Healthcare resource utilization and costs in patients with a newly confirmed diagnosis of lupus nephritis in the United States over a 5-year follow-up period.

BACKGROUND: We aimed to describe healthcare resource utilization (HCRU) and healthcare costs in patients with newly confirmed lupus nephritis (LN) in the United States over a 5-year follow-up period. METHODS: This retrospective, longitudinal cohort study (GSK Study 214102) utilized administrative claims data to identify individuals with a newly confirmed diagnosis of LN between August 01, 2011, and July 31, 2018, based on LN-specific International Classification of Diseases diagnosis codes. Index was the date of first LN-related diagnosis code claim. HCRU, healthcare costs, and incidence of systemic lupus erythematosus (SLE) flares were reported annually among eligible patients with at least 5 years continuous enrollment post-index. RESULTS: Of 2,159 patients with a newly confirmed diagnosis of LN meeting inclusion and exclusion criteria, 335 had at least 5 years continuous enrollment post-index. HCRU was greatest in the first year post-LN diagnosis across all categories (inpatient admission, emergency room [ER] visits, ambulatory visits, and pharmacy use), and trended lower, though remained substantial, in the 5-year follow-up period. Among patients with LN and HCRU, the mean (standard deviation [SD]) number of ER visits and inpatient admissions were 3.7 (4.6) and 1.8 (1.5), respectively, in Year 1, which generally remained stable in Years 2-5; the mean (SD) number of ambulatory visits and pharmacy fills were 35.8 (25.1) and 62.9 (43.8), respectively, in Year 1, and remained similar for Years 2-5. Most patients (≥ 91.6%) had ≥ 1 SLE flare in each of the 5 years of follow-up. The proportion of patients who experienced a severe SLE flare was higher in Year 1 (31.6%) than subsequent years (14.3-18.5%). Total costs (medical and pharmacy; mean [SD]) were higher in Year 1 ($44,205 [71,532]) than subsequent years ($29,444 [52,310]-$32,222 [58,216]), driven mainly by inpatient admissions (Year 1: $21,181 [58,886]; subsequent years: $7,406 [23,331]-$9,389 [29,283]). CONCLUSIONS: Patients with a newly confirmed diagnosis of LN have substantial HCRU and healthcare costs, particularly in the year post-diagnosis, largely driven by inpatient costs. This highlights the need for improved disease management to prevent renal damage, improve patient outcomes, and reduce costs among patients with renal involvement.

Hereditary factor X deficiency in America survey: impact on quality of life and burden of disease in patients and caregivers.

Hereditary factor X deficiency (HFXD) is a rare bleeding disorder causing delayed haemostasis and potentially life-threatening bleeds. Patient/caregiver burden and diagnosis path have not been well characterized. THE AIM OF THIS STUDY WAS TO: describe the diagnosis path, disease burden, and HFXD impact on quality of life (QoL) in patients and caregivers.This was a prospective, cross-sectional, web-based survey of patients with HFXD and caregivers addressing the patient/caregiver experience, QoL, humanistic and unmet needs.Thirty patients and 38 caregivers completed the survey with mean ages 24.7 and 44.6 years, respectively. Mean age at diagnosis was 4.1 years. The diagnostic process was somewhat/very difficult for 23% of patients and 26% of caregivers. Approximately half (53%) received single factor replacement (SFR) as prophylaxis or on-demand. Most patients (71%) reported regular prophylaxis treatment. Over one-fourth (27%) reported treatment with fresh frozen plasma. Bleeding episodes were less common in patients using SFR versus non-SFR: three bleeds or fewer were reported by 92% SFR and 75% non-SFR patients. HFXD patients reported low well being in work/school/social activities with mean HFXD-adapted Hemophilia Well being Index. Patient symptoms negatively impacted caregiver burden with a mean HFXD-adapted Hemophilia Caregiver Index (±SD) of 15.9 (4.6), but also unexpectedly had a positive impact on self-worth and inner strength.To our knowledge, this is the first study to assess patient and caregiver burden of HFXD and impact on QoL. Improvements in symptom recognition, prompt diagnosis, and adherence to expert recommendations for treatment could improve QoL and decrease burden on HFXD patients and caregivers.

Health Care Costs With Sustained Oral Corticosteroid Use in Systemic Lupus Erythematosus.

