Home Health Beta-thalassemia and myelodysplastic syndromes: approved innovative therapy that reduces blood transfusions

Beta-thalassemia and myelodysplastic syndromes: approved innovative therapy that reduces blood transfusions

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A longer and simpler life, without having to resort to continuous blood transfusions and avoiding the possible complications of iron accumulation. Today it is possible thanks to a new innovative therapy that has received the approval of the Italian Medicines Agency (Aifa). It is luspatercept for adult patients with blood transfusion-dependent anemia due to beta-thalassemia or myelodysplastic syndromes. This is good news concerning in Italy, one of the most affected countries in the world, about 7,000 patients with beta-thalassemia and about 3,000 new cases of myelodysplastic syndromes, especially in the elderly over 70, which are estimated every year. The data was presented today at a press conference sponsored by Celgene now part of Bristol Myers Squibb.

Symptoms of beta-thalassemia

Beta-thalassemia is a genetic, inherited disease caused by a defect in the production of hemoglobin, the protein responsible for transporting oxygen throughout the body. “If both genes of the beta chains that form hemoglobin are mutated, we have beta-thalassemia major, which presents a severe clinical picture, with severe anemia,” he explains. Gianluca Forni, Director of Hematology Center for Microcythemia and Congenital Anemia, Galliera Hospital of Genoa. “The symptoms of this form of the disease appear 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 complications, endocrine and cardiovascular “.

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The advantages of the new therapy

Until the 1960s, patients with beta-thalassemia major did not survive beyond 10-15 years. “Today – continues Forni – thanks to the combination of transfusion and iron chelation therapy, their life expectancy has greatly improved. 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 “. That’s why the reimbursement of an innovative therapy like luspatercept can really change their life. “This therapy – explains Forni – allows to decrease the transfusion requirement, reduce the accumulation of iron and the consequent comorbidities, with a hope of improvement in survival. The data deriving from ongoing trials may provide further evidence in this regard, but the international Believe study, published in the ‘New England Journal of Medicine’ and which involved 336 patients with transfusion-dependent thalassemia, has already shown that 70% of patients treated with luspatercept reduces transfusion requirements by 33% “.

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Myelodysplastic syndromes

The results of the Medalist study on 153 patients with myelodysplastic syndromes were also significant: 47% were free from transfusions for about 2 months (8 weeks). The approval of Aifa concerns people with very low, low and intermediate risk myelodysplastic syndromes, who have ring sideroblasts with unsatisfactory response or unsuitable for erythropoietin-based therapy. “Myelodysplasias are blood cancers, defined syndromes for their heterogeneity – he explains Matteo Della Porta, Head of the Leukemia and Myelodysplasia Unit, Humanitas Cancer Center, Milan. They are caused by a ‘defect’ in the bone marrow stem cell, which produces red, white blood cells and platelets. As a consequence of this pathological condition, blood values ​​are lowered, in particular those of red blood cells, causing anemia, and the risk of the disease evolving into acute myeloid leukemia, a more aggressive blood cancer, increases ”.

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The efficacy on myelodysplastic syndromes

The insufficiency of red blood cells, in almost all patients, becomes severe over time and needs support with regular blood transfusions, which can also become very frequent. “Treating severe transfusion-dependent anemia effectively – continues Della Porta – is the key to improving the quality and life expectancy of patients with myelodysplastic syndrome. 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 ”.

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A new therapeutic class

Luspatercept is able to substantially reduce the transfusion requirement: it counteracts the ineffective erythropoiesis of the marrow, or the lack or insufficient production of mature red blood cells, the cause of the severe anemia that can characterize these two haematological diseases. Although the initial damage is of a different nature in the two pathologies, in both of them there is, as a final consequence, an inadequate production of hemoglobin and, therefore, a picture of anemia. Luspatercept is the first and only erythroid maturation agent approved in Europe and therefore represents a new therapeutic class, also available for patients in Italy. “Luspatercept – he explains Cosimo Paga, Executive Country Medical Director, Bristol Myers Squibb – has an innovative mechanism of action, consisting of a subcutaneous injection every 21 days. 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 these drugs are marketed on the national territory. Thanks to these programs, to date, over 350 patients have been treated with luspatercept in Italy “.

Overcoming bureaucratic difficulties

Patients also express satisfaction: “Our long wait is finally over – he says Raffaele Vindigni, president of United Onlus (National Federation of Associations, Thalassemia, Sickle Cell Disease and Rare Anemias). 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. This, however, has not yet been followed by the implementing decree to secure the network and thus allow the diagnosis and treatment carried out in recent years to continue ”.

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The role of the network of centers

Networks are also essential in the treatment of myelodysplastic syndromes. “They were among the first blood diseases in which there was a national mobilization for the creation of specific pathology networks, to guarantee patients uniform access to an appropriate diagnosis and adequate treatment – underlines Della Porta. Pathology networks provide an immediate connection with the reference center closest to the patient’s home, which is 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 since patients with myelodysplastic syndromes have very specific clinical and care needs, therefore they must be treated in specialized centers “.

Bringing assistance to the territory

“It is unthinkable that the elderly patient who has to undergo treatment for severe anemia should move and be forced to travel hundreds of kilometers from their home to access a quality service – he concludes 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. 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 really change the lives of patients ”.


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