Real-world treatment utilization and economic implications of lupus nephritis disease activity in the United States.

BACKGROUND: Lupus nephritis (LN) is a common and severe complication of systemic lupus erythematosus (SLE), with approximately 40% of patients with SLE developing LN. Even with treatment, 10%-30% of patients will progress to end-stage renal disease (ESRD). Although many studies have assessed the clinical value of low disease activity in LN, the economic implications are less defined. OBJECTIVE: To evaluate treatment utilization and health care costs associated with active disease, low disease activity, and ESRD in patients with LN. METHODS: A retrospective analysis of Optum pharmacy and medical claims data from 2015 to 2019 was performed and included patients with a diagnosis of SLE (International Classification of Diseases, Ninth Revision or Tenth Revision codes 710.0 or M32, respectively) and additional prespecified criteria for LN. Total health care payer costs for medical and pharmacy services and treatment utilization for commonly prescribed medications were determined for periods of low disease activity, active disease, or ESRD. RESULTS: A total of 21,251 patients (mean age 60.3 years; 87% female; 55% White patients and 18% Black patients) with a mean follow-up period of 30.6 months were included; the majority of patients had active disease (67.3%), followed by low disease activity (51.3%), and ESRD (10.5%). Glucocorticoids were used 2 times more often and mycophenolate mofetil was used 4 times more often in patients with active disease vs low disease activity. Glucocorticoids, mycophenolate mofetil, and tacrolimus were more commonly used in patients with ESRD vs those with low disease activity. Mean medical costs were $4,777 per month in active disease and $18,084 per month in ESRD vs $2,523 per month in low disease activity. CONCLUSIONS: Treatment burden and costs are high for patients with active disease and ESRD in LN. Treatments that allow patients to achieve and maintain low disease activity may help improve patient outcomes and reduce medication use and overall health care costs. DISCLOSURES: Maria Dall'Era and Kenneth Kalunian are consultants of Aurinia Pharmaceuticals. Eric Turowski, Vanessa Birardi, Neil Solomons, Simrat Randhawa, and Paola Mina-Osorio are employees and stockholders of Aurinia Pharmaceuticals. Michael Eaddy is a former employee of Xcenda, LLC. Augustina Ogbonnaya and Eileen Farrelly are employees of Xcenda, LLC, which was contracted by Aurinia Pharmaceuticals to assist in the conduct of this study and the writing of this manuscript. Aurinia Pharmaceuticals provided funding for this study and the preparation of the manuscript. Aurinia Pharmaceuticals had a role in writing the report and decision to submit for publication.

Hospital Readmissions and Mortality Among Intubated and Mechanically Ventilated Adult Subjects With Pneumonia Due to Gram-Negative Bacteria.

BACKGROUND: Ventilator-associated pneumonia (VAP) is one of the most common hospital-acquired infections in ICUs and is associated with significant morbidity and mortality. Gram-negative bacteria cause 55-85% of hospital-acquired pneumonia and are associated with increased mortality. METHODS: This study sought to describe mortality rates and 30-d readmission rates among intubated and mechanically ventilated subjects with Gram-negative pneumonia and to explore associated risk factors for mortality and rehospitalization using data from the 2013 Healthcare Cost and Utilization Project (HCUP) National Readmission Database. The study sample included adults age ≥ 18 y who were hospitalized with invasive, continuous mechanical ventilation; were discharged between February 1, 2013, and November 30, 2013; and had a primary or secondary diagnosis of Gram-negative bacterial pneumonia. Logistic regression was used to identify subject characteristics significantly associated with mortality and readmissions. RESULTS: Using the HCUP projected sample of 32,683 intubated and mechanically ventilated subjects with Gram-negative pneumonia, the mortality rate during the index hospitalization was 24.3%. More than one fifth of subjects (22.9%) who survived the index hospitalization were readmitted within 30 d of discharge. Among subjects with readmissions, 18% occurred within 3 d of discharge, 39% occurred within 7 d of discharge, and 65% occurred within 14 d of discharge. Subjects with prior hospitalization within 30 d of the index hospitalization had a higher risk of readmission with an odds ratio of 1.70 (95% CI 1.48-1.94). CONCLUSIONS: Mortality was high and readmissions were substantial among intubated and mechanically ventilated subjects with Gram-negative pneumonia.

