Real-world outcomes in patients with first-line and second-line therapy for advanced esophageal squamous cell carcinoma.

Background: Little is known about real-world outcomes for first-line and anti-PD-1 second-line treatment for advanced/metastatic esophageal squamous cell carcinoma (ESCC). Patients & methods: Retrospective data of advanced/metastatic ESCC patients treated between 2011 and 2021 were collected from Flatiron Health. Median duration of therapy (mDoT) and median overall survival (mOS) were evaluated for patients initiating first-line and anti-PD-1 second-line therapy. Results: Among patients receiving first-line therapy (n = 948), mDoT was 1.4 months and mOS was 16.0 months, with mOS of 16.0 and 18.0 months for the non-immunotherapy and immunotherapy cohorts, respectively. Among patients receiving anti-PD-1 second-line therapy (n = 60), mDoT was 5.7 months and mOS was 10.1 months. Conclusion: Patients with advanced/metastatic ESCC have short duration of therapy, and overall survival remains limited. This real-world study underscores the need for efficacious treatments for advanced/metastatic ESCC in the first- and second-line setting. Direct comparisons of emerging therapies in the real world are urgently needed.

Determinants of patient and physician treatment satisfaction in moderate-to-severe psoriasis: a multinational survey of psoriasis patients.

There is a lack of validated information of both physician and patient-reported treatment satisfaction, and association with outcomes in psoriasis. Data from the 2015 Adelphi Psoriasis Disease Specific Programme were used to compare self-reported satisfaction with biologic and non-biologic therapy for psoriasis in physicians and their consulting patients in the United States (USA) and five European countries (EU5). Disease severity and health-related quality of life (HRQoL) were assessed using Body Surface Area (BSA) affected by psoriasis and the Dermatology Life Quality Index (DLQI), respectively. Patients satisfied with biologic therapy reported better HRQoL than unsatisfied patients, whereas a greater proportion of unsatisfied patients on biologic therapy had moderate-to-severe psoriasis (USA: 95.1% versus 52.4%, EU5; 86.4% versus 43.1%, P<0.0001). Multivariate logistic regression indicated that having a BSA affected by psoriasis of >10% was associated with lower likelihoods of physician and patient treatment satisfaction versus <3% (P<0.0001). A one-unit increase in the DLQI score lowered the likelihood of a patient being satisfied by approximately 20% (P<0.0001). Patients were ~60% more likely to be satisfied on biologic therapy than non-biologic therapy (P=0.0012). Physician and patient-reported treatment satisfaction was associated with greater HRQoL and lesser disease severity.

Treatment Patterns in US Patients Receiving First-Line and Second-Line Therapy for Metastatic Pancreatic Ductal Adenocarcinoma in the Real World.

INTRODUCTION: Metastatic pancreatic ductal adenocarcinoma (mPDAC) is a common cancer with poor survival outcomes. Although treatment options are limited, real-world treatment patterns and outcomes are not well understood, particularly beyond first-line treatment. This study described real-world treatment patterns and outcomes for mPDAC in the USA. METHODS: This retrospective analysis used electronic health record-derived de-identified data of patients with mPDAC diagnosed between January 1, 2014 and June 30, 2021. Treatments were classified into six groups: (1) standard combination chemotherapy; (2) nonstandard combination chemotherapy; (3) single-agent chemotherapy; (4) targeted therapy; (5) clinical study drugs; and (6) off-label therapies. Analyses were descriptive in nature. Treatment utilization and switching, and time on treatment and time to discontinuation, were described by first-line (1LOT) and second-line (2LOT) treatment groups. Median overall survival (mOS) from 1LOT and 2LOT was stratified by treatment group, and for 1LOT on the basis of whether patients received further treatment. RESULTS: 1LOT included 6979 patients, 3241 (46%) of whom received further 2LOT. Standard combination chemotherapy was the most common 1LOT (70%) and 2LOT (46%). Nonstandard combination chemotherapy was used more as 2LOT (35%) than 1LOT (11%). First-line time on treatment was generally higher than second-line time on treatment, and time to discontinuation was lower than time on treatment. mOS in days (months) from 1LOT was 271 (8.9), 252 (8.3), 219 (7.2), 170 (5.6), 280 (9.2), and 182 (6.0), and mOS from 2LOT was 202 (6.6), 193 (6.3), 186 (6.1), 193 (6.3), 179 (5.9), and 97 (3.2), for groups 1-6, respectively. Within group 1, mOS from 1LOT was 318 days (10.4 months) for FOLFIRINOX and 241 days (7.9 months) for gemcitabine and nab-paclitaxel. CONCLUSION: Most patients with mPDAC received 1LOT in line with clinical practice guidelines, yet mOS remains poor. This study highlights the need for novel therapies to demonstrate improved patient survival compared with therapies in current clinical practice guidelines.

