In a nutshell
This study aimed to investigate whether venetoclax (Venclexta) is effective and well tolerated in patients with chronic lymphocytic leukemia (CLL) in a real-world setting.
This study concluded that venetoclax is safe and effective in the real-world setting in these patients.
Venetoclax is a type of targeted therapy called a BCL2 inhibitor. It blocks BCL2 which cancer cells use to grow. It has proven high effectiveness in clinical trials with patients with relapsed/refractory (R/R) CLL. However, in clinical trials, patients are carefully selected and are usually younger, fitter, and healthier compared to the real-world population. Therefore, the safety and effectiveness of venetoclax in patients with CLLin the real-world setting (outside drug trials) are still under investigation.
Methods & findings
This study involved 60 patients with CLL and 7 patients with Richter syndrome (RS). RS is the development of aggressive lymphoma in patients with CLL. All patients were treated with venetoclax and were followed up for an average of 12 months.
74% of patients had TP53 disruption and 58% had complex karyotype. These are adverse genetic features often found in cancer patients.
The overall response rate (ORR) to venetoclax treatment was 75% in patients with CLL. Of the 7 patients with RS, 2 responded to treatment. 61% of patients with CLL were alive without cancer worsening after 1 year. The 1-year overall survival (OS) was 70%. The OS was 90% in patients who responded to treatment compared to 29% in those who did not respond.
22% of patients experienced tumor lysis syndrome (TLS) as a side effect. TLS is a massive release of tumor cells into the bloodstream as a result of cancer cells dying. This can lead to the accumulation of toxic substances into the blood. 24% of patients were hospitalized for severe infection.
The bottom line
This study concluded that venetoclax is a safe and effective treatment for CLL patients in a real-world setting.
The fine print
This study included a very small number of participants. Many of the patients had genetic abnormalities that can respond differently to treatment. The follow-up period was also very short.
Published By :
Annals of Hematology
Jan 25, 2021