PURPOSE: The goal of this study was to compare health care costs, health care resource utilization, and adverse events associated with sustained oral corticosteroid (OCS) use versus no OCS use in systemic lupus erythematosus. METHODS: This retrospective cohort study used claims data (January 1, 2006-July 31, 2019) from patients with systemic lupus erythematosus aged ≥5 years with ≥24 months of continuous enrollment. Health care costs, health care resource utilization, and OCS-related adverse events were assessed. The sustained OCS cohort (defined as ≥12 months of continuous OCS use) was divided into exposure categories based on the number of 6-month classification periods with >5 mg/d OCS (0, 1-2, or 3-4). FINDINGS: Of the 6234 patients in the sustained OCS use cohort, there were 1587 (25.5%) patients with 0 periods of >5 mg/d OCS use, 2087 (33.5%) patients with 1 to 2 periods of >5 mg/d OCS use, and 2560 (41.1%) patients with 3 to 4 periods of >5 mg/d OCS use; the no OCS use cohort included 7828 patients. Adjusted health care cost differences (95% CIs) were significantly greater for patients with 0, 1 to 2, and 3 to 4 periods of OCS use >5 mg/d versus the no OCS use cohort ($7774 [5426-10,223], $21,738 [18,898-25,321], and $30,119 [26,492-33,774], respectively). A higher proportion of patients in all OCS exposure categories required health care resource utilization (≥99.7% vs 93.4%) and experienced OCS-related adverse events (94.3%-96.8% vs 82.6%) versus the no OCS use cohort, with more periods of OCS use >5 mg/d associated with increased health care resource utilization and adverse events. IMPLICATIONS: Sustained OCS use in systemic lupus erythematosus was associated with high economic burden, health care resource utilization, and OCS-related adverse events. These data highlight the need for health care providers to carefully consider OCS use in systemic lupus erythematosus.

Clinical and Economic Burden in Patients With Systemic Lupus Erythematosus During the First Year After Initiating Oral Corticosteroids: A Retrospective US Database Study.

OBJECTIVE: To characterize health care resource utilization (HCRU), health care costs, and adverse events (AEs) among patients with systemic lupus erythematosus (SLE) initiating oral corticosteroids (OCS) versus patients without OCS use. METHODS: In this retrospective cohort study (GSK Study 213061), eligible patients (aged ≥5 years at first OCS claim) with SLE from the IQVIA Real-World Data Adjudicated Claims-US database (January 2006 to July 2019) had continuous enrollment during the 6-month preindex (baseline) and 12-month postindex (observation) periods and one or more inpatient or emergency department SLE diagnosis codes or two or more outpatient SLE diagnosis codes during baseline. The "OCS-initiator cohort" comprised patients with one or more OCS pharmacy claims during the study period and no evidence of preindex OCS use and was classified into three exposure categories based on the number of 6-month periods of more than 5 mg/day of OCS use (0, 1, 2). The "no-OCS-use cohort" comprised patients without OCS claims, although patients may have received OCS prior to the study period. Clinical and economic outcomes were reported over the observation period. RESULTS: Adjusted health care costs differed significantly ($6542 [95% confidence interval (CI): $5761-$7368], $19,149 [95% CI: $16,954-$21,471], $28,985 [95% CI: $25,546-$32,885]). HCRU incidence rates were significantly greater for all OCS-initiator exposure categories (n = 16,216) versus the no-OCS-use cohort (n = 11,137; adjusted incidence rate ratios [95% CI]: 1.22 [1.19-1.24], 1.39 [1.34-1.43], 1.66 [1.60-1.73]). OCS-related AEs were experienced by 67.1% to 74.1% of patients with OCS initiation, most commonly affecting the immune system. CONCLUSION: Within 12 months of OCS initiation, patients with SLE experienced substantial clinical and economic burden, which may imply a need to minimize OCS use.

Healthcare Resource Utilization and Associated Costs in Patients With Systemic Lupus Erythematosus Diagnosed With Lupus Nephritis.

