(Adnkronos) - "Fino al 2000 non esistevano farmaci per ridurre i sintomi della malattia che, essendo cronica, non può essere curata ma può essere trattata. Ora si possono controllare i suoi sintomi ma, nel momento in cui un farmaco non risponde in maniera completa al meccanismo patogenetico che ha definito la malattia, il paziente può continuare ad avere una condizione di emolisi, ovvero di rottura dei suoi globuli rossi, quindi conseguentemente di anemia e di quello stato di stanchezza, fatigue, che caratterizza tutte le forme di anemia e quindi anche quella dell'Emoglobinuria parossistica notturna”. Così ai microfoni dell’Adnkronos Salute, Anna Paola Iori, dirigente medico dipartimento Ematologia, Oncologia e Dermatologia Policlinico Umberto I di Roma, in occasione di un incontro su "Emoglobinuria parossistica notturna: verso un controllo migliore della malattia".
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00:00The main symptoms are anemia, the patient feels tired, exhausted and this is most of the time the reason that leads him to the doctor.
00:15Other times the symptoms are particular, like thrombotic events, so the arrival is more dramatic, let's say, to the emergency room or to the doctor because of the thrombotic event that has led the patient to go to the doctor.
00:33Other times, even worse, there can be a picture of peripheral pancytopenia, that is, all the cell lines, red blood cells, white blood cells, platelets, are low, because in fact hemoglobinuria periossistica nocturnae in that case is also associated with what we call middle aplasia.
00:56So all these varieties of clinical manifestations of the disease can be the symptoms that can bring the most frequent, at least bring the patient to the doctor. Less frequent are dysphagia, erectile dysfunction, abdominal pain, which are always clinical manifestations of the disease, but less frequently.
01:21In all diseases, not always the drugs solve all the problems of the disease, so also the availability of drugs that until 2000 there was nothing to cure the disease, indeed to reduce the symptoms of the disease, to treat the disease, because even today it is not cured, in the English sense of healing hemoglobinuria periossistica nocturnae, but its symptoms are checked.
01:49Well, when a drug does not fully respond to the pathogenic mechanism that has defined the disease, the patient can continue to have a condition of hemolysis, that is, the rupture of his red blood cells, then consequently of anemia, then consequently of that state of fatigue that characterizes everything.
02:19All forms of anemia and therefore also that of hemoglobinuria periossistica nocturnae.
02:24Certainly everything that makes you tired has an impact on the quality of life, because fatigue reduces the performance of anyone in terms of work, in terms of relationship, of family life and therefore there is also an impact on those who live with the person who in a chronic and constant way feels particularly tired.
02:49In addition, the fact that the patient may have to receive treatments in a hospital environment implies that a dependency is created from the hospital and therefore it is clear that when a patient has a lifestyle that requires long journeys for work or for personal choice or in any case general movements,
03:16the possibility of being autonomous in the supply of the drug is certainly a point of strength that will improve his quality of life in the sense of no longer depending on the hospital, but this must also always be considered and evaluated case by case.