Università degli studi di Modena e Reggio Emilia

A new treatment option for children and adolescents with narcolepsy: results of a study coordinated by Unimore in Lancet Neurology

A multicentre, double-blind clinical study demonstrates the efficacy of pitolisant in the treatment of paediatric narcolepsy symptoms, offering a valuable therapeutic option for children and adolescents suffering from a disease that profoundly affects their daily life and school learning.

A study on the treatment of paediatric narcolepsy has just been published online in the April 2023 issue of Lancet Neurology entitled  “Safety and efficacy of pitolisant in children aged 6 years or older with narcolepsy with or without cataplexy: a double blind, randomised, placebo-controlled trial.” (Yves Dauvilliers, et al. www.thelancet.com/neurology) .

This is a multicentre, randomised, double-blind clinical trial that compared the efficacy of pitolisant with that of placebo in the treatment of narcolepsy symptoms in children and adolescents aged 6 to under 18 years. Narcolepsy is a disease that often occurs in childhood, although the diagnosis may be made after many years because the referral of patients to centres specialised in diagnosis is sometimes not timely.

Professor Giuseppe Plazzi, Full Professor of Child Neuropsychiatry and Director of the School of Specialisation in Child Neuropsychiatry at Unimore, with one of the largest case histories of paediatric narcolepsy in the world, coordinated the study involving Italy, France, Holland, Finland and Russia.

'Although this new paediatric indication represents a small step for the treatment of an often paediatric and severely disabling disease such as narcolepsy,' comments Professor Giuseppe Plazzi, 'it does in fact testify to a new and important interest in this rare sleep disorder. Narcolepsy is undoubtedly one of the most interesting neurological diseases in neuroscience research today, and cases are now more easily recognised and diagnosed even in adolescents; having drugs with a specific indication, proven to be effective and safe, will reassure patients and prescribers, facilitate rapid access to treatment, and prevent young patients from the devastating consequences of not receiving treatment early on."

The clinical study published in Lancet Neurology, preceded by a pharmacokinetic study, enrolled 110 children over the age of 6 years with narcolepsy type 1 or type 2. The duration of the experimental therapy was eight weeks.

Pitolisant has long been used in the treatment of this disease in adults, in which it has proven to be effective on several symptoms and well tolerated. Scales specific to these age groups, such as the Ullanlinna Narcolepsy Scale, have been used to assess efficacy in children and adolescents, in addition to the usual variables, the weekly frequency of cataplexy episodes and objective instruments such as the Maintenance of Alertness Test (MWT).

Of the 110 randomised patients, 72 were treated with pitolisant and 38 received placebo; the results indicated that both excessive daytime sleepiness and the frequency of cataplexy improved statistically significantly in the group receiving the active drug, compared to the placebo group. The verdict on the effect of the therapy expressed by doctors and patients themselves also confirmed the significantly greater effectiveness of pitolisant, compared to placebo.

Regarding tolerability, no differences were observed in the frequency of undesirable effects between the two treatment groups, adverse events were almost always mild, headache and insomnia, as in adults.

In their conclusions, the study authors pointed out that the results confirm the good benefit/risk balance of pitolisant in paediatric patients with narcolepsy. Also on the basis of the data collected in this research, the Committee for Medicinal Products for Human Use of the European Medicines Agency (EMA) has given a positive opinion on the use of pitolisant in the treatment of paediatric narcolepsy symptoms, adding a valuable therapeutic option for children and adolescents suffering from a disease that profoundly impairs them in their daily life and school learning.

Giuseppe Plazzi, a neurologist, is Full Professor of Child Neuropsychiatry, Director of the School of Specialisation in Child Neuropsychiatry at the University of Modena and Reggio Emilia, and Director of the Sleep Centre at the IRCCS-Institute of Neurological Science in Bologna. He is Past-President of the Italian Association of Sleep Medicine and President of the European Narcolepsy Network. He sits on the Board of several International Scientific Societies and is author/co-author of more than 450 scientific papers published in international, peer-reviewed and indexed journals on neurology, epilepsy, sleep medicine.

Categorie: International - english