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This study aimed to examine the reasons underlying treatment changes among pediatric patients with attention-deficit/hyperactivity disorder (ADHD).Data were obtained through online medical chart abstraction (08/2021-09/2021). Eligible patients with ADHD had initiated a treatment regimen at ages 6-17 and within 1-5 years of chart abstraction. Reasons contributing to treatment changes were analyzed for a randomly selected treatment episode. ADHD/treatment-related complication rate was also described. Results were reported overall and among children (ages 6-12) and adolescents (ages 13-17), separately. Physicians' perspective on adherence among their child, adolescent, and adult patients was assessed through an online survey.A total of 156 physicians abstracted 434 patient charts (235 children + 199 adolescents). Mean patient age was 11.3 years, and 68.7% were male. Inadequate/suboptimal symptom management was the most common reason for treatment discontinuation (50/83 [60.2%]), add-on (17/21 [81.0%]), and dose increase (189/237 [79.7%]). Patient/parent/family attitude/dislike of medication and ADHD/treatment-related complications were common reasons for treatment discontinuation, add-on, switch, and dose decrease. Overall, 42.4% of patients had ≥ 1 documented ADHD/treatment-related complication, insomnia/sleep disturbances being the most common (9.7%). Among patients with ≥ 1 complication, 75.5% reported the experience/fear of complications had a negative impact on their treatment adherence. Results were similar among children and adolescents. Physicians reported taking actions toward patients' non-adherence by further educating patients, closer monitoring, and changing the prescribed ADHD medication.Lack of effectiveness and ADHD/treatment-related complications are important reasons for treatment changes among children and adolescents with ADHD, highlighting the need for more effective and tolerable treatments to mitigate the burden of ADHD.

To provide a comprehensive evaluation of the economic burden associated with attention-deficit/hyperactivity disorder (ADHD) among children and adolescents from a US societal perspective.Direct healthcare costs of children (5-11 years) and adolescents (12-17 years) with ADHD were obtained using claims data from the IBM MarketScan Research Databases (01/01/2017-12/31/2018). Direct non-healthcare and indirect costs were estimated based on literature and government publications. Each cost component was estimated using a prevalence-based approach, with per-patient costs extrapolated to the national level.The total annual societal excess costs associated with ADHD were estimated at $19.4 billion among children ($6,799 per child) and $13.8 billion among adolescents ($8,349 per adolescent). Education costs contributed to approximately half of the total excess costs in both populations ($11.6 billion [59.9%] in children; $6.7 billion [48.8%] in adolescents). Other major contributors to the overall burden were direct healthcare costs ($5.0 billion [25.9%] in children; $4.0 billion [29.0%] in adolescents) and caregiving costs ($2.7 billion [14.1%] in children; $1.6 billion [11.5%] in adolescents).Cost estimates were calculated based on available literature and/or governmental publications due to the absence of a single data source for all costs associated with ADHD. Thus, the quality of cost estimates is limited by the accuracy of available data as well as the study populations and methodologies used by different studies.ADHD in children and adolescents is associated with a substantial economic burden that is largely driven by education costs, followed by direct healthcare costs and caregiver costs. Improved intervention strategies and policies may reduce the clinical and economic burden of ADHD in these populations.

BACKGROUND: Attention-deficit/hyperactivity disorder (ADHD) is associated with substantial clinical burden as individuals transition to adulthood, including higher rates of comorbidities, mortality, incarceration, and psychiatric hospitalizations than in individuals without ADHD. These higher rates likely contribute to substantial economic burden as well. OBJECTIVE: To provide a comprehensive evaluation of the economic burden associated with ADHD in the US adult population. METHODS: Direct health care costs were obtained by using claims data from the IBM MarketScan Research Databases (January 1, 2017, through December 31, 2018). Direct non-health care costs and indirect costs were estimated on the basis of the literature and government publications. Excess costs incurred by adults with ADHD during 2018 were evaluated from a societal perspective; per-patient costs were extrapolated to the national level. RESULTS: An estimated 8.7 million adults live with ADHD in the United States, resulting in a total societal excess cost attributable to ADHD of $122.8 billion ($14,092 per adult). Excess costs of unemployment ($66.8 billion; 54.4%) comprised the largest proportion of the total, followed by productivity loss ($28.8 billion; 23.4%) and health care services ($14.3 billion; 11.6%). CONCLUSIONS: ADHD in adults is associated with substantial economic burden. DISCLOSURES: This study was funded by Otsuka Pharmaceutical Development & Commercialization, Inc. (Otsuka). The study sponsor contributed to and approved the study design, participated in the interpretation of data, and reviewed and approved the manuscript. Schein is an employee of Otsuka. Gagnon-Sanschagrin, Davidson, Kinkead, Cloutier, Guerin, and Lefebvre are employees of Analysis Group, Inc., a consulting company that provided paid consulting services to Otsuka to develop and conduct this study and write the manuscript. Adler has received research support from Shire/Takeda, Sunovion, and Otsuka; consulting fees from Bracket, Shire/Takeda, Sunovion, Otsuka, the State University of New York (SUNY), the National Football League (NFL), and Major League Baseball (MLB); and royalty payments (as inventor) from New York University (NYU) for license of adult ADHD scales and training materials. Childress has received research support from Allergan, Takeda/Shire, Emalex, Akili, Ironshore, Arbor, Aevi Genomic Medicine, Neos Therapeutics, Otsuka, Pfizer, Purdue, Rhodes, Sunovion, Tris, KemPharm, Supernus, and the US Food and Drug Administration; was on the advisory board of Takeda/Shire, Akili, Arbor, Cingulate, Ironshore, Neos Therapeutics, Otsuka, Pfizer, Purdue, Adlon, Rhodes, Sunovion, Tris, Supernus, and Corium; received consulting fees from Arbor, Ironshore, Neos Therapeutics, Purdue, Rhodes, Sunovion, Tris, KemPharm, Supernus, Corium, Jazz, and Tulex Pharma; received speaker fees from Takeda/Shire, Arbor, Ironshore, Neos Therapeutics, Pfizer, Tris, and Supernus; and received writing support from Takeda/Shire, Arbor, Ironshore, Neos Therapeutics, Pfizer, Purdue, Rhodes, Sunovion, and Tris. Part of the material in this study was presented as a poster at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) 2021 Virtual Meeting; May 17-20, 2021.

To assess treatment patterns in adults with attention-deficit/hyperactivity disorder (ADHD) and associated healthcare costs in a real-world US setting. Claims data from the IBM MarketScan Commercial Subset (Q1/2014-Q4/2018) was used to identify adults diagnosed with ADHD who newly initiated on ADHD treatment (index date). Treatment sequences were defined using an algorithm; for each sequence, the regimen comprised all ADHD-related agents observed within 30 d of the first agent during the 12-month study period. Treatment changes included discontinuation, switch, add-on, and drop. Treatment characteristics were described for the first treatment regimen observed. Total adjusted annual healthcare costs were compared between patients with no treatment change and patients with 1, 2, and ≥3 treatment changes. Among 122,881 adults with ADHD, the majority initiated a stimulant (95.1%) as their first treatment regimen observed; 9.3% of patients initiated combination therapy of ≥2 ADHD-related agents, and 34.9% of patients had psychotherapy. After an average first treatment regimen duration of 7.1 months, 50.2% of patients experienced a treatment change (22.5% discontinued, 17.5% switched, 5.3% had an add-on, and 4.6% had a treatment drop). Among those who discontinued, 44.8% did so within the first month of initiation. Mean annual healthcare costs were higher among patients with at least 1 treatment change compared to those with no treatment changes; excess costs increased with each additional treatment change. Treatment changes were commonly observed and were associated with excess healthcare cost, emphasizing the unmet treatment needs of adults with ADHD in the US.

Objectives To describe characteristics and compare clinical outcomes including falls, fractures, infections, and neuropsychiatric symptoms (NPS) among long-term care residents with dementia with and without agitation. Methods A cross-sectional secondary analysis of administrative healthcare data was conducted whereby residents with dementia residing in a long-term care facility for ≥12 months were identified from the AnalytiCare LLC database (10/2010-06/2014) and were classified into mutually exclusive cohorts (Agitation Cohort or No-Agitation Cohort) based on available agitation-related symptoms. Entropy balancing was used to balance demographic and clinical characteristics between the two cohorts. The impact of agitation on clinical outcomes was compared between balanced cohorts using weighted logistic regression models. Results The study included 6,265 long-term care residents with dementia among whom, 3,313 were included in the Agitation Cohort and 2,952 in the No-Agitation Cohort. Prior to balancing, residents in the Agitation Cohort had greater dementia-related cognitive impairment and clinical manifestations compared to the No-Agitation Cohort. After balancing, residents with and without agitation, respectively, received a median of five and four distinct types of medications (including antipsychotics). Further, compared to residents without agitation, those with agitation were significantly more likely to have a recorded fall (OR = 1.58), fracture (OR = 1.29), infection (OR = 1.18), and other NPS (OR = 2.11). Conclusions Agitation in long-term care residents with dementia was associated with numerically higher medication use and an increased likelihood of experiencing falls, fractures, infections, and additional NPS compared to residents without agitation, highlighting the unmet need for effective management of agitation symptoms in this population.