BACKGROUND: Lupus nephritis (LN) is among the most severe organ manifestations of systemic lupus erythematosus (SLE), affecting between 31% and 48% of patients, usually within five years of SLE diagnosis. SLE without LN is associated with a high economic burden on the healthcare system, and although data are limited, several studies have shown that SLE with LN could increase this burden.  Aim: We aimed to compare the economic burden of LN versus SLE without LN among patients managed in routine clinical practices in the USA and describe the clinical course of these patients. MATERIALS AND METHODS: This was a retrospective observational study of patients with commercial or Medicare Advantage health insurance. It included 2310 patients with LN and 2310 matched patients who had SLE without LN; each patient was followed for 12 months after diagnosis (the patient's index date). Outcome measures included healthcare resource utilization (HCRU), direct healthcare costs, and SLE clinical manifestations.  Results: In all healthcare settings, the mean (SD) use of all-cause healthcare resources was significantly higher in the LN versus SLE without LN cohort, including the mean number of ambulatory visits (53.9 (55.1) vs 33.0 (26.0)), emergency room visits (2.9 (7.9) vs 1.6 (3.3)), inpatient stays (0.9 (1.5) vs 0.3 (0.8)), and pharmacy fills (65.0 (48.3) vs 51.2 (42.6)) (all p<0.001). Total all-cause costs per patient in the LN cohort were also significantly higher compared with the SLE without LN cohort ($50,975 (86,281) vs $26,262 (52,720), p<0.001), including costs for inpatient stays and outpatient visits. Clinically, a significantly higher proportion of patients with LN experienced moderate or severe SLE flares compared with the SLE without LN cohort (p<0.001), which may explain the difference in HCRU and healthcare costs. CONCLUSION: All-cause HCRU and costs were higher for patients with LN than for matched patients with SLE without LN, highlighting the economic burden associated with LN.

Prolonged oral corticosteroid treatment in patients with systemic lupus erythematosus: An evaluation of 12-month economic and clinical burden.

BACKGROUND: Prolonged, high-dose corticosteroid treatment for systemic lupus erythematosus (SLE) is associated with substantial health care costs, health care resource utilization (HCRU), and adverse events (AEs). OBJECTIVE: To compare all-cause health care costs, HCRU, and oral corticosteroid (OCS)-related AEs among patients with prevalent OCS use and patients without OCS use. METHODS: This retrospective, longitudinal cohort study (GSK study 214100) used claims data from the IQVIA Real-World Data Adjudicated Claims - US, IQVIA, Inc, database between January 1, 2006, and July 31, 2019, to identify patients with SLE. Patients with at least 1 OCS pharmacy claim during the study period and continuous OCS use during the 6-month pre-index (baseline) period (index date is the date of the first OCS claim following 6 months' continuous use) formed the "prevalent OCS use cohort." This cohort was subdivided based on the level of OCS exposure during the 12-month observation period, ie, the number of 6-month periods of greater than 5 mg/day OCS use (0, 1, or 2). Patients without OCS claims formed the "no OCS use cohort." All patients had continuous enrollment during the baseline and observation periods, had at least 1 inpatient or at least 2 outpatient SLE diagnosis codes during baseline, and were aged at least 5 years at index. A 2-part model, a generalized linear regression model with a negative binomial distribution, and a multivariate logistic regression model were used to compare health care costs, HCRU, and the odds of developing an OCS-related AE between cohorts, respectively. RESULTS: The no OCS use and prevalent OCS use cohorts included 21,517 and 16,209 patients, respectively. Adjusted health care cost differences (95% CI) were significantly lower for the no OCS use cohort vs all prevalent OCS use exposure categories ($5,439 [$4,537-$6,371] vs $17,856 [$16,368-$19,498]), driven by inpatient stays and outpatient visits; HCRU was also significantly lower (adjusted incidence rate ratios vs no OCS use cohort [95% CI]: 1.20 [1.16-1.23] vs 1.47 [1.41-1.52]). Health care costs and HCRU increased with increasing length of OCS exposure. OCS-related AEs occurred more frequently for all prevalent OCS use exposure categories vs the no OCS use cohort (odds ratio [95% CI]: 1.39 [1.25-1.55] vs 2.32 [2.02-2.68]), driven by hematologic/oncologic and immune system-related AEs. The mean (SD) average daily dose of OCS increased with increasing periods of prevalent OCS use (2.5 [1.3], 6.9 [31.1], and 34.6 [1,717.3] mg/day, respectively, for patients with 0, 1, and 2 periods of OCS use). CONCLUSIONS: Prevalent OCS use incurs a substantial clinical and economic burden, highlighting the need for restricted OCS doses and durations. DISCLOSURES: This study (GSK Study 214100) was funded by GSK. GSK was involved in designing the study, contributing to the collection, analysis, and interpretation of the data, supporting the authors in the development of the manuscript, and funding the medical writing assistance. All authors, including those employed by GSK, approved the content of the submitted manuscript and were involved in the decision to submit the manuscript for publication. Dr DerSarkissian, Dr Duh, and Mr Benson are employees of Analysis Group, which received research funding from GSK to conduct this study. Dr Wang, Ms Gu, and Mr Vu are former employees of Analysis Group. Mr Bell is an employee of GSK and holds stocks and shares in the company. Ms Averell and Dr Huang are former employees of GSK and held stocks and shares in the company at the time of the study.

