Ruling on once-a-day, oral therapy gives hope for patients with severe disease when other treatments have failed. Patients in Scotland living with chronic immune thrombocytopenic purpura (ITP), have access to Revolade® (eltrombopag), a once-daily oral treatment.

The Scottish Medicines Consortium (SMC) has accepted eltrombopag for restricted use within NHS Scotland. Eltrombopag treatment should remain under the supervision of a physician who is experienced in the treatment of haematological diseases. Indication under review: Eltrombopag is indicated for adult chronic immune (idiopathic) thrombocytopenic purpura (ITP) splenectomised patients who are refractory to other treatments (e.g. corticosteroids, immunoglobulins). Eltrombopag may be considered as second-line treatment for adult non splenectomised patients where surgery is contraindicated.

SMC restriction: in both the splenectomised and non-splenectomised patient populations, restricted to use in patients with severe symptomatic ITP or a high risk of bleeding. Eltrombopag has been shown to be significantly more effective than placebo in raising and maintaining platelet counts at (or above) a minimum target level in previously treated patients with ITP.

Scotland is the first place in the UK to have recommended eltrombopag, as a cost effective treatment for the NHS, which is positive news for patients with this rare disease who have few treatment options available. Eltrombopag is the first oral thrombopoietin receptor agonist (TRA) licensed for this patient population. It has an innovative mode of action which increases platelet production; a different therapeutic approach to traditional ITP treatments, which reduce platelet destruction.

Dr Mark Drummond PhD FRCPath, Consultant Haematologist & Honorary Senior Clinical Lecturer, Gartnavel General Hospital, commented:

This is fantastic news for patients in Scotland and we are delighted with the decision from the SMC. Eltrombopag is an innovative treatment option which represents a big step forward in the management of ITP - it is relatively easy to take and offers a reduced risk of bleeding during treatment, something that we know worries patients and has an impact on their quality of life.

Chronic ITP is a serious condition, where patients have low platelet levels either because the body does not produce enough platelets or destroys too many. Platelets are essential to normal clotting, so patients with ITP are at increased risk of bleeding and may develop bruises, experience nose or gum bleeds and other types of bleeding that are difficult to stop. Although a very rare occurrence, patients with ITP can suffer from bleeding in the brain which is potentially fatal. Quality of life is adversely affected in patients with chronic ITP, with a fear of bleeding limiting patients' daily activities, such as participating in sports or manual work. Fatigue and depression are also often associated with ITP.

Eltrombopag received marketing authorisation in Europe in March 2010 based on results from two Phase III, randomised, double-blind, placebo-controlled clinical trials and two open-label studies involving previously-treated adult patients with chronic ITP. These studies showed that the patients treated with eltrombopag experienced significant increases in platelet counts and a reduction in the incidence of bleeding, compared with those taking placebo. Treatment with eltrombopag also allowed patients to reduce the dose of their concomitant medications, such as steroids.

Results from one of the clinical trials (RAISE) showed that the odds of responding over the 6-month treatment period were 8 times greater in eltrombopag-treated subjects than in placebo-treated subjects (p