Prognostic Understanding, Goals of Care, and Quality of Life in Hospitalized Patients with Leukemia or Multiple Myeloma.

Background: Prior studies reveal a lack of illness understanding and prognostic awareness among patients with hematological malignancies. We evaluated prognostic awareness and illness understanding among patients with acute leukemia and multiple myeloma (MM) and measured patient-hematologist discordance. Methods: We prospectively enrolled patients with acute leukemia and MM at Mount Sinai Hospital or Yale New Haven Hospital between August 2015 and February 2020. Patients were administered a survey assessing prognostic awareness, goals of care (GOC), and quality of life. Hematologists completed a similar survey for each patient. We assessed discordance across the cohort of patients and hematologists using the likelihood-ratio chi-square test and within patient-hematologist pairs using the kappa (κ) statistic. Results: We enrolled 185 patients (137 with leukemia and 48 with MM) and 29 hematologists. Among patients, 137 (74%) self-identified as White, 27 (15%) as Black, and 21 (11%) as Hispanic. Across the entire cohort, patients were significantly more optimistic about treatment goals compared with hematologists (p = 0.027). Within patient-hematologist pairs, hematologists were significantly more optimistic than patients with respect to line of treatment (κ = 0.03). For both leukemia and MM cohorts, patients were significantly more likely to respond "don't know" or deferring to a faith-based response with 88 (64%) and 34 (71%), respectively, compared with only 28 (20%) and 11 (23%) of hematologists, respectively. Conclusions: We observed significant discordance regarding prognosis and GOC among patients with hematological malignancies and their hematologists. These data support future interventions to improve prognostic understanding among this patient population to facilitate informed treatment choices.

Panel Interview of ONcology practices with Emergent Experience of teclistamab in the Real world: the TecPIONEER Study.

OBJECTIVE: To understand clinicians' current teclistamab step-up dosing (SUD) model and how they envision future administration models, as well as perceived barriers and facilitators to these models in day-to-day clinical practice. METHODS: Interviews of clinicians with RW experience administering teclistamab, with a subsequent roundtable discussion to discuss interview findings. Topics of interest included managing adverse events (AE), and handling logistics of SUD and transition of care (ToC). RESULTS: 20 clinicians representing 19 practices participated. Of 14 practices administering inpatient teclistamab SUD, 12 (86%) utilized a single admission. A day 1-3-5 dosing schedule with a 7-day length of stay was planned in 10/14 (71%). The remaining 5 practices employed outpatient or hybrid SUD. SUD models depended on cellular therapy experience, patient volume, and monitoring capabilities. Clinicians desired to administer SUD outpatient for convenience and reduced healthcare resource use. 11% of practices reported using tocilizumab for cytokine release syndrome (CRS) prophylaxis, whilst it was uniformly used to treat grade 2+ CRS. Corticosteroids were the preferred treatment for neurotoxicity. Infection prophylaxis with intravenous immunoglobulin was reported by 89% of practices. Patient- and institution-level factors affected decision-making of transitioning patients back to referring sites after SUD. CONCLUSION: The results consolidated practice-based experiences and indicated diverse RW SUD models and patient management strategies in practices with familiarity with teclistamab AE management and ToC protocols. Inpatient SUD is common, with expectations that approaches will evolve toward outpatient or community-based administration. Further research is needed to investigate outcomes of different care models and AE management strategies.

Multiple myeloma is a blood cancer that forms in plasma cells. Teclistamab is a new treatment for patients with multiple myeloma who have received prior treatment but for whom their multiple myeloma has come back or stopped responding to treatment multiple times. Because teclistamab works differently than other existing multiple myeloma treatments, there is a need to understand how oncologists who have experience with teclistamab are managing their patients in order to inform best practices for use by more healthcare providers. We interviewed oncologists that treat patients with multiple myeloma to understand their experiences with teclistamab, including how they manage initial dosing (step-up dosing) processes, treat adverse events, and transition patients to outpatient or external clinics for continued care. Most practices were administering step-up dosing of teclistamab in an inpatient setting soon after teclistamab became a treatment option, with a high level of desire to move the initial dosing to an outpatient setting in the near future. Those that were already administering step-up dosing in an outpatient setting had models unique to their practice. Oncologists described numerous processes for monitoring and managing adverse events of the treatment, including treating patients with preventative medications and regularly monitoring vital signs throughout step-up dosing. Oncologists expected that their teclistamab administration processes will likely evolve over time as they gain more familiarity with the treatment, and will need to consider patient-level factors to administer step-up dosing in an outpatient setting.