Describe symptoms associated with ADHD/treatment-related adverse side effects among adults with ADHD in the US and assess their impact on quality of life (QoL) and work productivity.An online survey among adults receiving ADHD medications in the US was conducted to collect information relating to symptoms associated with ADHD/treatment-related adverse side effects. Participants were recruited from the panel of a well-established market research firm, Dynata, from 26 July to 30 July 2021 and were included in the study if they met the eligibility criteria and were willing to participate in the survey. Correlations between symptoms and key outcomes (QoL/employment/work impairment) were estimated using linear regression analyses.Of 585 participants, 95.2% experienced ≥1 symptom associated with ADHD/treatment-related adverse side effects in the past month (average = 5.8 symptoms). The number of symptoms was significantly correlated with reduced QoL, reduced probability of being employed, and increased work/activity impairment. Among subgroups with insomnia/other sleep disturbances and emotional impulsivity/mood lability, 50.4% and 44.7% reported their symptoms had "a lot" or "extremely" negative impact on their overall well-being, respectively.Symptoms associated with ADHD/treatment-related adverse side effects are common and have a substantial negative impact on QoL and reduces patients' probability of employment. Improved management of ADHD/treatment-related adverse side effects and more tolerable treatment options have the potential to improve QoL and work productivity among adults with ADHD.

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 Nestle 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 Nestle 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.

Introduction This study described patients hospitalized for acute heart failure (AHF) in Japan who received intravenous (IV) diuretics and/or vasodilators as the initial therapy. Methods The Japan Medical Data Vision database was used to identify adult patients hospitalized for AHF during 2013–2017, who were hemodynamically stable at presentation and treated with IV diuretics and/or IV vasodilators as initial therapy. Treatment patterns and use of cardiac rehabilitation, as well as outcomes (e.g., length of stay [LOS], in-hospital mortality, HF-readmission) were reported overall and by year of AHF hospitalization. Results Of 30,360 patients (mean age = 80.0 years; 52.2% male), 87.0% were treated during the hospitalization with IV diuretics, 63.9% with IV vasodilators, and 13.8% with intensified therapies. On average, the duration of IV therapy was 10.6 days. In-hospital cardiac rehabilitation was utilized by 51.7% of the patients for 11.7 days on average. Mean LOS was 23.3 days, while in-hospital mortality and 30-day HF readmission post-discharge were 13.2 and 9.5%, respectively. Hospitalization outcomes remained stable between 2013 and 2017 despite important changes in AHF management such as a decrease in carperitide use (55.9–40.0% in 2017), and increases in use of tolvaptan (from 14.2% in 2013 to 31.3% in 2017) and of cardiac rehabilitation (from 43.2% in 2013 to 56.1% in 2017). Patients with intensified therapies had the longest IV therapy duration (mean 23.8 days vs. 5.5–9.9 days), the highest cardiac rehabilitation services use (60.2 vs. 38.3–57.0%), the longest LOS (mean 36.7 vs. 16.3–22.2 days), and the highest in-hospital mortality (37.4 vs. 3.1–12.4%) compared to the other treatment groups. Conclusions Contemporary treatment for AHF hospitalization in Japan comprises a long duration of IV therapy followed by extended use of oral medications and in-hospital cardiac rehabilitation prior to discharge. Patients requiring intensified therapies had much longer LOS and higher in-hospital mortality. Supplementary Information The online version contains supplementary material available at 10.1007/s40119-021-00212-y.

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.

Introduction: Venetoclax has demonstrated deep responses and sustained progression-free survival and is well tolerated in patients (pts) with previously untreated or relapsed/refractory (R/R) chronic lymphocytic leukemia/small lymphocytic leukemia (CLL/SLL) in clinical trials. Some early studies provided initial insight of venetoclax utilization in the real-world (RW), but RW evidence focusing on venetoclax effectiveness and safety is still limited. This study assessed venetoclax effectiveness, safety, and treatment patterns in CLL pts treated in clinical practice. Methods: The CLL Collaborative Study of Real-World Evidence (CORE), a large multicenter chart review across 21 institutions in 4 countries, enrolled adult pts who initiated a first line (1L) therapy on/after 01/01/2012 or a new line of therapy (LOT) for R/R CLL/SLL on/after 02/12/2014 (current data cut through 06/08/2020). Clinical responses were abstracted from the medical records as assessed by treating physicians or investigators (iwCLL criteria were provided as reference). Overall response rate (ORR) was calculated as the proportion of pts with complete response (CR) or partial response (PR) out of pts with documented response assessments. Treatment patterns included choice of regimens and sequences, dose interruption, dose reduction, and discontinuation. Treatment discontinuation was defined as ending therapy for any reason(s) other than the completion of planned duration of therapy. Interruption was defined as a gap in the same LOT. All analyses were descriptive. Results: Of the 1231 CLL pts in the CORE data, 155 received venetoclax (21 in 1L and 134 in R/R) and were included in this study. Most of the sample were male (65%) and the median age at venetoclax initiation was 67.5 (range 37-91). For high-risk features among pts with available data, 33% (43/132 pts) had del(17p) or TP53 mutation, 24% (31/131 pts) had complex karyotype (≥ 3 abnormalities), and 77% (61/79 pts) had unmutated IGHV. At venetoclax initiation, 46%, 40%, and 14% of 151 pts with available data had low, medium, and high tumor burden, respectively. Median follow-up time from venetoclax initiation across all lines was 7.0 (0.1-43.1) months. Median number of prior LOT for R/R pts was 2 (1-5); the largest proportion of pts (36%) received venetoclax in 2L followed by 3L (26%). In 1L, venetoclax was most frequently used (62%) in combination (Figure 1), while in R/R setting venetoclax monotherapy was more commonly used (61%) followed by venetoclax + rituximab (25%; Figure 2). Majority of R/R pts (63%) received Bruton's tyrosine kinase inhibitors (BTKi) prior to venetoclax (Figure 2). Response was assessed and documented for 114 pts (74%). Among these, ORR was 75% (CR=40%, PR=35%) overall. Pts ≥65 years old had an ORR of 79% (CR=41%, PR=38%, n=74), and pts Overall, 48% of pts had adverse events (AEs) recorded. Five R/R pts (3.2%) had TLS events (defined by Howard or Cairo-Bishop criteria), of which 3 (1.9%) were clinical. Of the 5 pts, 2 had high tumor burden, and 3 had medium burden. Other AEs of interest include neutropenia (17%), thrombocytopenia (9%), and diarrhea/colitis (5%). Overall, 32% of pts discontinued venetoclax; 13% of all pts discontinued venetoclax due to AEs (primarily neutropenia [n=2] and thrombocytopenia [n=2]), followed by disease progression (8%). Venetoclax interruption rate was 14%, and dose reduction rate was 22% overall. Conclusions: Consistent with trial experiences, venetoclax demonstrates high response rates, including high-risk groups, and a manageable safety profile with low rates of TLS in clinical practice. Despite limitations of RW research, this study provides useful insights into treatment practices with venetoclax. Future research with longer follow-up is warranted to better understand the long-term clinical outcomes associated with venetoclax regimens. Disclosures Zheng: AbbVie: Current Employment. Sail:AbbVie Inc.: Current Employment, Current equity holder in publicly-traded company. Roeker:Abbott Laboratories: Other: spouse with minority ownership interest ; American Society of Hematology: Research Funding; AbbVie: Other: spouse with minority ownership interest . Manzoor:AbbVie: Current equity holder in publicly-traded company. Tuncer:AbbVie: Membership on an entity's Board of Directors or advisory committees; AstraZeneca: Consultancy, Membership on an entity's Board of Directors or advisory committees. Allan:Abbvie, Janssen, AstraZeneca, Pharmacyclics: Honoraria; Celgene, Genentech, Janssen, TG Therapeutics: Research Funding; Acerta, Genentech, Abbvie, Sunesis, Ascentage, Pharmacyclics, Janssen, AstraZeneca, BeiGene: Consultancy. Ujjani:Gilead/Kite: Consultancy, Research Funding; AstraZeneca: Consultancy, Honoraria, Research Funding; Genentech: Consultancy, Honoraria; MorphoSys: Consultancy; Atara: Consultancy, Honoraria; Verastem Oncology: Consultancy, Honoraria; Abbvie: Consultancy, Honoraria, Research Funding. Barr:Genentech: Consultancy; Merck: Consultancy; Abbvie/Pharmacyclics: Consultancy, Research Funding; Janssen: Consultancy; Celgene: Consultancy; Seattle Genetics: Consultancy; Verastem: Consultancy; TG therapeutics: Consultancy, Research Funding; Morphosys: Consultancy; Gilead: Consultancy; AstraZeneca: Consultancy, Research Funding. Brown:Novartis: Consultancy; Nextcea: Consultancy; Octapharma: Consultancy; MEI Pharma: Consultancy; Eli Lilly and Company: Consultancy; Astra-Zeneca: Consultancy; TG Therapeutics: Consultancy; Sunesis: Consultancy; Loxo: Consultancy, Research Funding; Sun: Research Funding; Rigel Pharmaceuticals: Consultancy; Pfizer: Consultancy; Catapult: Consultancy; Dynamo Therapeutics: Consultancy; Pharmacyclics: Consultancy; Morphosys: Consultancy, Membership on an entity's Board of Directors or advisory committees, Other; Gilead: Consultancy, Research Funding; Genentech: Consultancy; BeiGene: Consultancy; Invectys: Membership on an entity's Board of Directors or advisory committees, Other: DSMC; Janssen: Honoraria; AbbVie: Consultancy; Kite: Consultancy; Juno/Celgene: Consultancy; Verastem: Consultancy, Research Funding; Acerta: Consultancy. Eyre:AbbVie: Consultancy, Honoraria, Other: travel support; KITE, AZ, Loxo Oncology at Lilly: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Janssen: Consultancy, Honoraria, Other: travel support; Gilead: Consultancy, Honoraria, Other: travel support. Skarbnik:Abbvie: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Jazz Pharmaceuticals: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Kite Pharma: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Celgene: Consultancy, Membership on an entity's Board of Directors or advisory committees; Janssen: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; CLL Society: Membership on an entity's Board of Directors or advisory committees; AstraZeneca: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Gilead: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Alexion: Consultancy; Beigene: Speakers Bureau; Seattle Genetics: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Novartis: Speakers Bureau; Verastem: Speakers Bureau; Pharmacyclics: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Genentech: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau. Bannerji:Regeneron Pharmaceuticals: Research Funding; F. Hoffmann-La Roche Ltd/Genentech, Inc and Pharmacyclics LLC, an AbbVie Company: Research Funding; Sanofi-Pasteur: Other: Spouse is employee; AbbVie: Research Funding. Eichhorst:BeiGene: Consultancy, Honoraria, Other: travel support, Research Funding; AbbVie: Consultancy, Honoraria, Other: travel support, Research Funding; Janssen-Cilag: Consultancy, Honoraria, Other: travel support, Research Funding; F. Hoffmann-LaRoche: Consultancy, Honoraria, Other: travel support, Research Funding; ArQule: Consultancy, Honoraria, Other: travel support, Research Funding; Gilead: Consultancy, Honoraria, Other: travel support, Research Funding; AstraZeneca: Consultancy, Honoraria, Other: travel support, Research Funding; Oxford Biomedica: Consultancy, Honoraria, Other: travel support, Research Funding; Novartis: Consultancy, Honoraria, Other: travel support, Research Funding; Celgene: Consultancy, Honoraria, Other: travel support, Research Funding. Brander:Verastem: Consultancy, Honoraria, Other, Research Funding; NCCN: Other; Genentech: Consultancy, Honoraria, Other, Research Funding; Juno/Celgene/BMS: Other, Research Funding; MEI Pharma: Other, Research Funding; Pharmacyclics LLC, an AbbVie Company: Consultancy, Honoraria, Other, Research Funding; Pfizer: Consultancy, Other; TG Therapeutics: Consultancy, Membership on an entity's Board of Directors or advisory committees, Other, Research Funding; NCCN: Other; Novartis: Consultancy, Other; Teva: Consultancy, Honoraria; Tolero: Research Funding; Ascentage: Other, Research Funding; ArQule: Consultancy, Other, Research Funding; AbbVie: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other, Research Funding; Tolero: Research Funding; Teva: Consultancy, Honoraria; Novartis: Consultancy, Other; DTRM: Other, Research Funding; AstraZeneca: Consultancy, Honoraria, Other, Research Funding; BeiGene: Other, Research Funding. Sharmokh:AbbVie: Current Employment, Current equity holder in publicly-traded company. Jiang:AbbVie: Current Employment, Other: may hold stock or options. Pena:AbbVie: Current Employment, Current equity holder in publicly-traded company. Kamalakar:AbbVie: Current Employment, Other: may hold stock or other options. Emechebe:AbbVie: Current Employment, Current equity holder in publicly-traded company. Pivneva:Novartis: Consultancy, Other: Irina Pivneva is an employee of Analysis Group, Inc which received consultancy fees from Novartis.. Burne:Analysis Group, Inc., which has received consultancy fees from AbbVie: Current Employment. Guerin:Abbvie: Consultancy, Other; Novartis Pharmaceuticals Corporation: Consultancy, Other: Annie Guerin is an employee of Analysis Group, Inc. which received consultancy fees from Novartis.; Sanofi Genzyme: Consultancy, Other: Annie Guerin is an employee of Analysis Group, Inc. which received consultancy fees from Sanofi Genzyme.. Davids:Celgene: Consultancy; Research to Practice: Honoraria; AbbVie: Consultancy; Sunesis: Consultancy; Genentech: Consultancy, Research Funding; Pharmacyclics: Consultancy, Research Funding; MEI Pharma: Consultancy, Research Funding; Surface Oncology: Research Funding; BeiGene: Consultancy; Novartis: Consultancy, Research Funding; AstraZeneca: Consultancy, Research Funding; Bristol Myers Squibb: Research Funding; Janssen: Consultancy; Adaptive Biotechnologies: Consultancy; Ascentage Pharma: Consultancy, Research Funding; Gilead Sciences: Consultancy; Eli Lilly: Consultancy; Syros Pharmaceuticals: Consultancy; Zentalis: Consultancy; Merck: Consultancy; TG Therapeutics: Consultancy, Research Funding; Verastem: Consultancy, Research Funding. Mato:AbbVie: Consultancy, Research Funding; Adaptive: Consultancy, Research Funding; Genentech: Consultancy, Research Funding; TG Therapeutics: Consultancy, Research Funding; AstraZeneca: Consultancy, Research Funding; Pharmacyclics: Consultancy, Research Funding; Loxo: Consultancy, Research Funding; Janssen: Consultancy, Research Funding.

