(Adnkronos) - L’Emoglobinuria Parossistica Notturna - Epn è una malattia debilitante che si verifica quando una parte del sistema immunitario, chiamata sistema del complemento, attacca i globuli rossi (Rbc) privi di alcune proteine regolatrici. Questa distruzione dei globuli rossi è chiamata emolisi. La Epn è cronica ed è dunque trattabile ma non curabile. Sul fronte terapeutico sono importanti le novità che arrivano dalla ricerca scientifica, capaci di migliorare non solo la gestione dei sintomi ma anche la qualità di vita del paziente. Se ne è discusso al media tutorial "Emoglobinuria Parossistica Notturna: verso un controllo migliore della malattia" organizzato da Novartis Italia a Milano.
Category
🗞
NewsTranscript
00:00It affects men and women in equal measure, with symptoms of gravity widely variable.
00:09Although the pathology can develop at any age, it is often diagnosed in people
00:13of age between 30 and 40 years old.
00:15It is hemoglobinuria paroxystica nocturna, a rare, chronic and serious blood disease
00:20that occurs when a part of the immune system, called the complement system, attacks
00:26the red blood cells, devoid of some regulatory proteins.
00:28It is estimated that in the world about 10-20 people out of a million are affected.
00:33This has been discussed in the course of the Media Tutorial of Novartis Italy, entitled
00:37Hemoglobinuria paroxystica nocturna, towards a better control of the disease.
00:42The main symptoms are anemia.
00:45The patient feels tired, exhausted, and this is often the reason that leads him to the doctor.
00:54Other times, the symptoms are particular, such as thrombotic events, and therefore the
01:01arrival is more dramatic, let's say, to the emergency room, from the doctor, because
01:07it is the thrombotic event that has led the patient to go to the health care.
01:11Other times, even worse, there can be a picture of peripheral pancytopenia,
01:18that is, all the cell lines, red blood cells, white blood cells, platelets, are low,
01:25because, in fact, Hemoglobinuria paroxystica nocturna, in that case, is also associated
01:30with what we call middle aplasia.
01:34Thanks to scientific research, they are working on the therapeutic panorama, drugs
01:38capable of revolutionizing the standard of care.
01:41These drugs have reduced hemolysis, increased hemoglobin levels, therefore improved
01:46anemia, and, above all, reduced the risk of thrombosis in our patients, improving
01:52survival.
01:53They are now at the doorstep of new treatments that, in addition to making the patient survive
01:57more, can have an improving effect also on their quality of life,
02:03because they are able to further increase the values of hemoglobin, also solve residual anemia
02:09related to the incomplete functioning of these inhibitors of the C5, and therefore
02:16allow the almost normalization of hemoglobin levels, and therefore of the quality of life
02:22of our patients.
02:23Novartis has been working for more than 20 years in the field of hematological research.
02:28We know that today, with the available treatments, more than 80% of patients
02:37do not reach satisfactory fatigue levels, they continue to manifest a level of constant fatigue,
02:46and 30% of patients still constantly need blood transfusions.
02:55The therapeutic proposal of Novartis is aimed at trying to fill these two important
03:04needs, reducing on the one hand the fatigue and on the other the need for blood transfusions.