Everolimus (Votubia) for angiomyolipoma associated with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis first line or post surgery

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1 Everolimus (Votubia) for angiomyolipoma associated with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis first line or post surgery April 2011 This technology summary is based on information available at the time of research and a limited literature search. It is not intended to be a definitive statement on the safety, efficacy or effectiveness of the health technology covered and should not be used for commercial purposes. The National Horizon Scanning Centre Research Programme is part of the National Institute for Health Research

2 Everolimus (Votubia) for angiomyolipoma associated with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis first line or post surgery Target group Angiomyolipoma (AML): at least one AML 3cm in diameter; associated with tuberous sclerosis complex (TSC) or sporadic lymphangioleiomyomatosis (LAM) first line or following surgery. Background TSC is a multisystem, autosomal dominant disorder characterised by benign tumours (hamartomas) in multiple organ systems. It results from inactivating mutations in either the TSC1 or TSC2 genes in over 85% of cases, and lesions occur in the brain, kidneys, heart, liver, lungs and skin, causing renal and/or pulmonary complications, and disabling neurological disorders such as epilepsy, learning difficulties and autism 1,2. Pulmonary LAM is a rare disorder characterised by widespread pulmonary proliferation of abnormal smooth-muscle and cystic changes within the lung parenchyma 3. LAM can occur as an isolated form (sporadic) or as a pulmonary manifestation of TSC 2. AMLs are found in both TSC and LAM 1. AMLs, although rare, are the most common benign renal tumours and are composed of adipose tissue, muscle cells, and vascular structures 1. The majority of adult patients with TSC present with multiple bilateral lesions which are usually asymptomatic 2, but can cause life-threatening haemorrhage and impaired renal function 1. Symptoms may develop in patients once tumour size reaches 4cm or greater 4,5 and may include a palpable abdominal mass, haematuria and flank pain 6. There is a higher incidence of haemorrhage and increased morbidity in patients with large AMLs >8cm 7. Technology description Everolimus (Votubia, Afinitor, ABSORB, Certican, RAD001) is an orally active immunosuppressant analogue of sirolimus, a macrolide antibiotic produced by Streptomyces hygroscopicus. These compounds are also known as mammalian Target of Rapamycin (mtor) inhibitors. Everolimus is intended to be used as first line therapy or post surgery, and is administered orally. Everlolimus for AML and LAM is being administered at 10mg daily in the current phase III clinical trial. Everolimus is currently licensed in the EU for coronary artery restenosis, heart transplant rejection, renal cancer (second line therapy) and renal transplant rejection. Everolimus is in the following phases of clinical trials for the stated indications: Phase III Breast cancer. Diffuse large cell B lymphoma. Gastric cancer (late stage disease, second line therapy and third line therapy). Liver transplant rejection. Polycystic kidney disease. Advanced neuroendocrine tumours. Phase II Gastrointestinal stromal tumours (combination therapy and second line therapy). Renal cancer (first line and monotherapy). 2

3 Some common recognised adverse effects of everolimus include: diarrhoea, nausea, abnormal taste, vomiting, rash, pruritus, infections, fatigue, hypertension, insomnia, dyspnoea and epitaxis 8,9. Innovation and/or advantages If licensed for this indication everolimus may offer a new, less invasive treatment option for this patient group, who currently have limited treatment options. Developer Novartis Oncology. Availability, launch or marketing dates, and licensing plans In phase III trials. NHS or Government priority area This topic is relevant to the National Service Framework for Long-term Conditions (2005). Relevant guidance The Tuberous Sclerosis Association. Clinical guidelines for the assessment of behaviour and learning in tuberous sclerosis complex. September The Tuberous Sclerosis Association. Clinical guidelines for the care of patients with tuberous sclerosis complex. April Clinical need and burden of disease Population based studies in the UK report that at an estimated 1 in 1,200 to 1 in 1,400 children under 10 years of age have TSC 2, with incidence at birth of around 1 in 6, It is estimated that there are there are currently around 8,000 people living with TSC in the UK 12. LAM primarily affects premenopausal women between years of age; however, rare case reports of LAM in men exist, primarily in men with TSC 5. No study has yet defined the frequency of LAM in the general population, but it affects approximately 1-3% of people with TSC 2. Multiple bilateral AMLs are found in about 40-80% of adult patients with TSC 13, and are often more symptomatic in women. Renal complications are the most frequent cause of TSC- related death 2. Renal AMLs are present in around 32% of patients with sporadic LAM 2. Existing comparators and treatments Treatment for AML is based on the patient's symptoms and the size of the lesions. Prophylactic treatment would probably be indicated if AML >4cm and growing. Current treatment options include 2a : Interventional measures: for bleeding tumours the preferred method of treatment is tumour embolisation. Other options include: laproscopic partial nephrectomy, cryotherapy, radio-frequency ablations and laproscopic abalation. Total nephrectomy is only indicated when all other treatment options have failed. Sirolimus: in a clinical trial, treatment with sirolimus for a year resulted in a decrease in the size of AMLs in adults with TSC and sporadic LAM 5.Treatment with sirolimus is not currently licensed for this indication in the UK. a Expert opinion. 3

