Present address: University of Chicago.
An open-label, single-arm, phase 2 study of single-agent carfilzomib in patients with relapsed and/or refractory multiple myeloma who have been previously treated with bortezomib
Article first published online: 30 JUL 2012
© 2012 Blackwell Publishing Ltd
British Journal of Haematology
Volume 158, Issue 6, pages 739–748, September 2012
How to Cite
Vij, R., Siegel, D. S., Jagannath, S., Jakubowiak, A. J., Stewart, A. K., McDonagh, K., Bahlis, N., Belch, A., Kunkel, L. A., Wear, S., Wong, A. F., Orlowski, R. Z. and Wang, M. (2012), An open-label, single-arm, phase 2 study of single-agent carfilzomib in patients with relapsed and/or refractory multiple myeloma who have been previously treated with bortezomib. British Journal of Haematology, 158: 739–748. doi: 10.1111/j.1365-2141.2012.09232.x
- Issue published online: 28 AUG 2012
- Article first published online: 30 JUL 2012
- Manuscript Accepted: 6 JUN 2012
- Manuscript Received: 20 APR 2012
- Multiple myeloma;
- proteasome inhibitor;
- phase 2
Carfilzomib is a next-generation proteasome inhibitor that selectively and irreversibly binds to its target. In clinical studies, carfilzomib has shown efficacy in patients with relapsed and/or refractory multiple myeloma (MM) and has demonstrated a tolerable safety profile. In this phase 2, open-label, multicentre clinical trial, 35 patients with relapsed and/or refractory MM following 1–3 prior therapies, including at least one bortezomib-based regimen, received carfilzomib 20 mg/m2 in a twice-weekly, consecutive-day dosing schedule for ≤12 monthly cycles. The best overall response rate (ORR) was 17·1% and the clinical benefit response rate (ORR + minimal response) was 31·4%. The median duration of response was >10·6 months and the median time to progression was 4·6 months. The most common adverse events were fatigue (62·9%), nausea (60·0%), and vomiting (42·9%). No exacerbation of baseline peripheral neuropathy was observed. Single-agent carfilzomib was generally well tolerated for up to 12 treatment cycles and showed activity in patients with relapsed and/or refractory MM who had received prior treatment with bortezomib. These data, combined with an acceptable toxicity profile, support the potential use of carfilzomib in patients with relapsed and/or refractory MM and warrant continued investigation of carfilzomib as single agent or in combination with other agents.