Societal impact for patients with psoriasis: A nationwide Swedish register study.

BACKGROUND: Psoriasis is an immune-mediated chronic inflammatory disease having a significant negative health impact. Psoriasis has societal impact; loss of productivity has been estimated at approximately 10% and it may influence the patient's financial status. Relationships between quality of life, disease severity, and cost of care need exploration. Understanding the disease burden is important for health policy and research allocation. Few studies address the research gaps in socioeconomics, comorbidity, and medication use. OBJECTIVE: Observing differences in education, income, employment status, marital status, health care consumption, and drug utilization between patients with psoriasis and matched controls. METHODS: Cohort study following socioeconomics and health care consumption for all psoriasis patients from the Swedish patient register. All individuals with a first diagnosis of psoriasis in outpatient or inpatient care from 2002 to 2013 were followed until death, emigration, or end of the study. RESULTS: Overall, 109,803 patients were included (mean age 51.2 years, 53% women) and matched with 1.08 million controls. The levels of education and income were similar, but the proportion employed was significantly lower for patients with psoriasis. There was a tendency for fewer patients with psoriasis to be married. LIMITATIONS: Generalizability, lack of primary care diagnoses, and lack of early treatments (available from 2005). CONCLUSION: Understanding of the socioeconomic impact of psoriasis is extended by showing reductions in employment.

The Occurrence of Metabolic Risk Factors Stratified by Psoriasis Severity: A Swedish Population-Based Matched Cohort Study.

PURPOSE: To assess the relative risk of metabolic risk factors in patients stratified by psoriasis severity compared with population controls. PATIENTS AND METHODS: A retrospective cohort study was conducted using national Swedish registers. Adult patients with psoriasis were selected if they had a dispensing of anti-psoriasis prescription (2007-2013) and at least one diagnosis within five years before the dispensing date. The patients with psoriasis were matched 1:10 to controls from the general population on birth year, sex, and county. The cohort was further divided into three disease severity groups (mild, moderate, or severe) based on their dispensed anti-psoriasis medication. Subjects were followed from the index date until censoring. We applied flexible parametric modeling to understand the risks of the incident comorbidities hypertension, hypercholesterolemia, and diabetes mellitus among patients with psoriasis from 6 months through 10 years. Hazard ratios and predicted risk (ie, 1 minus the survival probability) of comorbidities were reported. RESULTS: The hazard of hypertension, hypercholesterolemia, and diabetes mellitus is higher among psoriasis patients compared with population controls, and the hazard ratio increases with psoriasis severity. For example, HRs of hypertension for patients with mild, moderate, and severe psoriasis are 1.29 (95% CI: 1.27-1.32), 1.35 (95% CI: 1.32-1.38), and 1.73 (95% CI: 1.64-1.82), respectively. The predicted risk of hypertension, hypercholesterolemia, and diabetes mellitus among patients with severe psoriasis at year ten was 0.58 (95% CI: 0.56, 0.59), 0.33 (95% CI: 0.32, 0.35), and 0.21 (95% CI: 0.20, 0.23), respectively, while it was 0.42 (0.41, 0.43), 0.23 (0.22, 0.23), 0.11 (0.10, 0.11) among controls, respectively. The predicted risk at year ten was similar among patients with mild or moderate psoriasis. CONCLUSION: The HRs and predicted risks of metabolic risk factors are higher among patients with psoriasis compared with matched controls and are more prominent among the severe psoriasis group.

Young patients with risk factors prevalent in the elderly - differences in comorbidity depending on severity of psoriasis: a nationwide cross-sectional study in Swedish health registers.