Rosacea Treatment Satisfaction: Matching Adjusted Indirect Treatment Comparison Analysis of Metronidazole Gel or Cream vs Azelaic Acid Foam.

OBJECTIVE: To assess differences in patient-reported treatment side effects and concerns associated with azelaic acid 15% foam (AAF) vs metronidazole cream (MC) and metronidazole gel (MG). METHODS: This study used matching-adjusted indirect comparison (MAIC) to compare patient-reported outcomes from survey data evaluating rosacea treatments. Outcomes of interest included percentages of patients reporting concerns and side effects and measures of importance of the concerns and tolerability of the side effects. Patients in each analysis (MG vs AAF and MC vs AAF) were matched using stabilized inverse propensity scores. RESULTS: When compared to AAF, MG-treated patients more frequently reported concerns with treatment efficacy (54% vs 4%), application (7% vs 3%), and treatment side effects. MC-treated patients more frequently reported concerns with treatment efficacy (61% vs 5%) and dryness (8% vs 5%). AAF-treated patients more frequently reported concerns with cost of treatment compared with MG (7% vs 1%) and MC (9% vs 4%). Among patients reporting concerns, level of importance associated with these concerns was similar for AAF-treated patients compared with MG- and MC-treated patients. When compared to AAF-treated patients, MG-treated patients more frequently reported side effects of dryness (26% vs 15%) and uneven skin tone (3% vs 0%), and MC-treated patients more frequently reported side effects of burning (7% vs 3%), itching (7% vs 5%), and redness (7% vs 5%). MG- and MC-treated patients indicated greater intolerance for reported side effects than AAF-treated patients. CONCLUSIONS: MG- and MC-treated patients more frequently reported treatment concerns and side effects than AAF-treated patients, and tolerability of those side effects was higher for patients treated with AAF. While treatment cost is a more frequent concern in patients treated with AAF, these patients less frequently reported concerns with treatment efficacy and reported similar or greater tolerance to side effects than patients treated with either MC or MG. J Drugs Dermatol. 2020;19(3): doi:10.36849/JDD.2020.3679.

Treatment with Repository Corticotropin Injection in Patients with Rheumatoid Arthritis, Systemic Lupus Erythematosus, and Dermatomyositis/Polymyositis.

PURPOSE: Repository corticotropin injection (RCI) is indicated for a number of autoimmune-mediated diseases including rheumatoid arthritis (RA), systemic lupus erythematosus (SLE), and dermatomyositis (DM)/polymyositis (PM). To better understand the practice patterns and outcomes of RCI in patients with RA, SLE, or DM/PM, we conducted a retrospective medical record analysis. PATIENTS AND METHODS: Participating providers selected deidentified medical records of patients meeting the inclusion criteria (age ≥18 years; physician-reported diagnosis of RA, SLE, or DM/PM; initiation of treatment with RCI between 1/1/2011 and 2/15/2016; ≥3 in-office visits with same site/provider). Collected data spanned 12 months before and after the first prescription date for RCI. Analyses included patient demographics and clinical history, RCI treatment patterns, and physician's impression of change. RESULTS: Data from 54 patients with RA, 30 patients with SLE, and 8 patients with DM/PM were analyzed. The most frequently reported reasons for initiating RCI were lack of efficacy with prior treatment, acute exacerbation of disease, and use as add-on to ongoing therapy. The most common initial RCI dosing, 80 U twice weekly, was used for 84% of patients with RA, 75% with SLE, and 86% with DM/PM. The mean duration of treatment was 4.8, 6.5, and 6.8 months for RA, SLE, and DM/PM, respectively. Among the 57 patients with data on physician's impression of change with RCI, 78.1% of patients with RA, 94.7% with SLE, and 66.7% with DM/PM had a rating of "improved," and the mean time to best impression of change was 3.4, 4.3, and 3.4 months for RA, SLE, and DM/PM, respectively. CONCLUSION: This study reports the real-world patient profile, use patterns, and outcomes of patients who used RCI for the treatment of RA, SLE, and DM/PM. These data can inform appropriate use and clinical expectations when using RCI.

Cost analysis of COPD exacerbations and cardiovascular events in SUMMIT.