Clinical and Economic Burden of Systemic Lupus Erythematosus in the Years Preceding End-Stage Kidney Disease Diagnosis: A Retrospective Observational Study.

INTRODUCTION: This study aimed to describe the clinical burden, healthcare resource utilisation (HCRU) and healthcare costs for patients with systemic lupus erythematosus (SLE) in the 12-60 months preceding an end-stage kidney disease (ESKD) diagnosis in the USA. METHODS: This retrospective observational study identified adult patients with SLE with newly diagnosed ESKD between 1 March 2012 and 31 December 2018 using administrative claims data. Clinical characteristics, mean all-cause HCRU (i.e. any HCRU visit and pharmacy fill) and total all-cause healthcare costs (comprising medical and pharmacy costs in 2019 US dollars) were assessed during the 12 months pre-ESKD diagnosis and yearly during the 5 years pre-ESKD diagnosis among patients with ≥ 5 years of continuous health plan enrolment. RESULTS: Of the 1356 patients included, 51.2% had severe SLE, 71.2% had lupus nephritis (LN) and 20.6% underwent kidney biopsy during the 12 months pre-ESKD. The mean (standard deviation [SD]) number of HCRU visits during the 12 months pre-ESKD was 78.0 (64.1) per patient. The mean (SD) total healthcare costs per patient in the 12 months pre-ESKD diagnosis was $64,887 (106,822), driven by medical costs $51,764 (96,458). The proportions of patients with severe SLE, LN and those undergoing biopsy increased from year 5 to year 1 pre-ESKD diagnosis. The mean (SD) number of HCRU visits increased from year 5 (61.6 [54.0]) to year 1 (83.2 [62.1]) pre-ESKD. Mean (SD) total healthcare costs rose year on year from year 5 ($34,890 [74,346]) to year 1 ($73,236 [114,584]) pre-ESKD. CONCLUSION: There were substantial clinical burden and healthcare costs among patients with SLE in the 12 months pre-ESKD diagnosis. The clinical burden and healthcare costs generally increased with each year approaching ESKD diagnosis. Early interventions for patients with SLE could prevent the development of ESKD, mitigating the burden of the disease.

Assessment of Quality of Life Among Patients With Recurrent Clostridioides difficile Infection Treated with Investigational Oral Microbiome Therapeutic SER-109: Secondary Analysis of a Randomized Clinical Trial.

