Dr Ehninger presented the results of a phase II randomised study on the safety and efficacy of Sorafenib at EHA 2015.

Despite the success of tyrosine kinase inhibitors in some forms of leukaemias such as chronic myeloid leukaemia and acute lymphoblastic leukaemia, until now a kinase inhibitor had yet to demonstrate activity in acute myeloid leukaemia (AML).

Insights on the variety of mutations that fuel AML have led researchers to study sorafenib, an investigational oral tyrosine kinase inhibitor that blocks the activity of several mutated enzymes that can drive growth of the disease.

To better determine the safety and efficacy of sorafenib, 267 AML patients 18-60 years of age were enrolled in a phase II study and randomised to receive either sorafenib (134 patients) or placebo (133 patients) in addition to a standard protocol.

After three years of follow-up, sorafenib-treated patients had a median event-free survival of 20.5 months and a three-year relapse-free survival rate of 56 percent.

By comparison, patients receiving placebo had a median event-free survival of 9.2 months and a three-year relapse-free survival of 38 percent.

Sorafenib treatment increased the likelihood of side effects such as fever, rash and bleedings, but was generally well tolerated.

“The positive, lasting responses we observed in AML patients receiving sorafenib represent the first randomised evidence for a clinical benefit of a tyrosine kinase inhibitor in this type of leukaemia,” said lead study author Professor Gerhard Ehninger, of University Hospital Dresden in Germany.

Watch the video interview and press conference for more information.

Source: EHA