4 Efficacy and safety Trial EXIST-2, NCT ; adults; everolimus or placebo; phase III. Sponsor Novartis Pharmaceuticals. Status Ongoing. Source of Trial registry 14. information Location EU (inc UK), USA, Canada and other countries. Design Randomised, placebo-controlled. Participants and schedule n=99 (planned); adults; TSC diagnosis or sporadic LAM; at least 1 AML of 3cm. Randomised to everolimus 10mg daily or placebo. Follow-up Follow up at week 12, 24, 48 and annually thereafter. Primary AML response rate. outcome Secondary outcomes Time to AML progression, skin lesion response rate, biomarkers, renal function and lung function in participants with LAM. No quality of life outcome measures Expected reporting date included in trial outcomes Estimated primary completion date Q3 2011, with publication anticipated Q Estimated cost and cost impact The cost of everolimus for AML associated with TBC and LAM is not yet finalised, however, everolimus containing afinitor tablets cost 75 per 5mg tablet. Claimed or potential impact speculative Patients Reduced mortality or increased length of survival Other: Services Increased use: drug level monitoring may be required. Decreased use: if licensed, everolimus may reduce the need for emobolisation procedures and support services for deteriorating renal and pulmonary function. Reduction in associated morbidity or Improved quality of life for patients and/or carers Service organisation Other: Quicker, earlier or more accurate diagnosis or identification of disease Staff requirements Costs Increased unit cost compared to alternative Increased costs: more patients coming for treatment Increased costs: capital investment needed New costs: new treatment option. Savings: Other: if licensed, everolimus may reduce the need emobolisation procedures and support services for deteriorating renal and pulmonary function. Other issues Clinical uncertainty or other research question identified: Clinical expert opinion has highlighted uncertainties in the use of everolimus in combination with other drugs used for this indication, and whether trial outcomes will correspond to clinically meaningful reductions in haemorrhage and chronic renal failure. 4

5 References 1 Yates JRW. Tuberous Sclerosis. European Journal of Human Genetics 2006;14: Curatolo P, Bombardieri R and Jozwiak, S. Tuberous sclerosis. The Lancet 2008;372: Sam R, Khalid S, Brecklin C et al. A case of lymphangioleiomyomatosis with membranous nephropathy and likely systemic lupus. Clinical and Experimental Nephrology 2009;13: Blick C, Patel AB, Wilson L et al. Renal angiomyolipoma in a solitary functioning kidney discovered post partum. The Internet Journal of Urology 2006;3. Accessed 3 February Bissler J J, Mc Cormack F X, Young L R et al. Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis. The New England Journal of Medicine 2008;358: Nasir K and Ahmad A. Giant renal angiomyolipomas and pulmonary lymphangioleiomyomatosis. Saudi Journal of Kidney Disease and Translpantation 2010; 21: Dickson M, Ruckle H, Beaghler M et al. Renal angiomyolipoma: optimal treatment based on signs and symptoms. Clinical Nephrology 1998;49L Electronic Medicines Compendium. Everolimus Summary of Product Characteristics. Accessed 3 February British Medical Association and Royal Pharmaceutical Society of Great Britain. British National Formulary. BMJ Group and RPS Publishing. London; September Tuberous Sclerosis Association. Clinical guidelines for the assessment of behaviour and learning in tuberous sclerosis complex. Birmingham; September The Tuberous Sclerosis Association. Clinical guideline for the care of patients with tuberous sclerosis complex. Birmingham; April The Tuberous Sclerosis Association. What is tuberous sclerosis complex (TSC)? Accessed 14 February Bishoy A, Cayed MD, Bruce L et al. Resection of a staggering 36cm angiomyolipoma. Canadian Urological Association Journal 2010;4:E ClinicalTrials.gov. Efficacy and safety study of RAD001 in patients aged 18 or over with angiomyolipoma associated with tuberous sclerosis complex (TSC) or lymphangioleiomyomatosis (LAM) (EXIST-2) Accessed 3 February The National Institute for Health Research National Horizon Scanning Centre Research Programme is funded by the Department of Health. The views expressed in this publication are not necessarily those of the NHS, the NIHR or the Department of Health The National Horizon Scanning Centre, Department of Public Health and Epidemiology University of Birmingham, 90 Vincent Drive, Edgbaston, Birmingham, B15 2SP, England Tel: +44 (0) Fax +44 (0)

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