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.

b 4th Department of Internal Medicine Hematology Charles University Faculty Hospital and Faculty of Medicine Hradec Králové Czech Republic

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

Department of Haematooncology University Hospital Ostrava and Faculty of Medicine and Faculty of Science University of Ostrava Poruba Czech Republic

Department of Internal Medicine Charles University Prague 1st Faculty of Medicine and General Teaching Hospital Hradec Králové Czech Republic

e Department of Hemato Oncology University Hospital Olomouc and Faculty of Medicine and Dentistry Palacky University Olomouc Olomouc Czech Republic

f Department of Internal Medicine and Hematology University Hospital Kralovske Vinohrady Praha Czech Republic

g Department of Hematology and Transfusion Medicine Pelhrimov Hospital Pelhřimov Czech Republic

h Department of Clinical Hematology Hospital Ceske Budejovice České Budějovice Czech Republic

i Department of Hematology General Hospital Liberec Liberec Czech Republic

j Department of Clinical Hematology Silesian Hospital Opava Opava Czech Republic

Janssen Health Economics and Market Access EMEA Statistics and Modelling Beerse Belgium

k Department of Hematology Hospital Novy Jicin Nový Jičín Czech Republic

l Department of Clinical Hematology General Hospital Havirov Havířov Czech Republic

m Institute of Biostatistics and Analyses Faculty of Medicine and Faculty of Science Masaryk University Brno Czech Republic

o Janssen Health Economics and Market Access EMEA Athens Greece

p Janssen Cilag s r o Smíchov Anděl Czech Republic

q Janssen Health Economics and Market Access EMEA High Wycombe UK

r Department of Haematooncology University Hospital Ostrava and Faculty of Medicine University of Ostrava Ostrava Czech Republic

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