Professor Adrian Newland, Clinical Director for Pathology, The Royal London Hospital commented: “I was very pleased to see that NICE has reco
Adult 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
- 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)
- National Institute of Clinical Excellence (NICE): Eltrombopag for the treatment of chronic immune (idiopathic) thrombocytopenic purpura (rev TA205): Technology Appraisal Guidance. July 2013. http://guidance.nice.org.uk/TA293
- Revolade Summary of Product Characteristics (SPC): June 2012.
- Stasi R. Immune thrombocytopenic purpura: the treatment paradigm. Eur J Haem 2009; 82 (suppl. 71): 13-19.
- National Institute for Health and Care Excellence. Final appraisal determination: Romiplostim for the treatment of chronic immune (idiopathic) thrombocytopenic purpura. Available at: http://www.nice.org.uk/nicemedia/live/12027/53541/53541.pdf (accessed 29 May 2013).
- Provan D. Characteristics of immune thrombocytopenic purpura: a guide for clinical practice. Eur J Haem 2009; 82 (suppl.71): 8-12.
- Bennett D et al, Prevalence of Diagnosed Adult Immune Thrombocytopenia in the United Kingdom. Adv Ther 2011; 28 (12): 1096-1104.