Importance: Recurrent Clostridioides difficile infection (CDI) is a debilitating disease leading to poor health-related quality of life (HRQOL), loss of productivity, anxiety, and depression. The potential association of treatment with HRQOL has not been well evaluated. Objectives: To explore the association of SER-109 compared with placebo on HRQOL in patients with recurrent CDI up to week 8. Design, Setting, and Participants: This study was a secondary analysis of a randomized, double-blind, placebo-controlled trial that took place at 56 sites in the US and Canada from July 2017 to April 2020 and included 182 patients randomized to SER-109 or placebo groups. Interventions: SER-109 or placebo (4 capsules once daily for 3 days) following antibiotics for CDI. Main Outcomes and Measures: Exploratory analysis of HRQOL using the disease specific Clostridioides difficile Quality of Life Survey (Cdiff32) assessed at baseline, week 1, and week 8. Results: In this study, 182 patients (109 [59.9%] female; mean age, 65.5 [16.5] years) were randomized to SER-109 (89 [48.9%]) or placebo (93 [51.1%]) groups and were included in the primary and exploratory analyses. Baseline Cdiff32 scores were similar between patients in the SER-109 and placebo groups (52.0 [18.3] vs 52.8 [18.7], respectively). The proportion of patients with overall improvement from baseline in the Cdiff32 total score was higher in the SER-109 arm than placebo at week 1 (49.4% vs 26.9%; P = .012) and week 8 (66.3% vs 48.4%; P = .001).Greater improvements in total and physical domain and subdomain scores were observed in patients in the SER-109 group compared with placebo as early as week 1, with continued improvements observed at week 8. Among patients in the placebo group, improvements in HRQOL were primarily observed in patients with nonrecurrent CDI while patients in the SER-109 group reported improvements in HRQOL, regardless of clinical outcome. Conclusions and Relevance: In this secondary analysis of a phase 3 clinical trial, SER-109, an investigational microbiome therapeutic was associated with rapid and steady improvement in HRQOL compared with placebo through 8 weeks, an important patient-reported outcome. Trial Registration: ClinicalTrials.gov Identifier: NCT03183128.

Validation of a Health-Related Quality of Life Questionnaire in Patients With Recurrent Clostridioides difficile Infection in ECOSPOR III, a Phase 3 Randomized Trial.

BACKGROUND: Debilitating symptoms of recurrent Clostridioides difficile infection (rCDI) often lead to long-term effects on health-related quality-of-life (HRQOL). In ECOSPOR III, SER-109, an investigational oral microbiome therapeutic, was superior to placebo in reducing rCDI. We investigated the validity, reliability, and responsiveness of a 32-item, CDI-specific questionnaire-the Clostridium difficile Quality of Life Survey (Cdiff32)-across mental, physical, and social domains in patients with rCDI. METHODS: In this post hoc analysis of a phase 3 clinical trial, 182 outpatients with rCDI completed Cdiff32 and EQ-5D at baseline and at 1 and 8 weeks. Cdiff32 was evaluated for item performance, internal reliability, and convergent validity. To assess known-groups validity, Cdiff32 scores were compared by disease recurrence status at week 1; internal responsiveness was evaluated in the nonrecurrent disease group by 8 weeks by means of paired t test. RESULTS: All 182 patients (mean age [standard deviation], 65.5 [16.5] years; 59.9% female) completed baseline Cdiff32. Confirmatory factor analysis identified 3 domains (physical, mental, and social relationships) with good item fit. High internal reliability was demonstrated (Cronbach α = 0.94 with all subscales >0.80). Convergent validity was evidenced by significant correlations between Cdiff32 subscales and EQ-5D (r = 0.29-0.37; P < .001). Cdiff32 differentiated patients by disease recurrence status at week 1 (effect sizes, 0.38-0.42; P < .05 overall), with significant improvement from baseline through week 8 in patients with nonrecurrent disease at week 1 (effect sizes, 0.75-1.02; P < .001 overall). CONCLUSIONS: Cdiff32 is a valid, reliable, and responsive disease-specific HRQOL questionnaire that is fit for purpose for interventional treatment trials. The significant improvement in patients with nonrecurrent disease by 8 weeks demonstrates the negative impact of rCDI on HRQOL.

The cost of flares among patients with systemic lupus erythematosus with and without lupus nephritis in the United States.

