databases. SETTING: National Ambulatory Medical Care Survey (NAMCS, 2012-2016) and State Ambulatory Surgery and Services Databases (SASD, 2012-2015) in available states. METHODS: In each analysis, we identified patients (≥18 years old) with a diagnosis of CRSwNP (ICD-9-CM: 471.x; ICD-10-CM: J33.x) in the visit record during the study period. CRS patients without polyps (CRSsNP: ICD-9-CM: 473.x, ICD-10-CM: J32.x; without CRSwNP codes) were identified for comparison. In the SASD, we focused on visits involving relevant sinus procedures. Outcomes included comorbidities, diagnostic testing, and prescribed medication (NAMCS) and surgery visit characteristics (SASD). RESULTS: We identified 2272 NAMCS records from physician offices (183 CRSwNP, 2089 CRSsNP). Most visits were for patients aged <65 years (78.8%, 80.6%) and privately insured (67.7%, 61.5%); CRSwNP visits had a male majority (56.3%, 35.4%). CRSwNP vs CRSsNP visits more often reported asthma (40.2%, 10.3%), allergic rhinitis (14.0%, 8.7%), and congestion (22.0%, 21.1%), with the use of glucocorticoids (21.0%, 17.7%) and nasal allergy medication (26.2%, 10.2%). In the SASD, 427,306 surgery visits were identified (71,195 CRSwNP, 356,111 CRSsNP); demographics were similar to NAMCS. CRSwNP surgeries involved more sinus types (59.3%, 41.4%). Surgeries were mostly elective (>99%) and completed quickly (<2 hours), without perioperative complications (>99%), followed by routine discharge (>91%); follow-up visits were common (14.9%, 13.9%). CONCLUSION: CRSwNP compared to CRSsNP patients have a distinct clinical experience, with moderately higher medication need and more extensive surgery.

The Clinical and Economic Burden of Newly Diagnosed Hereditary Transthyretin (ATTRv) Amyloidosis: A Retrospective Analysis of Claims Data.

INTRODUCTION: Little is known about the burden of hereditary transthyretin (ATTRv) amyloidosis, a genetic, progressive, and fatal disease caused by extracellular deposition of transthyretin amyloid fibrils. The study's aim was to estimate costs and disease burden associated with ATTRv amyloidosis in a real-world setting. METHODS: Using IBM® MarketScan® Commercial and Medicare Supplemental data, we identified patients at least 18 years of age with newly diagnosed ATTRv amyloidosis. Diagnosis required at least one medical claim with relevant diagnosis code (International Classification of Diseases, 9th Revision, Clinical Modification [ICD-9-CM] 277.30-.31, 277.39; ICD-10-CM E85.0-.4, E85.89, E85.9) between January 1, 2014 and December 31, 2016, and at least one additional criterion occurring during study period (2013-2017): at least 15 days diflunisal use without more than a 30-day gap; liver transplant; or claim with codes E85.1 or E85.2. First diagnosis date was study index. Continuous enrollment 1-year pre-index (baseline) and post-index (follow-up) was required. Patients with baseline amyloidosis diagnosis were excluded. Outcomes of interest were comorbidities and 1-year follow-up healthcare utilization and costs (also reported quarterly). RESULTS: Among 185 qualifying patients, mean age was 59.2 years (standard deviation 15.2), 54.1% were female, and baseline Charlson comorbidity index was 2.2 (2.5). Neuropathy (30.3%), diabetes (27.0%), and cardiovascular-related comorbidities, including dyspnea (25.9%) and congestive heart failure (21.6%), were common during follow-up. Nearly a quarter of patients (24.9%) were hospitalized during follow-up. Most hospitalizations and emergency department visits occurred in the first quarter post-diagnosis (18.9%, 17.8%, respectively) and dropped in subsequent quarters. The annual mean total cost was $64,066, with inpatient services contributing the majority of the expenses ($34,461), followed by outpatient ($23,853), and then pharmacy ($5752). As with utilization, costs were highest in the first quarter post-diagnosis and dropped in subsequent quarters. CONCLUSION: Patients newly diagnosed with ATTRv amyloidosis have substantial healthcare utilization and costs in the first year, primarily the initial months, post-diagnosis. Further research should examine later costs associated with disease progression and end-of-life care.

Analysis of Safety, Medical Resource Utilization, and Treatment Costs by Drug Class for Management of Inflammatory Bowel Disease in the United States Based on Insurance Claims Data.

