A randomized phase III study of carfilzomib vs low-dose corticosteroids with optional cyclophosphamide in relapsed and refractory multiple myeloma (FOCUS)

R. Hájek, T. Masszi, M. T. Petrucci, A. Palumbo, L. Rosiñol, A. Nagler, K. L. Yong, A. Oriol, J. Minarik, L. Pour, M. A. Dimopoulos, V. Maisnar, D. Rossi, H. Kasparu, J. Van Droogenbroeck, D. B. Yehuda, I. Hardan, M. Jenner, M. Calbecka, M. DávidJ. De La Rubia, J. Drach, Z. Gasztonyi, S. Górnik, X. Leleu, M. Munder, M. Offidani, N. Zojer, K. Rajangam, Y. L. Chang, J. F. San-Miguel, H. Ludwig

Research output: Contribution to journalArticle

64 Citations (Scopus)

Abstract

This randomized, phase III, open-label, multicenter study compared carfilzomib monotherapy against low-dose corticosteroids and optional cyclophosphamide in relapsed and refractory multiple myeloma (RRMM). Relapsed and refractory multiple myeloma patients were randomized (1:1) to receive carfilzomib (10-min intravenous infusion; 20 mg/m 2 on days 1 and 2 of cycle 1; 27 mg/m 2 thereafter) or a control regimen of low-dose corticosteroids (84 mg of dexamethasone or equivalent corticosteroid) with optional cyclophosphamide (1400 mg) for 28-day cycles. The primary endpoint was overall survival (OS). Three-hundred and fifteen patients were randomized to carfilzomib (n=157) or control (n=158). Both groups had a median of five prior regimens. In the control group, 95% of patients received cyclophosphamide. Median OS was 10.2 (95% confidence interval (CI) 8.4-14.4) vs 10.0 months (95% CI 7.7-12.0) with carfilzomib vs control (hazard ratio=0.975; 95% CI 0.760-1.249; P=0.4172). Progression-free survival was similar between groups; overall response rate was higher with carfilzomib (19.1 vs 11.4%). The most common grade ≥3 adverse events were anemia (25.5 vs 30.7%), thrombocytopenia (24.2 vs 22.2%) and neutropenia (7.6 vs 12.4%) with carfilzomib vs control. Median OS for single-agent carfilzomib was similar to that for an active doublet control regimen in heavily pretreated RRMM patients.

Original languageEnglish
Pages (from-to)107-114
Number of pages8
JournalLeukemia
Volume31
Issue number1
DOIs
Publication statusPublished - Jan 1 2017

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research

Fingerprint Dive into the research topics of 'A randomized phase III study of carfilzomib vs low-dose corticosteroids with optional cyclophosphamide in relapsed and refractory multiple myeloma (FOCUS)'. Together they form a unique fingerprint.

  • Cite this

    Hájek, R., Masszi, T., Petrucci, M. T., Palumbo, A., Rosiñol, L., Nagler, A., Yong, K. L., Oriol, A., Minarik, J., Pour, L., Dimopoulos, M. A., Maisnar, V., Rossi, D., Kasparu, H., Van Droogenbroeck, J., Yehuda, D. B., Hardan, I., Jenner, M., Calbecka, M., ... Ludwig, H. (2017). A randomized phase III study of carfilzomib vs low-dose corticosteroids with optional cyclophosphamide in relapsed and refractory multiple myeloma (FOCUS). Leukemia, 31(1), 107-114. https://doi.org/10.1038/leu.2016.176