Elranatamab efficacy in MagnetisMM-3 compared with real-world control arms in triple-class refractory multiple myeloma.

Elranatamab efficacy in the single-arm, registrational MagnetisMM-3 trial (NCT04649359) was compared with that of physician's choice of treatment (PCT) for triple-class refractory multiple myeloma. MagnestisMM-3 eligibility criteria were applied to two USA-based oncology electronic health record databases, COTA and Flatiron Health (FH), to identify cohorts for this study (NCT05932290). Applied statistical techniques accounted for cohort imbalances. MagnetisMM-3 (BCMA-naive; n = 123) outcomes were compared with those from COTA (n = 239) and FH (n = 152). Elranatamab was associated with a significantly higher objective response rate (risk ratios, 1.88-2.25), significantly longer progression-free survival (hazard ratios [HRs], 0.37-0.57), and, across most analyses, significantly longer overall survival (HRs, 0.46-0.66) versus PCT. BCMA-naive patients who were treated with elranatamab exhibited significantly better outcomes than patients treated in real-world clinical practice.

Elranatamab is a new medicine for the treatment of people with multiple myeloma. In the ongoing clinical trial MagnetisMM-3, most people had fewer myeloma cells when treated with elranatamab. However, MagnetisMM-3 only looks at the effects of elranatamab without comparing it to other myeloma treatments. Therefore, a new study was designed to compare the effectiveness of elranatamab in the MagnetisMM-3 study with other treatments used in real-world clinical practice (not in a clinical trial). Data from people in MagnetisMM-3 was compared with data from two US databases (COTA and Flatiron Health) containing health records of patients treated for multiple myeloma in real-life clinical practice. The same criteria used to select patients for the MagnetisMM-3 trial (123 people) were used to identify people with similar characteristics in COTA (239 people) and Flatiron Health (152 people). More people treated with elranatamab had fewer myeloma cells in their bodies after treatment than people who received their doctor's choice of treatment in clinical practice. In fact, six out of ten people treated with elranatamab had fewer myeloma cells versus about three in ten people from each real-world database. People treated with elranatamab versus physician's choice of treatment lived longer without their disease getting worse and lived longer overall. In conclusion, this study found that more people treated with elranatamab responded to treatment and lived longer than similar people from the COTA and Flatiron Health databases who were given treatments available in a real-world clinical setting.Clinical Trial Registration: NCT05932290 (ClinicalTrials.gov).

An economic evaluation of tofacitinib for the treatment of moderately-to-severely active ulcerative colitis: modeling the cost of treatment strategies in the United States.

Aims: To evaluate the cost differences between a treatment strategy including tofacitinib (TOFA) vs treatment strategies including adalimumab (ADA), golimumab (GOL), infliximab (IFX), and vedolizumab (VEDO) among all patients with moderate-to-severe ulcerative colitis (UC) (further stratified by patients naïve/exposed to tumor necrosis factor inhibitors [TNFis]). Materials and methods: An Excel-based decision-analytic model was developed to evaluate costs from the perspective of a third-party US payer over 2 years. Efficacy and safety parameters were taken from prescribing information and published trials. All patients started induction therapy on the first treatment in the strategy and continued if efficacy criteria were met and no major adverse event occurred (in which cases they proceeded to the next treatment in the strategy). Results: The cost per member per month (PMPM) of the TOFA->IFX->VEDO->GOL strategy ($1.11) was lower than that of the ADA->IFX->VEDO->GOL strategy ($1.34; Δ = $-0.23) among the TNFi-naïve population (n = 204 patients out of a plan of one million members). Similarly, the use of TOFA before ADA (i.e. TOFA->ADA->IFX-> VEDO) was also associated with lower PMPM costs than the use of ADA before TOFA (i.e. ADA->TOFA->IFX->VEDO): $1.15 vs $1.25 (Δ = $-0.10). Similar, and often larger, differences were observed in both the overall moderate-to-severe population and the TNFi-exposed population. Sensitivity analyses resulted in the same conclusions. Limitations: Our model relied on efficacy data from prescribing information and published trials, which were not head-to-head and slightly differed with respect to methods. Additionally, our model used representative minor and major ADRs (and the associated costs) to represent toxicity management, which was a simplifying assumption. Conclusions: This analysis, the first of its kind to evaluate TOFA vis-à-vis other advanced therapies in the US, suggests the early use of TOFA among both TNFi-naïve and TNFi-failure patients results in lower PMPM costs compared with other treatment alternatives.

