NICE says yes to blood disorder treatment in final guidance

NICE has recommended eltrombopag(Revolade, GlaxoSmithKline) as an option for treating some adults with the bleeding disorder chronic immune (idiopathic) thrombocytopenic purpuraiin final guidance.

 
This condition is caused by abnormally low levels of platelets, which are needed for the blood to clot.
Eltrombopag is licensed for use in adults with the condition who have had their spleen removed, and whose condition does not respond to other treatments (for example, corticosteroidsiiior immunoglobulinsiv).

 
Eltrombopag is also licensed as a second-line treatment in adults who have not had a splenectomy because surgery is not advisable. NICE has recommended eltrombopag as a treatment option for both of these patient groups, only if they have severe disease and a high risk of bleeding that needs frequent courses of rescue therapies, and if the manufacturer makes it available to the NHS under the terms agreed with the Department of Health as part of a patient access scheme.

 
The independent Appraisal Committee concluded that there are few treatment options licensed for people with chronic immune (idiopathic) thrombocytopenic purpura and, like romiplostim (which NICE recommended in 2011), eltrombopag is an effective treatment.

Professor Carole Longson, Health Technology Evaluation Centre Director at NICE, said: “People who have chronic immune thrombocytopenic purpura are at daily risk of nosebleeds or other bleeding that is hard to stop, which can have a significant impact on quality of life. Women with the condition may also experience heavier periods than normal. Some people may experience serious haemorrhaging, which can be fatal. Anxiety related to bleeding may affect work or leisure activities, and, in extreme situations, causes people to become housebound. NICE is, therefore, pleased to recommend eltrombopag as an option for treating people with this condition.”