TRUST-MM | TRANSLATING US REAL-WORLD DATA TO UK CLINICAL PRACTICE: TREATMENT PATTERNS AND OUTCOMES IN FIRST LINE RELAPSED/REFRACTORY MULTIPLE MYELOMA
Author(s)
Simon Chen, MSc1, Christina Zettler, MPH2, Benjamin Bray, PhD, MD1, Andrew Belli, MPH2, Laura Fernandes, PhD2, Godspower Toyin Oboli, MD1, Eric Hansen, MS2, Ching-Kun Wang, MD2, Andrew Thompson, PhD1.
1LCP Health, Lane Clark & Peacock LLP, London, United Kingdom, 2COTA, Inc., New York, NY, USA.
1LCP Health, Lane Clark & Peacock LLP, London, United Kingdom, 2COTA, Inc., New York, NY, USA.
OBJECTIVES: Treatment availability for multiple myeloma (MM) differs across countries, reflecting variation in regulatory and reimbursement decisions. We assessed whether US real-world data from patients with relapsed/refractory MM (RRMM) could generate evidence relevant to UK regulatory and clinical decision-making.
METHODS: We conducted a feasibility analysis to inform a retrospective cohort study using the COTA database, including patients with MM diagnosed between January 2015 and April 2025. Eligible patients had relapsed or refractory disease after first-line (1L) therapy, defined by initiation of second-line (2L) treatment using a prespecified algorithm. The main cohort comprised patients who received a UK standard-of-care (SOC) 1L regimen. Baseline characteristics and treatment patterns were described. Real-world overall survival (rwOS) from 2L initiation was estimated using Kaplan-Meier methods and stratified by stem cell transplant (SCT) during follow-up.
RESULTS: Of 3,562 patients with RRMM initiating 2L therapy, 1,532 (43.0%) received UK SOC in 1L and were included. Mean age at 2L initiation was 67 years (SD 11); 52.3% had IgG subtype and 70.0% had ECOG performance status 0-2. Common 1L regimens were bortezomib, dexamethasone and cyclophosphamide (48.1%) and bortezomib and dexamethasone (34.5%). Most patients did not receive UK SOC in 2L (76.5%); among those who did, lenalidomide- and carfilzomib-containing regimens predominated. Median rwOS from 2L initiation was 43.0 months (95% CI 40.3-46.2); 1-, 3-, and 5-year survival was 83.7%, 57.4%, and 36.8%, respectively. Median rwOS was 67.1 months (95% CI 59.1-76.1) in SCT recipients versus 33.2 months (95% CI 31.2-36.8) in non-recipients.
CONCLUSIONS: By assessing overlap in UK and US 1L MM treatments, we identified a comparable US cohort and estimated key real‑world outcomes. This approach is an important step in identifying UK relevant comparator populations for real world evidence studies and serves as the foundation for future evidence generation on the effectiveness of therapies not currently available in the UK.
METHODS: We conducted a feasibility analysis to inform a retrospective cohort study using the COTA database, including patients with MM diagnosed between January 2015 and April 2025. Eligible patients had relapsed or refractory disease after first-line (1L) therapy, defined by initiation of second-line (2L) treatment using a prespecified algorithm. The main cohort comprised patients who received a UK standard-of-care (SOC) 1L regimen. Baseline characteristics and treatment patterns were described. Real-world overall survival (rwOS) from 2L initiation was estimated using Kaplan-Meier methods and stratified by stem cell transplant (SCT) during follow-up.
RESULTS: Of 3,562 patients with RRMM initiating 2L therapy, 1,532 (43.0%) received UK SOC in 1L and were included. Mean age at 2L initiation was 67 years (SD 11); 52.3% had IgG subtype and 70.0% had ECOG performance status 0-2. Common 1L regimens were bortezomib, dexamethasone and cyclophosphamide (48.1%) and bortezomib and dexamethasone (34.5%). Most patients did not receive UK SOC in 2L (76.5%); among those who did, lenalidomide- and carfilzomib-containing regimens predominated. Median rwOS from 2L initiation was 43.0 months (95% CI 40.3-46.2); 1-, 3-, and 5-year survival was 83.7%, 57.4%, and 36.8%, respectively. Median rwOS was 67.1 months (95% CI 59.1-76.1) in SCT recipients versus 33.2 months (95% CI 31.2-36.8) in non-recipients.
CONCLUSIONS: By assessing overlap in UK and US 1L MM treatments, we identified a comparable US cohort and estimated key real‑world outcomes. This approach is an important step in identifying UK relevant comparator populations for real world evidence studies and serves as the foundation for future evidence generation on the effectiveness of therapies not currently available in the UK.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HSD10
Topic
Health Service Delivery & Process of Care, Real World Data & Information Systems
Disease
Oncology