OBJECTIVES: The Study to Understand Mortality and Morbidity in COPD (SUMMIT) trial compared the efficacy of once-daily fluticasone furoate/vilanterol (FF/VI) with placebo, FF monotherapy, and VI monotherapy on mortality in patients with moderate chronic obstructive pulmonary disease (COPD) and a history/increased risk of cardiovascular (CV) disease. We conducted a post hoc economic analysis using data from SUMMIT to evaluate the economic benefits of treating these patients with COPD and CV risk. STUDY DESIGN: Patients (aged 40-80 years, with ≥10 pack-years' smoking history and a risk of CV events) were randomized (1:1:1:1) to receive placebo, FF 100 mcg, VI 25 mcg, or FF/VI 100 mcg/25 mcg. METHODS: This was a post hoc economic analysis to assess the rates and associated costs of the composite end point (acute COPD exacerbations and revascularization/CV composite events) in the SUMMIT trial from a US healthcare payer perspective. RESULTS: Overall, 16,485 patients were evaluated; of these, 5246 (31.8%) experienced an on-treatment composite end point event (28.5% experienced a COPD exacerbation, 4.2% experienced a CV event, and 2.0% underwent a revascularization procedure). The mean estimated 1-year on-treatment combined end point cost was highest for placebo and lowest for FF/VI ($4220 vs $3482, respectively). The reductions in cost versus placebo were significant for all active treatments (P <.0001). The likelihood of experiencing an on-treatment combined end point event was lower for patients treated with FF/VI versus placebo (hazard ratio, 0.81; P <.001). CONCLUSIONS: One-year combined end point event costs were significantly lower for all active treatments versus placebo. Clinicians and payers may be able decrease costs by effectively managing patients' COPD in those with CV risk.

Treatment Patterns and Costs of Chronic Inflammatory Demyelinating Polyneuropathy: A Claims Database Analysis.

BACKGROUND: Corticosteroids, plasma exchange, and intravenous immunoglobulin (IVIG) have been standard-of-care treatments for chronic inflammatory demyelinating polyneuropathy (CIDP) for more than 2 decades. Despite guideline recommendations for best clinical practices, heterogeneity in patient presentation and the course of treatment for CIDP remains. There is limited literature regarding the real-world treatment patterns of and costs associated with CIDP. OBJECTIVE: To analyze and describe the real-world treatment patterns of and economic burden associated with CIDP. METHODS: This retrospective cohort study evaluated the treatment patterns and CIDP-related healthcare costs over a 2-year follow-up period for patients with newly diagnosed CIDP who had commercial insurance, using claims data from the IMS LifeLink PharMetrics Plus Claims database between 2009 through 2014. Treatment-naïve patients with newly diagnosed CIDP were evaluated for 2 years postdiagnosis, which captured the treatments used and the resource utilization. The patients were defined as receiving active CIDP therapy (ie, IVIG, immunosuppressants, oral or intravenous steroids, or plasma exchange) or active surveillance. RESULTS: Of the 525 patients identified with newly diagnosed CIDP, 55.2% of patients were prescribed only steroid therapy, and 25.3% of patients were prescribed an IVIG therapy during the 2-year follow-up. The median time to the initial treatment was shortest for patients receiving plasma exchange alone (0.03 months) or in combination with a steroid (0.03 months), followed by IVIG plus another therapy (0.53 months), and then IVIG alone (0.71 months). Initiating therapy with steroids alone took the longest mean time (6.51 months) to start the treatment. The median length of time to receive therapy was longest for the steroid plus plasma exchange cohort (21.8 months), followed by the steroid plus immunosuppressant cohort (10.1 months), and the 2 IVIG cohorts (9.04 months for IVIG alone and 9.82 months for IVIG plus another therapy). The mean total CIDP-specific 2-year follow-up costs were highest for the cohort that received IVIG alone ($119,928) or with an additional therapy ($133,334) and lowest for patients who received active surveillance ($3723) or steroids alone ($3101). CONCLUSIONS: Steroid therapy was initiated later and resulted in a shorter duration of treatment than other treatment options for patients with CIDP, which may reflect diagnostic uncertainty, disease severity or remission, therapeutic challenge to determine diagnosis, or the side-effect profile of steroids. The use of steroids alone was the most common prescribed treatment for CIDP. Further research is needed to understand the rationale for treatment decisions in this patient population and their potential impact on patients and health plans.

The real-world economic impact of home-based video electroencephalography: the payer perspective.