BACKGROUND: Association between psoriasis severity and cerebro- and cardiovascular comorbidities has rarely been investigated. AIM: We aimed to investigate differences in cerebro- and cardiovascular comorbidities by psoriasis severity. MATERIALS AND METHODS: Using Swedish nationwide health-care registers, new adult users of anti-psoriatic drugs (2007-2013) with a recorded diagnosis of psoriasis/psoriatic arthritis or a filled prescription for calcipotriol were included. Psoriasis severity was based on the type of anti-psoriatic treatment (topical/mild, non-biologic systemic/moderate-to-severe, and biologics/ severe). Age standardized prevalence rates of cerebro- and cardiovascular comorbidities and their risk factors were compared between the groups. RESULTS: We found that severe psoriasis patients (N=2147) were younger than moderate-to-severe (N=11,919) or mild (N=70,796) patients (median 44, 52, and 55 years). Prevalence of hypertension was 29.9%, 32.6%, and 36.5%, myocardial infarction was 2.5%, 2.3%, and 1.8%, and stroke was 2.4%, 2.2%, and 1.1% in mild, moderate-to-severe, and severe psoriasis patients, respectively. Diabetes prevalence was 7.6% in mild, 8.0% in moderate-to-severe, and 10.7% in severe psoriasis. CONCLUSION: Myocardial infarction and stroke were less common in patients with severe psoriasis while, despite being younger, they had a higher prevalence of diabetes and hypertension.

Determinants of healthcare provider recommendations for influenza vaccinations.

OBJECTIVE: Investigate determinants of receiving healthcare provider (HCP) recommendations for seasonal and H1N1 influenza vaccinations. METHODS: Using a United States national sample of adults 18 + from the National 2009 H1N1 Flu Survey, multivariate regression models estimated the likelihood of receiving a HCP recommendation. Covariates included demographics, socioeconomic status, and Advisory Committee on Immunization Practices (ACIP) priority groups. RESULTS: Adults age 55-64 and 65 + were more likely to report a HCP recommendation when compared to adults age 18-34 (OR: 1.483, 95%CI: 1.237-1.778 and OR: 1.738, 95%CI: 1.427-2.116, respectively). Chronically ill adults had 58.0% (95%CI: 1.414-1.765) higher odds of receiving a HCP recommendation than non-chronically ill adults. Patients visiting a doctor once and twice had 28.7% (95%CI: 0.618-0.821) and 17.1% (95%CI: 0.721-0.952) lower odds of receiving a HCP recommendation when compared to adults visiting their doctor at least four times. And, compared to Non-Hispanic Whites, Non-Hispanic Blacks had 28.4% (95%CI: 1.064-1.549) higher odds of receiving a recommendation. CONCLUSIONS: ACIP priority groups experienced higher rates of recommendations compared to non-ACIP groups. Racial differences in HCP recommendations cannot explain racial disparities in flu vaccination rates.

A review of the economic burden of glioblastoma and the cost effectiveness of pharmacologic treatments.

Grade IV glioma (glioblastoma) is one of the most common brain/central nervous system cancers. In 2005, the standard of care for adjuvant treatment was significantly changed with the approval of temozolomide. Carmustine wafers have also gained some popularity. Phase III trials are currently evaluating bevacizumab in conjunction with the standard temozolomide regimen. Despite these recent advances in pharmacotherapy, roughly two-thirds of patients do not survive longer than 2 years after diagnosis. Meanwhile, the costs of treatment are substantial. The goal of this study is to review the clinical, cost-of-illness, and cost-effectiveness literature relevant to treating glioblastoma. Estimates of the economic burden of glioblastoma within different healthcare systems were converted to 2013 US dollars. Temozolomide has demonstrated a 2.5-month increase in overall survival and a 1.9-month increase in progression-free survival, relative to radiotherapy alone. Carmustine wafers have also been shown to increase overall survival by 2.3 months, compared with placebo wafers. Cost-effectiveness studies of temozolomide have produced incremental cost-effectiveness ratios, adjusted to 2013 US dollars, with a range from US$73,586 per quality-adjusted life-year (QALY) (UK National Health Service perspective) to US$105,234 per QALY (US societal perspective). More research is needed to quantify the full societal burden of illness.

Novel methods of measuring clinical outcomes from psoriasis and psoriatic arthritis clinical trials.

Numerous instruments exist that measure the clinical and health related quality of life impact of psoriasis and psoriatic arthritis (PsA) in clinical trials. However, many of these instruments are not typically used in economic evaluations to inform decision problems facing health care decision makers. This study reviews the current state of psoriasis and PsA health outcome measures and evaluates their limitations in cost-effectiveness analyses (CEAs). We highlight the health related quality of life and clinical outcome measures that are typically used in CEAs, with special focus on studies with quality adjusted life years as a primary outcome measure. Despite the high prevalence of psoriasis and PsA health outcome measures in clinical trials, only a few are used in CEAs. The methods by which utilities are estimated from these measures vary across cost-effectiveness studies. These differences should be considered when conducting cost-effectiveness research in psoriasis and PsA.