Patient perspectives on BCMA-targeted therapies for multiple myeloma: a survey conducted in a patient advocacy group.

Background: Advances in multiple myeloma (MM) treatment have shifted the therapeutic landscape. Understanding patients' perspectives can assist physicians in helping patients make informed decisions. This study aimed to understand the patient decision-making process and gain insights into patient perspectives on B-cell maturation antigen (BCMA)-targeted therapies for MM. Methods: An 18-question survey was completed by patients with MM enrolled in HealthTree® Cure Hub, an online portal helping patients with plasma cell dyscrasias navigate their disease. Results: From October 28, 2022, to January 12, 2023, 325 patients with MM participated in the survey. The mean age (standard deviation) of the respondents was 66 (8) years; 54% were female and 90% were White. Among 218 patients with complete clinical records in the database, the median (min, max) lines of therapy (LOT) was 2 (1,16). Among 61 (28%) patients who had received ≥4 LOTs, 55 (90%) were triple-class exposed. Of the 290 patients who responded to the question about openness to new therapies, 76 (26%) were open to trying a new therapy immediately and 125 (43%) wanted more information on safety and efficacy. Most respondents reported likely or very likely to try a BCMA CAR T-cell therapy (60%) or a bispecific antibody (74%) and some needed more information to decide (16% for CAR T-cell therapy and 13% for bispecific antibody). The most requested information included efficacy, side effects (SEs), eligibility, and administration process for both CAR T-cell and bispecific therapies. When 2 therapies with the same efficacy and duration of response were offered, 69% of respondents would prefer the therapy with a lower risk of severe SEs but requires continuous dosing with no treatment-free interval, and 31% preferred a therapy given once followed by a treatment-free interval but with a potentially higher risk of severe SEs. To receive an effective therapy, the top acceptable trade-offs included frequent monitoring of SEs and initiating a new therapy in a hospital setting, and the least acceptable compromise was caregiver burden. Conclusions: This study found a high level of openness in patients with MM to try BCMA-targeted therapies. Information on efficacy, safety, availability, and eligibility may assist patients on decision-making.

A Comparison of Patients' and Neurologists' Assessments of their Teleneurology Encounter: A Cross-Sectional Analysis.

Background and Objectives: To better understand patients' and neurologists' assessments of their experiences regarding effectiveness of teleneurology encounters. Methods: Following an audio-video telehealth visit, neurologists asked patients to participate in a survey-based research study about the encounter, and then, the neurologists also recorded their own evaluations. Data were analyzed using standard quantitative and qualitative techniques for dichotomous and ordered-category survey responses in this cross-sectional analysis. Results: The study included unique encounters between 187 patients and 11 general neurologists. The mean patient age was 49 ± 17.5 years. Two thirds of the patients (66.8%, 125/187) were female. One third (33.2%; 62) were patients new to the NYU Langone Health neurology practices. The most common patient chief complaints were headache (69/187, 36.9%), focal and generalized numbness or tingling (21, 11.2%), memory difficulty (15, 8%), spine-related symptoms (12, 6.4%), and vertigo (11, 5.9%). Most patients (94.7%, 177/187) reported that the teleneurology encounter satisfied their needs. Patients and their neurologists agreed that the experience was effective in 91% (162/178) of encounters, regardless of whether the visit was for a new or established patient visit. Discussion: More than 90% of new and established patients and their neurologists agreed that teleneurology encounters were effective despite some limitations of the examination, the occasional need for patient assistance, and technical difficulties. Our results provide further evidence to justify and to expand the clinical use of teleneurology.

Chronic hepatitis B and HIV coinfection.

Hepatitis B virus (HBV) infection is common among people with HIV owing to shared modes of viral transmission. Compared with individuals with HBV infection alone, people with HIV/HBV coinfection experience an accelerated progression of liver disease, including increased risks for hepatocellular carcinoma, liver-related mortality, and all-cause mortality. Therefore, HBV screening and appropriate treatment are crucial for people with HIV. This article reviews the epidemiology, natural history, and management of HIV/HBV coinfection, as well as recommendations for prevention of HBV infection among people with HIV.

The Relative Value of Anti-Obesity Medications Compared to Similar Therapies.