Aims: Electroencephalography (EEG) is an established method to evaluate and manage epilepsy; video EEG (VEEG) has significantly improved its diagnostic value. This study compared healthcare costs and diagnostic-related outcomes associated with outpatient vs inpatient VEEG among patients with epilepsy in the US. Materials and methods: This study used Truven MarketScan Commercial and Medicare Supplemental claims databases. Patients with a VEEG between July 1, 2013 and December 31, 2016 were identified. Index event was the first VEEG claim, which was used to determine inpatient and outpatient cohorts. Continuous health plan enrollment 6 months pre- and 12 months post-index VEEG was required. Primary outcomes were costs during the index event and 12 months post index. A generalized linear model with gamma distribution and a log link was used to estimate adjusted index and post-index costs. Results: Controlling for baseline differences, epilepsy-related cost of index VEEG was significantly lower for the outpatient ($4,098) vs the inpatient cohort ($13,821; p < 0.0001). The cost differences observed at index were maintained in the post-index period. The 12-month post-index epilepsy-related costs were lower in the outpatient cohort ($6,114 vs $12,733, p < 0.0001). Time from physician referral to index VEEG was significantly shorter in the outpatient cohort (30.6 vs 42.5 days). Patients in the inpatient cohort were also more likely to undergo an additional subsequent follow-up inpatient VEEG (p < 0.0001). Limitations: Administrative claims data have limitations, including lack of data on clinical presentation, disease severity, and comprehensive health plan information. Generalizability may be limited to a US insured population of patients who met study criteria. Conclusions: Index VEEG was less costly in an outpatient vs inpatient cohort, and costs were lower during the follow-up period of 12 months, suggesting that outpatient VEEG can be provided to appropriate patients as a less costly option. There were fewer follow-up tests in the outpatient cohort with similar pre- and post-index diagnoses.

Two comparative assessments of intravenous immunoglobulin therapy switching patterns in the treatment of chronic inflammatory demyelinating polyneuropathy in the US.

Purpose: For chronic inflammatory demyelinating polyneuropathy (CIDP) patients, each branded intravenous immunoglobulin (IVIG) treatment differs in production processes, virus elimination, formulation, and composition. Given the limited availability of real-world data comparing IVIGs for CIDP, this study evaluated switching patterns between IVIG products in 2 separate retrospective databases. Patients and methods: Two independent analytic teams retrospectively evaluated IVIG treatment-naïve patients with an ICD diagnosis code for CIDP. Study 1 used integrated healthcare claims from IMS LifeLink PharMetrics Plus™ and Study 2 used the Truven MarketScan® Database. All analyses were descriptive, with outcomes assessed during the 2-year post-index period. Results: One-quarter of IVIG patients switched therapies within the 2-year study period. In both studies, switching rates were lowest for IVIG-G (Gamunex®-C) (Study 1: 9.8%, Study 2: 8.9%), followed by IVIG-F (Flebogamma®) (Study 1: 25.0%, Study 2: 18.2%), and highest for IVIG-other (Octagam®/Gammaplex®) (Study 1: 50.0%, Study 2: 33.3%). When patients were switched, most switched to IVIG-G (Study 1: 51.6%, Study 2: 54.3%). Conclusion: The small proportion of CIDP switchers in 2 independent studies suggests that IVIG therapy is generally well tolerated. However, differences existed in switch rates for different IVIG products. The reason for low switching rates could not be assessed in this study; therefore, further studies are required to detect possible relevant differences in effectiveness and tolerability.

Cost differential of immuno-oncology therapy delivered at community versus hospital clinics.

