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.
Comments