Purpose: To demonstrate a need for improved health insurance coverage for anti-obesity medications (AOMs) by comparing clinical and economic benefits of obesity treatments to covered medications for selected therapeutic areas. Methods: Using a grey literature search, we identified and prioritized therapeutic areas and treatment analogues for comparison to obesity. A targeted literature review identified clinical and economic outcomes research across the therapeutic area analogues. Associated comorbidities, clinical evidence, indirect costs (ie, absenteeism and productivity loss), and direct medical costs were evaluated to determine the relative value of treating obesity. Results: Four therapeutic areas/treatment analogues were selected for comparison to obesity: smoking cessation (varenicline), daytime sleepiness (modafinil), migraines (erenumab), and fibromyalgia (pregabalin). Obesity was associated with 17 comorbidities, more than migraine (9), smoking (8), daytime sleepiness (5), and fibromyalgia (2). Economic burden was greatest for obesity, followed by smoking, with yearly indirect and direct medical costs totaling $676 and $345 billion, respectively. AOMs resulted in cost savings of $2586 in direct medical costs per patient per year (PPPY), greater than that for varenicline at $930 PPPY, modafinil at $1045 PPPY, and erenumab at $468 PPPY; pregabalin utilization increased costs by $924 PPPY. AOMs were covered by 10-16% of United States health insurance plans, compared to 45-59% for the four comparators. Conclusion: Compared to four therapeutic analogues, obesity represented the highest economic burden and was associated with more comorbidities. AOMs provide greater cost savings compared to selected analogues. However, AOMs have limited formulary coverage. Improved coverage of AOMs may increase access to these treatments and may help address the clinical and economic burden associated with obesity and its comorbidities.

Clinical and Cost Benefits of Anti-Obesity Medication for US Veterans Participating in the MOVE! Weight Management Program.

Abstract This study investigated the clinical and economic impact of anti-obesity medications (AOMs; orlistat, liraglutide, phentermine/topiramate extended-release [ER], naltrexone ER/bupropion ER) among United States Veterans with obesity participating in Motivating Overweight/Obese Veterans Everywhere! (MOVE!), a government-initiated weight management program. The study population was identified from electronic medical records of the Veterans Health Administration (2010-2020). Clinical indices of obesity and health care resource utilization and costs were evaluated at 6, 12, and 24 months after the initial dispensing of an AOM in the AOM+MOVE! cohort (N = 3732, mean age 57 years, 79% male) or on the corresponding date of an inpatient or outpatient encounter in the MOVE! cohort (N = 7883, mean age 58 years, 81% male). At 6 months postindex, the AOM+MOVE! cohort had better cardiometabolic indices (eg, systolic blood pressure, diastolic blood pressure, total cholesterol, low-density lipoprotein cholesterol, hemoglobin A1c) than the MOVE! cohort, with the trends persisting at 12 and 24 months. The AOM+MOVE! cohort was significantly more likely than the MOVE! cohort to have weight decreases of 5%-10%, 10%-15%, and >15% and lower body mass index at 6, 12, and 24 months. The AOM+MOVE! cohort also had fewer inpatient and emergency department visits than the MOVE! cohort, which was associated with lower mean total medical costs including inpatient costs. These results suggest that combining AOM treatment with the MOVE! program could yield long-term cost savings for the Veterans Affairs network and meaningful clinical improvements for Veterans with obesity.

Burden of illness associated with overweight and obesity in patients hospitalized with COVID-19 in the United States: analysis of the premier healthcare database from April 1, 2020 to October 31, 2020.

BACKGROUND: SARS-CoV-2 (COVID-19) continues to be a major public health issue. Obesity is a major risk factor for disease severity and mortality associated with COVID-19. OBJECTIVE: This study sought to estimate the healthcare resource use and cost outcomes in patients hospitalized with COVID-19 in the United States (US) according to body mass index (BMI) class. METHODS: Retrospective cross-sectional study analyzing data from the Premier Healthcare COVID-19 database for hospital length-of-stay (LOS), intensive care unit (ICU) admission, ICU LOS, invasive mechanical ventilator use, invasive mechanical ventilator use duration, in-hospital mortality, and total hospital costs from hospital charge data. RESULTS: After adjustment for patient age, gender, and race, patients with COVID-19 and overweight or obesity had longer durations for mean hospital LOS (normal BMI = 7.4 days, class 3 obesity = 9.4 days, p < .0001) and ICU LOS (normal BMI = 6.1 days, class 3 obesity = 9.5 days, p < .0001) than patients with normal weight. Patients with normal BMI had fewer days on invasive mechanical ventilation compared to patients with overweight and obesity classes 1-3 (6.7 days vs. 7.8, 10.1, 11.5, and 12.4, respectively, p < .0001). The predicted probability of in-hospital mortality was nearly twice that of patients with class 3 obesity compared to patients with normal BMI (15.0 vs 8.1%, p < .0001). Mean (standard deviation) total hospital costs for a patient with class 3 obesity is estimated at $26,545 ($24,433-$28,839), 1.5 times greater than the mean for a patient with a normal BMI at $17,588 ($16,298-$18,981). CONCLUSIONS: Increasing levels of BMI class, from overweight to obesity class 3, are significantly associated with higher levels of healthcare resource utilization and costs in adult patients hospitalized with COVID-19 in the US. Effective treatment of overweight and obesity are needed to reduce the burden of illness associated with COVID-19.

