AML LI-1

AML16 and LI-1 together have evaluated (or are in the process of evaluating) 11 novel therapies with more treatments due to come on board later in 2016.

Acute myeloid leukaemia (AML) is a condition which affects about 2,400 people every year in the UK. The median age of diagnosis is 67. With intensive chemotherapy, about half of patients aged under 60 and 20% of patients aged over 60 are alive at 5 years. However, it has been estimated that anything up to two-thirds of patients over the age of 65 are not suitable for intensive chemotherapy, and only about 10% of such patients live for 2 years following their diagnosis.

There is a clear need to improve the outcome of these patients. A number of treatments are available, so it is important to evaluate these treatments in the most efficient manner possible.

In 2006, we developed the AML16 trial using a novel design called Pick-A-Winner. The aspiration is to double survival at 2 years from around 11% to about 22%. This requires about 400 patients to be randomised between a new treatment and current standard of care, which is low-dose ara-C. In Pick-A-Winner a number of new treatments are evaluated at the same time, and treatments which are unlikely to deliver a doubling in 2-year survival are dropped early. This allows new, promising treatments to be included in the trial. LI-1 is the successor to AML16 in patients not suitable for intensive chemotherapy, and initially ran from 2011-14, with the programme renewed for five years in 2014.

So far, AML16 and LI-1 together have evaluated (or are in the process of evaluating) 12 novel therapies.