Objectives This study sought to characterize in-hospital treatment patterns and associated patient outcomes among patients hospitalized for heart failure (HF) in U.S. clinical practice. Background Hospitalizations for HF are common and associated with poor patient outcomes. Real-world patterns of in-hospital treatment, including diuretic therapy, in contemporary U.S. practice are unknown. Methods Using Optum de-identified Electronic Health Record data from 2007 through 2018, patients hospitalized for a primary diagnosis of HF (ejection fraction ≤40%) and who were hemodynamically stable at admission, without concurrent acute coronary syndrome or end-stage renal disease, and treated with intravenous (IV) diuretic agents within 48 h of admission were identified. Patients were categorized into 1 of 4 mutually exclusive hierarchical treatment groups defined by complexity of treatment during hospitalization (intensified treatment with mechanical support or IV vasoactive therapy, IV diuretic therapy reinitiated after discontinuation for ≥1 day without intensified treatment, IV diuretic dose increase/combination diuretic treatment without intensified treatment or IV diuretic reinitiation, or uncomplicated). Results Of 22,677 patients hospitalized for HF with reduced ejection fraction (HFrEF), 66% had uncomplicated hospitalizations without escalation of treatment beyond initial IV diuretic therapy. Among 7,809 remaining patients, the highest level of therapy received was IV diuretic dose increase/combination diuretic treatment in 25%, IV diuretic reinitiation in 36%, and intensified therapy in 39%. Overall, 19% of all patients had reinitiation of IV diuretic agents (26% of such patients had multiple instances), 12% were simultaneously treated with multiple diuretics, and 61% were transitioned to oral diuretic agents before discharge. Compared with uncomplicated treatment, IV diuretic reinitiation and intensified treatment were associated with significantly longer median length of stay (uncomplicated: 4 days; IV diuretic reinitiation: 8 days; intensified: 10 days) and higher rates of in-hospital (uncomplicated: 1.6%; IV diuretic reinitiation: 4.2%; intensified: 13.2%) and 30-day post-discharge mortality (uncomplicated: 5.2%; IV diuretic reinitiation: 9.7%; intensified: 12.7%). Conclusions In this contemporary real-world population of U.S. patients hospitalized for HFrEF, one-third of patients had in-hospital treatment escalated beyond initial IV diuretic therapy. These more complex treatment patterns were associated with highly variable patterns of diuretic use, longer hospital lengths of stay, and higher mortality. Standardized and evidence-based approaches are needed to improve the efficiency and effectiveness of in-hospital HFrEF care.

Background: Few studies have examined patient characteristics and treatment patterns among patients with dementia and agitation in the United States (US). Objective: To examine real-world treatment patterns and characteristics of patients with agitation related to dementia who were treated with antipsychotics in US residential care and community-based settings. Methods: This retrospective chart review collected US physician-level data from patients 55 to 90 years old initiated on an antipsychotic medication for the treatment of agitation related to dementia from January 2018 to May 2018. Clinical characteristics and treatment patterns were assessed overall and stratified by residential care and community-based settings. Results: A total of 313 participating physicians, 59.5% of whom were primary care physicians, abstracted 801 patient charts (residential care: n = 312; community-based: n = 489). Of patients with agitation who were initiated on an antipsychotic, most patients (74.5%) were initiated within 3 months of the onset of their studied agitation episode, and 62.8% experienced multiple agitation episodes before initiation. While non-pharmacological therapies are recommended first-line approach for agitation in dementia, use of non-pharmacological therapy before initiation of antipsychotics was reported for only 37.8% of patients in residential care and 21.3% in community-based settings. Conclusion: Most patients were initiated on an antipsychotic treatment after multiple episodes of agitation and largely without initial non-pharmacological therapy, suggesting that current treatment guideline recommendations for first-line non-pharmacological intervention may not be adequately followed in clinical practice. Understanding the clinical burden and treatment patterns among dementia patients with agitation is imperative for effective disease management.

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.

