(Adnkronos) - “La mielofibrosi, che può essere primaria o rappresentare l’evoluzione di una precedente neoplasia mieloproliferativa cronica, è considerata un tumore raro del quale è difficile rilevare la reale incidenza. Si stima ci siano circa 500-600 nuove diagnosi ogni anno in Italia. È una malattia importante e la sopravvivenza mediana globale è di 6-7 anni, anche se l’introduzione dei Jak inibitori ha portato un miglioramento nella sopravvivenza globale della malattia”. Lo spiega Alessandro Vannucchi, professore di Ematologia dell’università di Firenze e direttore della SOD Ematologia dell'Aou Careggi, illustrando le caratteristiche della mielofibrosi in occasione della conferenza stampa organizzata a Milano da Gsk per annunciare il via libera di Aifa alla rimborsabilità di momelotinib, inibitore selettivo delle proteine JAK1 e JAK2, per il trattamento della mielofibrosi, un tumore del sangue particolarmente aggressivo che colpisce il midollo osseo, impedendogli di formare correttamente globuli rossi, globuli bianchi e piastrine.
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00:00What is myelofibrosis?
00:04Myelofibrosis, which can be primary or represent the evolution of a previous chronic myeloproliferative neoplasia,
00:11which is the name with which a family of stem cell tumors is identified,
00:16is considered a rare tumor.
00:18It is difficult to establish the real incidence.
00:21We consider that in Italy there are probably between 500 and 600 new cases that are diagnosed every year.
00:29It is an important disease because the global average survival rate of patients with myelofibrosis
00:36is estimated to still be around 6-7 years,
00:39although the introduction and use of inhibitors has led to an improvement in the global survival rate of the disease.
00:47The fact remains that the only therapeutic approach to be able to cure the disease of myelofibrosis
00:54is the transplant of stem cells, which is reserved for younger patients
00:59and for patients who have particularly unfavorable characteristics of the disease itself.
01:05What has changed a lot in recent years
01:10has been the introduction of a family of new drugs, which are the DGEC inhibitors.
01:18In fact, myelofibrosis is characterized by three main genetic mutations
01:23that directly or indirectly concern the DGEC-STAT signaling pathway.
01:29This, in turn, is responsible for the main signs and symptoms of the disease,
01:35that is, for the altered growth of the blood cells,
01:38for the increased volume of the myeloma
01:41and for the important inflammatory condition that characterizes these patients,
01:46which is responsible for the systemic symptoms of the disease,
01:49ranging from weight loss to fever and night sweats.
01:53Therefore, having discovered the role of the DGEC-STAT pathway,
01:57it has allowed the development of inhibitor drugs of this same pathway,
02:01which have proven to be effective in reducing sphenomegaly and improving symptoms.
02:07However, these drugs also have, as part of their mechanism of action,
02:11inhibited the ability of the myeloma to produce red blood cells and platelets.
02:16In this sense, the third DGEC inhibitor that we have available in Italy,
02:22which is Mumelotinib, represents an important step forward.
02:26This is a molecule that has a double inhibitor activity compared to DGEC-2
02:30and a receptor, the receptor of Activin,
02:33thus influencing another intracellular signaling pathway,
02:37which is the SMAD protein pathway,
02:39which, in the end, improves the ability of the myeloma to produce red blood cells.
02:44Therefore, Mumelotinib, maintaining its effectiveness
02:47compared to sphenomegaly and systemic symptomatology,
02:50is effective in anemic patients,
02:53improves anemia in patients who begin therapy with anemia,
03:00but above all, it also has less toxicity than other DGEC inhibitors,
03:05less toxicity in the production of red blood cells by the myeloma.