The emergence of targeted therapies known as tyrosine kinase inhibitors (TKIs) has improved prognoses for certain leukaemia patients.

Despite the success of TKIs in some forms of the disease such as chronic myeloid leukaemia and a small subset of patients with acute lymphocytic leukaemia, until now a TKI had yet to clearly demonstrate improved outcomes in acute myeloid leukaemia (AML).

Insights into the variety of mutations that drive AML have led researchers to study sorafenib, an oral tyrosine kinase inhibitor currently approved for kidney and liver cancer that blocks the activity of several mutated enzymes that can drive growth of AML.

To better determine the safety and efficacy of sorafenib in combination with standard chemotherapy, researchers enrolled 267 AML patients ages 18-60 in a Phase II study and randomised them 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 rate of 38 percent.

The treatment combination was generally well tolerated; however, the sorafenib-treated patients experienced higher rates of certain events such as fever and bleeding.

Sorafenib did not lead to improved overall survival in the treatment group when compared to those patients receiving placebo.

“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 Christoph Röllig, MD, of University Hospital Dresden in Germany.

“In addition to validating these promising results in a larger trial, it will be useful to further evaluate the genetic markers that may predispose some patients to respond better than others to this treatment to fully maximise its potential.”

Watch the press conference and the interview for more.

Source: ASH