OBJECTIVE: Assess healthcare costs associated with systemic lupus erythematosus (SLE) flares among patients with and without lupus nephritis (LN). METHODS: This retrospective cohort study used medical and pharmacy claims data from the United States-based Optum Clinformatics database to identify adults with SLE between 1 January 2016, and 31 December 2018. Index was the date of a patient's earliest SLE diagnosis claim during the identification period. Patients were categorized based on ICD-9/-10 diagnosis codes into one of two cohorts: SLE with LN (LN) and SLE without LN (non-LN). Baseline characteristics were assessed in the 12 months preceding index (baseline period). The presence, severity, and healthcare costs (in 2019 US dollars) of flares were determined in the 12 months following index (follow-up period). RESULTS: Overall, 11,663 patients with SLE were included (LN, n = 2916; non-LN, n = 8747). During the baseline period, a greater proportion of patients in the LN cohort versus non-LN cohort had a Charlson Comorbidity Index score ≥4 (72.5% vs 13.7%) and inpatient stays (41.0% vs 17.0%). A total of 12,190 flares were identified during the follow-up period (LN, 3494; non-LN, 8696). A greater proportion of flares experienced by patients with LN versus those without LN were moderate (61.2% vs 53.6%) and severe (10.6% vs 5.4%). The mean (standard deviation [SD]) number of moderate and severe flares per patient was greater among the LN cohort than the non-LN cohort (moderate: LN, 1.8 [1.2] and non-LN, 1.4 [1.2]; severe: LN, 0.2 [0.6] and non-LN, 0.1 [0.3]). The mean (SD) total healthcare costs associated with SLE flares of any severity were greater for patients with LN (LN, $5842 [9604]; non-LN, $2600 [4249]). The mean (SD) cost per flare increased with severity (mild: LN, $2753 [4640] and non-LN, $1606 [2710]; moderate: LN, $4561 [7156] and non-LN, $2587 [3720]; severe: LN, $29,148 [27,273] and non-LN, $14,829 [19,533]). CONCLUSIONS: Patients with SLE with LN have greater healthcare costs than those without LN. Flares among patients with LN were more frequent, severe, and costly than among patients without LN. This highlights the need for treatments that prevent or reduce flares among patients with SLE, both with and without LN.

A 5-Year Retrospective, Observational Study Assessing Rheumatoid Arthritis Disease Outcome Measures to Characterize Systemic Lupus Erythematosus Burden in the USA.

INTRODUCTION: We evaluated the use of rheumatoid arthritis (RA) disease measures in patients with systemic lupus erythematosus (SLE) in a US community-based rheumatology physician network over 5 years. METHODS: This retrospective, observational cohort study (GSK Study 213818) of patients with SLE utilized electronic medical records (01 January 2010-31 December 2019) from the United Rheumatology Normalized Integrated Community Evidence database. The index was the date of first SLE diagnosis recorded in the database; the observation period was 5 years post-index. RA disease measures evaluated were: Pain Index, Multi-Dimensional Health Assessment Questionnaire (MD-HAQ), Patient Global Assessment (PtGA), Physician Global Assessment (PGA), Swollen Joint Count (SJC), Tender Joint Count (TJC), Routine Assessment of Patient Index Data 3 (RAPID3), Clinical Disease Activity Index (CDAI), Simplified Disease Activity Index (SDAI), and Disease Activity Score 28 (DAS-28). The number of patients with measures utilized, the score on each measure, and proportion of patients per disease activity category were assessed. RESULTS: Overall, 5990 patients with SLE were included. The most frequently used measures were Pain Index, SJC, TJC, MD-HAQ, PtGA, RAPID3, and PGA (cumulative use over Years 1-5: 23.9-71.3%). For all measures, frequency of use was lowest in Year 1, followed by a general increase from Year 1 to Year 5. Scores remained relatively stable for most measures, and the proportion of patients in remission or with low/moderate disease activity per RAPID3 increased. CONCLUSION: RA disease measure utilization in SLE was generally infrequent but increased over time. Pain Index and MD-HAQ were the most commonly applied cumulatively across 5 years of follow-up. The rationale for the increased use of these measures in SLE over time requires further exploration. In the absence of a clinically applicable SLE-specific measure, the use of RA measures, for example in conjunction with SLE measures, may provide an alternative approach for measuring disease activity, representing an opportunity to improve patient outcomes.

Health care costs and utilization prior to diagnosis of antineutrophil cytoplasmic antibody vasculitis in Medicare beneficiaries.

