Clinical and economic burden of medicare beneficiaries with multiple myeloma and renal impairment: An observational study.

Evidence on real-world clinical and economic outcomes in patients with multiple myeloma (MM) and renal impairment (RI) is limited in the United States. This retrospective study aimed to generate an updated comprehensive assessment of the clinical and economic outcomes of MM patients with RI using the Medicare research identifiable files data with Part D linkage, which might assist in assessing the total clinical and socioeconomic burden of these high-risk and challenging-to-treat patients. Treatment patterns and clinical and economic outcomes in first line (1L) to fourth line (4L) therapy were described in Medicare beneficiaries (2012 to 2018) for MM patients with RI (RI MM cohort). For reference purposes, information on a general cohort of MM patients was generated and reported to highlight the clinical and economic burden of RI. Since the goal was to describe the burden of these patients, this study was not designed as a comparison between the 2 cohorts. Compared with the general MM cohort (n = 13,573), RI MM patients (24.9%) presented high MM-associated comorbidities. In the RI MM cohort, bortezomib-dexamethasone (45.7%), bortezomib-lenalidomide (18.6%), lenalidomide (12.3%), and bortezomib-cyclophosphamide (12.1%) were the most prevalent regimens in 1L; carfilzomib and pomalidomide were mostly received in 3L to 4L; and daratumumab in 4L. Across 1L to 4L, the RI MM cohort presented shorter median real-world progression-free survival (1L: 12.9 and 16.4 months) and overall survival (1L: 31.1 and 46.8 months) and higher all-cause healthcare resource utilization (1L incidence rate of inpatient days: 12.1 and 7.8 per person per year) than the general MM cohort. In the RI MM cohort, the mean all-cause total cost increased from 1L to 4L ($14,549-$18,667 per person per month) and was higher than that of the general MM cohort. RI MM patients presented higher clinical and economic burdens across 1L to 4L than the general MM patients in real-world clinical practice.

Real-world clinical outcomes and healthcare costs in patients with Crohn's disease treated with vedolizumab versus ustekinumab in the United States.

OBJECTIVE: To compare real-world treatment persistence, dose escalation, rates of opportunistic or serious infections, and healthcare costs in patients with Crohn's disease (CD) receiving vedolizumab (VDZ) vs ustekinumab (UST) in the United States. METHODS: A retrospective observational study in adults with CD initiated on VDZ or UST on/after 26 September 2016, was performed using the IBM Truven Health MarketScan databases (1 January 2009-30 September 2018). Rates of treatment persistence, dose escalation, opportunistic or serious infection-related encounters, and healthcare costs per patient per month (PPPM) were evaluated. Entropy balancing was used to balance patient characteristics between cohorts. Event rates were assessed using weighted Kaplan-Meier analyses and compared between cohorts using log-rank tests. Healthcare costs were compared between cohorts using weighted 2-part models. RESULTS: 589 VDZ and 599 UST patients were included (172 [29.2%] and 117 [19.5%] were bio-naïve, respectively). After weighting, baseline characteristics were comparable between cohorts. No significant difference in rates of treatment persistence (12-month: VDZ, 76.5%; UST, 82.1%; p = .17), dose escalation (12-month: VDZ, 29.3%; UST, 32.7%; p = .97), or opportunistic or serious infection-related encounters were observed between VDZ and UST. Total mean healthcare costs were significantly lower for patients treated with VDZ vs UST (mean cost difference = -$5051 PPPM; p < .01). Findings were consistent in bio-naïve patients. CONCLUSIONS: In this real-world study, similar treatment persistence, dose escalation, and rates of opportunistic or serious infections were observed with VDZ- and UST-treated patients with CD. However, VDZ was associated with a significantly lower cost outlay for healthcare systems.

Crohn's disease (CD) causes inflammation in the digestive system. Vedolizumab (VDZ) and ustekinumab (UST) are therapies for patients with CD. Little is known about the clinical outcomes and healthcare costs of VDZ versus UST in the real world in the United States. We used health claims data and found that VDZ and UST had comparable real-world clinical outcomes. After 12 months of treatment, the proportions of patients with CD who stayed on treatment and those who needed to increase therapy dose were similar with VDZ and UST. The rate of infection was also similar between the two groups of patients. However, the monthly healthcare costs were $5051 less for patients treated with VDZ than with UST. This was mainly due to the lower cost of VDZ, which was almost half of that of UST. The lower treatment costs with VDZ may provide substantial savings for the healthcare system and patients specifically. Future cost-effectiveness studies on VDZ and UST are needed to aid treatment selection for patients with CD.

Real-World Treatment Patterns and Clinical Outcomes After Platinum-Doublet Chemotherapy and Immunotherapy in Metastatic Non-Small Cell Lung Cancer: A Multiregional Chart Review in the United States, Europe, and Japan.

