• Tue. Dec 7th, 2021

Beta-thalassemia and myelodysplasia drugs, transfusion-cutting therapy in Italy


Nov 24, 2021

The Italian Medicines Agency Aifa has approved the reimbursement of luspatercept in adult patients with blood transfusion-dependent anemia, due to beta-thalassemia or myelodysplasia. The therapy promises to “substantially” reduce transfusion requirements and represents “a substantial change in clinical practice”. This was explained by experts who spoke today at a virtual press meeting promoted by Celgene, now part of Bristol Myers Squibb (Bms). Luspatercept, they recalled, is “the first and only erythroid maturation agent approved in Europe”, the progenitor of a new therapeutic family. “In Italy, one of the countries in the world most affected by beta-thalassemia, there are about 7 thousand patients with this hereditary genetic disease, caused by a defect in the production of hemoglobin, the protein responsible for transporting oxygen in the body – explains Gianluca Forni, director of hematology, Center for microcythemia and congenital anemia, Galliera hospitals in Genoa – If both genes of the beta chains that form hemoglobin are mutated, there is beta-thalassemia major, which presents a severe clinical picture, with severe anemia. this form appears already in the first months of life and, if not treated with adequate therapies, the consequences can be severe anemia, bone deformations, enlargement of the spleen and liver, growth problems, hepatic, endocrine and cardiovascular complications. 60 patients with beta-thalassemia major did not survive beyond 10-15 years. Today, thanks to the combination of transfusion and iron chelation therapies, their expectation of life has improved a lot. However, patients are forced to undergo blood transfusions every 2-3 weeks for life and to take iron chelation therapy every day, to avoid the damage caused by the accumulation of iron to vital organs such as the heart, liver and pancreas “. “The reimbursement of an innovative therapy such as luspatercept can really change their lives because it allows to decrease the transfusion requirement, reduce the accumulation of iron and the consequent comorbidities, with a hope of improvement on survival”, underlines the specialist, recalling that ” the international Believe study, published in the ‘New England Journal of Medicine’ and which involved 336 patients with transfusion-dependent thalassemia, showed that 70% of those treated with luspatercept reduce the need for transfusion by 33% “. “The results of the Medalist study on 153 patients with myelodysplastic syndromes are also significant”, blood cancers defined as syndromes for their o heterogeneity: “47% were free from transfusions for about 2 months (8 weeks)”. Hence the Aifa ok for patients with very low, low and intermediate risk myelodysplastic syndromes, presenting ring sideroblasts with unsatisfactory response or unsuitable for erythropoietin-based therapy. bone, which produces red, white blood cells and platelets – highlights Matteo Della Porta, head of the Leukemia and Myelodysplasia Unit, Humanitas Cancer Center, Milan – As a consequence, blood values ​​are lowered, in particular those of red blood cells, causing anemia, present in almost It also increases the risk of the disease developing into acute myeloid leukemia, a more aggressive blood cancer Anemia is responsible for many symptoms, including paleness of the skin and mucous membranes, fatigue, breathlessness and rapid heart rate. Red blood cell insufficiency in nearly all patients becomes severe over time and needs support with regular transfus blood ions, which can also become very frequent. Treating severe transfusion-dependent anemia effectively is the key to improving patients’ quality and life expectancy. Luspatercept has been shown in clinical studies to substantially reduce the transfusion requirement in patients with a particular subtype of myelodysplastic syndrome, characterized by the presence of ring sideroblasts. This is a truly decisive step forward in therapy “.” With Celgene, for over 30 years we have been focused on haematological diseases – declares Cosimo Paga, Executive Country Medical Director, Bms – Luspatercept has an innovative mechanism of action, it consists of a subcutaneous injection every 21 days and has been shown to be effective in two different haematological diseases. We are committed to research on serious pathologies and we make our attention to patients concrete also through the activation of compassionate therapeutic use programs, which, thanks to the free supply of drugs carried out during the price and reimbursement negotiation period with Aifa, allow patients to access therapies before such drugs are marketed on the national territory. Thanks to these programs, to date, over 350 patients have been treated with luspatercept in Italy “.” The long wait for patients is finally over – commented Raffaele Vindigni, president of United Onlus, National Federation of Thalassemia, Sickle Cell Disease and Rare Anemia Associations – A important achievement capable of decreasing the need for blood and making them more free from transfusions. However, it is essential not to stop in front of the bureaucratic difficulties linked to the approval processes at the regional level, which create strong territorial discrepancies. The still open question of the network of centers must also be resolved as soon as possible. In 2017, the Italian network of thalassemia and hemoglobinopathies was established through a specific law. However, this has not yet been followed by the implementing decree to secure the network and allow the diagnosis and treatment carried out in recent years to continue “. Myelodysplastic syndromes” were among the first blood diseases in which was a national mobilization for the creation of specific pathology networks, to guarantee patients uniform access to an appropriate diagnosis and adequate treatment – observes Della Porta – Pathology networks provide an immediate connection with the most important reference center. close to the patient’s home, essential to ensure territorial continuity. We are trying, through collaboration with patient associations, to spread the knowledge of the disease and of the diagnostic and treatment aids included in the pathology networks at the widest possible level. Patients with myelodysplastic syndromes have very specific clinical and care needs, therefore they must be treated in specialized centers “.” In Italy, about 3 thousand new cases are estimated every year, especially in the elderly over 70 “, but” in reality it is a figure on the downside – specifies the expert – because many people affected by the disease do not receive a correct and timely diagnostic classification. Prognosis is also difficult to determine, as it varies widely. For myelodysplastic syndromes, a specific pathology code has been identified, that of ‘rare neoplastic disease’, which is very useful because it allows us to determine the exact epidemiological impact at the national level. Furthermore, it is an important recognition in terms of defining specific health strategies. “” It is unthinkable that the elderly patient who has to undergo treatment for severe anemia has to travel and be forced to travel hundreds of kilometers from their home to access a quality service – affirms Paolo Pasini, president of Aipasim (Italian association of patients with myelodysplastic syndrome) Onlus – The purpose of the networks is also to distribute skills throughout the territory at the service of patients. The care and psychological burden of a person suffering from myelodysplastic syndromes is not less than that of a patient with acute myeloid leukemia. Yet, in the past, they were undervalued, as if they were ‘taken for granted’ with old age. The reality is that they are crippling diseases. Autonomy and functionality are very limited, especially when the anemia is severe. Having the need for frequent blood transfusions, patients often have to go to hospital and their life, in fact, revolves around the specialist center. This condition ends up weighing on the family and requires, on the part of the health system, the activation of continuous assistance services. The innovation brought by luspatercept and research can truly change patients’ lives. “

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