Real-world treatment patterns, healthcare resource use and disease burden in patients with multiple myeloma in Europe.

Aim: To investigate treatment patterns, healthcare resource utilization and disease burden in patients with multiple myeloma (MM). Methods: Point-in-time survey of physicians and their patients presenting in a real-world clinical setting, collected across Europe between May and November 2021. Results: In total, 173 physicians provided data for 2179 patients with MM. Treatments received became more diverse as line of therapy increased, dictated by previous treatment choices. Overall, 25% of all patients were tri-exposed, and experienced a higher degree of healthcare resource utilization, disease burden and impairment than non-tri-exposed patients. Conclusion: The treatment landscape in MM is complex and evolving. There is an unmet need for more effective therapies to reduce disease burden, particularly in tri-exposed patients.

There are many new treatments available for patients with multiple myeloma. While outcomes such as survival, symptoms and health problems experienced have improved, patients still continue to relapse and fall ill again. This means their current treatment stops working and they have to change to a new treatment to prevent their disease from developing further. Patients who have received three different types of treatment are classed as being 'tri-exposed', and they experience greater problems with their health. To better understand this course of events, we used information from a survey of doctors and their patients with multiple myeloma across Europe in 2021. We looked at patient's symptoms, the treatments they received, how and when they accessed healthcare (including hospital visits and tests) and the overall difficulties experienced due to their illness. We found that patients were broadly treated according to the most recent European guidelines, although differences were seen between countries. When patients had to switch therapy, the type of treatment received next depended on what they had previously been prescribed, meaning that treatment choices became increasingly complicated. Overall, 25% of patients in our study were classed as tri-exposed, and had more hospitalisations, required more hospital tests, had greater health problems and experienced more difficulties at work than those who were not tri-exposed. Despite recent developments in the treatment of multiple myeloma, there is still a need for more effective therapies. This is especially true for patients who are tri-exposed, who have limited treatment options and experienced greater health problems.

Adjusted comparison of outcomes between patients from CARTITUDE-1 versus multiple myeloma patients with prior exposure to proteasome inhibitors, immunomodulatory drugs and anti-CD38 antibody from the prospective, multinational LocoMMotion study of real-world clinical practice.

Ciltacabtagene autoleucel (cilta-cel) is a chimeric antigen receptor T-cell therapy studied in patients with multiple myeloma exposed to three classes of treatment in the single-arm CARTITUDE-1 study. To assess the effectiveness of cilta-cel compared to real-world clinical practice (RWCP), we performed adjusted comparisons using individual patients' data from CARTITUDE-1 and LocoMMotion, a prospective, multinational study of patients with multiple myeloma triple-class exposed of treatment. Comparisons were performed using inverse probability weighting. In CARTITUDE-1, 113 patients were enrolled, and 97 patients were infused with cilta-cel. In LocoMMotion, 248 patients were enrolled, and 170 patients were included in the comparisons versus infused patients. Ninety-two unique regimens were used in LocoMMotion, most frequently carfilzomib-dexamethasone (13.7%), pomalidomide-cyclophosphamide-dexamethasone (13.3%) and pomalidomidedexamethasone (11.3%). Adjusted comparisons showed that patients treated with cilta-cel were 3.12-fold more likely to respond to treatment than those managed by RWCP (response rate, 3.12, 95% confidence interval [95% CI]: 2.24-4.00), had their risk of progression or death reduced to by 85% (progression-free survival hazard ratio=0.15, 95% CI: 0.08-0.29), and a risk of death lowered by 80% (overall survival hazard ratio HR=0.20, 95% CI: 0.09-0.41). The incremental improvement in healthrelated quality of life from baseline for cilta-cel versus RWCP at week 52, as measured by EORTC QLQ-C30 Global Health Status, was 13.4 (95% CI: 3.5-23.6) and increased to 30.8 (95% CI: 21.8-39.8) when including death as additional information regarding patients' health status. Patients treated with cilta-cel experienced more adverse events than those managed with RWCP (any grade: 100% vs. 83.5%). The results from this study demonstrate improved efficacy outcomes of cilta-cel versus RWCP and highlight its potential as a novel and effective treatment option for patients with multiple myeloma triple-class exposed of antimyeloma treatment. CARTITUDE-1 is registered with clinicaltrials gov. Identifier: NCT03548207. LocoMMotion is registered with clinicaltrials gov. Identifier: NCT04035226.

The Economic Burden of Obesity in Germany: Results from the Population-Based KORA Studies.

