Anakinra (Kineret®), an interleukin-1 (IL-1) inhibitor, is now available in the UK for treatment of cryopyrin-associated periodic syndromes (CAPS) in children and adults.
It is the first CAPS treatment licensed for use in infants as young as 8 months by the Medicines and Healthcare Products Regulatory Agency (MHRA).
Anakinra (Kineret®), an interleukin-1 (IL-1) inhibitor, is now available in the UK for treatment of cryopyrin-associated periodic syndromes (CAPS) in children and adults.
It is the first CAPS treatment licensed for use in infants as young as 8 months by the Medicines and Healthcare Products Regulatory Agency (MHRA).
CAPS is an umbrella term for a number of ultra-rare, life-long, severely debilitating autoinflammatory diseases, which cause rash, headache, fever, joint pain and other inflammatory symptoms. Patients suffer substantial pain and progressive reduction in their quality of life.
The most severe form of the condition causes chronic aseptic meningitis. Late complications include hydrocephalus, developmental delay, mental retardation and hearing loss.(1)
People with rare diseases: ‘significant and underserved population’
Despite severe symptoms, children with CAPS typically experience a long journey to diagnosis. This plight is common for people with rare diseases, and it is hoped that the national rare disease strategy being implemented by the four home governments will help reduce delays to diagnosis and ensure that more patients are offered coordinated care close to home. Although numbers of patients with individual rare diseases are small, when grouped together their sufferers are a significant and underserved population; one in 17 people in the UK will be diagnosed with rare disease.(2) CAPS affects approximately one in 1,000,000 people in the UK.(3)
Professor Taunton Southwood, Professor of Paediatric Rheumatology, University of Birmingham and Paediatric Rheumatologist at Birmingham Children’s Hospital welcomed the approval of anakinra, saying: “While the number of patients with CAPS is small, it has a heavy burden on the lives of those affected. These are patients who often wait years for a diagnosis, and as a result of the rarity of their disease, may have to work hard to get appropriate treatment. Clinicians in secondary care should suspect an autoinflammatory condition such as CAPS – and consider referring to a tertiary rheumatology centre – if they encounter unexplained fevers, rash, headaches and joint pain, especially if symptoms do not respond to standard care.”
Until now, the only licensed treatment was for patients over 2 years old, and patients have to travel to London to receive this treatment. Patients can now choose to have their therapy managed at home with daily injections, monitored by their local specialist.
Nicky Freeman, Paediatric Rheumatology Nurse Specialist at Birmingham Children’s Hospital commented: “Working closely with CAPS patients and their families you see that travelling long distances for treatment isn’t always feasible, which is why it’s great to have the option to manage their treatment at home, with shared care at a local hospital coordinated by the tertiary hospital.”
To facilitate use in paediatric patients, anakinra is now supplied in a syringe with marked graduations to allow for dose adjustments. Anakinra has a well-documented safety profile. It inhibits IL-1, an inflammatory cytokine, which is implicated in a number of rare inflammatory conditions.
References
1. Hashkes PJ, Autoinflammatory disease. Pediatr Clin North Am 2012;59:447–70.
2. http://www.raredisease.org.uk/about-rare-diseases.htm (accessed 4 April 2014).
3. National Specialised Commissioning Team. National treatment service for cryopyrin associated periodic fever syndromes (CAPS). Service Specification 2012/13.
