Bms, myelodysplasia therapy frees 48% of patients from transfusions for 8 weeks

Almost half (48%) of patients with myelodysplastic syndromes presenting with ring sideroblasts treated with a new therapy (luspatercept) are free from blood transfusions for at least 8 weeks. A very important result for people forced to go to transfusion centers frequently, even every week. The advantages, especially for those with severe anemia, are reflected in the quality of life and the possibility of reducing organ damage related to the accumulation of iron introduced with transfusions. These issues – causes, symptoms and innovative treatments capable of transforming the lives of patients – on the occasion of the World Day dedicated to myelodysplastic syndromes on 25 October, were discussed today in Rome, during a press conference promoted by Bristol Myers Squibb (Bms). Myelodysplastic syndromes, a heterogeneous group of blood cancers – recalls a note – are still little known and their impact on the daily lives of patients, often elderly, over 70 years old, and with other associated pathologies is underestimated. The most frequent symptoms caused by hemoglobin deficiency are: fatigue, weight loss, breathing difficulties and a rapid heart rate. “Myelodysplastic syndromes are blood cancers that affect the stem cells of the bone marrow, which give rise to all peripheral blood cells, that is red, white blood cells and platelets. This leads to an alteration of the maturation process of the marrow and a qualitative and quantitative defect in the production of peripheral blood elements ”, explains Pellegrino Musto, UOC director of Hematology with transplantation of the consortium hospital-university Polyclinic of Bari and full professor of Hematology at the ‘Aldo Moro’ University of Bari.” The symptoms and the course “of these syndromes,” vary significantly according to the type of blood cell affected – continues Musto -. Low-risk forms may have anemia (reduction in red blood cells), neutropenia (reduction in white blood cells) or thrombocytopenia (reduction in platelets). The most frequent clinical manifestation is anemia and, therefore, the need for blood transfusions, but these patients are also susceptible to infections and bleeding. Myelodysplastic syndromes, in the most severe forms, can develop into acute myeloid leukemia, a more aggressive tumor. In most cases – adds the hematologist – the causes of the disease are unknown, that is, the mechanism that triggers the process of modification of the DNA of one of the stem cells of the bone marrow is not known. There are also secondary forms, linked to exposure to occupational risk factors, for example to chemicals such as benzene, lead or solvents. In addition, some patients may develop the disease following previous treatments with chemotherapy or radiotherapy, used to treat other neoplasms ”. On the need for a cultural change in the approach of these syndromes, the head of the Leukemia and Myelodysplasie Unit, Humanitas Cancer Center, Milan and Professor of Hematology at Humanitas University, Matteo Della Porta, states: “In Italy, about 3 thousand new cases are estimated every year . Many patients do not receive a correct and timely diagnosis. The mean age at diagnosis is 75 years. In more than 90% of cases, the first symptom is anemia. Unfortunately, being elderly people – he continues – anemia is often underestimated or considered a physiological condition, almost inevitable. Hence the delays in diagnosis. Anemia in the elderly – even when it is not severe – should always be investigated, precisely because it can hide haematological diseases such as myelodysplasias. Furthermore, responses to therapies and resulting clinical benefits are better if anemia is treated early. For this, a cultural change is also needed in the clinical medical community. Luspatercept – he observes – is the first example of personalized therapy in low-risk myelodysplasias with ring sideroblasts ”. “Luspatercept is the result of Bristol Myers Squibb’s research in hematology, an area in which we hold a leadership position – explains Cosimo Paga, Executive Country Medical Director, Bms – The drug has an innovative mechanism of action, it consists of a subcutaneous injection every 21 days and has been shown to be effective also in beta-thalassemia, a genetic disease that involves ineffective erythropoiesis. We are engaged in research on serious diseases and we make concrete our attention to patients also through the activation of compassionate use programs. that allow patients to access therapies before reimbursement “. “Aifa – underlines Della Porta – in December 2021, approved the reimbursement of luspatercept in adult patients with transfusion-dependent anemia due to myelodysplastic syndromes at very low, low and intermediate risk, presenting ring sideroblasts with an unsatisfactory or unsatisfactory response. suitable for erythropoietin-based therapy. The ‘Medalist’ pivotal study showed that 48% of patients treated with luspatercept – he adds – are free from blood transfusions for intervals of at least 8 weeks, with the possibility of multiple periods of response, during the entire duration of treatment. Not only. These results were also confirmed on over 200 ‘real life’ patients not selected and included in the compassionate program launched in our country. A very important result, which will be presented in December at the American hematology congress in New Orleans. “” Innovation is the right word in the protection of the patient with myelodysplasia. Ten years ago – underlines the president of Aipasim (Italian association of patients with myelodysplastic syndrome onlus), Paolo Pasini – the pathology was not recognized, today there is more awareness among patients, clinicians and health authorities, representing a new paradigm of personalized therapy. Innovation has taken on various forms, from basic and applied research, to genetics up to clinical trials. Aipasim – he continues – was born 5 years ago to fill a void of knowledge and intervention. With this in mind, recognizing the potential value of luspatercept for people with myelodysplasia, we are committed to achieving its full availability in the healthcare system. And we are happy with the result achieved last year with reimbursement. World Pathology Day is celebrated tomorrow – he concludes -. It is an opportunity to take stock of the results obtained in the treatment and those expected. We will analyze a hitherto unexplored area, namely the quality of life in illness “.

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