PURPOSE: To characterize treatment patterns and real-world clinical outcomes of patients with metastatic non-small cell lung cancer (mNSCLC) who developed progression on an anti-PD-1/anti-PD-L1, herein referred to as anti-PD-(L)1, and platinum-doublet chemotherapy. METHODS: Eligible oncologists/pulmonologists in the United States, Europe (France, Germany, and United Kingdom), and Japan completed electronic case report forms for patients with mNSCLC (no evidence of EGFR/ALK/ROS1 alterations). Eligible patients had disease progression on/after an anti-PD-(L)1 and platinum-doublet chemotherapy (received concurrently or sequentially), initiated a subsequent line of therapy (LOT) between 2017 and 2021, and had an Eastern Cooperative Oncology Group (ECOG) performance status 0-2 at this subsequent LOT initiation (index date). Overall survival (OS), time to treatment discontinuation (TTD), and real-world progression-free survival (rwPFS) after index were assessed using Kaplan-Meier analysis. RESULTS: Overall, 160 physicians (academic, 54.4%; community, 45.6%) provided deidentified data from 487 patient charts (United States, 141; Europe, 218; Japan, 128; at mNSCLC diagnosis: median age 66 years, 64.7% male, 81.3% nonsquamous, 86.2% de novo mNSCLC; at line of interest initiation: 86.0% ECOG 0-1, 39.6% liver metastases, 18.9% brain metastases, 79.1% smoking history). The most common treatment regimens upon progression after anti-PD-(L)1/platinum-doublet chemotherapy were nonplatinum chemotherapy (50.5%), nonplatinum chemotherapy plus vascular endothelial growth factor receptor inhibitor (12.9%), and platinum-doublet chemotherapy (6.6%). Median OS was 8.8 months (squamous, 7.8 months; nonsquamous, 9.5 months). Median TTD was 4.3 months (squamous, 4.1 months; nonsquamous, 4.3 months). Median rwPFS was 5.1 months (squamous, 4.6 months; nonsquamous, 5.4 months). CONCLUSION: In this multiregional, real-world analysis of pooled patient chart data, patients with mNSCLC who had disease progression after anti-PD-(L)1/platinum-doublet chemotherapy had poor clinical outcomes with various treatment regimens, demonstrating an unmet clinical need for effective options after failure on anti-PD-(L)1 and platinum-doublet chemotherapy treatments.

Delayed treatment initiation of oral anticoagulants among Medicare patients with atrial fibrillation.

Study objective: This study aimed to identify factors associated with delayed oral anticoagulant (OAC) treatment initiation among atrial fibrillation (AF) patients in United States (US) clinical practice. Participants: Medicare beneficiaries newly diagnosed with AF without moderate-to-severe mitral stenosis or a mechanical heart valve, were aged ≥65 years and prescribed OAC on or after 10/1/2015 through 2019 were included. Delayed and early OAC initiation were defined as >3 months and 0-3 months initiation from first AF diagnosis, respectively. Main outcome measures: Association between delayed OAC initiation and patient demographics, clinical and index OAC coverage and formulary characteristics was examined using multivariable logistic regression. Results: A total of 446,441 patients met the inclusion criteria; 30.0 % (N = 131,969) were identified as delayed and 70.0 % (N = 314,472) as early OAC initiation. Median age for both cohorts was 78 years. In the early and delayed OAC cohorts, 47.1 % and 47.6 % were male and 88.8 % and 86.6 %, were White, respectively. Factors associated with delayed OAC initiation (odds ratio; 95 % confidence interval) included Black race (1.29; 1.25 to 1.33), west region (1.29; 1.26 to 1.32), comorbidities such as dementia (1.27; 1.23 to 1.30), recent bleeding hospitalization (1.22; 1.18 to 1.27), prior authorization (1.69; 1.66 to 1.71), tier 4 formulary for index OAC at AF diagnosis (1.26; 1.22 to 1.30). Conclusion: Our study revealed that nearly one-third of Medicare patients with AF experienced delayed OAC initiation. Key patient characteristics found to be associated with delayed OAC initiation included race and ethnicity, comorbidities, and formulary restrictions.

Disease Burden, Treatment Patterns, and Economic Impact of Rectovaginal Fistulas in Patients with Crohn's Disease: Findings from a Retrospective, Observational, Longitudinal Study Based on US Claims Databases.

BACKGROUND: Crohn's-related rectovaginal fistulas (RVF) greatly impact quality of life and are notoriously difficult to treat. The aim of this study was to assess the burden of recurrent episodes of care for RVF and its economic impact. METHODS: A retrospective observational cohort study of administrative US claims databases was conducted. Eligible patients were female adults, with a diagnosis code for Crohn's disease with or without a diagnosis/procedural code for RVF. For the RVF cohort, rates of recurrence of RVF episodes of care were estimated using Kaplan-Meier analyses. Healthcare resource utilization (HCRU) and direct healthcare costs were compared between the RVF cohort and RVF-free cohort. RESULTS: Mean ages in the RVF cohort (n = 963) and RVF-free cohort (n = 56,564) were 47.2 and 50.8 years, with a mean follow-up period of 58.7 and 49.8 months, respectively. For the RVF cohort, the probability of having a second RVF episode of care within 2 years of the first one was estimated to be 35.9% and of having a third episode within 2 years of the second was 47.8%. During the first 2 years, the RVF cohort had 67% more inpatient admissions than the RVF-free cohort with each RVF episode of care being associated with 16% more admissions. The estimated incremental cost associated with having RVF was US$17,561, with an incremental cost of US$11,607 for each additional RVF episode of care. CONCLUSIONS: This real-world study highlights the significant impact of RVF in patients with Crohn's disease with regard to repeat interventions and associated HCRU and direct healthcare costs, suggesting novel therapeutics are needed in this patient population.