OBJECTIVES: The site of cancer care delivery has been shown to be associated with the total cost of care. The magnitude of this effect in patients receiving expensive immuno-oncology (I-O) therapies has not been evaluated. We evaluated cost differentials between community-based and hospital-based outpatient clinics among patients receiving I-O therapies. STUDY DESIGN: This was a retrospective analysis utilizing Truven MarketScan Commercial and Supplemental Medicare claims databases. METHODS: Cost data for 3135 patients with non-small cell lung cancer, squamous cell carcinoma of the head and neck, bladder cancer, renal cell carcinoma, or melanoma who received pembrolizumab, nivolumab, and/or ipilimumab between January 1, 2015, and February 14, 2017, were analyzed as cost per patient per month (PPPM). Patients treated within a community setting were matched 2:1 with those treated at a hospital clinic based on cancer type, specific I-O therapy, receipt of radiation therapy, evidence of metastatic disease, gender, age, and evidence of surgery in the preindex period. RESULTS: Mean (SD) total (medical plus pharmacy) PPPM cost was significantly lower for patients treated in a community- versus hospital-based clinic ($22,685 [$16,205] vs $26,343 [$22,832]; P <.001). Lower PPPM medical cost in the community versus hospital setting ($21,382 [$15,667] vs $24,831 [$22,102]; P <.001) was the major driver of this cost differential. Lower total cost was seen regardless of cancer type or I-O therapy administered. CONCLUSIONS: Treatment with I-O therapies in community practice is associated with a lower total cost of care compared with that in hospital-based outpatient practices. With the expanding indications of these agents, future research is needed.

Evaluation of treatment patterns and survival among patients with diffuse large B-cell lymphoma in the USA.

AIM: To evaluate treatment patterns of diffuse large B-cell lymphoma (DLBCL). PATIENTS & METHODS: First-line and relapsed/refractory treatment patterns and survival outcomes following first-line therapy in adult patients newly diagnosed with DLBCL were evaluated. RESULTS: A total of 1436 DLBCL patients initiated treatment and mainly received a combination regimen versus monotherapy (92.1 vs 7.9%). Patients who received monotherapy were older with more comorbidities and had shorter progression-free survival than patients receiving combination therapy (median: 31.3 vs 55.8 months). In the second-line setting (n = 164), rituximab-based combination regimens were most common; 25% underwent stem cell transplantation, and were younger with fewer comorbidities. CONCLUSION: These results illustrate the need for new treatment options for patients unable to tolerate initial combination therapy and transplant-ineligible patients who require salvage therapy.

A retrospective study evaluating treatment patterns and survival outcomes in elderly patients with acute myeloid leukemia treated in the United States with either 7+3 or a hypomethylating agent.

Intensive treatment for newly diagnosed acute myelogenous leukemia (ND-AML) patients are reserved for "fit" patients. While guidelines recommend evaluation of age, performance status and comorbidities, there is no consensus on the definition of "fitness" or optimal therapy for elderly AML patients. This retrospective study evaluated characteristics and survival outcomes of 274 patients (age ≥60 years) with ND-AML treated with 7 + 3 (cytarabine + an anthracycline) vs. hypomethylating agents (HMAs). Most patients received 7 + 3 (60.2%) vs. HMAs (39.8%) in first-line therapy (1 L T); more HMA patients were ≥75 years old and had more comorbidities. Median progression-free survival (PFS) following 1 L T was longer for patients who received 7 + 3 vs. HMAs (6.7 months [95% confidence interval (CI)]: 4.9, 11.1) vs. 4.1 months (95% CI: 2.8, 4.9, respectively). Median overall survival (OS) following 1 L T was also longer for patients who received 7 + 3 vs. HMAs (14.7 months [95% CI: 11.0, not estimated] vs. 4.3 months [95% CI: 3.2, 5.8], respectively). An age-adjusted Charlson Comorbidity Index score of ≥4 vs. < 4 negatively affected PFS and OS irrespective of treatment. Overall, choosing an HMA over 7 + 3 in elderly patients with ND-AML may be influenced by age and comorbidities; patients receiving 7 + 3 had longer survival than those on an HMA.

Treatment Patterns and Survival Outcomes in Patients With Follicular Lymphoma: A 2007 to 2015 Humedica Database Study.