The COVID-19 pandemic has caused many people to be seriously ill. People who are overweight are more likely to get sicker from COVID-19 infection and to require hospitalization.In our study, we compared patients who have normal weight to people who have overweight or obesity to understand how excess weight affects their experiences with COVID-19. We looked at: (1) how overweight and obesity is related to how long patients with COVID-19 stay in the hospital, (2) if they stayed in the intensive care unit (ICU) and how long they spent there, (3) whether they needed help breathing with the use of a ventilator and how long they needed a ventilator, (4) if they died during their hospital stay, and (5) how much their hospital stay cost.We found that people who have overweight or obesity stayed in the hospital longer, were more likely to need to stay in the ICU, and were in the ICU longer. They were also more likely to need help breathing with the use of a ventilator and needed that help for a longer time. People who have overweight or obesity died during their hospital stay more often than people with a normal BMI. The costs associated with people who have overweight or obesity were higher than people who have a normal BMI.Overall, this study shows that having overweight or obesity is a significant risk factor for poor outcomes from COVID-19 infection. Treatment for obesity and overweight is needed to help improve outcomes from future pandemics.

Considerations for Drug Development in Myelodysplastic Syndromes.

Myelodysplastic syndromes (MDS) have historically been challenging diseases for drug development due to their biology, preclinical modeling, and the affected patient population. In April 2022, the FDA convened a panel of regulators and academic experts in MDS to discuss approaches to improve MDS drug development. The panel reviewed challenges in MDS clinical trial design and endpoints and outlined considerations for future trial design in MDS to facilitate drug development to meaningfully meet patient needs. Challenges for defining clinical benefit in patients with MDS include cumbersome response criteria, standardized transfusion thresholds, and application and validation of patient reported outcome instruments. Clinical trials should reflect the biology of disease evolution, the advanced age of patients with MDS, and how patients are treated in real-world settings to maximize the likelihood of identifying active drugs. In patients with lower-risk disease, response criteria for anemic patients should be based on baseline transfusion dependency, improvement in symptoms, and quality of life. For higher-risk patients with MDS, trials should include guidance to prevent dose reductions or delays that could limit efficacy, specify minimal durations of treatment (in the absence of toxicity or progression), and have endpoints focused on overall survival and durable responses. MDS trials should be designed from the outset to allow the practicable application of new therapies in this high-needs population, with drugs that can be administered and tolerated in community settings, and with endpoints that meaningfully improve patients' lives over existing therapies.

Neurologists' Evaluations of Experience and Effectiveness of Teleneurology Encounters.

Background and Objectives: To better understand neurologists' assessments of the experiences and effectiveness of teleneurology encounters. Methods: After completing an audio-video telehealth visit with verbally consenting patients, neurologists recorded their evaluations of the encounter. Data were analyzed using standard quantitative and qualitative techniques. Results: The study included unique encounters between 187 patients and 11 neurologists. The mean patient age was 49 ± 17.5 years. Two thirds of patients (66.8%, 125/187) were female. One third of patients (33.2%; 62) were new patients. The most common patient complaints were headache (69/187, 36.9%), focal and generalized numbness or tingling (21, 11.2%), memory difficulty (15, 8%), spine-related symptoms (12, 6.4%), and vertigo (11, 5.9%). Neurologists reported that they completed a virtual examination that provided enough information for medical decision-making in 94.9% of encounters (169/178, 9 missing responses). Fourteen of 25 examination elements important for medical decision-making could be performed sufficiently during virtual encounters. Examination assistance was needed for 16.4% (30/183) of patients, who were, on average, 17.3 years older than those who did not require assistance (62.9 years vs. 45.6 years, p = 0.0002). In 19.1% (34/178) of encounters, neurologists learned clinically relevant information from seeing patients in their homes. Neurologists' assessments of the effectiveness of encounters were not related to the presence (97.2%, 35/36 effective) or absence (95%, 134/141 effective) of technical difficulties (p = 0.5729) in 177 encounters (10 missing responses). Discussion: Neurologists reported that nearly 95% of teleneurology encounters were effective despite limitations of the virtual examination, occasional need for patient assistance, and technical difficulties.

