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.
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