(Adnkronos) - "L'emoglobinuria parossistica notturna è una malattia rara, cronica, grave, che è caratterizzata da una triade sintomatologica, costituita da una severa anemia emolitica, una propensione dei pazienti ad eventi trombotici, a volte anche gravi, e a un difetto del midollo osseo. Negli ultimi 10-15 anni la storia naturale di questa malattia è cambiata grazie all'avvento di farmaci specifici per la malattia, che rendono quindi la gestione per noi medici e per i pazienti più agevole”. Lo ha detto Antonella Sau, dirigente medico dell’Uoc di Oncoematologia pediatrica della Asl di Pescara, a margine del 51° congresso nazionale Sie - Società italiana di ematologia, in svolgimento al MiCo di Milano dal 23 al 25 settembre e al quale è presente anche Sobi, azienda biofarmaceutica con focus su malattie ematologiche rare o poco conosciute, e oncoematologia.
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00:00What is Hemoglobinuria Paroxysmica Nocturna?
00:04Hemoglobinuria Paroxysmica Nocturna is a rare, chronic, serious disease
00:10characterized by a so-called symptomatic triad
00:15constituted by a severe hemolytic anemia,
00:19a propensity of patients to thrombotic events, sometimes even serious,
00:24and a defect of the bone marrow,
00:28in terms of the possibility of developing middle aplasias or melodysplastic syndromes.
00:35The main symptoms are those characterized by anemia
00:40and all the symptoms related to anemia, fatigue, pallor,
00:44difficulty in performing normal life activities.
00:48Eventually, the outbreak can also be caused by a thrombotic event
00:54in various districts of the body, even in so-called rare districts,
01:00such as at the cerebral or abdominal level,
01:04and also other coronary symptoms, such as abdominal pain,
01:08disorders with deglutition,
01:11all symptoms that make the pathology serious
01:17and with an important compromise of the patient's quality of life.
01:22In the last 10-15 years, the natural history of this disease has changed a bit
01:29thanks to the advent of specific drugs for the disease
01:34that make the management easier for doctors and patients.
01:41However, we should not forget, neither doctors nor the entire scientific community,
01:47that this is a chronic disease that does not tend to heal
01:51and with which the patient is forced to live for the rest of his life.
01:57Therefore, quality of life is certainly the main objective of the next scientific progress.
02:09If a patient does not get the expected response to the so-called first-line treatment,
02:17fortunately today we have the opportunity to offer the patient alternatives
02:23in order to obtain a very high percentage of response and control of the disease.
02:29In particular, today we can offer the patient Pegcetacoplan,
02:35a so-called second-line drug,
02:39which can guarantee the patient an optimal response and control of the disease
02:46in the absence of side effects.