BACKGROUND: Few studies have evaluated real-world treatment patterns and survival in follicular lymphoma (FL). This study evaluated these outcomes among newly diagnosed patients with FL in routine clinical care. PATIENTS AND METHODS: A retrospective study was conducted in newly diagnosed patients with FL from Humedica, a large United States electronic medical record database, from January 1, 2008 to July 31, 2015. Patients were followed from treatment initiation until death, loss to follow-up, or end of study (September 30, 2015). Treatment patterns were assessed in the follow-up period. Progression-free survival (PFS) and overall survival (OS) at 2 years were evaluated in the overall population using Kaplan-Meier analyses. OS was also compared between patients with and without evidence of disease progression within 2 years following first-line therapy (ie, early progressors vs. non-early progressors). RESULTS: A total of 1346 patients were included in the study, with most patients receiving rituximab-based regimens. Fewer early progressors received rituximab-based regimens. Across all lines, combination therapies predominated, particularly bendamustine + rituximab. Following first-line therapy, OS was 86.9% at 2 years, and median OS was not reached. Two-year PFS after first-line therapy was 64.6%, and median PFS was 48.1 months (95% confidence interval, 39.4-58.4 months). OS at 2 years was 76.8% among early progressors versus 90.4% among non-early progressors (P < .001); the median OS was not reached in both groups. CONCLUSION: In routine clinical practice, rituximab-based regimens predominated; however, utilization of these regimens differed among early and non-early progressors. The assessment of survival outcomes also highlights the negative impact of early progression on OS in the rituximab-era.

Real-world treatment patterns and outcomes of patients with small cell lung cancer progressing after 2 lines of therapy.

OBJECTIVES: To evaluate treatment patterns, physician-assessed overall response rate (ORR), progression-free survival (PFS), and overall survival (OS) among third-line (3L)-plus small cell lung cancer (SCLC) patients. MATERIALS AND METHODS: Retrospective analysis of a United States (US)-based community oncology electronic medical record (EMR) database was conducted. Target sample included SCLC patients ≥18 years of age whose disease progressed after at least 2 prior treatments. Treatment patterns captured systemic therapy and best supportive care (BSC) in 3L, fourth-line (4L), and fifth-line (5L) settings. ORR, PFS, and OS were evaluated for each line of systemic therapy and OS was also evaluated for BSC. RESULTS: 334 3L SCLC patients received systemic therapy (n = 249) or BSC (n = 85). Mean age (standard deviation [SD]) was 63.7 (9.5), with 72% having extensive disease at initiation of first-line therapy. Of 3L patients, 41% and 12% went on to 4L and 5L, respectively. ORR for systemic therapy in 3L and 4L averaged around 21% while 5L was 12%. Median PFS in 3L systemic therapy was 2.3 months (95% confidence interval [CI]: 1.9, 2.5), which decreased in 4L and 5L. Median OS for 3L systemic therapy was 4.4 months (95% CI: 4.0, 5.5), with 6- and 12-month survival rates of 37% and 11%, respectively. In contrast, median OS for 3L BSC was 0.9 months (95% CI: 0.6, 1.2), with 9% survival rate at 6 months. CONCLUSION: Current treatments utilized in the 3L-plus setting yield limited survival benefit. Furthermore, patients left untreated and placed on BSC typically live less than 1 month. New therapeutic options are thus needed for these patients, where no approved options exist.

A Theory-Driven Investigation of the Association Between Emotion Dysregulation and Suicide Risk in a Clinical Adolescent Sample.

OBJECTIVE: Emotion dysregulation has been consistently linked to suicide ideation and attempt, but an explanatory model for this relationship has not been adequately investigated in adolescents. This study examined the concurrent relationship among emotion dysregulation, variables from the Interpersonal-Psychological Theory of Suicide (IPTS), and suicide risk (operationalized as a continuous variable that increases in intensity from nonspecific to active suicide ideation to suicide ideation with a plan) in a clinical adolescent sample. METHOD: A total of 151 adolescents (aged 12-17) were recruited from an inpatient psychiatry unit. Cross-sectional analyses were conducted to determine whether the relationship between emotion dysregulation and suicide risk was explained by the variables of perceived burdensomeness (PB), thwarted belongingness, and capability for suicide, as proposed by the IPTS. RESULTS: As hypothesized, the relationship between emotion dysregulation and suicide risk was explained by PB and capability for suicide. Depressive symptoms had an independent relationship with suicide risk after controlling for IPTS variables. CONCLUSIONS: The results from this study suggest that effective treatment strategies that reduce negative cognition tied to PB and depressive symptoms would address the most proximal variables related to suicide risk in adolescents. Enhancing emotion management would serve to maintain low levels of proximal influences on risk.

Transfusion-free interval is associated with improved survival in patients with higher-risk myelodysplastic syndromes engaged in routine care.

