Abstract
Background: Multiple myeloma (MM) is a heterogeneous hematologic malignancy with significant variability in treatment approaches across countries and healthcare systems. Real-world evidence is essential to understand how treatment access, practice patterns, and biomarker testing influence patient outcomes in routine care. This study examined differences in MM management and progression-free survival (PFS) using electronic health record (EHR)-derived datasets from Germany and the UK.
Methods: We conducted a retrospective cohort study using the Flatiron Health Research Database MM datasets from the UK and Germany. These datasets are derived from EHRs collected from routine clinical care in each country. Data were curated through technology-enabled abstraction and underwent rigorous quality assurance. Key variables include patient demographics, disease characteristics, cytogenetic testing, and treatment patterns across lines of therapy. The cohort included patients diagnosed with MM from January 1, 2011 to March 31, 2025. Real-world PFS (rwPFS) was derived using laboratory based algorithms leveraging M-spike and light chain dynamics. Kaplan-Meier methods were used to estimate rwPFS and subgroup analyses were stratified by autologous stem cell transplantation (ASCT) status and clinical risk factors.
Results: The UK and Germany cohorts included 616 and 466 patients, respectively. In both countries, 75% of patients were diagnosed at ISS stage II or III. Cytogenetic testing was reported for 59% of patients in Germany, compared to 60% of the UK cohort, with 1q21 gain/amplification and del13q being the most commonly reported positive tests in both countries. Extramedullary disease was documented in 6% (UK) and 8% (Germany). Most common first-line therapy was daratumumab, lenalidomide, and dexamethasone in Germany (13%), and cyclophosphamide, bortezomib, and dexamethasone (24%) in the UK. Use of ASCT was more frequent in Germany (38%) than UK (25%). PFS was influenced by ASCT status, with median PFS of 3.3 years for Germany patients with ASCT and 1.5 years for Germany patients without ASCT; in the UK, median PFS was 1.9 years with ASCT and 1.2 years without ASCT. Longer PFS was also associated with low ISS stage, absence of extramedullary disease, and absence of cytogenetic risk factors. Discussion: We observed important differences in MM management between Germany and the UK, including variations in cytogenetic testing rates, treatment patterns, and ASCT utilization. These variations may reflect differences in health system infrastructure, drug availability, and clinical guideline implementation. The observed PFS differences by ASCT status and risk profile are consistent with prior real-world and clinical trial data (eg, EMN02, IFM2009, Czech MM Registry), but may also be influenced by patient selection. Growing patient counts and longitudinal data collection will help validate these findings and map the uptake of minimal residual disease measurement and advanced therapies.