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.