OBJECTIVE: To estimate the excess costs of obese compared to normal-weight persons in Germany based on self-reported resource utilisation and work absence. METHODS: Five cross sectional surveys of cohort studies in southern Germany were pooled resulting in 9,070 observations for 6,731 individuals (31-96 years). BMI was measured in the study centre. Self-reported health care utilisation and work absence was used to estimate direct and indirect costs for the year 2011 based on unit costs. Using regression analyses, adjusted costs for different BMI groups were calculated. RESULTS: Overweight and obese people showed significantly higher odds of health care utilisation and productivity losses compared with normal-weight people in most categories. Total direct/indirect costs were significantly higher with increasing severity of obesity (pre-obese (1.05 (0.90-1.23) / 1.38 (1.11-1.71)), obesity level I (1.18 (1.00-1.39) / 1.33 (1.02-1.73)), obesity level II (1.46 (1.14-1.87) / 1.77 (1.18-2.65)) or level III (2.04 (1.40-2.97) / 1.99 (1.20-3.30)) compared to normal-weight participants. In particular, higher obesity classes were significantly associated with increased costs for medication, general practitioner utilisation and work absence. CONCLUSION: Our results show that overweight and obesity are associated with enormous societal direct and indirect costs in Germany. This supports the evidence from previous top-down studies, but provides important new information based on a large pooled data set and measured BMI.

Service use in adolescents at risk of depression and self-harm: prospective longitudinal study.

PURPOSE: Although depression and self-harm are common mental health problems in adolescents, there are barriers to accessing help. Using a community-based sample, this study investigates predictors of service contacts for adolescents at high risk of depression and self-harm. METHODS: Three thousand seven hundred and forty-nine (3,749) 12- to 16-year-olds in UK secondary (high) schools provided baseline and 6 months' follow-up data on mood, self-harm and service contacts with a range of primary and secondary healthcare services. RESULTS: Although most adolescents at high risk of depression or self-harm had seen their general practitioner (GP) in the previous 6 months, less than one-third had used primary or secondary healthcare services for emotional problems. 5 % of adolescents who reported self-harm had seen specialist child and adolescent mental health services in the previous 6 months. In longitudinal analyses, after adjustment for confounders, both depression and self-harm predicted the use of any healthcare services [adjusted odds ratio (AOR) = 1.34 (95 % CI 1.09, 1.64); AOR = 1.38 (95 % CI 1.02, 1.86), respectively] and of specialist mental health services [AOR = 5.48 (95 % CI 2.27, 13.25); AOR = 2.58 (95 % CI 1.11, 6.00), respectively]. Amongst those with probable depression, 79 % had seen their GP and 5 % specialist mental health services in the preceding year. CONCLUSIONS: Most adolescents at high risk of depression or self-harm see their GP over a 6-month period although only a minority of them access specialist mental health services. Their consultations within primary care settings provide a potential opportunity for their identification and for signposting to appropriate specialist services.

A systematic review of the quality of economic models comparing thrombosis inhibitors in patients with acute coronary syndrome undergoing percutaneous coronary intervention.

BACKGROUND: Thrombosis inhibitors can be used to treat acute coronary syndromes (ACS). However, there are various alternative treatment strategies, of which some have been compared using health economic decision models. OBJECTIVE: To assess the quality of health economic decision models comparing thrombosis inhibitors in patients with ACS undergoing percutaneous coronary intervention, and to identify areas for quality improvement. DATA SOURCES: The literature databases MEDLINE, EMBASE, EconLit, National Health Service Economic Evaluation Database (NHS EED), Database of Abstracts of Reviews of Effects (DARE) and Health Technology Assessment (HTA). STUDY APPRAISAL AND SYNTHESIS METHODS: A review of the quality of health economic decision models was conducted by two independent reviewers, using the Philips checklist. RESULTS: Twenty-one relevant studies were identified. Differences were apparent regarding the model type (six decision trees, four Markov models, eight combinations, three undefined models), the model structure (types of events, Markov states) and the incorporation of data (efficacy, cost and utility data). Critical issues were the absence of particular events (e.g. thrombocytopenia, stroke) and questionable usage of utility values within some studies. LIMITATIONS: As we restricted our search to health economic decision models comparing thrombosis inhibitors, interesting aspects related to the quality of studies of adjacent medical areas that compared stents or procedures could have been missed. CONCLUSIONS: This review identified areas where recommendations are indicated regarding the quality of future ACS decision models. For example, all critical events and relevant treatment options should be included. Models also need to allow for changing event probabilities to correctly reflect ACS and to incorporate appropriate, age-specific utility values and decrements when conducting cost-utility analyses.