Real-world upper endoscopy utilization patterns among patients with gastroesophageal reflux disease, Barrett esophagus, and Barrett esophagus-related esophageal neoplasia in the United States.

This study fills a gap in literature by providing contemporary real-world evidence on the prevalence of patients with gastroesophageal reflux disease (GERD), Barrett esophagus (BE), and Barrett esophagus-related neoplasia (BERN) and their upper endoscopy utilization patterns in the United States. A retrospective cohort study design was used: adults with GERD, nondysplastic Barrett esophagus (NDBE), and BERN (indefinite for dysplasia [IND], low-grade dysplasia [LGD], high-grade dysplasia [HGD], or esophageal adenocarcinoma [EAC]) were identified from the MarketScan databases (January 01, 2015-December 31, 2019). For each disease stage, prevalence of adults in commercial claims by calendar year, annual number of upper endoscopies per patient and time between upper endoscopies were reported. In 2019, in commercial claims (N = 12,363,227), the annual prevalence rate of GERD was 13.7% and 0.70% for BE/BERN, among which, 87.1% had NDBE, 6.8% had IND, 2.3% had LGD, 1.0% had HGD, and 2.8% had EAC. From 2015-2019, the study included 3,310,385 patients with GERD, 172,481 with NDBE, 11,516 with IND, 4332 with LGD, 1549 with HGD, and 11,676 with EAC. Annual mean number of upper endoscopies was 0.20 per patient for GERD, 0.37 per patient for NDBE, 0.43 for IND, 0.58 for LGD, and 0.87 for HGD. Median time (months) to second upper endoscopy was 38.10 for NDBE, 36.63 for IND, 22.63 for LGD, and 11.90 for HGD. Upper endoscopy utilization increased from GERD to BE to BERN, and time between upper endoscopies decreased as the disease stage progressed from BE to BERN, with less frequent utilization in BERN than what would be expected from guideline recommendations for surveillance.

Healthcare Resource Utilization and Costs Among Patients With Gastroesophageal Reflux Disease, Barrett's Esophagus, and Barrett's Esophagus-Related Neoplasia in the United States.

Background: Gastroesophageal reflux disease (GERD) is a risk factor for Barrett's esophagus (BE) and BE-related neoplasia (BERN). Objectives: This study aimed to evaluate healthcare resource utilization (HRU) and costs associated with GERD, BE, and BERN in the United States. Methods: Adult patients with GERD, nondysplastic BE (NDBE), and BERN (including indefinite for dysplasia [IND], low-grade dysplasia [LGD], high-grade dysplasia [HGD] or esophageal adenocarcinoma [EAC]), were identified from a large US administrative claims database, the IBM Truven Health MarketScan® databases (Q1/2015-Q4/2019). Patients were categorized into the corresponding mutually exclusive EAC-risk/diagnosis cohorts based on the most advanced stage from GERD to EAC using diagnosis codes in medical claims. Disease-related HRU and costs (2020 USD) were calculated for each cohort. Results: Patients were categorized into the following EAC-risk/diagnosis cohorts: 3 310 385 into GERD, 172 481 into NDBE, 11 516 into IND, 4332 into LGD, 1549 into HGD, and 11 676 into EAC. Disease-related annual mean number of inpatient admissions, office visits, and emergency department visits by cohort were 0.09, 1.45, and 0.19 for GERD; 0.08, 1.55, and 0.10 for NDBE; 0.10, 1.92, and 0.13 for IND; 0.09, 2.05, and 0.10 for LGD; 0.12, 2.16, and 0.14 for HGD; and 1.43, 6.27, and 0.87 for EAC. Disease-related annual mean total healthcare costs by cohort were $6955 for GERD, $8755 for NDBE, $9675 for IND, $12 241 for LGD, $24 239 for HGD, and $146 319 for EAC. Discussion: Patients with GERD, BE, and BERN had important HRU and costs, including inpatient admissions and office visits. As patients progressed to more advanced stages, there was substantially higher disease-related resource utilization, with associated costs being 16 times higher in patients with EAC than those with NDBE. Conclusions: Findings suggest the need for early identification of high-risk individuals prior to progression to EAC to potentially improve clinical and economic outcomes in this population.

Real-world experience of pediatric patients treated with peanut (Arachis hypogaea) allergen powder-dnfp.

