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To demonstrate the measurement properties of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Hepatocellular Carcinoma 18-question module (EORTC QLQ-HCC18) within a previously treated, unresectable hepatocellular carcinoma (HCC) clinical trial population that was distinct from the published QLQ-HCC18 validation population. Analyses were conducted using data from BGB-A317-208, an open label, international, clinical trial assessing efficacy and safety of the monoclonal antibody tislelizumab in adult HCC patients. The EORTC Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) and QLQ-HCC18 instruments were assessed at baseline and weeks 3 and 9 follow-up visits. Per US Food and Drug Administration guidance, psychometric validation of the QLQ-HCC18 included reliability (internal consistency and test–retest), construct validity (convergent and discriminant validity and known-groups validity), ability to detect change, and meaningful within-patient change (MWPC). Known-groups validity and MWPC analyses were also stratified on several pre-defined subgroups. A total of 248 patients were included. Only the QLQ-HCC18 fatigue, nutrition, and index domains demonstrated acceptable internal consistency; acceptable test–retest reliability was found for fatigue, body image, nutrition, pain, sexual interest, and index domains. The QLQ-HCC18 fatigue domain achieved the pre-specified criterion defining acceptable convergent and discriminant validity for 13 of 16 correlations, whereas the index domain achieved the pre-specified criterion for 14 of 16 correlations. Clear differentiation of the QLQ-HCC18 change scores between improvement and maintenance anchor groups were observed for body image, fatigue, pain, and index domains, whereas differentiation between deterioration and maintenance anchor groups were observed for fever and fatigue domains. MWPC point estimates defining improvement for the QLQ-HCC18 fatigue and index domains were-7.18 and-4.07, respectively; MWPC point estimates defining deterioration were 5.34 and 3.16, respectively. The EORTC QLQ-HCC18 fatigue and index domains consistently demonstrated robust psychometric properties, supporting the use of these domains as suitable patient-reported endpoints within a previously treated, unresectable HCC patient population.

Background Gastroesophageal junction carcinoma (GEJC) and esophageal squamous cell carcinoma (ESCC) are increasing in Australia and other Western countries. We aimed to investigate past trends and predict the future direction of GEJC and ESCC cases. Methods Data on GEJC and ESCC were extracted from the Australian Cancer Database, and the National Mortality Database for the Australian states of Victoria, Queensland, Tasmania, and the Australian Capital Territory. The new number of cases were predicted up to 2039 by fitting a least squares linear regression using the Australian incidence rates trend from 2009 to 2018. Past trends were analyzed for prevalence and mortality. Kaplan-Meier curves generated the survival trend for up to 10 years. Results Between 2009 and 2018, the overall incidences of GEJC showed an increasing trend (annual change [β 1 ] = 0.87, P  < 0.05). For GEJC, the rates of incidence, prevalence, and mortality were higher in men (β 1  = 1.86, 95% CI: 1.29–2.43, P  < 0.05; β 1  = 14.45, 95% CI: 12.93–15.98, P  < 0.05; β 1  = 1.04, 95% CI: − 0.68 to 2.76, P  = 0.195, respectively) than in women. By 2039, it is estimated that 6 in 100,000 population will be newly diagnosed with GEJC. Notably, the rate of new GEJC cases decreased in women during the study period (β 1 =–0.08, 95% CI: − 0.39 to 0.24, P  = 0.596). For ESCC, the incidence rate increased, albeit at a slower rate compared with GEJC (β 1  = 0.87, 95% CI: 0.6–1.15, P  < 0.05; β 1  = 0.03, 95% CI: − 0.28 to 0.34, P  = 0.805, respectively). New cases of ESCC in men declined (β 1 =–0.03, 95% CI: − 0.46 to 0.4, P  = 0.884). By 2039, it is predicted that 2 in 100,000 people will be newly diagnosed. The 10-year survival rate for GEJC and ESCC was low (11% and 20%, respectively), while the survival rate for women was relatively higher than for men. Conclusion The incidence, prevalence, and mortality would be predicted to increase in 2039 if there were no significant changes in risk factors, diagnosis, treatment, and management compared with 2009–2018. Strategies to identify early signs and enable earlier diagnosis and early and new treatment options are essential in GEJC and ESCC.