The burden of insomnia in Japan.

OBJECTIVES: Several studies have suggested that patients who experience insomnia report a number of significant impairments. However, despite this literature, fewer studies have focused on the burden of insomnia among patients in Japan. The objective of the current study is to extend this work in Japan to further understand the effect of insomnia on health-related quality of life (hrQOL). Further, another objective is to understand general predictors of hrQOL among patients with insomnia. METHODS: Data from the 2012 Japan National Health and Wellness Survey, an annual, cross-sectional study of adults aged 18 years or older, were used (N=30,000). All National Health and Wellness Survey respondents were categorized based on the incidence of self-reported insomnia diagnosis and prescription medication usage (clinical insomniacs under treatment versus [vs] good sleepers without insomnia or insomnia symptoms). Comparisons among different groups were made using multiple regression models controlling for demographics and health history. RESULTS: Clinical insomniacs (n=1,018; 3.4%) reported significantly worse hrQOL compared with good sleepers (n=20,542) (mental component summary: 34.2 vs 48.0; physical component summary: 48.0 vs 52.8; health utilities: 0.61 vs 0.76; all P<0.05). Health behaviors (smoking, exercise, alcohol use) and comorbidities were the strongest predictors of health utilities for clinical insomniacs. For all three clinical insomniac subgroups of interest, those with a physical comorbidity but not a psychiatric one, those with a psychiatric comorbidity but not a physical one, and those without either a physical or psychiatric comorbidity, large decrements in health utilities were observed for respondents who did not engage in any positive health behaviors (0.61, 0.57, 0.64, respectively) relative to good sleepers (0.78). However, the gap in health utility scores between these subgroups and good sleepers diminishes with an increasing number of positive health behaviors (eg, clinical insomniacs with a physical comorbidity but not a psychiatric comorbidity performing all three positive health behaviors =0.67 vs good sleepers =0.78). DISCUSSION: A significant burden remains for those with insomnia who are treated. Given the particularly low levels of hrQOL among treated insomnia patients who have poor health behavior profiles and have psychiatric comorbidities, physicians should place particular emphasis on these patients who are most in need of intervention. Improved treatments may help to address the unmet needs of these patient populations.

The impact of menopausal symptoms on quality of life, productivity, and economic outcomes.

BACKGROUND: The aim of this study was to investigate the impact of menopausal symptoms and menopausal symptom severity on health-related quality of life (HRQoL), work impairment, healthcare utilization, and costs. METHODS: Data from the 2005 United States National Health and Wellness Survey were used, with only women 40-64 years without a history of cancer included in the analyses (N=8,811). Women who reported experiencing menopausal symptoms (n=4,116) were compared with women not experiencing menopausal symptoms (n=4,695) on HRQoL, work impairment, and healthcare utilization using regression modeling (and controlling for demographics and health characteristic differences). Additionally, individual menopausal symptoms were used as predictors of outcomes in a separate set of regression models. RESULTS: The mean age of women in the analysis was 49.8 years (standard deviation,±5.9). Women experiencing menopausal symptoms reported significantly lower levels of HRQoL and significantly higher work impairment, and healthcare utilization than women without menopausal symptoms. Depression, anxiety, and joint stiffness were symptoms with the strongest associations with health outcomes. CONCLUSIONS: Menopausal symptoms can be a significant humanistic and economic burden on women in middle age.

Patient characteristics, treatment patterns, and health outcomes among COPD phenotypes.

