In the treatment of epileptic seizures associated with Dravet syndrome, characterized by a very complex and drug-resistant epilepsy, “fenfluramine, with an innovative mechanism of action, is undoubtedly one more card to improve the quality of life of patients. In more than 50% it allows excellent control of seizures over time and a number of patients are seizure-free. These results, in addition to opening up the possibility of use in other forms of developmental epileptic encephalopathies, are very important because they totally change the quality of life of the patient and of the families, with an extremely positive impact on other aspects that are part of the pathology: behavioral disturbance, cognitive problems “and others. Thus Francesca Darra, head of Uoc Child Neuropsychiatry, Integrated University Hospital of Verona, speaking in Milan at a meeting with the press on the occasion of Aifa’s approval of the reimbursement of Ucb’s drug for the treatment of epileptic seizures associated with Dravet syndrome as add-on therapy to other anti-seizure drugs, for patients 2 years of age and older.
Dravet syndrome is a rare epileptic encephalopathy that usually occurs within the first year of life. It is characterized by severe and frequent sudden and prolonged seizures typically triggered by even minimal changes in body temperature, emotions or flashing lights. The disease also causes severe cognitive, behavioral and motor impairments, which persist into adulthood. People with this syndrome often have resistance to antiepileptic drugs.
“The innovative drug – explains Darra – has a different mechanism of action from other antiepileptics”. It acts on two systems by controlling both “neuronal excitability” and “cognitive aspects”. Compared to other anti-epileptic seizure drugs, continues the specialist, fenfluramine, “acting on different receptors, it increases the amount of serotonin and at the same time acts on the Sigma-1 receptor which is involved in various functions: it regulates ion exchanges and ecittability neuronal and mechanisms related to memory and cognitive functions. After all, the serotonergics that control neuronal excitability also have effects on cognitive aspects and appetite: one effect of the drug is the reduction of the sense of hunger “.
The new drug therefore helps to control the disease on the one hand by reducing the neural hyperexcitability at the origin of the convulsive manifestations, on the other by acting on the cognitive and behavioral aspects. This combined action, which reduces the risk of sudden and unexpected death from epilepsy (Sudep), opens up the possibility of exploring its use in other epileptic encephalopathies.
Currently, “with the compassionate use of fenfluramnine, 52 patients have been treated in Italy for a long period – points out Darra – The positive result of the 75% reduction of crises was recorded in 50% of patients and 11% had lived without convulsion for a prolonged period. The observational study involving Italian patients and other countries has shown that the effectiveness is comparable in the different age groups: small children, adolescents and older children. We also have hopes for the subjects adults “.
The specialist’s wish is to “see a drastic reduction in the number of incapacitating crises and drugs used in polypharmacy, which lead to a poor quality of life and a difficult daily management of the child and adolescent who cannot attend school and other activities. The fact that the stability from the clinical point of view improves the behavioral aspects, which is unexpected, pushes us to consider the use of the drug in other forms of developmental epileptic encephalopathies and in particularly drug-resistant forms “.