Real-world experience of novel multiple myeloma treatments in a large, single-center cohort in Finland

• Published in: EJHaem Vol. 4; no. 4; pp. 1019 - 1029
• Main Authors: Loponen, Heidi, Mehtälä, Juha, Ylisaukko-Oja, Tero, Brück, Oscar, Porkka, Kimmo, Koskenvesa, Perttu, Saukkonen, Kirsi, Lievonen, Juha
• Format: Journal Article
• Language: English
• Published: United States John Wiley & Sons, Inc 01.11.2023; Wiley
• Subjects: Age; Bortezomib; carfilzomib; Clinical trials; Cytogenetics; Electronic health records; Medical prognosis; Multiple myeloma; novel treatments; Patients; Response rates; Stem cell transplantation; treatment‐related outcomes; Working groups; Finland; treatment‐related outcomes; multiple myeloma; novel treatments; carfilzomib
• ISSN: 2688-6146; 2688-6146
• DOI: 10.1002/jha2.802

In this single-center study, we aimed to describe the characteristics, treatment patterns, and outcomes of patients with multiple myeloma (MM) following treatment with bortezomib, carfilzomib, daratumumab, ixazomib, lenalidomide or pomalidomide-based regimens. Data were collected retrospectively from a study cohort of patients receiving a MM treatment in the Hospital District of Helsinki and Uusimaa (HUS) in Finland between 2016-2020. In total, 472 patients were included in the study. Median age was 68.2 years and nearly 25% had a high cytogenetic risk according to the International Myeloma Working Group categorization. In 2018-2020, the spectrum of regimens used as third- or later-line therapy was notably broader than in 2016-2017. The overall response rates for patients who received the most novel regimens (available ≤ 5 years) in second or third line of therapy (  = 67/430) and fourth line or later (  = 78/151) were 53.3% and 25.0%, respectively. In this real-world MM patient cohort, the response rates for these novel agents were lower compared to those reported in clinical trials. Given the higher cytogenetic risk profile and more advanced disease stage at the time when treated with novel agents, patients could have benefited from effective novel therapies earlier in their treatment pathway. What is the NEW aspect of your work? (ONE sentence) This study characterized the treatment of Finnish multiple myeloma patients during the era of most novel therapies (after 2016) and also included information on the cytogenetic risk profile of this real-world population.What is the CENTRAL finding of your work? (ONE sentence) There are clear differences between real-world populations treated with most novel combinations and those of randomized controlled trials (RCTs), which is reflected by the poorer treatment outcomes in the real-world setting.What is (or could be) the SPECIFIC clinical relevance of your work? (ONE sentence) Given the high cytogenetic risk profile and advanced disease stage at the time when treated with novel agents, patients could have benefited from effective novel therapies earlier in their treatment pathway.