Tomáš Jelínek1, Vladimír Maisnar2, Luděk Pour3, Ivan Špička4, Jiří Minařík5, Evžen Gregora6, Petr Kessler7, Michal Sýkora8, Hana Fraňková9, Dagmar Adamová10, Marek Wróbel11, Peter Mikula12, Jiří Jarkovský13, Joris Diels14, Xenia Gatopoulou15, Šárka Veselá16, Hervé Besson14, Lucie Brožová13, Tetsuro Ito17, Roman Hájek18. 1. a Department of Haematooncology, University Hospital Ostrava and Faculty of Medicine and Faculty of Science , University of Ostrava , Poruba , Czech Republic. 2. b 4th Department of Internal Medicine - Hematology, Charles University Faculty Hospital and Faculty of Medicine , Hradec Králové , Czech Republic. 3. c Department of Internal Medicine, Hematology and Oncology , University Hospital Brno and Faculty of Medicine Masaryk University , Jihlavská 340/20, 625 00 Brno-Bohunice-Brno-Starý Lískovec , Czech Republic. 4. d Department of Internal Medicine , Charles University in Prague, First Faculty of Medicine and General Teaching Hospital , Hradec Králové , Czech Republic. 5. e Department of Hemato-Oncology , University Hospital Olomouc and Faculty of Medicine and Dentistry, Palacky University Olomouc , Olomouc , Czech Republic. 6. f Department of Internal Medicine and Hematology , University Hospital Kralovske Vinohrady , Praha , Czech Republic. 7. g Department of Hematology and Transfusion Medicine , Pelhrimov Hospital , Pelhřimov , Czech Republic. 8. h Department of Clinical Hematology , Hospital Ceske Budejovice , České Budějovice , Czech Republic. 9. i Department of Hematology , General Hospital Liberec , Liberec , Czech Republic. 10. j Department of Clinical Hematology , Silesian Hospital Opava , Opava , Czech Republic. 11. k Department of Hematology , Hospital Novy Jicin , Nový Jičín , Czech Republic. 12. l Department of Clinical Hematology , General Hospital Havirov , Havířov , Czech Republic. 13. m Institute of Biostatistics and Analyses, Faculty of Medicine and Faculty of Science , Masaryk University , Brno , Czech Republic. 14. n Janssen Health Economics & Market Access EMEA Statistics & Modelling , Beerse , Belgium. 15. o Janssen Health Economics & Market Access EMEA , Athens , Greece. 16. p Janssen - Cilag s.r.o. , Smíchov-Anděl , Czech Republic. 17. q Janssen Health Economics & Market Access EMEA , High Wycombe , UK. 18. r Department of Haematooncology, University Hospital Ostrava and Faculty of Medicine , University of Ostrava , Ostrava , Czech Republic.
Abstract
OBJECTIVES: We conducted an adjusted comparison of progression-free survival (PFS) and overall survival (OS) for daratumumab monotherapy versus standard of care, as observed in a real-world historical cohort of heavily pretreated multiple myeloma patients from Czech Republic. METHODS: Using longitudinal chart data from the Registry of Monoclonal Gammopathies (RMG) of the Czech Myeloma Group, patient-level data from the RMG was pooled with pivotal daratumumab monotherapy studies (GEN501 and SIRIUS; 16 mg/kg). RESULTS: From the RMG database, we identified 972 treatment lines in 463 patients previously treated with both a proteasome inhibitor and an immunomodulatory drug. Treatment initiation dates for RMG patients were between March 2006 and March 2015. The most frequently used treatment regimens were lenalidomide-based regimens (33.4%), chemotherapy (18.1%), bortezomib-based regimens (13.6%), thalidomide-based regimens (8.0%), and bortezomib plus thalidomide (5.3%). Few patients were treated with carfilzomib-based regimens (2.5%) and pomalidomide-based regimens (2.4%). Median observed PFS for daratumumab and the RMG cohort was 4.0 and 5.8 months (unadjusted hazard ratio [HR], 1.14; 95% confidence interval [CI], 0.94-1.39), respectively, and unadjusted median OS was 20.1 and 11.9 months (unadjusted HR, 0.61; 95% CI, 0.48-0.78), respectively. Statistical adjustments for differences in baseline characteristics were made using patient-level data. The adjusted HRs (95% CI) for PFS and OS for daratumumab versus the RMG cohort were 0.79 (0.56-1.12; p = .192) and 0.33 (0.21-0.52; p < .001), respectively. CONCLUSIONS: Adjusted comparisons between trial data and historical cohorts can provide useful insights to clinicians and reimbursement decision makers on relative treatment efficacies in the absence of head-to-head comparison studies for daratumumab monotherapy.
OBJECTIVES: We conducted an adjusted comparison of progression-free survival (PFS) and overall survival (OS) for daratumumab monotherapy versus standard of care, as observed in a real-world historical cohort of heavily pretreated multiple myelomapatients from Czech Republic. METHODS: Using longitudinal chart data from the Registry of Monoclonal Gammopathies (RMG) of the Czech Myeloma Group, patient-level data from the RMG was pooled with pivotal daratumumab monotherapy studies (GEN501 and SIRIUS; 16 mg/kg). RESULTS: From the RMG database, we identified 972 treatment lines in 463 patients previously treated with both a proteasome inhibitor and an immunomodulatory drug. Treatment initiation dates for RMG patients were between March 2006 and March 2015. The most frequently used treatment regimens were lenalidomide-based regimens (33.4%), chemotherapy (18.1%), bortezomib-based regimens (13.6%), thalidomide-based regimens (8.0%), and bortezomib plus thalidomide (5.3%). Few patients were treated with carfilzomib-based regimens (2.5%) and pomalidomide-based regimens (2.4%). Median observed PFS for daratumumab and the RMG cohort was 4.0 and 5.8 months (unadjusted hazard ratio [HR], 1.14; 95% confidence interval [CI], 0.94-1.39), respectively, and unadjusted median OS was 20.1 and 11.9 months (unadjusted HR, 0.61; 95% CI, 0.48-0.78), respectively. Statistical adjustments for differences in baseline characteristics were made using patient-level data. The adjusted HRs (95% CI) for PFS and OS for daratumumab versus the RMG cohort were 0.79 (0.56-1.12; p = .192) and 0.33 (0.21-0.52; p < .001), respectively. CONCLUSIONS: Adjusted comparisons between trial data and historical cohorts can provide useful insights to clinicians and reimbursement decision makers on relative treatment efficacies in the absence of head-to-head comparison studies for daratumumab monotherapy.
Authors: Dina Oksen; Patricia Prince; Emmanuelle Boutmy; Elizabeth M Garry; Barbara Ellers-Lenz; Adina Estrin; Andreas Johne; Patrice Verpillat; Nicolle M Gatto Journal: Clin Transl Sci Date: 2022-06-11 Impact factor: 4.438