Bisphosphonate therapy is a well-established and effective treatment for postmenopausal osteoporosis and the prevention of osteoporotic fracture. However, poor adherence to and poor persistence with bisphosphonate therapy may reduce its benefits. Previous studies have documented the poor rates of adherence and persistence among postmenopausal women with osteoporosis. The objective of this systematic literature review was to evaluate adherence, persistence, and the impact of adherence and persistence on fracture risk in postmenopausal women with diagnosed osteoporosis. Articles eligible for review included observational studies of the real-world use of bisphosphonates in 23 countries and were identified by using MEDLINE, EMBASE, IMSEAR (Index Medicus for South-East Asia Region), and LILACS (Latin American and Caribbean Health Sciences Database). We identified and evaluated 10 studies that assessed bisphosphonate adherence by measuring medication possession ratio (MPR), persistence, and/or the impact of adherence and persistence on fracture risk. Mean MPR at 1 year ranged from 54% to 71% in the 3 studies that reported this assessment of adherence, and 40%–85% of patients at 1 year were adherent, defined as an MPR ≥80%, in the 8 studies that reported this end point. At 1 year, rates of persistence ranged from 28% to 74%. Rates of adherence and persistence were highest with agents requiring less frequent administration and typically declined over time. Fracture rates were significantly lower among adherent women with MPRs ≥80% compared with women with MPRs <80%. Our results show that suboptimal adherence to and persistence with bisphosphonate therapy in postmenopausal women are common and increase the risk of fracture. Additional research is needed to identify and incorporate effective strategies for improving adherence to bisphosphonates in postmenopausal women.

Background Mature B‐cell neoplasms chronic lymphocytic leukaemia/small lymphocytic lymphoma (CLL/SLL) and waldenström macroglobulinaemia (WM) are highly prevalent in older populations. Aims This study quantified the incidence, prevalence and relative survival/mortality rate in Australia for CLL/SS and WM and reported the past trends. Materials & Methods All CLL/SLL and WM cases registered from January 2009 to December 2018 in Victoria, Tasmania, Australian Capital Territory and Queensland were identified. Incidence rates over the observed period (2009–2018) were calculated and then projected to 2038 using linear regression. Kaplan–Meier (KM) curves were used to estimate survival rates from 2009 to 2018. Results Between 2009 and 2018, the annual age‐standardised incidence rates of CLL/SLL (range, 600.05–887.92 cases per 107 person‐years) and WM (range, 41.48–78.19 cases per 107 person‐years) showed an increasing trend (coefficient: 26.98 [p = 0.023] and 3.20 [p = 0.009], respectively). A similar trend was seen in age‐standardised prevalence proportions by sex and age group. KM curves showed 10‐year survival rates of 53% (CLL/SLL) and 42% (WM) at the end of the available data period (2018). Differences in survival between sexes were not statistically significant in the log‐rank test, but univariable analysis showed male sex and older age were associated with a higher risk of mortality in both condition. Discussion The change in survival over time may reflect disease characteristics and recent advances in treatment. Conclusion Given the increasing incidence and relatively high survival of CLL/SLL and WM, strategic planning for the future management is warranted in the context of Australia’s ageing population.