Development of an Assessment Tool for Completion by Patients with Overweight or Obesity.

INTRODUCTION: Discussions of weight-management strategies between patients and healthcare providers can yield positive outcomes for people with overweight or obesity. Nonetheless, people with overweight or obesity encounter communication challenges and other barriers to pursuing effective weight-management strategies with their healthcare providers. The aim of this study was to develop a new self-completed assessment tool to initiate and facilitate conversations related to weight management between patients and healthcare providers. METHODS: Developing the assessment tool involved a series of steps and draft versions of the tool, based on feedback from key opinion leaders in the field of obesity (N = 4) and input from people with overweight or obesity (N = 18). Three iterative rounds of qualitative interviews were conducted in the USA. A targeted review of prior qualitative research was conducted to identify common and important impacts of obesity on patients' functioning. Standard qualitative analytical methods were used to identify concepts of importance in a concept elicitation exercise during the interviews and were evaluated for potential inclusion in the tool. Potential problems with the tool were flagged during cognitive debriefing of the draft tool. RESULTS: During 18 individual interviews, participants referenced the impact of their weight on their lives, including health and comorbidities, physical function, emotional/mental functioning, social life, and physical appearance. Over the course of the tool's development, 24 common and important impacts of obesity on patients' functioning were reduced to a final set of eight concepts in the final tool that were deemed important and relevant to both patients and key opinion leaders. CONCLUSIONS: The assessment tool is a five-item, self-completed measure expected to foster patient self-advocacy for individuals with overweight or obesity by giving them an opportunity to define their weight-management goals and discuss these, along with various medical interventions, with a healthcare provider.

Effect of Bacteriocin-Like Inhibitory Substance (BLIS) from Enterococcus faecium DB1 on Cariogenic Streptococcus mutans Biofilm Formation.

The aim of the study was to investigate the effect of bacteriocin-like inhibitory substance (BLIS) from Enterococcus faecium DB1 on cariogenic Streptococcus mutans biofilm. Crystal violet staining, fluorescence, and scanning electron microscopy analyses demonstrated that the BLIS from Enterococcus faecium DB1 (DB1 BLIS) inhibited S. mutans biofilm. When DB1 BLIS was co-incubated with S. mutans, biofilm formation by S. mutans was significantly reduced (p<0.05). DB1 BLIS also destroyed the preformed biofilm of S. mutans. In addition, DB1 BLIS decreased the viability of S. mutans biofilm cells during the development of biofilm formation and in the preformed biofilm. DB1 BLIS significantly decreased the growth of S. mutans planktonic cells. Furthermore, S. mutans biofilm on the surface of saliva-coated hydroxyapatite discs was reduced by DB1 BLIS. Taken together, DB1 BLIS might be useful as a preventive and therapeutic agent against dental caries caused by S. mutans.

Health care utilization and costs associated with functional status in patients with psoriatic arthritis.