Background Autosomal dominant polycystic kidney disease (ADPKD) is one of the most common inherited kidney diseases characterized by progressive development of renal cysts and numerous extra-renal manifestations, eventually leading to kidney failure. Given its chronic and progressive nature, ADPKD is expected to carry a substantial economic burden over the course of the disease. However, there is a paucity of evidence on the impact of ADPKD from a societal perspective. This study aimed to estimate the direct and indirect costs associated with ADPKD in the United States (US). Methods A prevalence-based approach using data from scientific literature, and governmental and non-governmental organizations was employed to estimate direct healthcare costs (i.e., medical services, prescription drugs), direct non-healthcare costs (i.e., research and advocacy, donors/recipients matching for kidney transplants, transportation to/from dialysis centers), and indirect costs (i.e., patient productivity loss from unemployment, reduced work productivity, and premature mortality, caregivers’ productivity loss and healthcare costs). The incremental costs associated with ADPKD were calculated as the difference between costs incurred over a one-year period by individuals with ADPKD and the US population. Sensitivity analyses using different sources and assumptions were performed to assess robustness of estimates and account for variability in published estimates. Results The estimated total annual costs attributed to ADPKD in 2018 ranged from $7.3 to $9.6 billion in sensitivity analyses, equivalent to $51,970 to $68,091 per individual with ADPKD. In the base scenario, direct healthcare costs accounted for $5.7 billion (78.6%) of the total $7.3 billion costs, mostly driven by patients requiring renal replacement therapy ($3.2 billion; 43.3%). Indirect costs accounted for $1.4 billion (19.7%), mostly driven by productivity loss due to unemployment ($784 million; 10.7%) and reduced productivity at work ($390 million; 5.3%). Total excess direct non-healthcare costs were estimated at $125 million (1.7%). Conclusions ADPKD carries a considerable economic burden, predominantly attributed to direct healthcare costs, the majority of which are incurred by public and private healthcare payers. Effective and timely interventions to slow down the progression of ADPKD could substantially reduce the economic burden of ADPKD.

ObjectiveTo describe patient characteristics and healthcare costs associated with uveitic macular oedema (UME) in US clinical practices from a commercial payer’s perspective.Methods and analysisThe IBM MarketScan Commercial Subset (1 October 2015–31 March 2020) was used to identify patients with non-infectious uveitis (NIU), with or without UME. Patients with UME at any time were further classified into subgroups of patients who received a UME diagnosis during the study period and those who received a UME diagnosis and local steroid injection (LSI) during the study period. Demographic and clinical characteristics, NIU-related treatments and healthcare costs were described for each cohort and subgroup during the most recent 12 months of continuous health plan enrolment. Healthcare costs were also described by vision status among all patients with NIU.ResultsA total of 36 322 patients with NIU were identified, of whom 3 301 (9.1%) had UME and 33 021 (90.9%) had no UME. Patients with UME more frequently received NIU-related treatment compared with those without UME (64.6% vs 45.0%), particularly LSI treatment (12.5% vs 0.7%). Mean total all-cause healthcare costs per-patient-per-year (PPPY) were higher among patients with UME ($19 851) than patients without UME ($16 188) and were especially high among those with bilateral UME ($24 162). Further, vision loss was more commonly observed in those with UME versus those without UME (5.7% vs 2.2%) and a trend of increasing healthcare costs with increasing vision loss was observed.ConclusionNIU is associated with substantial clinical and economic burden, particularly when UME is present.

This study synthesized evidence regarding the prevalence of post-traumatic stress disorder (PTSD) in the United States (US).A systematic literature review (SLR) identified recently published (2015-2019) observational studies of PTSD prevalence in the USData from 38 identified articles were categorized by population, diagnostic criteria, and lookback period. Among civilians, point prevalence ranged from 8.0% to 56.7%, 1-year prevalence from 2.3% to 9.1%, and lifetime prevalence from 3.4% to 26.9%. In military populations, point prevalence ranged from 1.2% to 87.5%, 1-year prevalence from 6.7% to 50.2%, and lifetime prevalence from 7.7% to 17.0%. Within these ranges, several estimates were derived from relatively high quality data; these articles are highlighted in the review. Prevalence was elevated in subpopulations including emergency responders, refugees, American Indian/Alaska Natives, individuals with heavy substance use, individuals with a past suicide attempt, trans-masculine individuals, and women with prior military sexual trauma. Female sex, lower income, younger age, and behavioral health conditions were identified as risk factors for PTSD.PTSD prevalence estimates varied widely, partly due to different study designs, populations, and methodologies, and recent nationally representative estimates were lacking. Efforts to increase PTSD screening and improve disease awareness may allow for a better detection and management of PTSD.

Introduction: Venetoclax (VEN), a novel BCL-2 inhibitor, received accelerated FDA approval for the treatment of newly diagnosed (ND) acute myeloid leukemia (AML) in adults aged 75 years or older, or who have comorbidities precluding use of intensive chemotherapy, in combination with hypomethylating agents (HMAs: azacitidine [AZA] or decitabine [DEC]), or low-dose cytarabine (LDAC). As Phase III trials have demonstrated relative efficacy of VEN combinations, this real-world initiative provides insight into the characteristics and outcomes of ND AML patients (pts) treated with VEN combinations vs non-VEN-based regimens in clinical practice. Methods: The AML Real world evidenCe (ARC) Initiative is a multicenter chart review study of adult pts with ND AML treated with VEN ≥ 4-11-2016 (VEN cohort) or non-VEN-based regimens ≥ 5-15-2015 (control cohort) from different academic sites in the US. Pts in the control cohort were matched to pts in the VEN cohort based on age ( Results: At time of interim data analysis, 33 VEN and 33 control ND AML pts were included. Combinations in the VEN cohort included HMA (AZA [26 pts, 78.8%], DEC [6 pts, 18.2%]) or LDAC (1 pt, 3.0%); the control cohort received high- (18 pts, 54.5%) or low-intensity regimens (15 pts, 45.5%; HMA: 12 pts; enasidenib: 2 pts; LDAC: 1 pt). Median age was 75 years in both VEN and control cohorts, 39.4% and 42.4% were female, respectively, and 63.6% had de novo AML in both cohorts. The majority had ECOG performance status of 1 (VEN: 66.7%; control: 63.3%) and adverse-risk ELN classification (66.7% in both cohorts). All pts with favorable-risk in the VEN cohort (100.0%) had mutated NPM1 without FLT3-ITD or with low allelic ratio ( In the high-intensity therapy matched subgroup (VEN: n=18; control: n=18), median age was 74 and 69 in VEN versus control, respectively. The majority of pts had ECOG performance status of 1 (VEN: 76.5%; control: 75.0%) and adverse-risk ELN classification (61.1% in both cohorts). High-intensity treatment regimens used for the control group were: CPX-351 (n=10; 55.6%), 7+3 (n=4; 22.2%), 7+3 + gemtuzumab ozogamicin (n=3; 16.7%), cytarabine + mitoxantrone (n=1; 5.6%). In the VEN matched high-intensity subgroup, 15 (83.3%) received VEN + AZA, 2 (11.1%) received VEN + DEC, and 1 (5.6%) received VEN + LDAC. Ten (55.6%) initiated VEN as an inpatient treatment. Outcomes for the full sample and subgroup are presented in Table 1. In the full sample, the overall response rate (ORR: CR+CRi+CRh) was 69.7% in the VEN cohort and 45.5% in the control cohort. The 1-year OS was 67.0% in the VEN cohort and 44.2% in the control cohort for the full sample; and 71.3% and 55.4%, respectively, in the high-intensity therapy matched subgroups. Conclusions: The ongoing ARC Initiative demonstrates real-world treatment effectiveness of VEN combinations in clinical practice. The real-world outcomes for VEN combinations for ND AML pts appear consistent with results reported from clinical trials, though a small number of pts were assessed. Further analyses of comparisons to matched controls, both overall and in the high-intensity therapy matched subgroup, and continuing data collection will allow a more robust assessment of the comparative effectiveness of VEN combinations in the real-world setting. Disclosures Pollyea: Syndax: Consultancy; Syros: Consultancy; Novartis: Consultancy; Karyopharm: Consultancy; Janssen: Consultancy; 47: Consultancy, Research Funding; Genentech: Consultancy; Takeda: Consultancy; Celgene/BMS: Consultancy; Agios: Consultancy; Glycomimetics: Other; Amgen: Consultancy; Abbvie: Consultancy, Research Funding; Daiichi Sankyo: Consultancy; Pfizer: Consultancy. Talati:BMS: Honoraria; Jazz: Speakers Bureau; AbbVie: Honoraria; Pfizer: Honoraria; Astellas: Speakers Bureau. Lai:Abbvie: Consultancy; Jazz: Speakers Bureau; Astellas: Speakers Bureau; Macrogenics: Consultancy; Agios: Consultancy. Abedin:Helsinn Healthcare: Honoraria; Agios: Honoraria; Jazz Pharmaceuticals: Honoraria; Pfizer: Research Funding; Helsinn Healthcare: Research Funding; Actinium Pharmaceuticals: Research Funding. Zeidner:Sumitomo Dainippon Pharma Oncology, Inc.: Research Funding; Merck: Research Funding; Forty-Seven: Other: Travel Reimbursement, Research Funding; AROG: Research Funding; AsystBio Laboratories: Consultancy; Daiichi Sankyo: Honoraria; Takeda: Consultancy, Honoraria, Other: Travel Reimbursement, Research Funding; Pfizer: Honoraria; Genentech: Honoraria; Celgene: Consultancy, Honoraria, Research Funding; AbbVie: Honoraria, Other: Independent Review Committee; Agios: Honoraria. Xavier:Kite/Gilead: Honoraria, Membership on an entity's Board of Directors or advisory committees; AbbVie: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; AstraZeneca: Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Takeda: Honoraria, Membership on an entity's Board of Directors or advisory committees; Morphosys: Honoraria, Membership on an entity's Board of Directors or advisory committees; TG therapeutics: Honoraria, Membership on an entity's Board of Directors or advisory committees; Verastem: Honoraria, Membership on an entity's Board of Directors or advisory committees; Acrotec: Consultancy, Speakers Bureau; Biegene: Speakers Bureau; Genentech: Speakers Bureau; Seattle Genetics: Speakers Bureau; Celgene/BMS: Speakers Bureau; Epizyme: Speakers Bureau; Astellas: Speakers Bureau; Incyte: Speakers Bureau. Wolach:AbbVie: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Fees for lectures and Consultancy, Research Funding; Pfizer: Consultancy, Honoraria; Janssen: Other: Fees for lectures and Consultancy; Amgen: Other: Fees for lectures and Consultancy; Astellas: Consultancy, Honoraria, Other: Fees for lectures and Consultancy; Novartis: Consultancy, Honoraria, Other: Fees for lectures and Consultancy. Moshe:Novartis: Membership on an entity's Board of Directors or advisory committees, Other: Lecturer; Astellas: Membership on an entity's Board of Directors or advisory committees, Other: lecturer; AbbVie: Membership on an entity's Board of Directors or advisory committees, Other: Fee for lec. Choi:AbbVie: Current Employment, Current equity holder in publicly-traded company. Bui:AbbVie: Current Employment, Current equity holder in publicly-traded company. Svensson:AbbVie: Current Employment, Current equity holder in publicly-traded company. Kye:AbbVie: Current Employment, Other: may hold stock or other options. Burne:Analysis Group, Inc., which has received consultancy fees from AbbVie: Current Employment. Guerin:Abbvie: Consultancy, Other; Sanofi Genzyme: Consultancy, Other: Annie Guerin is an employee of Analysis Group, Inc. which received consultancy fees from Sanofi Genzyme.; Novartis Pharmaceuticals Corporation: Consultancy, Other: Annie Guerin is an employee of Analysis Group, Inc. which received consultancy fees from Novartis.. Ma:Genentech, Inc.: Current Employment, Current equity holder in publicly-traded company. Montez:Genentech, Inc.: Current Employment; F. Hoffmann-La Roche: Current equity holder in publicly-traded company. Goldberg:Dava Oncology: Honoraria; Pfizer: Research Funding; Celularity: Research Funding; AROG: Research Funding; Aprea: Research Funding; ADC Therapeutics: Research Funding; Genentech: Consultancy, Membership on an entity's Board of Directors or advisory committees; Daiichi Sankyo: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Celgene: Consultancy; AbbVie: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Aptose: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding.