Background The EORTC QLQ-OES18 has previously demonstrated clinical validity; however, there are limited published psychometric data for patients with advanced esophageal squamous cell carcinoma (ESCC). We evaluated the measurement properties of the QLQ-OES18 in a clinical trial population of patients with advanced or metastatic ESCC. Methodology Analyses used data from RATIONALE 302 (NCT03430843), a randomized phase 3 study of tislelizumab versus investigator-chosen chemotherapy as second-line treatment for patients with advanced or metastatic ESCC. Psychometric validation of the QLQ-OES18 included tests of reliability, construct validity, ability to detect change, and estimation of anchor-based meaningful within-patient change (MWPC) thresholds—the latter two being exploratory given that the trial was not powered to detect efficacy in patient-reported outcome endpoints. Results In total, 512 patients were randomized to either tislelizumab or chemotherapy; the average age was 61.5 years, and 84.4% were male. Three of the 4 QLQ-OES18 multi-item scales (dysphagia, eating, and pain) and the index scale met the prespecified criterion for acceptable internal consistency as well as acceptable test-retest reliability. Associations between baseline QLQ-OES18 scores and convergent/discriminant validators were generally as expected (i.e., the QLQ-OES18 pain score had a strong positive correlation with the QLQ-C30 pain score). For known-groups validity, 88.6% of analyses demonstrated the hypothesized direction of effect, suggesting that the expected differences in baseline QLQ-OES18 scores between prespecified groups were observed. Ability to detect change analyses indicated that several QLQ-OES18 domain scores demonstrated sensitivity in detecting possible treatment effects, although many patients reported minimal symptoms at baseline, which limited the ability to detect significant improvement. Conclusion Overall, a collection of psychometric evidence indicated that the EORTC QLQ-OES18 reliably and validly measured symptom severity in the RATIONALE 302 population. Specifically, the dysphagia domain consistently demonstrated robust psychometric properties. Limitations in data reduced the interpretability of MWPC thresholds and are discussed in detail.

This study examined the health‐related quality of life (HRQoL) of patients with advanced non‐small cell lung cancer (NSCLC) receiving tislelizumab versus docetaxel in the open‐label, multicenter, Phase 3 trial called RATIONALE‐303 (NCT03358875). HRQoL was assessed with the EORTC QLQ‐C30, EORTC QLQ‐LC13, and the EQ‐5D‐5L instruments. A longitudinal analysis of covariance assessed the change from baseline to Week 12 and from baseline to Week 18. A time to deterioration analysis was also performed using the Kaplan–Meier method. Eight hundred and five patients were randomized to either tislelizumab (n = 535) or docetaxel, respectively (535 and 270 to tislelizumab and docetaxel, respectively). The tislelizumab arm improved while the docetaxel arm worsened in the QLQ‐C30 global health status/QoL scale score (difference LS mean change Week 18: 5.7 [95% CI: 2.38, 9.07, p = 0.0008]), fatigue (Week 12: ‐3.2 [95% CI: −5.95, −0.37, p < 0.0266]; Week 18: −4.9 [95% CI: −8.26, −1.61, p = 0.0037]), and QLQ‐LC13 symptom index score (Week 12: −5.5 [95% CI: −6.93, −4.04, P < 0.0001]; Week 18: −6.6 [95% CI: −8.25, −4.95, p < 0.0001]). The tislelizumab arm had improvements in coughing versus the docetaxel arm (Week 12: −4.7 [95% CI: −8.57, −0.78, p = 0.0188]; Week 18: −8.3 [95% CI: −13.02, −3.51, p = 0.0007]). The patients who received tislelizumab were less at risk for clinically meaningful worsening in the overall lung cancer symptom index scale (hazard ratio (HR): 0.24 [95% CI: 0.162, 0.356], p < 0.0001), dyspnea (HR: 0.74 [95% CI: 0.567, 0.958], p = 0.0109), coughing (HR: 0.74 [95% CI: 0.534, 1.019], p = 0.0309), and peripheral neuropathy (HR: 0.55 [95% CI: 0.370, 0.810] p = 0.0011). In general, tislelizumab versus docetaxel was associated with improved HRQoL and symptoms of lung cancer in patients who previously failed treatment with platinum‐containing chemotherapy. Patients with advanced non‐small cell lung cancer (NSCLC) have a poor prognosis and poor health‐related quality of life (HRQoL). This study found that patients treated with tislelizumab had greater improvements in the HRQoL measures of the EORTC QLQ‐C30 and EORTC lung cancer module (QLQ‐LC13) scales and scores compared with patients treated with docetaxel. These results support the favorable risk‐benefit ratio of tislelizumab as a second‐line therapy in patients with NSCLC who previously failed treatment with a platinum‐containing chemotherapy.