Most higher-risk myelodysplastic syndrome (HR-MDS) patients will become transfusion-dependent, leading to potential complications, including infections or end-organ dysfunction. Data correlating achievement of transfusion-free intervals (TFIs) during first-line therapy (1LT) with survival are sparse. We evaluated HR-MDS patients receiving 1LT diagnosed from 1/1/2008 to 7/31/2015 and the impact of a TFI (≥60-day interval without transfusions) on progression-free and overall survival (PFS, OS) using Cox proportional-hazard models. Two hundred and twenty-nine HR-MDS patients received 1LT; overall, median PFS/OS were 8.4 months and 14.7 months, respectively. Two-year PFS/OS were 22.3% and 34.6%, respectively. Median PFS/OS were longer for patients with vs. without a TFI (16.9 vs. 6.1 months and 26.1 vs. 11.8 months, respectively; p < .01 [both]). Two-year PFS (43.0% vs. 3.9%; p < .01) and 2-year OS (51.8% vs. 22.5%; p < .01) were also longer in patients with a TFI vs. not. Achievement of a TFI during 1LT appears to positively affect PFS and OS in HR-MDS patients.

Economic Burden of Patients Treated for Higher-Risk Myelodysplastic Syndromes (HR-MDS) in Routine Clinical Care in the United States.

BACKGROUND AND OBJECTIVE: Significant clinical burden is associated with higher-risk myelodysplastic syndromes (HR-MDS); however, the economic burden has not been fully examined. We examined cost of care and healthcare utilization (HCU) in HR-MDS patients engaged in routine care in the United States (US). METHODS: Adult US patients diagnosed with HR-MDS from 1/1/2008 to 10/31/2015 were identified from the Optum database. Patients were followed until death, progression to acute myeloid leukemia (AML), end of enrollment, or end of study (12/31/2015). Myelodysplastic syndrome (MDS)-related costs/HCU (including medical/pharmacy claims with a primary diagnosis of MDS, MDS-related treatment, or supportive care) and non-MDS-related costs/HCU were evaluated. Costs were calculated as per-patient per-month (PPPM) costs adjusted to 2015 US dollars. RESULTS: Of the 209 HR-MDS patients included, median follow-up was 9.9 months (interquartile range 4.6-17.9), and 69.4% had at least one inpatient admission, 56.9% had at least one emergency department visit, and nearly all patients had at least one outpatient visit. Average PPPM costs over follow-up were $17,361; year 1 versus year 2 costs were higher ($17,337 vs $12,976) following HR-MDS diagnosis. The majority of costs were for MDS-related medical services ($10,327 PPPM). MDS-related medical PPPM costs decreased from $10,557 (year 1) to $6530 (year 2). The main drivers of MDS-related medical costs and the decrease in year 2 were chemotherapy and supportive care costs. CONCLUSIONS: The economic burden of HR-MDS is considerable, particularly within the first year of diagnosis. Treatment/supportive care costs accounted for a significant portion of MDS-related costs. As HR-MDS treatment evolves, the economic impact and HCU need to be further investigated.

Cost Differences Associated With Oncology Care Delivered in a Community Setting Versus a Hospital Setting: A Matched-Claims Analysis of Patients With Breast, Colorectal, and Lung Cancers.

PURPOSE:: Access to high-quality cancer care remains a challenge for many patients. One such barrier is the increasing cost of treatment. With recent shifts in cancer care delivery from community-based to hospital-based clinics, we examined whether this shift could result in increased costs for patients with three common tumor types. METHODS:: Cost data for 6,675 patients with breast, lung, and colorectal cancer were extracted from the IMS LifeLink database and analyzed as cost per patient per month (PPPM). Patients treated within a community setting were matched (2 to 1) with those treated at a hospital clinic on the basis of cancer type, chemotherapy regimen, receipt of radiation therapy, presence of metastatic disease, sex, prior surgery, and geographic region. Approximately 84% of patients were younger than 65 years of age. RESULTS:: Mean total PPPM cost was significantly lower for patients treated in a community- versus hospital-based clinic ($12,548 [standard deviation {SD}, $10,507] v $20,060 [SD, $16,555]; P < .001). The PPPM chemotherapy cost was also significantly lower in the community setting ($4,933 [SD, $4,983] v $8,443 [SD, $10,391]; P < .001). The lower cost observed in community practice was irrespective of chemotherapy regimen and tumor type. CONCLUSION:: We observed significantly increased costs of care for our patient population treated at hospital-based clinics versus those treated at community-based clinics, largely driven by the increased cost of chemotherapy and provider visits in hospital-based clinics. If the site of cancer care delivery continues to shift toward hospital-based clinics, the increased health care spending for payers and patients should be better elucidated and addressed.

