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Juvenile idiopathic arthritis: study of oral therapy with tofacitinib by Gaslini of Genoa

By Cristiano Antonino Last updated Nov 16, 2021

Juvenile idiopathic arthritis: IRCCS Gaslini researchers demonstrate the efficacy of a new oral biological therapy

The study, which involved 225 young patients from 64 paediatric hospitals in 20 countries, led to the drug’s approval by the EMA and FDA for the treatment of juvenile polyarticular idiopathic arthritis and juvenile psoriatic arthritis.

The prestigious scientific journal The Lancet has published the results of a therapeutic study of an innovative biological drug called tofacitinib

The drug acts on the cells of the immune system by inhibiting the release of molecules that stimulate inflammation.

Data from the international study, coordinated by researchers at the IRCCS Istituto Giannina Gaslini in Genoa, show that the drug is effective and safe for the treatment of juvenile idiopathic arthritis.

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Juvenile idiopathic arthritis is a chronic autoimmune disease

It is of unknown origin, affects the paediatric population and often starts at a very early age.

It represents one of the most important causes of acquired disability in children.

It is estimated that around 60,000 children in Europe are affected by this disease.

The study, coordinated by Dr Nicola Ruperto, paediatrician and researcher at the IRCCS Istituto Giannina Gaslini, involved 225 young patients from 64 paediatric hospitals in 20 countries organised in two international research networks: Paediatric Rheumatology International Trials Organisation (PRINTO) and Pediatric Rheumatology Collaborative Study Group (PRCSG).

“Polyarticular juvenile idiopathic arthritis is a chronic disabling disease that limits children’s participation in daily activities and has a major impact on their quality of life,” Dr Nicola Ruperto stressed.

“Although several effective and safe therapies are already available, about one third of patients do not respond satisfactorily.

Furthermore, tofacitinib represents an interesting alternative to traditional biological drugs, also because it can be administered orally, thus avoiding the inconvenience for children and their families of injections or infusions of the drug, which often have to be carried out in a hospital environment.

Oral administration offers important advantages: it makes the therapy more acceptable to children, especially the youngest ones, and facilitates the parents’ task”.

Polyarticular juvenile idiopathic arthritis: the excellence of Gaslini in Genoa

“The article confirms the international excellence of the Gaslini Institute researchers and encourages us to work to extend this coordination ability also to other sectors in which our IRCCS has a leadership role” comments the General Director of Gaslini Dr. Renato Botti.

Our paediatric hospital, which is a Scientific Institute for Hospitalisation and Care,” says the President of the Liguria Region and Health Councillor Giovanni Toti, “is a jewel in the crown for Liguria, for the country and at international level, not only in the care of young patients who come to Genoa from different parts of Italy and the world, but also in scientific research aimed at improving the lives of children with rare and disabling diseases and their families.

The fact that Gaslini professionals led this study as the leader of 64 paediatric hospitals from 20 countries fills us with pride”.

Thanks to this study, tofacitinib has been approved by both the European Medicines Agency (EMA) and the Food and Drug Administration (FDA) in the United States for the treatment of polyarticular juvenile idiopathic arthritis and juvenile psoriatic arthritis.

Children aged two years and older who have responded inadequately to previous treatment with anti-rheumatic drugs can be treated.

“The article was considered so relevant by the Lancet that it deserved the editorial commentary, aimed at highlighting the revolutionary significance of the availability of the first oral biological therapy for the treatment of juvenile idiopathic arthritis,” says the scientific director of the Gaslini Institute, Prof. Angelo Ravelli.