BACKGROUND: Recent literature has suggested that emphysema and chronic bronchitis, traditionally considered to be entities overlapping within chronic obstructive pulmonary disease (COPD), may be distinct disorders. Few studies have examined the differences in patient characteristics and health outcomes between these conditions. This study examined whether COPD phenotypes represent distinct patient populations, in a large nationally representative US sample. METHODS: Data were obtained from the 2010 US National Health and Wellness Survey (NHWS). NHWS respondents (n = 75,000) were categorized as a COPD phenotype based on their self-reported diagnosis of COPD only (n = 970), emphysema only (n = 399), or chronic bronchitis only (n = 2071). Phenotypes were compared on demographics, health characteristics, treatment patterns, health outcomes, work productivity, and resource use. Variables were compared using Chi-square and analysis of variance tests for categorical and continuous outcomes, respectively. Health outcomes were also examined using regression modeling, controlling for demographic and health characteristic covariates. RESULTS: Patients with chronic bronchitis were significantly younger (51.38 years versus 63.24 years for COPD versus 63.30 years for emphysema, P < 0.05) and more likely to be employed (46.98% versus 23.81% for COPD versus 28.33% for emphysema, P < 0.05). Relative to the other phenotypes, patients with chronic bronchitis were also significantly more likely to be female, nonwhite, and to exercise currently (all P < 0.05), and were significantly less likely to be a current or former smoker (P < 0.05). Controlling for demographic and health characteristics, patients self-identified as having COPD only reported significantly worse physical quality of life (adjusted mean 36.69) and health utilities (adjusted mean 0.65) and significantly more absenteeism (adjusted mean 7.08%), presenteeism (adjusted mean 30.73%), overall work impairment (adjusted mean 34.06%), and activity impairment (adjusted mean 46.59%) than the other phenotypes (all P < 0.05). CONCLUSION: These results suggest considerable heterogeneity among different COPD phenotypes with respect to demographics, health characteristics, disease characteristics, treatment patterns, and health outcomes. Research aimed at understanding the differences in patient characteristics and disease presentation of these phenotypes could be used to guide treatment recommendations.

Burden of gastrointestinal disease in the United States: 2012 update.

BACKGROUND & AIMS: Gastrointestinal (GI) diseases account for substantial morbidity, mortality, and cost. Statistical analyses of the most recent data are necessary to guide GI research, education, and clinical practice. We estimate the burden of GI disease in the United States. METHODS: We collected information on the epidemiology of GI diseases (including cancers) and symptoms, along with data on resource utilization, quality of life, impairments to work and activity, morbidity, and mortality. These data were obtained from the National Ambulatory Medical Care Survey; National Health and Wellness Survey; Nationwide Inpatient Sample; Surveillance, Epidemiology, and End Results Program; National Vital Statistics System; Thompson Reuters MarketScan; Medicare; Medicaid; and the Clinical Outcomes Research Initiative's National Endoscopic Database. We estimated endoscopic use and costs and examined trends in endoscopic procedure. RESULTS: Abdominal pain was the most common GI symptom that prompted a clinic visit (15.9 million visits). Gastroesophageal reflux was the most common GI diagnosis (8.9 million visits). Hospitalizations and mortality from Clostridium difficile infection have doubled in the last 10 years. Acute pancreatitis was the most common reason for hospitalization (274,119 discharges). Colorectal cancer accounted for more than half of all GI cancers and was the leading cause of GI-related mortality (52,394 deaths). There were 6.9 million upper, 11.5 million lower, and 228,000 biliary endoscopies performed in 2009. The total cost for outpatient GI endoscopy examinations was $32.4 billion. CONCLUSIONS: GI diseases are a source of substantial morbidity, mortality, and cost in the United States.

Incorporating indirect costs into a cost-benefit analysis of laparoscopic adjustable gastric banding.

OBJECTIVES: The objective of this study was to estimate the time to breakeven and 5-year net costs of laparoscopic adjustable gastric banding (LAGB) taking both direct and indirect costs and cost savings into account. METHODS: Estimates of direct cost savings from LAGB were available from the literature. Although longitudinal data on indirect cost savings were not available, these estimates were generated by quantifying the relationship between medical expenditures and absenteeism and between medical expenditures and presenteeism (reduced on-the-job productivity) and combining these elasticity estimates with estimates of the direct cost savings to generate total savings. These savings were then combined with the direct and indirect costs of the procedure to quantify net savings. RESULTS: By including indirect costs, the time to breakeven was reduced by half a year, from 16 to 14 quarters. After 5 years, net savings in medical expenditures from a gastric banding procedure were estimated to be $4970 (±$3090). Including absenteeism increased savings to $6180 (±$3550). Savings were further increased to $10,960 (±$5864) when both absenteeism and presenteeism estimates were included. CONCLUSIONS: This study presented a novel approach for including absenteeism and presenteeism estimates in cost-benefit analyses. Application of the approach to gastric banding among surgery-eligible obese employees revealed that the inclusion of indirect costs and cost savings improves the business case for the procedure. This approach can easily be extended to other populations and treatments.

The burden of illness for patients with viral hepatitis C: evidence from a national survey in Japan.