Introduction Infants with spinal muscular atrophy (SMA) type 1 typically face a decline in motor function and a severely shortened life expectancy. Clinical trials for SMA type 1 therapies, onasemnogene abeparvovec (AVXS-101) and nusinersen, demonstrated meaningful improvements in efficacy (e.g., overall survival) but there were no head-to-head clinical trials assessing comparative efficacy. This study estimated the treatment effects of AVXS-101 relative to nusinersen for the treatment of SMA type 1. Methods Overall survival, event-free survival (no death or need to use permanent assisted ventilation), improvement in motor function [increase of ≥ 4 points in Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score from baseline], and motor milestone achievements (head control, rolling over, and sitting unassisted) reported in the onasemnogene abeparvovec (AVXS-101-CL-101; NCT02122952) and nusinersen (ENDEAR; NCT02193074) clinical trials were indirectly compared using frequentist and Bayesian approaches. Results Among symptomatic infants with SMA type 1, the number needed to treat (NNT) to prevent one more death with AVXS-101 instead of nusinersen was 6.2 [95% confidence intervals (CI) = 4.1–12.2], and the probability of preventing death was 20% higher for patients treated with AVXS-101 than nusinersen [risk ratio (RR) = 1.2, 95% CI 1.1–1.3]. For event-free survival, the NNT to prevent one more event was 2.6 (95% CI 2.0–3.6) and RR was 1.6 (95% CI 1.4–1.9). For improvement in motor function, NNT was 3.5 (95% CI 2.6–5.3) and RR was 1.4 (95% CI 1.2–1.6). For milestone achievements, the NNTs were 1.4 (95% CI 1.1–1.9), 1.5 (95% CI 1.1–2.5), and 1.2 (95% CI 1.0–1.5); RRs 4.2 (95% CI 2.6–6.7), 7.8 (95% CI 3.6–17.0), and 11.2 (95% CI 5.1–24.5) for head control, rolling over, and sitting unassisted, respectively. Results were similar using the Bayesian approach. Conclusion This indirect comparison (AVXS-101-CL-101 vs. ENDEAR) among symptomatic SMA type 1 infants suggests that AVXS-101 may have an efficacy advantage relative to nusinersen for overall survival, independence from permanent assisted ventilation, motor function, and motor milestones. Funding AveXis.

Background: Recently, a novel class of treatments, CDK4/6 inhibitors, has been approved, and is now recommended for pre-menopausal women with HR+/HER2- metastatic breast cancer (mBC). This study examined prevailing treatment patterns and sequencing among premenopausal women with mBC treated in clinical practice. Methods: Patient-level data were collected from patient charts in May 2018 from 30 oncologists, mostly from community practice, in the US. Treatment sequences and patterns were assessed for pre-menopausal women diagnosed with HR+/HER2- mBC between January 2015 and January 2017 (with a minimum of 1 year of follow-up). Results: Data were collected on 201 pre-menopausal women with HR+/HER2- mBC. In first-line therapy for mBC, 52.7% of the patients received a CDK4/6 inhibitor-based regimen, 23.4% received endocrine monotherapy, 20.9% received a chemotherapy-based regimen, and the remaining 3.0% received an everolimus-based regimen. The majority of patients who received a CDK4/6 inhibitor received it in combination with an AI (73.6%), fulvestrant (11.3%), or tamoxifen (6.6%). Approximately half of all patients (51.2%) received an ovarian suppression agent during first-line therapy. Overall, median time on treatment from Kaplan Meier (KM) analysis for first-line therapy was 16.1 months. Most common reason for discontinuing first line was disease progression or suboptimal response (79.0% of patients who discontinued); another common reason was the completion of the planned duration of therapy (12.6%). Among the 106 patients who received a CDK4/6 inhibitor in the first line, median time on treatment from KM analysis was 26.8 months. Main reason for CDK4/6 inhibitor discontinuation was disease progression or suboptimal response (90.2% of patients who discontinued). For the 109 patients for whom we observed a second-line therapy, treatment sequences are presented in Table 1. Median time on treatment for second and third line therapy was 9.6 and 7.8 months, respectively. Conclusion: Following the introduction of novel CDK4/6 inhibitor treatments in the mBC setting, we observed that approximately half of pre-menopausal patients received a CDK4/6-based regimen in the first line of therapy. Top 5 treatment sequences in pre-menopausal HR+/HER2- mBC patients (n=109)Treatment SequenceN(%)CDK4/6 - ET -> Everolimus - ET21(19.3%)Chemo -> Chemo16(14.7%)CDK4/6 - ET -> Chemo13(11.9%)ET -> CDK4/6 - ET13(11.9%)Chemo -> CDK4/6 - ET10(9.2%)ET: endocrine therapy; Chemo: chemotherapy; -> indicates a change to the next line of therapy. Percentages calculated among patients with at least 2 lines of therapy Citation Format: Dalal AA, Goldschmidt D, Romdhani H, Kelkar S, Guerin A, Wang H, Caria N, Sawhney A, O'Shaughnessy J. Treatment patterns and sequences among pre-menopausal women with HR+/HER2- metastatic breast cancer: A chart review study [abstract]. In: Proceedings of the 2018 San Antonio Breast Cancer Symposium; 2018 Dec 4-8; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2019;79(4 Suppl):Abstract nr P6-18-38.

Objectives Dry eye disease (DED) is a complex multifactorial condition of the ocular surface characterized by symptoms of ocular discomfort, irritation, and visual disturbance. Data previously reported from this study showed an increase in prevalence and incidence of DED with age and over time. The objective of this study was to compare the ranking of DED prevalence among other ocular conditions that led patients to seek eye care. Methods In this population-based study using the US Department of Defense Military Health System claims database of >9.7 million beneficiaries, indicators of DED and other ocular conditions were analyzed over time. The overall prevalence (2003-2015) and annual incidence (2008-2012) of DED and other ocular conditions were estimated using an algorithm based on two independent indicators derived from selected diagnostic and procedure codes and prescriptions for cyclosporine ophthalmic emulsion for DED and diagnostic codes for the indicators of other common ocular conditions. Results In 2003-2015, the most common ocular conditions were disorders of refraction and accommodation (25.84%), cataracts (17.14%), glaucoma (7.27%), disorders of the conjunctiva (6.76%), other retinal disorders (5.94%), and DED (5.28%). DED was the fifth most prevalent ocular condition in women (7.78%) and ninth most prevalent in men (2.96%). In 2012, DED had the third highest annual incidence (0.87%), behind disorders of refraction/accommodation (1.87%) and cataracts (1.50%). Conclusion This study provided further epidemiologic evidence for DED as a commonly occurring condition that drives patients to seek treatment.