BACKGROUND: Peanut (Arachis hypogaea) allergen powder-dnfp (PTAH) is the first oral immunotherapy indicated for children aged 4 to 17 years with peanut allergy. There are limited real-world data on patients treated with PTAH. OBJECTIVE: To characterize pediatric patients treated with PTAH and associated treatment patterns in US clinical practice. METHODS: US-based physicians with allergy and immunology training treating patients with peanut allergy aged 4 to 17 years with PTAH were recruited from an existing physician panel and completed an online case report form (October to December 2021) with data abstracted from patient medical charts. Physician practice circumstances, patient characteristics, and PTAH treatment patterns were reported. Time to reach the 300-mg dose and treatment persistence were assessed using Kaplan-Meier analysis. RESULTS: A geographically balanced sample of 43 physicians contributed data for 118 demographically diverse pediatric patients. Patients had heterogeneous diagnostic test results, with a wide range of peanut-specific immunoglobulin E levels; 6.8% received an oral food challenge. During the updosing phase, there were no temporary interruptions and 5.1% of the patients required downdosing. Patients reached the 300-mg dose at a median of 21.3 weeks post-initiation. The rate of PTAH persistence at 24 weeks was 93.4%. Only 1 patient discontinued treatment because of treatment-related systemic allergic symptoms, and the remaining discontinuations were for reasons other than treatment-related symptoms. Prophylactic antihistamines were used by 33.9% of the patients to prevent PTAH adverse effects. CONCLUSION: PTAH was prescribed in demographically diverse patients with a wide range of peanut-specific immunoglobulin E levels. Treatment persistence with PTAH was high in this study population, with a small number of patients experiencing treatment modification.

Chronic Myeloid Leukemia: Part II-Cost of Care Among Patients in Advanced Phases or Later Lines of Therapy in Chronic Phase in the United States from a Commercial Perspective.

Background: Tyrosine kinase inhibitors (TKIs) are the standard-of-care treatment for chronic myeloid leukemia in chronic phase (CML-CP). Despite advances in therapy, there remains a proportion of patients with CML-CP that are refractory/intolerant to TKIs, and these patients cycle through multiple lines of therapy. Moreover, even with TKIs, some patients progress to accelerated phase/blast crisis (AP/BC), which is associated with particularly poor clinical outcomes. Objectives: To describe real-world treatment patterns, healthcare resource utilization (HRU), and costs of patients with CML-CP reaching later lines of therapy or progressing to AP/BC in the United States. Methods: Adult CML patients from administrative claims data (January 1, 2000-June 30, 2019) were classified by health state: on third-line (CML-CP On Treatment), on fourth or later lines (CML-CP Post-Discontinuation), or progressed to AP/BC (CML-AP/BC). Outcomes were assessed by health state. Results: There were 296 (4620 patient-months), 83 (1644 patient-months), and 949 (25 593 patient-months) patients classified in the CML-CP On Treatment, CML-CP Post-Discontinuation, and CML-AP/BC cohorts, respectively. Second-generation TKIs (nilotinib, dasatinib, and bosutinib) were most commonly used in the CML-CP On Treatment (69.1% of patient-months) and CML-CP Post-Discontinuation cohorts (59.1% of patient-months). Three-month outpatient incidence rates (IRs) were 7.6, 8.3, and 7.0 visits in the CML-CP On Treatment, CML-CP Post-Discontinuation, and CML-AP/BC cohort, respectively, with mean costs of $597 per service. Three-month inpatient IRs were 0.6, 0.7, and 1.4 days in the CML-CP On Treatment, CML-CP Post-Discontinuation, and CML-AP/BC cohort, respectively, with mean costs of $5892 per day. Mean hematopoietic stem cell transplantation cost was $352 333; mean 3-month terminal care cost was $107 013. Discussion: Cost of CML care is substantial among patients with CML reaching third-line, fourth or later lines, or progressing to AP/BC, suggesting that the disease is associated with a significant economic and clinical burden. From third-line to fourth or later lines, HRU was observed to increase, and the incidence of inpatient days was particularly high for those who progressed to AP/BC. Conclusion: In this study, patients with CML cycling through TKIs in later lines of therapy or progressing to AP/BC experienced substantial HRU and costs, suggesting unmet treatment needs.

Chronic Myeloid Leukemia: Part I-Real-World Treatment Patterns, Healthcare Resource Utilization, and Associated Costs in Later Lines of Therapy in the United States.

Background: Despite advances in tyrosine kinase inhibitor (TKI) therapy for chronic myeloid leukemia in chronic phase (CML-CP), a sizeable proportion of patients with CML-CP remains refractory or intolerant to these agents. Objectives: Treatment patterns, healthcare resource utilization (HRU), and costs were evaluated among patients with CML who received third or later lines of therapy (3L+), a clinical population that has not been previously well-studied, with unmet treatment needs as TKI therapy has repeatedly failed. Methods: Adult patients with CML who received 3L+ were identified in the IBM® MarketScan® Databases (January 1, 2001-June 30, 2019) and the SEER-Medicare-linked database (January 1, 2006-December 31, 2016). Treatment patterns were observed from CML diagnosis. HRU and direct healthcare costs (payer's perspective, 2019 USD) were measured in a 3L+ setting. Results: Among 296 commercially insured patients with 3L+ (median age, 58.5 years; female, 49.7%), the median duration of first-line (1L), second-line (2L), and 3L therapy was 8.5, 4.2, and 8.3 months, respectively. The annual incidence rate during 3L+ was 3.4 for inpatient days, 30.8 for days with outpatient services, and 1.2 for emergency department visits. Mean per-patient-per-month (PPPM) total healthcare costs (pharmacy + medical costs) were $18 784 in 3L+, $15 206 in 3L, and $19 546 in 4L, with inpatient costs driving most of the difference between 3L and 4L (mean [3L] = $2528 PPPM, mean [4L] = $6847 PPPM). Among 53 Medicare-insured patients with 3L+ (median age, 72.0 years; female, 39.6%), the median duration of 1L, 2L, and 3L therapy was 9.7, 5.0, and 7.0 months, respectively. During 3L+, the annual incidence rate was 10.3 for inpatient days, 61.9 for days with outpatient services, and 1.5 for emergency department visits. Mean PPPM total healthcare costs were $14 311 in 3L+, $15 100 in 3L, and $16 062 in 4L. Discussion: Patients with CML receiving 3L+ rapidly cycled through multiple lines. Costs increased from 3L to 4L; in commercially insured patients, inpatient costs were responsible for most of the cost increase between 3L and 4L, underlying these patients' continued need for care. Conclusions: These findings support the need for better treatment options in patients with CML undergoing later lines of therapy.

