After 3.5 year process, NICE recommends Revolade (eltrombopag) for treatment of chronic immune thrombocytopenic purpura (ITP), UK

Date: Jul-24-2013Adult patients in England and Wales living with chronic immune (idiopathic) thrombocytopenic purpura (cITP), an immune disorder associated with low blood platelet counts, will now be able to access Revolade® (eltrombopag) on the National Health Service (NHS), following final guidance (known as Technology Appraisal Guidance - TAG) issued today by the National Institute for Health and Care Excellence (NICE).[1]

Professor Adrian Newland, Clinical Director for Pathology, The Royal London Hospital commented: "I was very pleased to see that NICE has recognised the clinical value and cost-effectiveness of eltrombopag in their guidance. With their recommendation on the use of eltrombopag within its licensed indication, we now have an important addition to the treatment options for patients with severe or refractory disease. When added to conventional immunosuppressive therapy, eltrombopag, an oral thrombopoietin receptor agonist, increases response rates compared to placebo and in some patients allows reduction or discontinuation of concomitant treatments for chronic ITP. The option to prescribe this agent will be of benefit to patients with chronic ITP and access to an oral agent is welcome."

The NICE TAG recommends eltrombopag within its licensed indication for the treatment of adults with cITP, if:[1]
their condition is refractory to standard active treatments and rescue therapies, or
they have severe disease and a high risk of bleeding that needs frequent courses of rescue therapies
and
the manufacturer provides eltrombopag with the discount agreed in the patient access scheme.

Eltrombopag is an oral thrombopoietin receptor agonist (TPO-RA) taken at home as a once-daily tablet.[2] Eltrombopag stimulates the growth and maturation of cells in the bone marrow (megakaryocytes) that produce platelets, thereby increasing platelet production.[3] The only other licensed TPO-RA, which is also recommended by NICE, is N-plate (romiplostim); romiplostim is given in the form of a weekly injection.[4]

Erik Van Snippenberg, General Manager, GlaxoSmithKline (GSK) UK commented: "This has been a lengthy three and a half year long appraisal process. We are pleased that NICE has recommended eltrombopag and that the small number of cITP patients in England and Wales are granted access to an alternative treatment option offering the benefit of oral convenience. With eltrombopag we hope to ultimately make a meaningful difference in the quality of life of cITP patients and contribute to potential savings for the NHS."

Adverse effects associated with eltrombopag include headache, insomnia, paraesthesia, cataract, dry eye, nausea, diarrhoea, constipation, upper abdominal pain, hepatobiliary disorders, rash, pruritus, alopecia, arthralgia, myalgia, muscle spasm, bone pain, fatigue and oedema peripheral. Other serious side effects include bleeding after stopping treatment, high platelet counts and risk of blood clots, as well as liver and bone marrow problems.[2]

In patients with cITP, the immune system prematurely destroys platelets or impairs platelet production so that platelets are lost from the circulation faster than they can be replaced from the bone marrow, where they are made.[5] This results in a shortage of platelets (thrombocytopenia). While some patients are asymptomatic or develop only mild bruising, others may have serious bleeding, which affects their quality of life and in some instances may be fatal.[5] It is estimated that ITP currently affects 50 in 100,000 people in the UK.[6]

About eltrombopag

Eltrombopag is indicated for adult chronic immune (idiopathic) thrombocytopenic purpura (cITP) splenectomised (spleen has been removed) 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.[4]

Eltrombopag treatment should remain under the supervision of a physician who is experienced in the treatment of haematological diseases.[4]

Further information can be found in the Summary of Product Characteristics.

About cITP (per GSK's submission to NICE)

Primary immune thrombocytopenia (ITP), formerly known as idiopathic thrombocytopenic purpura, is an orphan disease. The prevalence of ITP in the UK is estimated at 50 in 100,000 people.[6]

It is uncertain what causes ITP, in which increased platelet destruction and impaired platelet production, lead to low platelet counts and impaired blood clotting.[2] The resulting bleeding symptoms range from mild bruising to serious haemorrhage, which can be fatal.[2]

Patients who have had ITP for more than 12 months are considered to have cITP.[7] Quality of life is adversely affected in patients with cITP, with a fear of bleeding limiting patients' daily activities.[8]

The aims of treatment are to reduce the risk of bleeding by elevating platelet count, whilst minimising treatment related side effects.[9] The management of ITP is complex; following first line treatment with corticosteroids or immunoglobulins there is no clearly defined treatment pathway and the clinical trial evidence is scarce.[3] It is generally accepted that management of ITP is tailored to the individual patient depending on their symptoms, platelet count, lifestyle and the adverse events associated with the different therapies.[3,9] Splenectomy (removal of the spleen) is a potentially curative treatment option for cITP but is invasive, irreversible and not appropriate for all patients.[10] Splenectomy provides an initial haemostatic response in 80 per cent of patients and around 66 per cent will have a sustained complete response.[7] About 14 per cent of patients do not respond and approximately 20 per cent of responders relapse following splenectomy.[7] Patients typically cycle through several therapy options, some of which have significant side effects and most of which are not licenced as treatments for ITP.[3]

Eltrombopag is one of two thrombopoietin receptor agonists (TPO-RAs) licensed for the treatment of cITP. The use of eltrombopag in cITP is supported by robust evidence from randomised controlled trials.[11-16]

Courtesy: Medical News Today Note: Any medical information available in this news section is not intended as a substitute for informed medical advice and you should not take any action before consulting with a health care professional.