Objective: This study was conducted to determine the incremental healthcare resource utilization (HRU) and costs associated with relapse or recurrence (R/R) in patients with major depressive disorder (MDD) treated with antidepressants (AD) in US clinical practice. Methods: In this retrospective cohort study, adult patients with MDD treated with a branded AD were selected from the Truven Health Analytics MarketScan Databases (January 1, 2004–March 31, 2015). Time to first indicator of R/R was described. Characteristics, HRU, and costs were compared between patients with and without R/R. Among patients with R/R, HRU and costs were also compared between the pre- and post-R/R period. Results: From the 22,236 selected patients, 5,541 had ≥ 1 indicator of R/R and 16,695 did not. The 3-year R/R rate varied between 21.3% and 36.4% based on pattern of AD use (continuous, switch/augmentation, or early discontinuation). Patients with and without R/R presented different characteristics—notably, more intensive prior AD use and a higher comorbidity burden. HRU and costs were high in both patients with and without R/R but substantially higher among those with R/R ($20,590 vs $12,368 per-patient-per-year (PPPY); adjusted difference [aDiff] = $7,037), mainly driven by increased inpatient (IP) services (adjusted incidence rate ratio IP days = 3.95; aDiff IP costs = $3,433 PPPY). Among patients with R/R, emergency department visits, IP days, and IP admissions were over 2-times higher during the post-R/R period and total costs increased by over 50% from $19,267 to $29,419 in the post-R/R period. Conclusions: The economic burden in MDD patients is substantial, but is significantly higher among those who experience R/R.

Introduction Agitation in individuals with Alzheimer's disease (AD) may predict institutionalization. This study assessed the incremental risk and costs associated with agitation in individuals with AD. Methods A retrospective analysis of the National Alzheimer's Coordinating Center Uniform Data Set (June 2005–February 2018) was conducted. Incremental risk of institutionalization associated with agitation was estimated and used with the number of institutionalized individuals with AD and agitation and costs of living by residential setting in the United States (literature-based), to estimate incremental institutionalization costs. Results The analysis included 11,348 individuals with AD: 6603 (58.2%) with and 4745 (41.8%) without agitation. Compared with individuals without agitation, those with agitation were 20% more likely to be institutionalized (odds ratio = 1.20; 95% CI = 1.08–1.33). Total incremental cost of institutionalization associated with agitation was $4.3 billion ($50,588/individual). Discussion Agitation is associated with a higher risk of institutionalization among patients with AD, which translates into a substantial economic burden., Highlights • Agitation in Alzheimer's disease portends higher risk of institutionalization. • Higher institutionalization risk translates into a substantial economic burden. • Agitation-related institutionalization cost was ∼$4.3 billion in the United States.

To assess healthcare resource utilization (HRU) and healthcare costs among women with hormone receptor-positive and human epidermal growth factor receptor 2-negative advanced breast cancer (HR+/HER2- aBC) treated with cyclin-dependent kinases 4 and 6 (CDK4/6) inhibitors. Women with HR+/HER2- aBC, initiating CDK4/6 inhibitor treatment were identified using IBM MarketScan® Commercial and Medicare Supplemental databases (Q1/2000-Q3/2018). Based on the first CDK4/6 patients received (index therapy), three cohorts were identified: abemaciclib, palbociclib, and ribociclib. The baseline period (6-months preceding treatment initiation) was used to describe patient characteristics. All-cause HRU and direct total healthcare costs (medical and pharmacy) from treatment initiation until the earliest of the end of index therapy, continuous health plan enrollment, or data availability, were compared for the ribociclib cohort versus the abemaciclib and palbociclib cohorts, separately, using weighted regression analyses balanced on baseline covariates. Average age at treatment initiation was ∼60 years and the majority of patients were postmenopausal (abemaciclib: 92%; palbociclib: 92%; ribociclib: 79%). Average follow-up duration was 3.9, 8.8, and 5.9 months for the abemaciclib, palbociclib, and ribociclib cohorts, respectively. After reweighting, HRU was not statistically different between the ribociclib and abemaciclib cohorts, however, the ribociclib cohort incurred significantly lower total healthcare costs (-$5,452; 95% CI: -$8,726; -$1,139, p = 0.01). Medical costs (driven by outpatient costs) and pharmacy costs (driven by CDK4/6 inhibitor costs) were significantly lower for the ribociclib cohort. Among the reweighted ribociclib and palbociclib cohorts, HRU and total healthcare costs were not statistically different, although the ribociclib cohort had lower outpatient costs per-patient-per-month (-$1,245, 95% CI: -$2,349; -$37, p = 0.04). Due to the retrospective, observational design, treatment cohorts were not randomly assigned. During CDK4/6 therapy, ribociclib patients tended to incur lower medical and pharmacy costs than abemaciclib patients. Among ribociclib and palbociclib patients, HRU and healthcare costs were similar.

Background: Hypomethylating agents (HMA), azacitidine (AZA) and decitabine (DEC), are the current standard of care in patients with intermediate-2 and high risk myelodysplastic syndromes (MDS) by IPSS prognostic category (higher-risk MDS) based on clinical trials showing increased response rates and overall survival with these agents. However, the outcomes of patients with MDS treated with HMAs have not been comprehensively assessed in real-world practice. This study describes real-world treatment patterns and outcomes among patients with MDS treated with HMAs. Methods: Adult patients with a confirmed MDS diagnosis between 01/2009-12/2015 treated with HMA were identified in the SEER-Medicare database (01/2006-12/2016). The index date was the date of HMA initiation; the first HMA was defined as the index HMA. Patients were required to have Medicare Parts A and B coverage for ≥12 months pre-index and ≥1 month post-index date. Patients with evidence of stem cell transplant (SCT) or acute myeloid leukemia (AML) pre-index, or enrollment in a clinical trial at any time were excluded. Patients meeting the aforementioned criteria formed the study population. An algorithm, based on timing of HMA initiation, timing of diagnosis of pancytopenia (low counts for red blood cells, white blood cells, and platelets), and diagnosis of refractory anemia with excess of blasts (RAEB)-1 and RAEB-2, was developed to categorize patients into specific MDS risk groups using claims data (Figure A). Patients with RAEB-1/2 diagnosis who initiated HMA within 1 month prior to or 4 months post-RAEB diagnosis, or pancytopenia diagnosis within 3 months prior to HMA initiation (regardless of types of MDS diagnosis) were classified into the higher-risk MDS group. Patients with non-RAEB MDS diagnosis and HMA initiation within 1 month prior to or 4 months post-MDS diagnosis were classified into the intermediate-1-risk MDS group; remaining, unclassified patients from the study population were assigned into the unknown-risk MDS group. OS and time to AML transformation (determined by a diagnosis of AML in claims) were assessed for each risk stratum post-index using Kaplan-Meier (KM) analyses. HMA treatment patterns were measured up to 12 months post-index or until the first event among the following: SCT, AML-like intensive chemotherapy use, AML, or end of data/Medicare Parts A and B coverage (follow-up period). Results: A total of 3,046 patients with MDS treated with HMA were included. On average, patients were aged 77.4 years, and 36.8% were female. The majority of patients were classified in the higher-risk MDS group (70.9%), 8.0% in the intermediate-1-risk MDS group, and 21.1% in the unknown-risk MDS group. Median OS was 11.6 months among patients in the higher-risk MDS group, whereas median OS was 18.4 and 19.1 months for patients in the intermediate-1 and unknown-risk MDS groups, respectively (Figure B). Median time to AML transformation was 19.3 months in the higher-risk MDS group, 50.4 months in the intermediate-1-risk MDS group, and was not reached for the unknown-risk MDS group (Figure C). Overall, patients received an average of 5.1 HMA cycles (median = 4.0 cycles) and the majority were complete cycles (90.9%; as indicated per label or, for AZA, also including the commonly used alternative regimens of 5-day 5-0-0 or 7-day with a weekend break 5-2-2). The mean duration of HMA cycles was 32.6 days (median = 28.0 days). As many as 45.3% of patients received Conclusions: In this US real-world observational study using claims data, risk of death or AML transformation among patients with MDS was high despite HMA treatment, especially for those with higher-risk MDS. More than 70% of patients with MDS treated with HMA were classified with higher-risk MDS. These patients had a poor prognosis, with a median OS shorter than 1 year and a time-to-AML transformation of less than 2 years from HMA initiation. These data highlight the unmet medical needs of patients with MDS treated with HMA, particularly for those in the higher-risk MDS group. Disclosures Stein: Agios: Consultancy, Membership on an entity's Board of Directors or advisory committees; Astellas Pharma US, Inc: Membership on an entity's Board of Directors or advisory committees; Celgene Corporation: Membership on an entity's Board of Directors or advisory committees; Daiichi Sankyo, Inc.: Membership on an entity's Board of Directors or advisory committees; Bioline: Membership on an entity's Board of Directors or advisory committees; Genentech: Membership on an entity's Board of Directors or advisory committees; Novartis: Membership on an entity's Board of Directors or advisory committees; PTC Therapeutics: Membership on an entity's Board of Directors or advisory committees; Syros: Membership on an entity's Board of Directors or advisory committees. Latremouille-Viau:Novartis: Other: I am an employee of Analysis Group, Inc., which provided paid consulting services to Novartis for the conduct of this study. Joseph:Amgen: Equity Ownership; Cigna: Equity Ownership; Pfizer: Equity Ownership; Novartis: Employment, Equity Ownership. Shi:Novartis: Other: I am an employee of Analysis Group, Inc., which provided paid consulting services to Novartis for the conduct of this study. Guerin:AbbVie: Other: employee of Analysis Group, Inc., which has received consultancy fees from AbbVie. Wu:Novartis: Other: I am an employee of Analysis Group, Inc., which provided paid consulting services to Novartis for the conduct of this study. Bonifacio:Novartis: Employment. Cao:Novartis: Employment, Equity Ownership.