Health-care resource utilization associated with peanut allergy management under allergen avoidance among commercially insured individuals.

Background: Until recently, the standard approach to care for individuals with peanut allergy (PA) was limited to allergen avoidance and treatment of reactions with emergency medicines. Objectives: To assess health-care resource utilization (HRU) and costs associated with PA management under allergen avoidance and to identify risk factors associated with peanut reactions that resulted in inpatient (IP) and/or emergency department (ED) visits. Methods: Privately insured individuals with PA diagnosis codes were identified from a large U.S. administrative claims data base (January 1, 1999, to March 31, 2017). PA-related HRU, indicated by a PA diagnosis and/or diagnostic procedure codes and by epinephrine autoinjectors (EAI) prescription fills in medical and pharmacy claims, respectively, and all-cause costs were described per patient-year (PPY). Risk factors associated with peanut reactions in an IP and/or ED setting were identified by using a multivariable logistic regression model. Results: A total of 86,483 patient-years from 14,136 individuals with PA were included. At the patient-year level, 28.1% were ages 0-3 years, 43.6% were ages 4-11 years, 13.7% were ages 12-17 years, and 14.5% were ages ≥ 18 years; 35.6% had PA-related outpatient visits; 50.6% had EAI fills; and 2.4% had PA-related IP and/or ED visits PPY. Younger individuals had more PA-related outpatient visits and EAI fills, with peak intensive use at ages 4-11 years. The proportion of individuals with PA-related IP and/or ED visits was highest among those aged ≥ 18 years. Mean all-cause costs were $3084 PPY; individuals with PA-related IP and/or ED visits incurred $8902 PPY ($17,451 for those with one or more IP visits). Risk factors associated with peanut reactions that resulted in IP and/or ED visits included young adults (odds ratio [OR] 3.19 [95% confidence interval {CI}, 2.66-3.83]), previous peanut reaction(s) (OR 1.66 [95% CI, 1.23-2.24]), asthma (OR 1.33 [95% CI, 1.18-1.51]), and male sex (OR 1.14 [95% CI, 1.01-1.28]). Conclusion: Individuals with PA and under allergen avoidance had significant HRU that varied across all age groups, with more PA-related outpatient visits during preschool and/or school age and PA-related urgent care among adults. Individuals with previous peanut reaction(s), asthma, and males had a higher risk of peanut reactions that resulted in IP and/or ED visits.

Tyrosine kinase inhibitor therapy discontinuation in patients with chronic myeloid leukemia in chronic phase in the United States after clinical practice guideline updates.

A physician survey (July 2019-August 2019) and a retrospective patient medical chart review (November 2019-December 2019) were conducted to assess TKI therapy discontinuation practice in patients with Ph + CML-CP in the US after the publication of practice guidelines updated with recommendations for TKI discontinuation. After guideline updates, 90% of physicians from the survey reported attempting TKI discontinuation and 24% of their patients discontinued TKI after achieving an adequate response. Although TKI therapy discontinuation practice is increasing, particularly in community-based practice, a little more than half of physicians were aware of these updated guidelines resulting in TKI discontinuation attempted under suboptimal conditions, mainly limited to first-line TKI therapy, with more than half of physicians without access to at least MR4.5 sensitivity level of detection monitoring. Stricter response criteria per guideline recommendations were observed to relate to lower relapse rates following TKI discontinuation, emphasizing the importance of communicating these recommendations and access to adequate monitoring tools.

Economic burden of peanut allergy in pediatric patients with evidence of reactions to peanuts in the United States.

