(Adnkronos) - "Noi abbiamo ad oggi dei nuovi inibitori prossimali del complemento, con diverse modalità di somministrazione. Il primo capostipite ad essere attivato è un farmaco sottocute a somministrazione bisettimanale, ma adesso abbiamo dei farmaci orali che permettono al paziente di essere del tutto libero dalla medicalizzazione. Fin dalle prime fasi, infatti, si potrà dare al paziente il farmaco così che possa autosomministrarselo. Sarà importantissimo in questo contesto educare il paziente all’aderenza terapeutica”. Così, Bruno Fattizzo, specialista ematologo al Policlinico di Milano, all’Adnkronos Salute, in occasione del media tutorial Dedicato all’Epn, l’moglobinuria parossistica notturna.
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00:00The hemoglobinuria parossistica notturna is a pathology characterized by a destruction
00:10of the red blood cells inside the circulatory torrent due to the aberrant activation of
00:16the complement system.
00:18The complement system is part of the immune system, it is a series of proteins capable
00:23of creating pores on the surface of bacteria, fungi, therefore external pathogens compared
00:30to our body and in hemoglobinuria the patient's red blood cells are particularly susceptible
00:35to this damage and therefore they are hemolyzed, they are destroyed by this complement system.
00:40The result is that the patient becomes anemic, it manifests hemoglobinuria, the hemoglobin
00:45is lost in the urine and in addition it has a risk of thrombosis, then venous thrombosis
00:50of the leg, pulmonary embolism, ictus, so in short, a pathology that can have a big
00:57impact on the patient's survival.
01:01The therapy of hemoglobinuria parossistica notturna has been until today based on the
01:05terminal inhibition of the complement, therefore on drugs that block these proteins of the
01:12complement, in particular the final activation phase that takes the name of C5, these drugs
01:17have reduced hemolysis, increased hemoglobin levels, therefore improved anemia and above all
01:24reduced the risk of thrombosis in our patients, improving survival.
01:28They are now at the doorstep of new treatments that in addition to making the patient survive
01:33more, they can have an improving effect also on his quality of life, because they are
01:39able to further increase the values of hemoglobin, also solve residual anemia
01:45linked to the incomplete functioning of these inhibitors of C5 and therefore really allow
01:53the almost normalization of hemoglobin levels and therefore of the quality of life of our
01:58patients.
01:59We have today new proximal inhibitors of the complement, those that I mentioned earlier,
02:05with different modes of supply, the first flagship to be activated is a drug
02:10under the guise of bi-weekly supply, but now we even have oral drugs that
02:16therefore allow the patient to be completely free from medicalization, from the first
02:21phases the drug can be given to the patient and he can self-supply it, so it will be
02:26very important to educate us clinicians and the patient about therapeutic adherence.