BACKGROUND: The Health Assessment Questionnaire Disability Index (HAQ-DI) has been validated and widely used in psoriatic arthritis (PsA) clinical trials for the assessment of patient functional status. Significant improvements in the HAQ-DI have been reported in response to therapeutic interventions; however, few US studies have evaluated the economic impact of functional disability in patients with PsA. OBJECTIVE: To evaluate the association of functional status with health care resource utilization (HCRU) and total health care costs in US patients diagnosed with PsA. METHODS: This retrospective study included adult patients with PsA enrolled in FORWARD between July 2009 and June 2019 who completed 1 or more HAQ-DI questionnaires between January 2010 and December 2019. Patient demographics, clinical characteristics, and patient-reported outcomes were collected from the most recent questionnaire. HCRU and total health care costs (2019 US dollars) for all hospitalizations, emergency department (ED) visits, outpatient visits, diagnostic tests, and procedures were assessed for the 6 months prior to survey completion. Negative binomial regression models (HCRU outcomes) and generalized linear models with γ distribution and log-link function (cost outcomes) were used to assess the relationship between HAQ-DI and HCRU and cost outcomes, respectively. RESULTS: A total of 828 patients with PsA who completed HAQ-DI questionnaires were included. The mean (SD) age was 58.5 (13.5) years, 72.3% were female, and 92.3% were White. The mean (SD) disease duration was 17.5 (12.4) years, and the mean (SD) HAQ-DI score at the time of the patients' most recent questionnaire was 0.9 (0.7). More severe functional disability, measured by higher HAQ-DI score, was significantly associated with increased risk (incident rate ratio [95% CI]) of hospitalizations (1.68 [1.11-2.55]), ED visits (2.09 [1.47-2.96]), outpatient visits (1.14 [1.05-1.24]), and diagnostic tests (1.42 [1.16-1.74]). There was also a significant positive association between greater HAQ-DI score and increased total annualized health care costs (incremental amount [95% CI], 1.13 [1.03-1.23]) and medical costs (1.38 [1.13-1.69]), but there was no significant association found with pharmacy costs. Total adjusted average patient medical costs increased with increasing HAQ-DI score. CONCLUSIONS: Among patients with PsA enrolled in FORWARD, more functional disability-as measured by higher HAQ-DI scores-was associated with greater HCRU and increased total health care costs. These results suggest that improving functional status in patients with PsA may reduce economic burden for health care payers and systems. DISCLOSURES: Dr Ogdie has received consulting fees from Amgen, AbbVie, Bristol Myers Squibb, Celgene, CorEvitas (formerly Corrona), Gilead, Janssen, Lilly, Novartis, Pfizer, and UCB and has received grant support from the National Institutes of Health/National Institute of Arthritis and Musculoskeletal and Skin Diseases, Rheumatology Research Foundation, National Psoriasis Foundation, Pfizer (University of Pennsylvania), Amgen (FORWARD), and Novartis (FORWARD). Dr Hwang has received consulting fees from Novartis and UCB and has received grant support (5KL2TR003168-03) from the University of Texas Health Science Center at Houston Center of Clinical and Translational Sciences KL2 program. Drs Veeranki and Shafrin were employees of PRECISIONheor at the time of this analysis. Ms Portelli and Mr Sison are employees of PRECISIONheor. Ms Pedro has nothing to disclose. Dr Hass is an employee of H. E. Outcomes, providing consulting services to Novartis. Dr Hur was an employee of Novartis at the time of this analysis. Dr Kim was a postdoctoral fellow at the University of Texas at Austin and Baylor Scott and White Health, providing services to Novartis at the time of this analysis. Dr Yi is an employee of Novartis. Dr Michaud received grant funding from the Rheumatology Research Foundation at the time of this analysis. This study was funded by Novartis Pharmaceuticals Corporation, East Hanover, NJ.

Association of health care utilization and costs with patient-reported outcomes in patients with ankylosing spondylitis.

BACKGROUND: Interventions for ankylosing spondylitis (AS) have improved patient-reported outcomes (PROs) in clinical studies. However, limited data exist associating these improvements with health care resource utilization (HCRU) or cost savings. Few studies have evaluated the economic impact of patient-reported physical status and related disease burden in patients with AS in the United States. OBJECTIVE: To assess the association of PRO measures with HCRU and health care costs in patients with AS from a national US registry. METHODS: This cohort study included adults with a diagnosis of AS enrolled in the FORWARD registry from July 2009 to June 2019 who completed at least 1 questionnaire from January 2010 to December 2019 and completed the Health Assessment Questionnaire Disability Index (HAQ-DI) (0-3) and/or Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) (0-10). Patient-reported data for demographics, clinical characteristics, and PROs were collected through questionnaires administered biannually and reported from the most recent questionnaire. Patient-reported HCRU and total health care costs (2019 US dollars) for hospitalizations, emergency department (ED) visits, outpatient visits, diagnostic tests, and procedures were captured during the 6 months prior to the most recent survey completion. The relationship between HAQ-DI or BASDAI and HCRU outcomes was assessed using negative binomial regression models, and the relationship between HAQ-DI or BASDAI and the cost outcomes was evaluated using generalized linear models with γ distribution and log-link function. RESULTS: Overall, 334 patients with AS who completed the HAQ-DI (n = 253) or BASDAI (n = 81) were included. The mean (SD) HAQ-DI and BASDAI scores at the time of patients' most recent surveys were 0.9 (0.7) and 3.7 (2.3), respectively. HAQ-DI score was positively associated with number of hospitalizations, ED visits, outpatient visits, and diagnostic tests, whereas BASDAI was not associated with HCRU outcomes. Overall annualized mean (SD) total health care, medical, and pharmacy costs for patients with AS were $44,783 ($40,595); $6,521 ($12,733); and $38,263 ($40,595), respectively. Annualized total health care, medical, and pharmacy costs adjusted for confounders increased by 35%, 76%, and 26%, respectively, for each 1.0-unit increase in HAQ-DI score (coefficient [95% CI]: 1.35 [1.15-1.58], 1.76 [1.22-2.55]; both P < 0.01 and 1.26 [1.04-1.52]; P < 0.05, respectively); BASDAI score was not significantly associated with cost outcomes. CONCLUSIONS: Higher HAQ-DI scores were associated with higher HCRU and total health care costs among patients with AS in FORWARD, but BASDAI scores were not. These findings indicate that greater functional impairment may impose an increased economic burden compared with other patient-reported measures of AS. DISCLOSURES: A. Ogdie has received consulting fees from Amgen, AbbVie, Bristol Myers Squibb, Celgene, CorEvitas (formerly Corrona), Gilead, Janssen, Lilly, Novartis, Pfizer, and UCB and has received grant support from the National Institutes of Health/National Institute of Arthritis and Musculoskeletal and Skin Diseases, Rheumatology Research Foundation, National Psoriasis Foundation, Pfizer (University of Pennsylvania), Amgen (FORWARD), and Novartis (FORWARD). M. Hwang has received consulting fees from Novartis and UCB and has received grant support (5KL2TR003168-03) from the University of Texas Health Science Center at Houston Center of Clinical and Translational Sciences KL2 program. P. Veeranki and J. Shafrin were employees of PRECISION-heor at the time of this analysis. A. Portelli and S. Sison are employees of PRECISION-heor. S. Pedro does not have anything to disclose. N. Kim was a postdoctoral fellow at the University of Texas at Austin and Baylor Scott and White Health, providing services to Novartis at the time of this study. E. Yi is an employee of Novartis. K. Michaud received grant funding from the Rheumatology Research Foundation at the time of this analysis. This study was funded by Novartis Pharmaceuticals Corporation, East Hanover, NJ.