To assess healthcare costs and hospitalization rates associated with rifaximin therapy versus lactulose alone among patients at risk for hepatic encephalopathy (HE). IBM Marketscan Commercial and Optum’s de-identified Clinformatics Data Mart databases were used separately to identify commercially insured HE patients treated with rifaximin or lactulose alone, using an algorithm developed with clinical experts. HE-related hospitalizations were defined based on an algorithm using diagnosis codes and diagnosis-related group codes. HE-related/all-cause hospital admissions/days and healthcare costs were compared between rifaximin and lactulose episodes using incidence rate ratios and adjusted cost differences. In Marketscan, there were 13,515 [Optum: 5,217] rifaximin episodes and 9,946 [4,897] lactulose alone episodes included. Yearly rates of HE-related hospital admissions decreased by 33% [34%] when treated with rifaximin versus lactulose alone, and rates of HE-related hospital days similarly decreased by 43% [57%]. Yearly rates of all-cause hospital admissions decreased by 27% [27%]; rates of all-cause hospital days decreased by 33% [37%] during rifaximin episodes versus lactulose alone. This translated to $2,417 [$2,301] and $173 [$397] lower total mean medical costs and HE-related hospital costs per-patient-per-month, respectively (p Patients incurred significantly lower rates of HE-related and all-cause hospitalizations during rifaximin versus lactulose episodes, resulting in lower facility and professional costs. Cost savings may be possible if rifaximin adherence is improved in HE patients. The study is subject to limitations common to claims-based analyses.

To build upon previous outdated studies by comprehensively assessing the direct healthcare burden of autosomal dominant polycystic kidney disease (ADPKD). Patients with ≥2 diagnoses for ADPKD (ADPKD cohort) were identified in the US fee-for-use IBM Truven Health Analytics MarketScan Commercial Claims and Encounters and IBM Truven Health Analytics MarketScan Medicare Supplemental databases (01 January 2015–31 December 2017) and matched (1:3) to controls without ADPKD (non-ADPKD cohort). The index date was the last calendar date followed by 12 months continuous enrollment (study period). Patients with ADPKD were stratified into one of seven mutually exclusive groups based on chronic kidney disease (CKD) stages (I–V), end-stage renal disease requiring renal replacement therapy (ESRD-RRT), and unknown stage. During the 12-month study period, patients with ADPKD incurred significantly higher total healthcare costs than those without ADPKD (mean cost difference = $22,879 per patient per year [PPPY]; p p Results may not be representative of the overall ADPKD US population; CKD stage was based on diagnosis and procedure codes; criteria used to identify ADPKD at risk of rapid progression did not rely on laboratory values; there may be billing inaccuracies and omissions in health insurance claims data. This study demonstrated the substantial healthcare costs associated with ADPKD, which increased as patients progressed through more severe CKD stages.

Aims: Adverse events (AEs) associated with treatments for metastatic colorectal cancer (mCRC) may compromise the course of treatment, impact quality-of-life, and increase healthcare resource utilization. This study assessed the direct healthcare costs of common AEs among mCRC patients in the US. Methods: Adult mCRC patients treated with chemotherapy or targeted therapies were identified from administrative claims databases (2009–2014). Up to the first three mCRC treatment episodes per patient were considered and categorized as with or without the AE system/organ category during the episode. Total healthcare costs (2014 USD) were measured by treatment episode and reported on a monthly basis. Treatment episodes with the AE category were matched by treatment type and line of treatment to those without the AE category. Adjusted total cost differences were estimated by comparing costs during treatment episodes with vs without the AE category using multivariate regression models; p-values were estimated with bootstrap. Results: A total of 4158 patients with ≥1 mCRC treatment episode were included (mean age = 59 years; 58% male; 60% with liver and 14% with lung metastases; 2,261 [54%] with a second and 1,115 [27%] with a third episode). On average, two treatment episodes were observed per patient with an average length of 166 days per episode. Adjusted monthly total cost difference by AE category included hematologic ($1,480), respiratory ($1,253), endocrine/metabolic ($1,213), central nervous system (CNS; $1,136), and cardiovascular ($1,036; all p Limitations: Claims do not include information on the cause of AEs, and potentially less severe AEs may not have been reported by the physician when billing the medical service. This study aimed to assess the association between costs and AEs and not the causation of AEs by treatment. Conclusions: The most costly AEs among mCRC patients were hematologic, followed by respiratory, endocrine/metabolic, CNS, and cardiovascular.

This study compared progression, progression-free survival (PFS), overall survival (OS), and treatment changes among chronic myelogenous leukemia patients in chronic phase (CML-CP) receiving nilotinib or dasatinib as second-line therapy. Information on CML-CP patients switched from imatinib to nilotinib or dasatinib as second-line therapy was collected retrospectively from 122 US hematologists and oncologists through an online medical chart review. Progression, PFS, and OS were compared using multivariate Cox proportional hazard models, and treatment changes using chi-square tests. Of 597 imatinib resistant or intolerant patients, 301 initiated nilotinib and 296 dasatinib as second-line therapy. Nilotinib was associated with a lower risk of progression (hazard ratio [HR] = 0.27; p = 0.021) and longer PFS (HR = 0.48; p = 0.030) than dasatinib, with a median follow-up time of 11 months for nilotinib and 10 months for dasatinib. Nilotinib patients had a lower estimated hazard of mortality than dasatinib patients, though not statistically significant (HR = 0.46; p = 0.067). When treatment changes were classified by the physicians’ justifications, fewer nilotinib patients had treatment changes due to ineffectiveness (2.0% vs. 5.1%, p = 0.041) or drug holidays due to intolerance (0.0% vs. 1.7%, p = 0.024) than dasatinib patients. Among CML-CP patients in this retrospective chart review who switched from imatinib to either nilotinib or dasatinib, nilotinib was associated with a significantly lower risk of progression and longer PFS than dasatinib. Nilotinib patients were also less likely than dasatinib patients to subsequently have treatment changes due to ineffectiveness or drug holidays due to intolerance. These findings could be subject to unobserved confounders.

Introduction Prior studies have reported that hidradenitis suppurativa (HS) is accompanied by a myriad of physical and mental conditions. However, given the small sample sizes and the limited number of pre-selected comorbidities, these studies do not provide a complete picture of the comorbidity burden of HS in the USA. Moreover, the relationship between HS severity and comorbidity burden has yet to be characterized. Using a large US claims database, we estimated the comorbidity burden associated with HS, stratified by disease severity. Methods A retrospective matched cohort design was used. Patients with HS were classified into two severity cohorts (milder and more severe) using an empirical algorithm based on treatments received. The comorbidity burden was compared between each HS cohort and their matched HS-free cohort, and between patients with milder vs. those with more severe forms of HS. Results Several physical and mental comorbidities were found to be more prevalent in both cohorts of patients with milder and more severe forms of HS than in their matched HS-free cohorts. The comorbidity burden also increased greatly as the disease progressed to more severe forms. Conclusions The results of this study highlight the complexity of the comorbidity burden of HS patients and the need for a multidisciplinary approach to optimize the management of HS and its numerous associated comorbidities. Funding AbbVie, Inc. Electronic supplementary material The online version of this article (10.1007/s13555-018-0264-z) contains supplementary material, which is available to authorized users.

Objective: To describe patient characteristics, treatment patterns, healthcare resource utilization (HRU), and costs among patients with anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC) receiving ceritinib in second or later line of therapy. Methods: Adult patients with NSCLC receiving ceritinib were identified from two large US claims databases (2006–2015). Patient characteristics, comorbidity profile, treatment patterns prior to ceritinib, and ceritinib dosing patterns were described. All-cause, HRU, and costs incurred during the observation period after ceritinib initiation were reported per patient per six months. Results: One hundred sixty-four patients were included (mean age 54.2 years, 57.3% female); the majority had metastatic disease (94.5%) and the average Charlson Comorbidity Index was 7.6. 150 (91.5%) patients received crizotinib prior to ceritinib – average crizotinib duration was 10.2 months and time between crizotinib discontinuation and ceritinib initiation was 2.1 months (median= 0; 25th–75th percentile= 0–0.8). Most patients (73.8%) initiated ceritinib on the recommended dose (750 mg) and maintained the dose until the end of the observation period (mean of 7.4 months) or ceritinib discontinuation; 61 (37.2%) patients discontinued ceritinib during the observation period. A total of 76 (46.3%) patients had at least one inpatient admission during the observation period after ceritinib initiation. Mean total healthcare cost per patient per six months was $111,468. Conclusions: Patients with ALK-positive NSCLC receiving ceritinib had a high comorbidity burden and generally started ceritinib on the recommended dose quickly after crizotinib discontinuation. Medical costs accounted for nearly a half of the total healthcare costs.