BACKGROUND: Antineutrophil cytoplasmic antibody (ANCA) vasculitis (AV) is a complex group of autoimmune disorders affecting blood vessels in multiple organ systems. Delays in diagnosis are common because AV symptoms can be nonspecific and present heterogeneously. This may result in increased health care utilization in the months preceding diagnosis. OBJECTIVE: To examine whether Medicare beneficiaries with AV experienced increased health care utilization and costs in the year before the first diagnosis recorded in claims, relative to beneficiaries without AV. METHODS: This retrospective cohort study used 2015-2016 Medicare Part A/B claims and Part D prescription drug data. Beneficiaries with newly diagnosed AV were identified by having 1 or more inpatient claims or 2 or more noninpatient claims 7 or more days apart in 2016 with an International Classification of Diseases, Tenth Revision, Clinical Modification code for AV, with no AV claims in the year prior. Beneficiaries with AV were matched 1:1 on age and sex to beneficiaries without any diagnoses for any type of systemic vasculitis in 2016. Beneficiaries with Part A/B coverage (AB, n = 1,460) and Part A/B/D coverage (ABD, n = 3,252) were analyzed separately. We estimated generalized linear mixed models with a negative binomial distribution to compare health care costs and utilization by AV status. RESULTS: Beneficiaries with AV had approximately 3 times higher Medicare Part A/B payments (incidence rate ratio [95% CI]: AB: 2.94 [2.44-3.53]; ABD: 2.95 [2.64-3.29]) and 2.5 times higher beneficiary Part A/B payments (AB: 2.47 [2.14-2.84]; ABD: 2.62 [2.40-2.87]) vs beneficiaries without AV. Beneficiaries with AV experienced significantly higher utilization across all categories, with the largest differences observed in hospital outpatient visits (AB: 2.69 [2.22-3.27]; ABD: 3.08 [2.73-3.47]). CONCLUSIONS: In the year prior to AV diagnosis, Medicare beneficiaries have significantly higher health care costs and utilization than beneficiaries without AV. DISCLOSURES: Dr Huang was supported by the University of North Carolina and GlaxoSmithKline Health Outcomes Fellowship during the time of the study and reports current employment at Horizon Therapeutics, Deerfield, IL. Dr Nguyen received predoctoral funding through a fellowship appointment sponsored by Bristol Myers Squibb during the time of the study and reports current employment at GlaxoSmithKline, Collegeville, PA. Dr Derebail receives personal fees from Travere Therapeutics, Bayer, and UpToDate, outside of the submitted work. The views expressed are those of the authors and do not represent the views of the Department of Veteran Affairs. This research did not receive any specific grant from funding agencies in the public, commercial, or not-for-profit sectors.

Real-World Effectiveness of Belimumab in Systemic Lupus Erythematosus: A Systematic Literature Review.

INTRODUCTION: Belimumab is a recombinant human monoclonal antibody that binds to soluble B-lymphocyte stimulator and inhibits its biological activity. Since receiving approvals for the treatment of systemic lupus erythematosus (SLE), several observational studies have investigated the effectiveness of belimumab in the real-world setting. This study reports a systematic review and meta-analysis of the literature to evaluate the real-world effectiveness of belimumab for the treatment of SLE. METHODS: A literature search following PRISMA Guidelines and limited to studies in English was performed (2014-2020) to identify relevant studies reporting effectiveness outcomes of belimumab in patients with SLE. A modified version of the Newcastle-Ottawa Scale was used to assess study quality. Outcomes, including SLE Disease Activity Index (SLEDAI) score, prednisone-equivalent use, and SLE flare were pooled and analyzed using statistical aggregation methods. RESULTS: The literature search identified 514 articles for initial review. Of these, 17 articles were suitable for data extraction and summary. Baseline characteristics of patients in real-world studies were generally similar to those of relevant clinical trials, including age, sex, disease duration, SLEDAI score, and prednisone-equivalent use. Real-world use of belimumab was associated with reductions in SLEDAI score (mean baseline score to month 6: 10.1-4.4; 57% reduction), prednisone-equivalent dosing (mean baseline dose to month 6: 12.1 mg/day to 6.9 mg/day; 43% reduction), and flare frequency (12 months prior to belimumab to 12 months after belimumab: 1.15-0.39 mean flares per patient per year; 66% reduction). Long-term data (up to 2 years post-treatment initiation) for SLEDAI score and prednisone-equivalent dose indicated that improvements in both outcomes continue over time among patients remaining on therapy. CONCLUSIONS: In the real-world setting, observed outcomes with belimumab for the treatment of SLE are consistent with those reported from randomized clinical trials. Improvements persist long-term for SLEDAI activity and prednisone-equivalent use with belimumab.

Benefit of Prompt versus Delayed Use of Single-Inhaler Fluticasone Furoate/Umeclidinium/Vilanterol (FF/UMEC/VI) Following a COPD Exacerbation.