Economic outcomes of antiobesity medication use among adults in the United States: A retrospective cohort study.

BACKGROUND: Obesity prevalence exceeds 40% in the US adult population, posing a substantial burden on the health care system. Antiobesity medication (AOM) is recommended for obesity management. However, little evidence exists estimating the economic impact of AOMs on health care costs over time. OBJECTIVE: To estimate the impact of AOMs indicated for long-term therapy on shortterm direct medical costs, by obesity class, in a commercially insured population. METHODS: For this retrospective cohort study, we used the IBM MarketScan Commercial Claims and Encounters Database to capture health care utilization between January 1, 2015, and December 31, 2019. Adults aged 18-63 years with a body mass index greater than or equal to 30 kg/m2 were categorized into 2 cohorts based on new AOM usage at cohort entry. New AOM users were taking 1 of 4 AOMs currently approved by the US Food and Drug Administration for long-term therapy, with greater than 112 days supply of medicine within 12 months after treatment initiation. AOM nonusers were those not taking an AOM indicated for long-term therapy during the baseline or follow-up period. We used difference-in-differences estimation to calculate the change in average annual total health care costs and cost of medications (excluding AOMs) over a 2-year follow-up period using inverse probability of treatment-weighted estimates. RESULTS: The study population included 219,971 patients, 1,405 AOM users and 218,566 AOM nonusers. Over 2 years, patients on treatment were more than twice as likely to be classified into a lower obesity class than AOM nonusers. Although the average yearly direct cost of care increased for both treatment groups in the first year of follow-up, by year 2, costs for untreated patients continued to rise while costs for patients on therapy remained stable or declined. The difference-in-differences of medication cost (excluding AOMs) and total health care cost (excluding AOMs) across all 3 obesity classes in year 2 ranged from $1,321 to $1,952 and $1,323 to $2,766, respectively, indicating a cost savings. Total cost of care, inclusive of AOMs, followed a similar trend. CONCLUSIONS: Use of AOMs is associated with the odds of moving to a lower obesity class and a general stabilization or reduction in health care costs in year 2 of follow-up. When considering change in health care costs over time, use of AOMs may be an effective strategy to mitigate the rising health care costs associated with obesity. DISCLOSURES: Dr Toliver is an employee of Novo Nordisk, Inc. Dr Watkins, Dr Kim, and Ms Whitmire were employees of Novo Nordisk at the time the study was conducted. Dr Garvey has served as a volunteer consultant on advisory committees for Jazz Pharmaceuticals, Boehringer Ingelheim, Eli Lilly, Novo Nordisk, and Pfizer; in each instance, he received no financial compensation, nor was there a financial relationship. He also has served as site principal investigator for clinical trials sponsored by his university and funded by Eli Lilly, Novo Nordisk, Epitomee, and Pfizer. Novo Nordisk funded the study and had a role in the study design, data collection, analysis, and interpretation of data, as well as writing support of the manuscript.