Background: Dose modification related to adverse event is common in the treatment of metastatic breast cancer (mBC). Based on the dosage form and strengths available, dose modification may lead to drug wastage when the dose cannot be split or saved for later use. This study aimed to describe dosing patterns and to estimate the economic burden of drug wastage associated with dose modifications in postmenopausal women with HR+/HER2- mBC receiving palbociclib. Methods: Postmenopausal adult women diagnosed with HR+/HER2- mBC were identified from the Truven administrative claims database (2006Q1–2015Q4). Regimens received following mBC diagnosis were identified – patients who received a palbociclib-based regimen during one of their first three lines of therapy for mBC were included in the study. Palbociclib starting daily dose, average daily dose, and dosing patterns (dosing modifications and sequences) were reported. A dose modification was defined as a change (decrease/increase) of ≥25mg daily compared to the preceding dose. The economic burden of drug wastage was estimated by multiplying the number of days with drug wastage (i.e., days with overlapping palbociclib prescriptions due to dosage change) by the average cost reimbursed by payers for one unit of palbociclib. Descriptive analyses were conducted separately by line of therapy for mBC. Results: A total of 473 patients received palbociclib in first (214), second (157), or third (120) line of therapy for mBC. Patients were observed to receive palbociclib for an average of 4.3 months in first line and 4.1 months in second and third line of therapy. The majority of patients started palbociclib on the recommended 125 mg dose and remained on that dose until the end of observation. Dosing patterns and sequences are summarized in Table 1. Among the 214 patients who used palbociclib in first line, 38 (17.8%) had a dose modification – among these, 6 (15.8%) patients had an overlap in prescription fills (average overlap of 9.2 days). This potential drug wastage resulted in an average cost of $4,376 per patient over a period of approximately 4 months following treatment initiation. Results were consistent in second and third lines of therapy, with higher proportions of patients with drug wastage in later lines of therapy. Conclusion: Over a short observation period, dose modifications, mostly dose reductions, were relatively frequent, and potential resulting drug wastage was associated with a substantial economic burden. Table 1: Palbociclib dosing patterns and sequences First lineSecond lineThird lineStarting dose (mg daily), N (%) 757 (3.3)3 (1.9)3 (2.5)10015 (7.0)17 (10.8)4 (3.3)125 - recommended dose192 (89.7)137 (87.3)113 (94.2)Average dose on treatment, mean±SD [median]119.52±11.27 [125]115.95±13.61 [125]116.85±13.30 [125]Dose sequencing (mg daily), N (%) 125163 (76.2)96 (61.1)74 (61.7)125→10021 (9.8)29 (18.5)26 (21.7)125→100→756 (2.8)8 (5.1)7 (5.8)125→100→1250 (0.0)1 (0.6)1 (0.8)125→752 (0.9)3 (1.9)4 (3.3)125→75→1000 (0.0)0 (0.0)1 (0.8)10010 (4.7)9 (5.7)1 (0.8)100→752 (0.9)7 (4.5)3 (2.5)100→1253 (1.4)0 (0.0)0 (0.0)100→75→1000 (0.0)1 (0.6)0 (0.0)753 (1.4)3 (1.9)3 (2.5)75→1002 (0.9)0 (0.0)0 (0.0)75→100→1251 (0.5)0 (0.0)0 (0.0)75→1251 (0.5)0 (0.0)0 (0.0) Citation Format: Dalal AA, Gagnon-Sanschagrin P, Burne R, Guerin A, Gauthier G, Small T, Niravath P. Dosing patterns and economic burden of drug wastage among postmenopausal women with HR+/HER2- metastatic breast cancer receiving palbociclib [abstract]. In: Proceedings of the 2017 San Antonio Breast Cancer Symposium; 2017 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2018;78(4 Suppl):Abstract nr P5-20-15.

Objective: To describe the setting, duration, and costs of induction and consolidation chemotherapy for adults with newly-diagnosed acute myeloid leukemia (AML), who are candidates for standard induction chemotherapy, in the US. Methods: Adults newly-diagnosed with AML who received standard induction chemotherapy in an inpatient setting were identified from the Truven Health Analytics MarketScan (2006–2015) and SEER-Medicare (2007–2011) databases. Patients were observed from induction therapy start to the first of hematopoietic stem cell transplant, 180 days after induction discharge, health plan enrollment/data availability end, or death. Induction and consolidation chemotherapy were identified using Diagnosis-Related Group codes (chemotherapy with acute leukemia) or procedure codes for AML chemotherapy administration. AML treatment episode setting (inpatient or outpatient), duration, and costs (2015 USD, payers’ perspective) were described for commercially insured patients and Medicare beneficiaries. Results: In total, 459 commercially insured patients and 563 Medicare beneficiaries (mean age = 54 and 66 years; 53% and 54% male; respectively) were identified. For induction therapy, mean costs were $145,189 for commercially insured patients and $85,734 for Medicare beneficiaries, and median inpatient duration was 31 days (both). Following induction, 64% of commercially insured patients and 53% of Medicare beneficiaries had ≥1 consolidation cycle; 75% and 65% of consolidation cycles were in an inpatient setting, respectively. For consolidation cycles, in the inpatient setting, mean costs were $28,137 for commercially insured patients and $28,843 for Medicare beneficiaries, median cycle duration was 6 days (both); in the outpatient setting, mean costs were $11,271 for commercially insured patients and $5,803 Medicare beneficiaries, median duration was 5 days (both). Limitations: Granular information on chemotherapy type administered was unavailable. Conclusions: This is the first exploratory study providing a complete picture of recent AML treatment patterns and management costs among commercially insured patients and Medicare beneficiaries. There is substantial heterogeneity in the management and costs of AML.

Background: Bendamustine-based regimens are often used in the management of patients with chronic lymphocytic leukemia (CLL) but few studies have analyzed the comorbidity- and/or adverse event (CAE)-related healthcare costs in patients receiving these regimens in a real-world setting. Aims: To describe all-cause and CAE-related healthcare costs in relapse/refractory (R/R) elderly patients with CLL treated with bendamustine-based regimens in a real-world setting. Methods: Adult patients with R/R CLL who received bendamustine-based regimens on/after January 2010 were selected from the Medicare Limited Data Set (LDS) 5% Standard Analytic Files. Selected patients were classified into cohorts based on the two most prevalent bendamustine-based regimens observed (index treatment): 1) bendamustine + rituximab (BR cohort) and 2) bendamustine monotherapy (B-mono cohort). For each cohort, all-cause and CAE-related healthcare costs, while on treatment, were reported per-patient-per-month (PPPM). Overall survival (OS) rates following initiation of the index treatment were described using age- and gender-adjusted Kaplan-Meier curves. Results: A total of 275 patients were included in the BR cohort and 100 patients in the B-mono cohort. Most patients were male and the mean age was approximately 75 years old. During treatment, total all-cause healthcare costs were $14,520 PPPM for the BR cohort and $13,125 PPPM for the B-mono cohort—outpatient costs (mainly driven by CLL-drug costs) represented 86.1% of the total all-cause healthcare costs for the BR cohort and 69.8% for the B-mono cohort. CAE costs accounted for 58.3% of the total all-cause healthcare costs for the BR cohort and 66.9% for the B-mono cohort. Median OS was 35 months in the BR cohort and 21 months in the B-mono cohort. Conclusion: In this population of elderly patients with R/R CLL treated with bendamustine-based regimens, CAEs were common and translated into important medical costs. Median OS was also relatively short suggesting an unmet medical need.

Background: Biologics have been shown to improve the outcomes of patients with psoriasis but their cost is an issue. Objective: Determine the number needed to treat (NNT) to achieve a 75%/90% reduction in the Psoriasis Area and Severity Index (PASI-75/90) and evaluate the incremental cost per PASI-75/90 responder (CPR) relative to placebo in Japan. Methods: A network meta-analysis was conducted to estimate the relative probabilities of achieving PASI-75/90 and NNTs. Drug costs were assessed based on Pharmaceutical and Medical Device Agency-approved dosing. The CPR was estimated for a short-term induction period and first year of treatment. Results: Compared with placebo, the PASI-75 NNT was 1.27 for adalimumab 80 mg, 1.29 for secukinumab 150 mg, 1.36 for secukinumab 300 mg, 1.57 for adalimumab 40 mg, 1.68 for ustekinumab 90 mg, 1.97 for ustekinumab 45 mg and 2.00 for infliximab 5 mg/kg. The short-term PASI-75 CPR relative to placebo was $5,062 for secukinumab 150 mg, $8209 for adalimumab 40 mg, $10,654 for secukinumab 300 mg, $11,754 for adalimumab 80 mg, $15,407 for ustekinumab 45 mg, $19,147 for infliximab 5 mg/kg and $26,257 for ustekinumab 90 mg. A similar ranking was observed for one-year PASI-75 CPRs and PASI-90 NNTs and CPRs. Conclusion: Adalimumab 40 mg/80 mg and secukinumab 150 mg/300 mg were the most efficacious and cost-efficient for patients with psoriasis in Japan.