OBJECTIVE: Viral hepatitis C (HCV) affects 170 million patients worldwide and 2 million patients in Japan. The objective of the current study was to examine the burden of HCV in Japan from a patient's perspective. METHODS: Using data from the 2008 and 2009 Japan National Health and Wellness Surveys, patients who reported an HCV diagnosis (n = 306) were compared with a propensity-score-matched control group (n = 306) on measures of quality of life (using the Medical Outcomes Study 12-Item Short Form Survey Instrument version 2), work productivity (using the Work Productivity and Activity Impairment questionnaire), and health-care resource use. All analyses applied sampling weights to project to the population. RESULTS: Prior to matching, patients with HCV had higher rates of hepatocellular carcinoma (4.88% vs. 0.02%) and cirrhosis (12.20% vs. 0.11%) than did subjects without HCV. The propensity-matching process eliminated differences between the two groups on demographics and patient characteristics. The postmatching analysis found significantly lower levels of quality of life for patients with HCV as measured by bodily pain (72.07 vs. 76.28), general health (44.64 vs. 48.61), and mental health (66.50 vs. 70.32) (all Ps < 0.05). Furthermore, compared with the matched group, the HCV group had significantly higher workplace absenteeism (8.59% vs. 4.12%), overall work impairment (26.08% vs. 17.32%), and health-care provider visits in the past 6 months (14.80 vs. 9.74). CONCLUSIONS: The results of this study suggest that HCV can be a substantial burden on patients in terms of quality of life in both physical and mental health measures. In addition, HCV can be a significant cost driver in terms of health-care use and lost productivity.

Direct and indirect costs and potential cost savings of laparoscopic adjustable gastric banding among obese patients with diabetes.

OBJECTIVE: To estimate the time to breakeven and 5-year net costs for laparoscopic adjustable gastric banding among obese patients with diabetes taking direct and indirect costs into account. METHODS: Indirect cost savings were generated by quantifying the cross-sectional relationship between medical expenditures and absenteeism and between medical expenditures and presenteeism (reduced on-the-job productivity) and simulating indirect cost savings based on these multipliers and reductions in direct medical costs available in the literature. RESULTS: Time to breakeven was estimated to be nine quarters with and without the inclusion of indirect costs. After 5 years, net savings increase from $26570 (±$9000) to $34160 (±$10 380) when indirect costs are included. CONCLUSION: This study presented a novel approach for incorporating indirect costs into cost-benefit analyses. Application to gastric banding revealed that inclusion of indirect costs improves the financial outlook for the procedure.

Musculoskeletal pain in the workforce: the effects of back, arthritis, and fibromyalgia pain on quality of life and work productivity.

OBJECTIVE: To investigate the impact of musculoskeletal pain on health-related quality of life and work productivity losses among US workers. METHODS: Data from the 2008 US National Health and Wellness Survey were used. Among those currently employed aged 20 to 64 years (N = 30,868), workers with arthritis (n = 2,670), back (n = 4,920), and fibromyalgia (n = 439) pain were compared with workers without those respective musculoskeletal pain conditions. RESULTS: Arthritis, back, and fibromyalgia pain were all associated with significantly lower levels of health-related quality of life, often at clinically meaningful levels. All pain conditions were associated with higher levels of work productivity loss, even after adjusting for demographic and health characteristics. CONCLUSIONS: Musculoskeletal pain conditions were highly prevalent and associated with a significant burden. Improved management of these conditions may lead to improved productivity, benefiting both employers and workers alike.

A computerized Stroop task to assess cancer-related cognitive biases.

Biases in processing information related to sources of stress have widely been demonstrated with the use of Stroop emotional color word tasks. One study reported such biases among women with histories of breast cancer in a first-degree relative (FH+) who were given a Stroop cancer word task. This study aimed to replicate and extend these findings with a computerized version of the task. Response latencies and errors were recorded during administration of the task to FH+ and FH- women. A cancer list and 5 comparison lists were administered. Results indicated that FH+ women exhibited longer response latencies for cancer words than did FH- women (p < 0.04), providing further support for cognitive biases in FH+ women. Confirming the psychometric properties of the task, lists exhibited high reliability for both latency (alphas 0.96-0.98) and error rate (alphas 0.61-0.79). In sum, results support the favorable psychometrics and predictive validity of the Stroop cancer word task.