In a nutshell
This study aimed to investigate the safety and effectiveness of ibrutinib (Imbruvica) combined with umbralisib for patients with released/unresponsive chronic lymphocytic leukemia (CLL).
This study concluded that the combination of umbralisib and ibrutinib is well tolerated and effective in relapsed/unresponsive CLL.
Some background
Chronic lymphocytic leukemia (CLL) is often treated with ibrutinib. This is a targeted therapy used for CLL. But some patients do not get a lasting benefit from this treatment. Umbralisib is an experimental targeted therapy.
It was not known if umbralisib used along with ibrutinib in patients with relapsed/unresponsive CLL would be safe and effective.
Methods & findings
This study involved 42 patients with blood cancers. 21 patients had relapsed/unresponsive CLL. Patients had an average of two previous treatments. Patients received umbralisib once a day in a dose of 400, 600 or 800mg. All patients also received 420mg ibrutinib once a day. Patients were treated until disease progression or unacceptable side effects.
No dose-limiting side effects were observed. The maximum tolerated dose of umbralisib was not reached. The maximum tolerated dose is the highest dose of a drug that does not cause unacceptable side effects. The recommended dose of umbralisib when given in combination with ibrutinib was 800mg once a day.
The most common side effects were diarrhea, nausea, infection, and fatigue. Serious side effects occurred in 29% of the 42 patients. These included lipase elevation (damage to pancreatic cells), abnormal heartbeat and low level of phosphate in the blood causing weakness and breathing problems.
Overall, 90% of the 21 patients with CLL responded to treatment. The average survival without cancer growing or spreading for patients with CLL after 2 years was 90%. The average 2-year overall survival was 95%.
The bottom line
This study concluded that the combination of umbralisib and ibrutinib is well tolerated and effective in relapsed/unresponsive CLL.
The fine print
This was the first study of its kind, with a small number of patients. Further studies are needed to test this treatment combination.
Published By :
The Lancet. Haematology
Date :
Dec 14, 2018