Background: Anti-tumor necrosis factor (TNF) biologic agents are effective in treating rheumatoid arthritis (RA). Information on patient persistence with biologic anti-TNF therapies is limited, and the effects of persistence on the costs of therapy are unknown. Objectives: The aims of this study were to compare treatment persistence with adalimumab, etanercept, or infliximab in combination withmethotrexate (MTX) and evaluate the effects of persistence on overall health care costs. Methods: This retrospective study used data from the PharMetrics managed care administrative claims database. Data from patients with RA who received combination treatment with an anti-TNF agent plus MTX and had ≥24 months of continuous plan eligibility were collected. The 3 anti-TNF cohorts were adalimumab + MTX (adalimumab group), etanercept + MTX (etanercept group), and infliximab + MTX (infliximab group). Treatment persistence was defined as the number of days between the first and last anti-TNF treatment and was reported as a percentage of the 1-year period after treatment initiation. Costs were compared between patients with treatment persistence rates ≥80% or <80%. Demographics, comorbidities, disease severity, and RA-related costs were assessed using descriptive statistics. Univariate and multivariate analyses were applied to identify differences in mean persistence between the 3 cohorts. Results: Data from 1242 patients were included (77.7% female; mean age, 50.0 years). The mean persistence rate in the overall population was 74.6%, and the mean treatment time was 272.3 days. The infliximab group had a higher persistence rate compared with the etanercept and adalimumab groups (78.0% vs 72.8% and 70.8%, respectively; P < 0.005). In all patients combined, those with treatment persistence ≥80% had higher mean total health care costs compared with those with treatment persistence <80% ($19,271.52 vs $15,598.46; P < 0.001), largely due to higher pharmacy costs. However, nonpharmacy costs were lower in the ≥80% persistence cohort ($3091 vs $4601; P = 0.015). Conclusions: In this population of patients with RA, overall treatment persistence was high, with patients treated with infliximab + MTX having significantly higher persistence compared with those treated with adalimumab + MTX or etanercept + MTX. While pharmacy costs were higher in patients with ≥80% persistence, nonpharmacy costs were lower.

Background Immune checkpoint inhibitors targeting the programmed cell death protein‐1 (PD‐1) and programmed death ligand‐1 (PD‐L1) axis (collectively referred to as PD[L]1i) have demonstrated clinical benefits in non‐small cell lung cancer (NSCLC) patients. The purpose of this United States‐based real‐world study is to examine changes in the landscape of first‐line therapies for NSCLC since the introduction of PD(L)1i. Methods Patients with NSCLC initiating first‐line treatment between May 1, 2017, and October 31, 2020, were identified in the IBM MarketScan® database. Patients were assigned groups based on first‐line therapy: PD(L)1i monotherapy, chemotherapy alone, PD(L)1i with chemotherapy, or targeted therapy for patients with actionable driver mutations. Results A total of 5431 patients with NSCLC starting first‐line treatment were identified: chemotherapy alone 2568 (47%), PD(L)1i with chemotherapy 1364 (25%), PD(L)1i monotherapy 790 (15%), and targeted therapy 709 (13%). The use of PD(L)1i monotherapy and targeted therapy remained consistent, while the percentage of patients receiving PD(L)1i with chemotherapy more than doubled. Over a third of patients in 2019 and 2020 received chemotherapy alone. Patients aged ≥65 years (odds ratio [OR]: 0.80; 95% confidence interval [CI]: 0.68–0.95), females (OR: 0.86; 95% CI: 0.74–0.98), and those with respiratory (OR: 0.82; 95% CI: 0.71–0.94) or kidney (OR: 0.56; 95% CI: 0.40–0.77) disease were less likely to have received PD(L)1i with chemotherapy than patients that received chemotherapy alone. Conclusions Since the approval of PD(L)1i for NSCLC, their use has significantly increased for first‐line treatment, especially when used in combination with chemotherapy. A significant proportion of patients received chemotherapy alone. Since the approval of PD(L)1i for NSCLC, their use has significantly increased for first‐line treatment, especially when used in combination with chemotherapy. A significant proportion of patients received chemotherapy alone.