BACKGROUND: The economic burden of food allergy is large; however, costs specific to individuals with peanut allergy experiencing reactions to peanuts remain to be evaluated. As the prevalence of peanut allergy continues to increase in children, a better understanding of the cost of care is warranted. OBJECTIVE: To assess the cost of care of peanut allergy among privately insured and Medicaid-insured pediatric patients in the United States. METHODS: This retrospective matched-cohort study included patients aged 4-17 years from the Optum Health Care Solutions and Medicaid Claims databases (January 1, 2007-March 31, 2017). Patients were classified into 2 cohorts: peanut allergy (with peanut allergy diagnosis codes and reactions triggering health care resource utilization [HRU]) and peanut allergy-free (no peanut allergy diagnosis codes in claims). Peanut allergy patients were matched 1:10 to peanut allergy-free patients based on baseline covariates. Comorbidities including anxiety and depression, HRU, and direct health care costs were compared between cohorts and reported for both perspectives separately. RESULTS: Compared with peanut allergy-free patients (n = 30,840 privately insured; n = 12,450 Medicaid), peanut allergy patients (n = 3,084 privately insured; n = 1,245 Medicaid) had higher prevalence of asthma, atopic dermatitis/eczema, other food allergies, allergic rhinitis, depression, and anxiety (all P < 0.01). Peanut allergy patients had higher HRU per patient per year (PPPY), including 90% more emergency department visits among both privately insured and Medicaid patients (P < 0.01) and higher direct health care costs PPPY, with incremental costs of $2,247 total or $1,712 excluding asthma-related costs for privately insured patients and $2,845 total or $1,844 excluding asthma-related costs for Medicaid patients (all P < 0.01). CONCLUSIONS: Pediatric patients in the United States with peanut allergy and reactions triggering HRU had significantly higher comorbidity burdens, HRU, and direct health care costs, regardless of asthma-related costs, versus those without peanut allergy. DISCLOSURES: This study was funded by Aimmune Therapeutics, a Nestlé Health Science company. The study sponsor was involved in several aspects of the research including the study design, the interpretation of data, the writing of the manuscript, and the decision to submit the manuscript for publication. Yu and Tilles are employees of Aimmune Therapeutics, a Nestlé Health Science company. Robison and Norrett were employees of Aimmune Therapeutics at the time this study was conducted. Blaiss, Meadows, and Hass provided paid consulting services to Aimmune Therapeutics. Guerin and Latremouille-Viau are employees of Analysis Group, a consulting company that provided paid consulting services to Aimmune Therapeutics. Parts of the results were presented at the AMCP Managed Care & Specialty Pharmacy Annual Meeting held March 25-28, 2019, in San Diego, CA, and at the ISPOR Annual Meeting held May 18-22, 2019, in New Orleans, LA.

Treatment patterns and outcomes in patients with myelodysplastic syndromes treated with hypomethylating agents: a SEER-Medicare analysis.

To describe real-world treatment patterns and outcomes among adult patients with myelodysplastic syndromes (MDS) treated with hypomethylating agents (HMA), patients were identified in the SEER-Medicare database (01/2006-12/2016); 3,046 patients with MDS treated with HMA were included. An algorithm was developed to categorize patients into MDS risk groups: the majority of patients were classified as Higher-risk (70.9%), 8.0% as Intermediate-risk, and 21.1% as Unknown-risk. Overall, 77.4% of patients initiated azacitidine and 22.6% decitabine; they received an average of 5.1 index-HMA cycles, of which 90.9% were complete with a median cycle duration of 28 days. Median survival was 11.6, 18.4, and 19.1 months for the Higher-risk, Intermediate-risk, and Unknown-risk groups, respectively. Median time-to-AML transformation was 19.3 months for the Higher-risk group and 50.4 months for the Intermediate-risk group (not reached for Unknown-risk). Data highlight the unmet medical needs of patients with MDS treated with HMA, particularly for the Higher-risk MDS group.

Healthcare resource utilization and costs in patients with myelodysplastic syndromes treated with hypomethylating agents: a SEER-Medicare analysis.

AIMS: To describe healthcare resource utilization (HRU) and costs in patients with myelodysplastic syndromes (MDS) treated with hypomethylating agents (HMA) based on HMA-treatment response. MATERIALS AND METHODS: SEER-Medicare data (January 2006-December 2016) were used to identify adults diagnosed with MDS (SEER: January 2009-December 2015) initiated on HMA (index date). HMA-treatment success (indicators: ≥7 HMA cycles, stem cell transplantation, and transfusion independence) or failure (indicators: acute myeloid leukemia [AML], AML-like treatment, and death) was determined using a claim-based algorithm. HRU and costs were assessed from the index date to 1-year post-index, overall and stratified by HMA-treatment success or failure. Among patients with HMA-treatment failure, HRU and costs were also assessed from failure to 1-year post-failure. RESULTS: The study included 3,046 patients (mean age: 77.4 years; females: 36.8%). Rates of HMA-treatment success and failure were 44.4% and 76.2%, respectively (20.6% had HMA-treatment success then failure). Overall, patients had 15.2 inpatient admissions per-100-patients-per-month (median follow-up: 5.9 months). Patients with HMA-treatment success had 7.5 inpatient admissions per-100-patients-per-month (median follow-up: 12.0 months), while those with HMA-treatment failure had 20.4 and 35.3 admissions per-100-patients-per-month pre- and post-HMA-treatment failure, respectively (median follow-up: 4.3 and 1.8 months, pre- and post-HMA-treatment failure, respectively). Mean total healthcare costs were $12,494 per-patient-per-month overall, $8,069 per-patient-per-month among patients with HMA-treatment success, and $13,809 and $19,242 per-patient-per-month pre- and post-HMA-treatment failure, respectively. Outpatient costs (68.3%) were the main contributor of total healthcare costs overall, while inpatient costs (80.3%) were the main cost driver post-HMA-treatment failure. LIMITATIONS: Without available laboratory test results, clinical indicators observed in claims were used to assess HMA-treatment response. CONCLUSIONS: Over 75% of patients with MDS failed HMA-treatment within 6 months of initiation and were observed with more inpatient admissions than those with HMA-treatment success, translating into substantially higher healthcare costs. HMA-treatment failure results in an important economic burden in MDS patients.