Cardiovascular comorbidities in a United States patient population with hemophilia A: A comprehensive chart review.

PURPOSE: Previous retrospective claims database analyses reported increased prevalence and earlier onset of cardiovascular comorbidities in patients with versus without hemophilia A. A comprehensive chart review was designed to further investigate previous findings. METHODS: This retrospective chart review study was conducted at Henry Ford Health System (Detroit, MI, USA). Baseline demographics, bleeding events, treatment parameters, coexisting diseases, hemophilia-associated events, Charlson Comorbidity Index score, and prevalence of 12 cardiovascular risk factors and associated diseases were compared between hemophilia A and control cohorts. P values from a chi-square test for categorical variables and a t test for continuous variables were calculated. Because of small sample sizes (N = 0-90, most <50), statistical differences between cohorts were also assessed using absolute standardized difference. RESULTS: Both groups were well matched by age, race, healthcare payer, and study year. The Charlson Comorbidity Index score was similar between groups. Prevalence of bleeds, hepatitis B and C, and HIV/AIDS was higher in the hemophilia cohort. Hemophilia A severity was severe, moderate, mild, or unknown in 52.7%, 10.8%, 10.8%, and 25.7% of patients, respectively. Prevalence of 12 cardiovascular risk factors and diseases was numerically higher in the control cohort, but differences were statistically significant (P ≤ 0.05) only for diabetes and hyperlipidemia. Meaningful statistical differences using standardized differences were not reached for venous and arterial thrombosis and atrial fibrillation. CONCLUSIONS: This retrospective chart review did not confirm statistically significant differences in cardiovascular comorbidities and their earlier onset in hemophilia A versus controls. Results suggest numerically higher comorbidities in controls.

Economic burden of elderly patients with acute myeloid leukemia treated in routine clinical care in the United States.

This retrospective claims database study examined healthcare utilization (HCU) and costs associated with acute myeloid leukemia (AML) in 237 elderly patients who received chemotherapy or a stem cell transplant (SCT) following AML diagnosis. Patients with secondary AML were excluded. Over the entire follow-up period, 92.0% of patients had ≥1 inpatient admission; 85.7% had ≥1 AML-related admission, and 42.6% had ≥1 non-AML-related admission. During inpatient admissions, 39.2% of patients had ≥1 intensive care unit (ICU) admission, with 20.7% having ≥1 AML-related ICU admission, and 27.8% having ≥1 non-AML-related ICU admission. Total mean per-patient per-month (PPPM) costs over the follow-up period were $25,243 (SD: $21,909), with costs from Year 1 ($27,756 [SD: $22,121]) more than double those in Year 2 ($12,953 [SD: $26,334]) following AML diagnosis. The majority of total costs were medical ($24,512 PPPM [SD: $21,704]), which included inpatient admissions ($6548 PPPM [SD: $10,777]), other outpatient visits ($5021 PPPM [SD: $7997]), supportive care ($3640 PPPM [SD: $5589], and chemotherapy administration ($2029 PPPM [SD: $2345]). Healthcare costs of treated elderly AML patients are substantial, particularly in the first year following diagnosis. Further research is needed to understand factors contributing to high costs in various settings of care for elderly AML patients.

Economic burden of patients with diffuse large B-cell and follicular lymphoma treated in the USA.

AIM: Evaluate healthcare costs and utilization of treated diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma (FL) patients. MATERIALS & METHODS: Adults with newly diagnosed DLBCL and FL between 1 January 2008 and 31 October 2015 were identified in the Optum™ claims database. Healthcare costs and utilization were assessed from diagnosis date until end of follow-up. RESULTS: A total of 1267 DLBCL- and 1595 FL-treated patients were identified. Mean per-patient, per-month cost during follow-up was US$11,890 for DLBCL and US$10,460 for FL. Healthcare costs and utilization decreased from year 1 to 2 following diagnosis, due to a decrease in chemotherapy services, inpatient admissions and other outpatient services. CONCLUSION: The economic burden of treated DLBCL and FL is considerable, especially in the first year following diagnosis.