(Adnkronos) - “Fenfluramina ha dimostrato un'efficacia significativa sia nei trial controllati a breve termine, sia nei trattamenti in aperto di real world effettuati anche per anni. Possiamo dire che almeno il 50% dei pazienti che hanno assunto” il farmaco “hanno tratto un beneficio estremamente significativo, sia sul piano di controllo delle crisi, sia sul piano della qualità di vita”. Così Bernardo Dalla Bernardina, già professore di Neuropsichiatria infantile dell’università degli studi di Verona e direttore scientifico del Centro di ricerca per le epilessie pediatriche dell’Aoui ospedale Donna e Bambino di Verona, all’incontro con la stampa organizzato da Ucb per annunciare il via libera di Aifa alla rimborsabilità di fenfluramina per il trattamento della sindrome di Lennox - Gastaut, una grave encefalopatia dello sviluppo ed epilettica.
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00:00Pharmaceuticals, until now available, have always obtained results that are both modest and transient,
00:15and therefore have never been able to modify the outcome, the evolution of these subjects,
00:23which is systematically unfavorable both on the level of persistence and crisis,
00:28as we have said, of all the associated handicaps.
00:33Today we have this new drug at our disposal, which has shown significant effectiveness
00:43both in controlled trials, in the short term,
00:47and in in-open real-world treatments that have also been carried out for years.
00:57And we can say that at least 50% of the patients who have taken this drug
01:05have had an extremely significant benefit.
01:10A benefit both on the level of crisis control and on the level of quality of life,
01:20partly because obviously, when there are no more crises, there is an improvement in quality of life,
01:30but also for a direct effect of phenfluramine on the cognitive skills of the behavioral language,
01:41on the quality of sleep.
01:44This is probably due to the fact that phenfluramine does not have a single mechanism of action,
01:52but has multiple mechanisms of action, and therefore is able to obtain results,
01:59even though it is a form that can be due to very different causes.
02:08So it acts on the mechanism of unbalancing that supports crises,
02:14regardless of the cause that has caused them,
02:18rebalancing the unbalancing that produces crises,
02:25through the mechanisms of action, in particular on the receptors of serotonin
02:32and on the receptors of sigma-1, which are significantly involved in the genesis,
02:40in the determination of many forms of epilepsy.