Ewch i’r prif gynnwys


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.


Chief Investigator(s)
Funder(s) Bloodwise
ISRCTN 40571019

Key facts

Start date 2011
End date 31 Dec 2020
Grant value £1.2 million
  • Recruiting

General enquiries

Ian Thomas
+44 (0)29 2251 0531