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NICE publishes final guidance on eltrombopag for the treatment of chronic immune (idiopathic) thrombocytopenic purpura

In final guidance published by NICE today (27 October 2010), eltrombopag (Revolade, GlaxoSmithKline) is not recommended for treating chronic immune (idiopathic) thrombocytopenic purpura[1] (ITP) in splenectomised adults[2] who do not respond to other treatments (for example, corticosteroids[3], immunoglobulins[4]), 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 ITP because the evidence presented regarding its long-term effectiveness is unclear. Eltrombopag was shown to raise platelet levels[5], 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 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.

Dr Carole Longson, Health Technology Evaluation Centre Director at NICE said: "The Appraisal Committee understands that people living with ITP are at daily risk of bleeding and subsequent bruising, and know 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 ITP."

The final guidance can be found at http://guidance.nice.org.uk/TA/Wave17/14

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Notes to Editors

About the appraisal

1. The final guidance is available at http://guidance.nice.org.uk/TA/Wave17/14

2. Eltrombopag (Revolade, GlaxoSmithKline) increases platelet production through activation of the thrombopoietin receptor. By stimulating platelet production, it helps to reduce bleeding.

3. Eltrombopag has UK marketing authorisation for the treatment of adult chronic immune (idiopathic) thrombocytopenic purpura (ITP) in splenectomised patients who are not responding to other treatments (e.g. corticosteroids, immunoglobulins) and as a second line treatment for adult non-splenectomised patients where surgery is contraindicated.

4. 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. For full details of dose and administration, see the SPC.

5. The price per 50 mg tablet of eltrombopag is £55.00. It is available in 28-tablet packs containing 25 mg tablets (£770.00) or 50 mg tablets (£1540.00). Therefore, the cost of a four week course of treatment is between £1540.00 and £2310.00, and the annual cost is between £20,020.00 and £33,030.00. Costs may vary in different settings because of negotiated procurement discounts.

6. About 24 per 100,000 adults have ITP. It is more common in women. Among both women and men, incidence is higher in older ages.

7. In adults, ITP comes on gradually and it usually does not follow a viral illness. There may be no symptoms, mild bruising or bleeding, or severe bleeding. ITP is not normally fatal but some rare cases may be life-threatening because of a risk of spontaneous haemorrhages

8. Because most adults with ITP do not have any symptoms, ITP 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.

About NICE

1. The National Institute for Health and Care Excellence (NICE) is the independent organisation responsible for providing national guidance on the promotion of good health and the prevention and treatment of ill health.

2. NICE produces guidance in three areas of health:

  • public health - guidance on the promotion of good health and the prevention of ill health for those working in the NHS, local authorities and the wider public and voluntary sector.
  • health technologies - guidance on the use of new and existing medicines, treatments, procedures and medical technologies within the NHS.
  • clinical practice - guidance on the appropriate treatment and care of people with specific diseases and conditions within the NHS.

Footnotes

[1] Chronic immune (idiopathic) thrombocytopenic purpura is a bleeding disorder caused by abnormally low levels of platelets in the blood. The disorder has no apparent cause.

[2] Splenectomised adults have had their spleen surgically removed; the spleen is an organ that is part of the lymphatic system. In some cases of ITP, a splenectomysplenectom is recommended, which can increase the platelet count in patients with the disorder.

[3] Corticosteroids are a type of steroid, which in turn is a type of hormone. Corticosteroids are commonly used to reduce inflammation, suppress the immune system, and replace hormones in the body.

[4] Immunoglobulins are also know as antibodies and are used by the immune system to identify and neutralise bacteria and viruses. They also reduce platelet destruction; therefore injecting them into the bloodstream is used as a treatment for ITP.

[5] Platelets are needed for the blood to clot. Normal platelet levels are between 150 and 400 × 109 per litre of blood. Low platelet counts (below 30 × 109 per litre) can result in bleeding and bruising.

This page was last updated: 27 October 2010

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Copyright 2014 National Institute for Health and Care Excellence. All rights reserved.

Accessibility | Cymraeg | Freedom of information | Vision Impaired | Contact Us | Glossary | Data protection | Copyright | Disclaimer | Terms and conditions

Copyright 2014 National Institute for Health and Care Excellence. All rights reserved.