INTRODUCTION: Conventional pharmaceutical interventions for inflammatory bowel disease (IBD) provide limited disease/symptom control and are associated with an increased risk of adverse events (AEs). These limitations increase patient morbidity, medical resource utilization (MRU), and costs. METHODS: The IQVIA™ Real-World Data Adjudicated Claims-US database was leveraged to identify adult patients (> 18 years) with Crohn's disease (Crohn's) or ulcerative colitis (UC), who were new and chronic users (≥ 60 days) of oral corticosteroids (OCS), immunosuppressants (IS), anti-tumor necrosis factor agents (anti-TNF) or combinations thereof. Using aminosalicylate-treated patients as a reference, we compared AE incidence, MRU, and medical costs across drug classes. RESULTS: The analysis included 30,676 patients (Crohn's: n = 14,528; UC: n  = 16,148). OCS monotherapy was the strongest predictor of any AE occurring [Crohn's: hazard ratio 1.62 (1.51-1.73); UC: hazard ratio 1.57 (1.49-1.66)]. A similar pattern was observed for severe infection and bone-related conditions. Patients with UC or Crohn's receiving OCS or IS plus OCS were more likely to have emergency department visits, IBD-related hospitalizations/visits/procedures, and gastrointestinal surgery than were patients receiving other therapies. Annualized total medical costs (pharmacy plus hospital service costs) were greatest for anti-TNF plus IS or anti-TNF therapy in both Crohn's and UC. Annualized medical service costs (excluding IBD drug costs) were highest for patients initiating OCS-containing therapies [Crohn's: OCS, $27,041 (24,882-29,200) and OCS plus IS, $23,332 (19,889-26,775); UC: OCS, $19,659 (17,977-21,340)]. CONCLUSION: Although biologic therapies have higher pharmacy costs, treatment decisions should consider the increased AE risks and long-term MRU costs associated with chronic use of OCS-containing therapies. FUNDING: This study was funded by F. Hoffmann-La Roche Ltd. The journal's Rapid Service Fee and Open Access publication were paid for by ApotheCom on behalf of Genentech, a member of the Roche group who funded the study.

Increased Clinical and Economic Burden Associated With Peripheral Intravenous Catheter-Related Complications: Analysis of a US Hospital Discharge Database.

The burden of complications associated with peripheral intravenous use is underevaluated, in part, due to the broad use, inconsistent coding, and lack of mandatory reporting of these devices. This study aimed to analyze the clinical and economic impact of peripheral intravenous-related complications on hospitalized patients. This analysis of Premier Perspective® Database US hospital discharge records included admissions occurring between July 1, 2013 and June 30, 2015 for pneumonia, chronic obstructive pulmonary disease, myocardial infarction, congestive heart failure, chronic kidney disease, diabetes with complications, and major trauma (hip, spinal, cranial fractures). Admissions were assumed to include a peripheral intravenous. Admissions involving surgery, dialysis, or central venous lines were excluded. Multivariable analyses compared inpatient length of stay, cost, admission to intensive care unit, and discharge status of patients with versus without peripheral intravenous-related complications (bloodstream infection, cellulitis, thrombophlebitis, other infection, or extravasation). Models were conducted separately for congestive heart failure, chronic obstructive pulmonary disease, diabetes with complications, and overall (all 7 diagnoses) and adjusted for demographics, comorbidities, and hospital characteristics. We identified 588 375 qualifying admissions: mean (SD), age 66.1 (20.6) years; 52.4% female; and 95.2% urgent/emergent admissions. Overall, 1.76% of patients (n = 10 354) had peripheral intravenous-related complications. In adjusted analyses between patients with versus without peripheral intravenous complications, the mean (95% confidence interval) inpatient length of stay was 5.9 (5.8-6.0) days versus 3.9 (3.9-3.9) days; mean hospitalization cost was $10 895 ($10 738-$11 052) versus $7009 ($6988-$7031). Patients with complications were less likely to be discharged home versus those without (62.4% [58.6%-66.1%] vs 77.6% [74.6%-80.5%]) and were more likely to have died (3.6% [2.9%-4.2%] vs 0.7% [0.6%-0.9%]). Models restricted to single admitting diagnosis were consistent with overall results. Patients with peripheral intravenous-related complications have longer length of stay, higher costs, and greater risk of death than patients without such complications; this is true across diagnosis groups of interest. Future research should focus on reducing these complications to improve clinical and economic outcomes.