Vaso-occlusive crises and costs of sickle cell disease in patients with commercial, Medicaid, and Medicare insurance - the perspective of private and public payers.

AIM: To characterize vaso-occlusive crises (VOCs) and describe healthcare costs among commercially-insured, Medicaid-insured, and Medicare-insured patients with sickle cell disease (SCD). MATERIALS AND METHODS: The IBM Truven Health MarketScan Commercial (2000-2018), Medicaid Analytic eXtract (2008-2014), and Medicare Research Identifiable Files (2012-2016) databases were used to identify patients with ≥2 SCD diagnoses. Study measures were evaluated during a 12-month follow-up period, stratified by annual number of VOCs (i.e. 0, 1, and ≥2). RESULTS: Among 16,092 commercially-insured patients (mean age = 36.7 years), 35.3% had 1+ VOCs. Mean annual total all-cause healthcare costs were $15,747, $27,194, and $64,555 for patients with 0, 1, and 2+ VOCs, respectively. Total all-cause healthcare costs were mainly driven by inpatient (0 VOC = 31.0%, 1 VOC = 53.1%, 2+ VOCs = 65.4%) and SCD-related costs (0 VOC = 56.4%, 1 VOC = 78.4%, 2+ VOCs = 93.9%). Among 18,287 Medicaid-insured patients (mean age = 28.5 years, fee-for-service = 50.2%), 63.9% had 1+ VOCs. Mean annual total all-cause healthcare costs were $16,750, $29,880, and $64,566 for patients with 0, 1, and 2+ VOCs, respectively. Inpatient costs (0 VOC = 37.2%, 1 VOC = 64.3%, 2+ VOCs = 72.9%) and SCD-related costs (0 VOC = 60.9%, 1 VOC = 73.8%, 2+ VOCs = 92.2%) accounted for a significant proportion of total all-cause healthcare costs. Among 15,431 Medicare-insured patients (mean age = 48.2 years), 55.1% had 1+ VOCs. Mean annual total all-cause healthcare costs were $21,877, $29,250, and $58,308 for patients with 0, 1, and ≥2 VOCs, respectively. Total all-cause healthcare costs were mainly driven by inpatient (0 VOC = 47.9%, 1 VOC = 54.9%, 2+ VOCs = 67.5%) and SCD-related costs (0 VOC = 74.9%, 1 VOC = 84.4%, 2+ VOCs = 95.3%). LIMITATIONS: VOCs managed at home were not captured. Analyses were descriptive in an observational setting; thus, no causal relationships can be inferred. CONCLUSIONS: A high proportion of patients experienced VOCs across payers. Furthermore, inpatient and SCD-related costs accounted for a significant proportion of total all-cause healthcare costs, which increased with VOC frequency.

Characteristics of Patients with Psoriatic Arthritis Receiving Secukinumab and Reasons for Initiation: A US Retrospective Medical Chart Review.

INTRODUCTION: Secukinumab is a fully human anti-interleukin 17A monoclonal antibody approved for the treatment of psoriatic arthritis (PsA) in the United States. Few studies have investigated prescribing patterns among rheumatologists who have initiated secukinumab for the treatment of patients with PsA in real-world settings. This US medical chart review describes clinical and treatment characteristics of patients with psoriatic arthritis (PsA) who were prescribed secukinumab and rheumatologist-reported reasons for prescribing secukinumab in clinical practice. METHODS: This US medical chart review included patients with physician-diagnosed PsA aged ≥ 18 years initiating secukinumab after January 15, 2016. Eligible rheumatologists used online forms to collect patient demographics, disease characteristics, comorbidity profiles, and treatment histories before or on the date of the first secukinumab prescription recorded in the medical chart. Information on reasons for secukinumab prescription and dosing was also collected. RESULTS: Medical charts from 153 patients with PsA who initiated secukinumab were reviewed by 46 rheumatologists between July 7, 2017, and August 11, 2017. Overall, 53.6% of patients were male, mean (standard deviation) age was 47.3 (11.5) years, and 24.8% were biologic naive. The most common reasons for secukinumab prescription among biologic-naive and biologic-experienced patients, respectively, were efficacy/effectiveness of secukinumab (84.2%) and failure of other prior biologics (80.9%). Nearly all patients (94.1%) received a loading regimen, including 150 mg every week (32.7%) and 300 mg every week (61.4%). Overall, 145 patients (94.8%) received ≥ 1 maintenance dose, of whom 49.7% received 150 mg every 4 weeks and 50.3% received 300 mg every 4 weeks. CONCLUSIONS: At the time of the chart review, most patients with PsA who initiated secukinumab were biologic experienced, although one-quarter received secukinumab as first-line biologic therapy. Efficacy/effectiveness of secukinumab and failure of other biologics were the most common reasons for initiating secukinumab. FUNDING: Novartis Pharmaceuticals Corporation, East Hanover, NJ. Plain language summary available for this article.