Purpose: Triple therapy (TT; inhaled corticosteroid, long-acting muscarinic antagonist, and long-acting ß2-agonist) is recommended for patients with chronic obstructive pulmonary disease (COPD) at risk of exacerbation, although the optimum timing of TT initiation remains unclear. This study evaluated the impact of prompt versus delayed initiation of single-inhaler TT (fluticasone furoate, umeclidinium, and vilanterol [FF/UMEC/VI]) following a COPD exacerbation. Patients and Methods: This retrospective cohort study used data from the IQVIA PharMetrics® Plus database. Patients initiating FF/UMEC/VI following a COPD exacerbation between September 18, 2017 and September 30, 2019 (exacerbation = index date) were categorized as prompt (within 30 days of index) or delayed (31-180 days after index) FF/UMEC/VI initiators. Patients were aged ≥40 years at index, had ≥12 months' continuous health insurance coverage before index (baseline), and ≥6 months' coverage after index (follow-up). Patients with a COPD exacerbation or claim for FF/UMEC/VI during baseline were excluded. Inverse probability weighting was used to adjust for differences in baseline characteristics between cohorts. Exacerbations (overall, moderate, and severe), healthcare costs, and readmissions were evaluated during follow-up. Results: A total of 1904 patients (prompt: 529; delayed: 1375) were included. After weighting, baseline characteristics were well balanced between cohorts. Patients in the prompt cohort had significantly lower rates per person-year (PPY) of overall (0.98 vs 1.23; rate ratio [RR] [95% CI] = 0.79 [0.65-0.94], p = 0.004), moderate (0.86 vs 1.03; RR [95% CI] = 0.84 [0.69-0.99], p = 0.038), and severe (0.11 vs 0.20; RR [95% CI] = 0.57 [0.37-0.79], p = 0.002) exacerbations, compared with delayed initiators. Mean all-cause and COPD-related healthcare costs were significantly lower among prompt initiators (all-cause: $26,107 vs $32,400 PPY, p = 0.014; COPD-related: $12,694 vs $17,640 PPY, p = 0.002). Conclusion: Prompt initiation of FF/UMEC/VI following a moderate or severe COPD exacerbation was associated with significant reductions in exacerbations and healthcare costs relative to delayed initiation.

Cost-effectiveness of implementing routine hearing screening using a tablet audiometer for pediatric cystic fibrosis patients receiving high-dose IV aminoglycosides.

BACKGROUND: Cystic fibrosis (CF) patients who receive high-dose aminoglycosides can acquire inner ear damage and subsequent hearing loss. There is no current standard protocol for assessing ototoxicity in CF centers in the United States. OBJECTIVE: To evaluate the cost-effectiveness of a pharmacist-implemented routine hearing screening for ototoxicity among pediatric patients using a clinically validated tablet audiometer to allow for earlier detection of hearing loss in an exploratory analysis. METHODS: A Markov decision-analytic model was developed to assess the cost-effectiveness of implementing routine screening with monthly cycles over a 3-year time horizon. The model measured the difference in promptly detected hearing loss, delayed detected hearing loss, and undetected hearing loss, compared with current screening practices. Model inputs were obtained through a comprehensive literature review. Primary model outcomes included total health care costs and quality-adjusted life-years (QALYs) gained with a 3% yearly discount. One-way, two-way, and probabilistic sensitivity analyses were conducted to evaluate model uncertainty. RESULTS: In a hypothetical cohort of 100 patients, routine screening using a tablet audiometer increased promptly detected hearing loss by 8 patients. There was an incremental gain of 3.2 QALYs at an increased cost of $333,826 compared with current screening practices. This resulted in an incremental cost-effectiveness ratio (ICER) of $103,771 per QALY. In the 1-way sensitivity analysis, the ICER ranged between $64,345 and $258,830 per QALY. CONCLUSIONS: Using a tablet audiometer for routine hearing screening appears to be a cost-effective option at a $150,000 per QALY willingness-to-pay threshold when only considering the immediate benefits gained. This analysis did not examine the long-term effects of early detection in language development for pediatric patients. DISCLOSURES: Huang reports funding from the University of North Carolina and GlaxoSmithKline Health Outcomes Fellowship. GlaxoSmithKline had no involvement in the study creation, analysis, or manuscript composition. The other authors have nothing to disclose.