The cost-effectiveness of temozolomide in the adjuvant treatment of newly diagnosed glioblastoma in the United States.

BACKGROUND: The objective of this work was to determine the cost-effectiveness of temozolomide compared with that of radiotherapy alone in the adjuvant treatment of newly diagnosed glioblastoma. Temozolomide is the only chemotherapeutic agent to have demonstrated a significant survival benefit in a randomized clinical trial. Our analysis builds on earlier work by incorporating caregiver time costs and generic temozolomide availability. It is also the first analysis applicable to the US context. METHODS: A systematic literature review was conducted to collect relevant data. Transition probabilities were calculated from randomized controlled trial data comparing temozolomide plus radiotherapy with radiotherapy alone. Direct costs were calculated from charges reported by the Mayo Clinic. Utilities were obtained from a previous cost-utility analysis. Using these data, a Markov model with a 1-month cycle length and 5-year time horizon was constructed. RESULTS: The addition of brand Temodar and generic temozolomide to the standard radiotherapy regimen was associated with base-case incremental cost-effectiveness ratios of $102 364 and $8875, respectively, per quality-adjusted life-year. The model was most sensitive to the progression-free survival associated with the use of only radiotherapy. CONCLUSIONS: Both the brand and generic base-case estimates are cost-effective under a willingness-to-pay threshold of $150 000 per quality-adjusted life-year. All 1-way sensitivity analyses produced incremental cost-effectiveness ratios below this threshold. We conclude that both the brand Temodar and generic temozolomide are cost-effective treatments for newly diagnosed glioblastoma within the US context. However, assuming that the generic product produces equivalent quality of life and survival benefits, it would be significantly more cost-effective than the brand option.

Cost effectiveness of moderate to severe psoriasis therapy with etanercept and ustekinumab in the United States.

BACKGROUND: Limited information is available on the cost effectiveness of ustekinumab and alternative biologic treatments in a United States (US) setting. Given the recent head-to-head clinical trial study of ustekinumab and etanercept, an economic model comparing the two treatments can be constructed. Etanercept and ustekinumab are indicated for the treatment of chronic moderate to severe plaque psoriasis in adult patients who are candidates for phototherapy or systemic therapy. OBJECTIVE: Clinical trials have evaluated the efficacy of ustekinumab, an anti-cytokine biologic, for the treatment of moderate to severe psoriasis. This study evaluated the cost effectiveness of ustekinumab compared with etanercept from a US societal perspective. METHODS: A Markov model was constructed to simulate the incremental cost per quality-adjusted life-year (QALY) gained every 12 weeks over a base-case 3-year time horizon. A hypothetical patient cohort was based on the characteristics of the phase III Active Comparator Psoriasis Trial (ACCEPT). The main outcome measures were costs and QALYs, which were estimated from the US societal perspective. Costs, utilities, treatment strategy, and resource use estimates were obtained from relevant literature. All costs were adjusted to 2011 US dollars. A 3 % annual discount rate was applied to costs and QALYs. Incremental cost-effectiveness ratios were in US dollars per QALY gained. RESULTS: For the base-case 3-year time horizon, the incremental cost-effectiveness ratio comparing ustekinumab 90 mg with etanercept 50 mg was US$384,401 per QALY gained. Ustekinumab 45 mg dominates etanercept 50 mg for the same time horizon. These results were robust to sensitivity analyses involving treatment strategy, transition probabilities, valuing outcomes, and resource use and costs. The probabilistic sensitivity analysis suggests ustekinumab 90 mg has a minimal (4 %) chance of being cost effective compared with etanercept 50 mg at a willingness-to-pay threshold of US$150,000 per QALY improvement. For the same threshold, ustekinumab 45 mg has a high (88 %) chance of being cost effective compared with etanercept 50 mg. CONCLUSION: Under typical US willingness-to-pay cutoffs, ustekinumab 90 mg is not cost effective compared with etanercept 50 mg therapy in moderate to severe psoriasis patients for the base-case 3-year time horizon. Ustekinumab 45 mg dominates etanercept 50 mg therapy for an equivalent patient psoriasis severity and time horizon.