Characterization of Patients with Ankylosing Spondylitis Receiving Secukinumab and Reasons for Initiating Treatment: A US Physician Survey and Retrospective Medical Chart Review.

OBJECTIVES: To characterize US patients with ankylosing spondylitis (AS) who were treated with secukinumab and to assess rheumatologist-reported reasons for prescribing treatment in clinical practice. METHODS: This descriptive analysis of data from a US retrospective medical chart review included patients aged ≥ 18 years diagnosed with AS who initiated secukinumab after 15 January 2016. Eligible rheumatologists used online forms to collect patient demographics, disease characteristics, co-morbidity profile, and treatment history prior to or on the index date, defined as the date of the first secukinumab prescription recorded in the medical chart. Information on physician-level characteristics and reasons for secukinumab prescription and dosing were also collected. RESULTS: Medical charts from 78 patients with AS who initiated secukinumab were reviewed by 25 rheumatologists between 7 July 2017 and 11 August 2017. Overall, 76.9% of patients were male, mean (SD) age was 39.8 (10.8) years, and 34.6% were biologic naïve. The most common reasons for secukinumab initiation among biologic-naïve and biologic-experienced patients, respectively, were efficacy/effectiveness (77.8%) and failure of other prior biologics (84.3%). Nearly all patients (94.9%) received a loading dose, including 150 mg every week (39.7%), 300 mg every week (53.8%), and other (1.3%). Overall, 73 patients (93.6%) received ≥ 1 maintenance secukinumab dose, of whom 56.2% and 43.8% received 150 mg and 300 mg, respectively, every 4 weeks. CONCLUSIONS: In this US medical chart review of patients with AS who initiated secukinumab, approximately one-third were biologic naïve, and secukinumab efficacy/effectiveness and failure of other biologics were the most common reasons for initiating secukinumab.

Tyrosine kinase inhibitor therapy treatment and discontinuation in patients with chronic myeloid leukemia in chronic phase in the United States: a clinical practice perspective.

Tyrosine kinase inhibitor (TKI) therapy discontinuation practice in patients with chronic myeloid leukemia chronic phase (CML-CP) was assessed in real-world practice prior to the release of recommendations on discontinuation. Data were collected from US oncologists/hematologists (through web-based physician survey and patient chart review) on TKI therapy discontinuation practice including monitoring, adequate response for discontinuation, relapse, and symptoms following discontinuation. From the physician survey, 34% of oncologists/hematologists attempted discontinuation, with two-thirds doing so outside of a trial. From the chart review, TKI therapy was discontinued in 3.4% of patients after they achieved an adequate response with the intention to remain CML-therapy-free until disease relapse. Among these patients, 21% relapsed and 17% had symptoms following discontinuation. There was a lack of consensus on the definition of adequate response suggesting that discontinuation was attempted without clear guidelines and under suboptimal conditions underscoring the need for physician education regarding guidelines for TKI therapy discontinuation.

Burden of Infections Among Chronic Myeloid Leukemia Patients Receiving Dasatinib or Nilotinib: A Real-World Retrospective Healthcare Claims Study in the United States.

INTRODUCTION: Tyrosine kinase inhibitors (TKI) have been demonstrated to prolong survival in patients with chronic myeloid leukemia (CML). However, TKIs may be associated with an increased risk of infections. This study compared healthcare resource utilization (HRU) and costs among patients with CML receiving dasatinib or nilotinib, with a focus on infection-related economic outcomes. METHODS: Two large administrative databases were used to identify adult patients newly diagnosed with CML who initiated dasatinib or nilotinib as first- (1L) or second-line (2L) therapy and were classified into the following 1L (dasatinib 1L/nilotinib 1L cohorts) or 2L (dasatinib 2L/nilotinib 2L) cohorts based on the initiated 1L/2L TKI therapy. Infection-related HRU and healthcare costs were compared between cohorts, separately for 1L and 2L. RESULTS: Cohorts included 1156 patients in the dasatinib 1L and 677 patients in the nilotinib 1L cohorts, 322 patients in the dasatinib 2L, and 207 in the nilotinib 2L cohorts. In 1L and 2L, infection-related HRU was higher for dasatinib than nilotinib cohorts. Infection-related inpatient (IP) days constituted a larger proportion of all-cause IP days in the 1L/2L dasatinib than 1L/2L nilotinib cohorts (dasatinib 1L/2L: 53%/58%; nilotinib 1L/2L: 50%/46%). Compared to the nilotinib cohort, the dasatinib cohort had higher all-cause total costs per patient per year by US$17,901 in 1L and $28,625 in 2L. Of the total cost difference, infection-related were $6048 (34%) in 1L and $28,192 (99%) in 2L, largely driven by IP cost differences (1L/2L: 96%/98%). CONCLUSIONS: Dasatinib was associated with higher HRU and healthcare costs compared to nilotinib, particularly related to infections. FUNDING: Novartis Pharmaceutical Corporation.