In a nutshell
This study investigated the safety and effectiveness of two different doses of carfilzomib (Kyprolis) for patients with relapsed or refractory multiple myeloma. This study concluded that once weekly carfilzomib is safe and more effective than the twice weekly dose for these patients.
Multiple myeloma is a type of cancer of the bone marrow that can lead to abnormal immune cells. After initial treatment, most patients with multiple myeloma eventually relapse. Patients who received multiple lines of therapy for refractory (does not respond to treatment) disease tend to have poorer outcomes. For these patients, treatment options are limited.
Carfilzomib is a targeted therapy. This type of treatment only targets cancer cells without damaging normal cells. This leads to cancer cell death with fewer side effects. Carfilzomib is usually given twice a week for 3 weeks. This dosing plan can be difficult for patients, leading to stopping. It is important to study if a simpler dosing plan is effective in patient with multiple myeloma.
Methods & findings
This study involved 478 patients with relapsed or refractory multiple myeloma. 58.2% of patients had stage 2 – 3 disease. Patients received carfilzomib either once a week (50.2%) or twice a week (49.2%). The average follow-up time ranged from 12.0 to 12.6 months.
The response rate was significantly higher in the once weekly group (62.9%) than the twice weekly group (40.8%). At follow-up, one-year overall survival (patients still alive 1 year later) was 76.6% (once weekly) and 71.9% (twice weekly).
The average progression-free survival (PFS – patients alive without a return of disease) was significantly higher in the once weekly group (11.2 months) than the twice weekly group (7.6 months). Time taken for disease to return (progression) was longer in the once weekly group (12.4 months) than the twice weekly group (8.5 months).
Overall, 95% (once weekly) and 97% (twice weekly) of patients had side effects. Severe or life-threatening side effects were more common in the once weekly group (68%) than the twice weekly group (62%). Of these, the most common included low red blood cell count (18%) and low platelet count (cells involved in blood clotting; 7%). 10% (once weekly) versus 7% (twice weekly) also reported pneumonia (lung infection).
21% (once weekly) and 13% (twice weekly) of patients reported serious treatment-related side effects. These included pneumonia and tumor lysis syndrome (a toxic side effect of dead cancer cells). 8% (once weekly) and 5% (twice weekly) of patients stopped treatment due to side effects.
The bottom line
This study concluded that once weekly carfilzomib is safe and more effective than the twice weekly dose for patients with relapsed or refractory multiple myeloma.
The fine print
This study received funding support from Amgen, the manufacturer of carfilzomib.
Published By :
The Lancet. Oncology
May 31, 2018
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