In a nutshell
This paper studied the effectiveness of venetoclax (Venclexta) for the treatment of relapsed or refractory multiple myeloma. Venetoclax at a daily dose up to 1200 mg showed promise in treating patients with relapsed or refractory multiple myeloma with the t(11;14) genetic mutation.
Some background
Multiple myeloma is a cancer cells in the bone marrow. Multiple myeloma is incurable, and after initial treatment, patients may relapse (return of cancer) or are refractory (do not respond to treatment). Venetoclax is a drug that has shown promise in treating chronic lymphocytic leukemia, acute myeloid leukemia and non-Hodgkin lymphoma. Initial studies have shown venetoclax to be sensitive to cells in multiple myeloma, especially those with the t(11;14) genetic mutation. About 15-20% of patients with multiple myeloma have the t(11;14) genetic mutation.
Methods & findings
66 patients with relapsed or refractory multiple myeloma were studied in this phase 1 trial. 46% of patients had the t(11;14) genetic mutation. Patients received venetoclax once daily at doses up to 1200 mg for an average 2.5 months. 83% of patients stopped the study, of which 76% were due to worsening disease and 5% due to side effects.
All patients had at least 1 adverse event (undesired effect of treatment). The most common adverse events were nausea (47%), diarrhea (36%) and vomiting (21%). 21% of patients had low neutrophils (type of white blood cell) and 14% had anemia (low red blood cell levels). 8% of patients had pneumonia, and 5% of patients had sepsis (severe infection).
21% of patients responded to venetoclax treatment. Of these, 85% had a t(11;14) genetic mutation. Among patients with the t(11;14) genetic mutation, 40% responded to venetoclax treatment.
The average duration of response was 9.7 months. Overall, the average time to worsening disease was 2.6 months. Among patients with the t(11;14) genetic mutation, the average time to worsening disease was 6.6 months. Among patients without the t(11;14) genetic mutation, the average time to worsening disease was 1.9 months.
The bottom line
The authors concluded that venetoclax was overall safe and showed promise in the treatment of relapsed or refractory multiple myeloma, especially those with t(11;14) genetic mutation.
The fine print
This was an early phase 1 trial. Furthur studies are necessary.
Published By :
Blood
Date :
Oct 10, 2017
