In final guidance published by NICE ( National Institute for Health and Clinical Excellence ) Eltrombopag ( Revolade ) is not recommended for treating chronic immune ( idiopathic ) thrombocytopenic purpura ( ITP ) in splenectomised adults who do not respond to other treatments ( for example, corticosteroids, immunoglobulins ), or as a second-line treatment for non-splenectomised adults when surgery is not advised.

The independent Appraisal Committee was not able to recommend Eltrombopag for idiopathic thrombocytopenic purpura because the evidence presented regarding its long-term effectiveness is unclear. Eltrombopag was shown to raise platelet levels, but its cost in relation to known and projected health benefits was also far greater than what is normally considered a cost effective use of NHS ( National Health Service ) resources. Eltrombopag provides health gains at a cost of £104,100 per quality adjusted life year ( QALY ) for splenectomised people, and £116,750 per QALY for non-splenectomised people.

The Appraisal Committee has understood that people living with idiopathic thrombocytopenic purpura are at daily risk of bleeding and subsequent bruising, and that this can have detrimental effect on quality of life, but they were not able to recommend Eltrombopag as a treatment for this chronic condition. From the clinical evidence presented, it is not clear how much long term health benefits Eltrombopag provides, compared with current alternative treatments available. This, coupled with the high cost of Eltrombopag in relation to the estimated health benefits, led the Committee to conclude that it could not recommend Eltrombopag for people with chronic idiopathic thrombocytopenic purpura.

Eltrombopag increases platelet production through activation of the thrombopoietin receptor. By stimulating platelet production, it helps to reduce bleeding. Eltrombopag is for oral administration. The summary of product characteristics ( SPC ) states that the recommended initial dose is 50 mg once daily. If after 2-3 weeks of initial therapy, the platelet counts are below the clinically targeted levels ( 50 × 109 per litre ), the dose may be increased to a maximum of 75 mg once daily. Treatment should be discontinued if the platelet count does not increase sufficiently to avoid clinically important bleeding after 4 weeks of therapy at 75 mg.

About 24 per 100,000 adults have idiopathic thrombocytopenic purpura. It is more common in women. Among both women and men, incidence is higher in older ages.
In adults, idiopathic thrombocytopenic purpura comes on gradually and it usually does not follow a viral illness. There may be no symptoms, mild bruising or bleeding, or severe bleeding.
Idiopathic thrombocytopenic purpura is not normally fatal but some rare cases may be life-threatening because of a risk of spontaneous haemorrhages.
Because most adults with ITP do not have any symptoms, idiopathic thrombocytopenic purpura is usually diagnosed on a routine blood test that has been done for other reasons. The full blood count shows a lower number of platelets than normal.

Source: NICE, 2010

XagenaMedicine2010


Link: Xapedia - Medical Encyclopedia