30.11.2009
Canakinumab
effective in children with cryopyrin associated periodic syndromes (Caps)
presented today in Padua during the National Congress of the Italian Society of Pediatrics, the preliminary results of a Phase III efficacy and security of biological therapy canakinumab (Ilaris ®) in children showing that the drug produces a rapid and sustained remission of symptoms in most children with a group of rare and life-threatening autoinflammatory diseases, known as cryopyrin-associated periodic syndromes ( CAPS).
The preliminary analysis of the study, which involved 19 patients with aged between 5 and 17 years, including Italian children also showed how the data of efficacy and safety in the pediatric population are similar to those found in adults. Over 80% of children treated with CAPS canakinumab every 8 weeks showed a complete response to therapy already the eighth day of treatment and 11 of 18 patients had no relapses.
"The demonstration of efficacy and safety of canakinumab in pediatric patients is particularly important because the news is a major step forward in the treatment of children with a group of very rare and debilitating diseases such as CAPS, which currently have very limited treatment options and invasive - says Dr. Mark Gattorno, pediatric rheumatologist at the Gaslini Hospital in Genoa and first author of the study - the results of studies indicate that this therapy offers the possibility of significantly improving the quality of life for patients, thanks to higher efficiency and infrequent administration. "
CAPS include three rare diseases of increasing severity that last a lifetime: familial cold autoinflammatory syndrome (FCAS), Muckle-Wells syndrome (MWS) and neonatal-onset multisystem inflammatory disease (NOMID) 1.2. These syndromes are associated with a gene mutation and characterized by overproduction of interleukin-1 β (IL-1β), a protein (or cytokine) which plays a pivotal role in determining the inflammation and destruction of tessuti1, 2,5,6. Patients with CAPS manifest debilitating fatigue, fever and chronic anemia from the first infanzia1, 2.9. The inflammation can affect the skin, eyes and bones, causing rash, conjunctivitis and arthritis distruttiva1, 2.9. Other serious complications of CAPS include progressive hearing loss, visual and intellectual impairment and amyloidosis, a disease in which the accumulation of proteins may lead to functional insufficiency of organs vitali1, 2,3,10. About 25% of CAPS patients develop systemic amyloidosis, which causes kidney failure and usually death within 5:00 to 10:00 anni3.
Canakinumab is a fully human monoclonal antibody that selectively blocks the IL-1β1 and durable, 5. Neutralizing IL-1β for a sustained period, canakinumab "off" all the symptoms of inflammation in CAPS 1,4,5.
The drug has been approved by the European Union on October 28 this year for the treatment of adults and children aged four years and over with CAPS.
canakinumab's success in the treatment of CAPS has meant that its effectiveness was investigated in other rare diseases such as systemic juvenile idiopathic arthritis (AIGS) or more municipalities, such as gout, chronic obstructive pulmonary disease (COPD) and type 2 diabetes 4,5,7,8.
Italy participates with a significant number of patients in the international study to evaluate the efficacy and safety of canakinumab in patients with CAPS in children. The Gaslini Institute of Genoa, in particular, is one of the centers of reference for autoinflammatory diseases such as CAPS. Currently, the Center coordinates the European project "Eurofever" for the creation of a European register of patients with autoinflammatory diseases, which aims to raise awareness among paediatricians to the rapid recognition of these diseases, to map all patients and to develop common guidelines for treatment.
http://www.salutedomani.com/il_weblog_di_antonio/2009/11/canakinumab-efficace-nei-bambini-affetti-da-sindromi-periodiche-associate-alla-criopirina-caps.html
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