NICE has recommended eltrombopag (Revolade, GlaxoSmithKline) as an option for treating some adults with the bleeding disorder chronic immune (idiopathic) thrombocytopenic purpura [i] in 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 [ii], and whose condition does not respond to other treatments (for example, corticosteroids[iii] or immunoglobulins [iv]). 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 [v], 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.”

This is NICE's final guidance on this technology and now replaces local recommendations across the country.

Ends

Notes to Editors

References and explanation of terms

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

ii. In some cases of ITP, removing the spleen, an organ that is part of the lymphatic system, and is responsible for cleaning the blood, destroying old red blood cells and fighting infection (the operation is called a splenectomy)splenectom, is recommended. This can increase the platelet count in patients with the disorder.

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

iv. Immunoglobulins are also known as antibodies and are used by the immune system to identify and fight bacteria and viruses. They also reduce platelet destruction; therefore injecting them into the bloodstream is used as a treatment for ITP.

v. Rescue therapies are treatment measures taken late in the course of a disease after other therapies have failed. For the treatment of chronic immune (idiopathic) thrombocytopenic purpura, these include corticosteroids, intravenous immunoglobulins and platelet infusions.

vi. 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.

About the final guidance

1. The guidance is available on the NICE website.
2. People currently receiving eltrombopag whose disease does not meet the criteria detailed in section 1.1 of the guidance should be able to continue treatment until they and their clinician consider it appropriate to stop.
3. NICE conducted a review of the original guidance, published in October 2010, because the manufacturer submitted a patient access scheme, which is a scheme proposed by a pharmaceutical company and agreed between the company and the Department of Health (with input from NICE) in order to improve the cost-effectiveness of a drug.
4. Eltrombopag (Revolade, GlaxoSmithKline) increases platelet production through activation of the thrombopoietin receptor. By stimulating platelet production, it helps to reduce bleeding.
5. 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 non-splenectomised patients where surgery is contraindicated.
6. Eltrombopag is taken orally. The summary of product characteristics (SPC) states that the recommended initial dose is 50 mg once daily (patients of East Asian ancestry should start eltrombopag at a reduced dose of 25 mg once daily). Patients should take eltrombopag at least 4 hours before or after any products such as antacids, dairy products (or other calcium-containing food products) or mineral supplements containing polyvalent cations (for example, iron, calcium, magnesium, aluminium, selenium and zinc). If, after initial therapy, platelet counts are below the clinically targeted level (50×10⁹ per litre), the dosage may be increased to a maximum of 75 mg once daily. Treatment should be stopped if the platelet count does not increase sufficiently to avoid clinically significant bleeding after 4 weeks of therapy at a dosage of 75 mg once daily. For full details of dosage and administration, see the summary of product characteristics.
7. The ‘British National Formulary' (BNF; edition 64) states that the net price of a 28-tablet pack of 25 mg eltrombopag is £770 (a single 25 mg dose costs £27.50). The net price of a 28-tablet pack of 50 mg eltrombopag is £1540 (a single 50 mg dose costs £55). The cost per patient will vary with dose adjustment and treatment duration. The manufacturer indicated that the average daily cost of eltrombopag (based on the mean dose of eltrombopag in the EXTEND study of 51.3 mg per day) is £56.43.
8. The manufacturer of eltrombopag (GlaxoSmithKline) has agreed a patient access scheme with the Department of Health that makes eltrombopag available with a discount. The size of the discount is commercial in confidence at the request of the manufacturer. The manufacturer has agreed that the patient access scheme will remain in place until any review of this NICE technology appraisal guidance is published.
9. For people with severe chronic ITP who are at high risk of bleeding and need frequent courses of rescue therapy (that is, the population for which romiplostim is recommended in NICE technology appraisal guidance 221), the Committee agreed that eltrombopag was less effective and less costly than romiplostim. The analysis that mirrored the Committee's preferred assumptions gave ICERs of more than £250,000 saved per QALY lost. The Committee noted that, in this situation, the higher the ICER, the more cost effective a treatment becomes. The Committee therefore concluded that eltrombopag can be considered a cost-effective use of NHS resources in this population.
10. The Committee concluded that there was no sufficiently robust cost-effectiveness evidence to make a recommendation for eltrombopag compared with the pathway of standard care alone. Therefore, it concluded that it could not recommend eltrombopag for patients who do not have severe disease and a high risk of bleeding (that is, the population for which romiplostim is not recommended in NICE technology appraisal guidance 221).
11. The UK incidence of adult ITP is estimated to be around 120 per year and 3000-3500 people are affected at any one time England and Wales. It is more common in women. Among both women and men, incidence increases with age.
12. 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 intercranial haemorrhages
13. 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.
14. NICE published guidance (TA237) in 2010 not recommending eltrombopag for the treatment of chronic immune or idiopathic thrombocytopenic purpura. Please see here.
15. Further information on the review process is given in the NICE Single Technology Appraisal process guide, section 6 here.
16. NICE technology appraisals apply across the NHS in England and Wales.
    Related published NICE guidance
17. Romiplostim for the treatment of chronic immune or idiopathic thrombocytopenic purpura. NICE technology appraisal guidance 221 (2011).
18. Eltrombopag for the treatment of chronic immune or idiopathic thrombocytopenic purpura. NICE technology appraisal guidance 205 (2010).

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