Cost-effectiveness analysis of semaglutide 2.4 mg for the treatment of adult patients with overweight and obesity in the United States.

BACKGROUND: The rising prevalence and associated public health burden of obesity has led to advancements in pharmaceuticals for weight management. Semaglutide 2.4 mg, an anti-obesity medication (AOM) recently approved by the US Food and Drug Administration, has demonstrated clinically relevant weight loss in its phase 3 clinical trials. Economic evaluation comparing semaglutide 2.4 mg with other available weight management therapies is essential to inform payers for decision-making. OBJECTIVES: To assess the cost-effectiveness of semaglutide 2.4 mg in the treatment of adult patients with obesity (ie, body mass index [BMI] ≥ 30) and adult patients who are overweight (ie, BMI 27-29.9) with 1 or more weight-related comorbidities from a US third-party payer perspective. METHODS: A cohort Markov model was constructed to compare semaglutide 2.4 mg with the following comparators: no treatment, diet and exercise (D&E), and 3 branded AOMs (liraglutide 3 mg, phentermine-topiramate, and naltrexone-bupropion). All AOMs, including semaglutide 2.4 mg, were assumed to be taken in conjunction with D&E. Changes in BMI, blood pressure, cholesterol level, experience of acute and chronic obesity-related complications, costs, and quality-adjusted life years (QALYs) were simulated over 30 years based on pivotal trials of the AOMs and other relevant literature. Drug and health care prices reflect 2021 standardized values. Cost-effectiveness was examined with a willingness-to-pay (WTP) threshold of $150,000 per QALY gained. Sensitivity analyses were conducted to test the robustness of the cost-effectiveness results to plausible variation in model inputs. RESULTS: In the base-case analysis, treatment with semaglutide 2.4 mg was estimated to improve QALYs by 0.138 to 0.925 and incur higher costs by $3,254 to $25,086 over the 30-year time horizon vs comparators. Semaglutide 2.4 mg is cost-effective against all comparators at the prespecified WTP threshold, with the incremental cost per QALY gained ranging from $23,556 to $144,296 per QALY gained. In the sensitivity analysis, extended maximum treatment duration, types of subsequent treatment following therapy discontinuation, and weight-rebound rates were identified as key drivers for model results. The estimated probability of semaglutide 2.4 mg being cost-effective compared with comparators ranged from 67% to 100% when varying model parameters and assumptions. CONCLUSIONS: As a long-term weight management therapy, semaglutide 2.4 mg was estimated to be cost-effective compared with no treatment, D&E alone, and all other branded AOM comparators under a WTP threshold of $150,000 per QALY gained over a 30-year time horizon. DISCLOSURES: Financial support for this research was provided by Novo Nordisk Inc. 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. Dr Kim and Ms Ramasamy are employees of Novo Nordisk Inc. Ms Kumar and Dr Burudpakdee were employees of Novo Nordisk Inc at the time this study was conducted. Dr Sullivan received research support from Novo Nordisk Inc for this study. Drs Wang, Song, Wu, Ms Xie, and Ms Sun are employees of Analysis Group, Inc, who received consultancy fees from Novo Nordisk Inc in connection with this study.

FDA Approval Summary: Decitabine and Cedazuridine Tablets for Myelodysplastic Syndromes.

On July 7, 2020, the Food and Drug Administration approved Inqovi (Otsuka Pharmaceutical Co.), an oral fixed-dose combination tablet comprising 35 mg decitabine, a hypomethylating agent, and 100 mg cedazuridine, a cytidine deaminase inhibitor (abbreviated DEC-C) for treatment of adult patients with myelodysplastic syndromes (MDS). Evidence of effectiveness of DEC-C was established in phase III ASTX727-02 (N = 133) in adults with MDS. The study involved a two-sequence crossover comparing DEC-C and intravenous (IV) decitabine 20 mg/m2 once daily for the first 5 days of each 28-day cycle in the first 2 cycles. From cycle 3 onward, patients received DEC-C. Five-day cumulative area under the curve (5-d AUC) of decitabine for DEC-C was similar to that of IV decitabine, with geometric mean ratio 0.99 (90% confidence interval: 0.93-1.06). Clinical benefit was supported by study ASTX727-02 and the similarly designed phase II study ASTX727-01-B (n = 80), with complete remission (CR) of 21% and 18% and median duration of CR 7.5 and 8.7 months, respectively. Adverse reactions were consistent with IV decitabine. Postmarketing assessments were issued to address the effect of cedazuridine on QT prolongation, food effect, moderate and severe hepatic impairment, and severe renal impairment on the pharmacokinetics and safety of DEC-C.