2023-12-18 08:36:56
by Vera Martinella
After decades of stalemate, something is moving: many studies on new drugs indicate improvements in the quality and life expectancy of patients with myelodysplastic syndrome
Limiting the risk of the disease evolving into acute leukemia and reducing the continuous need for transfusions caused by the severe anemia they suffer from: these are the biggest problems to face for those suffering from myelodysplastic syndrome and on both fronts there are important news. After decades of stalemate, scientific research has finally managed to make significant progress in this area and this is demonstrated by the results of various studies presented during the annual congress of the American Society of Hematology (Ash), just concluded in San Diego (California) – he comments Paolo Corradini, president of the Italian Society of Hematology (Sie) —. The term myelodysplastic syndrome, also called myelodysplasia, brings together a heterogeneous group of rare blood pathologies, very different from patient to patient, ranging from “indolent” conditions, i.e. slow progression that make no difference to the life expectancy of the person directly involved , to cases that rapidly progress to acute myeloid leukemia. The most relevant complications, especially in the initial stages of the disease, are anemia, thrombocytopenia and recurrent infections. Every year in Italy there are around 3 thousand new diagnoses, especially among people over 70, and the number of sick people is destined to grow together with the aging population.
The COMMANDS study: improving anemia
For all myelodysplasias, it always causes damage to the bone marrow stem cells which are unable to produce an adequate quantity of functional blood cells and this leads to a shortage of white blood cells, red blood cells and/or platelets. This is why almost all patients suffer from anemia at such a level that they are forced to require frequent transfusions, with a very negative impact not only on their quality of life, but also on their survival. The first step forward was in 2020 with the arrival of a new drug, luspatercept, already approved and also available in Italy, thanks to which we managed to reduce patients’ transfusion requirements, explains Matteo Della Porta, director of the Leukemia Unit at the Institute. Humanitas Clinic in Milan. The final data of the phase 3 COMMANDS study which recruited 354 people with myelodysplastic syndrome at low risk of evolving into leukemia, but with severe anemia for which they were dependent on transfusions: The results demonstrate the superiority of luspatercept compared to the current standard treatment, i.e. erythropoietin – he says Della Porta, main author of the trial -: the new treatment works in a greater number of people, over 60% compared to just over 30%, and with a longer duration (52 weeks versus 37). Finally, it is important to note that luspatercept does not present significant side effects and is therefore well tolerated by patients who are often elderly and frail.
The IMERGE study: independence from transfusions
Independence from transfusions was the goal he aimed for studio IMERGE the results of which were illustrated in San Diego and published in the prestigious scientific journal The Lancet: The conclusions of the trial on 178 participants indicate the effectiveness of a new drug (the first in its “category”, a telomaresis inhibitor) in the treatment of severe anemia of myelodysplasia and the treatment managed to make one free from transfusions for approximately one a significant portion of patients year – explains the specialist -. After 40 years in which we had nothing but erythropoietin (but 30% of patients are not eligible to receive it or are refractory, i.e. they do not benefit from it, and on another 30% it has a very limited effect over time) the panorama is expanding greatly of innovative treatments for anemia in myelodyslpastic syndromes. a void that is slowly filling, as several other studies with different new medicines have indicated. Precisely to explain the importance of participating in clinical trials and helping them seize the opportunity, theItalian Association of Patients with Myelodysplastic Syndrome (AIPaSiM) has just drawn up a free guide dedicated to patients and families with useful, concrete and clear information.
The VERONA study: reducing the risk of leukemia
Another relevant problem, when faced with myelodysplasia, is understanding whether the patient has a form at high risk of rapidly evolving into acute leukemia in order to be able to calibrate the therapies accordingly, which are established on the basis of various criteria. Hematopoietic stem cell transplantation is the only definitive cure, capable of leading to recovery, but often too heavy to tolerate for patients, who are mostly elderly and frail. We have treatments that manage to slow down the transformation of a myelodysplastic syndrome into acute leukemia, but they do not work for all patients or have a limited duration and when the leukemic phase reaches the average survival of about two years – concludes Della Porta -. Numerous studies therefore seek more effective solutions specifically for those with high-risk myelodysplasia and, among the many illustrated at the Ash 2023 congress, those of the trial VERONA which must be viewed with caution because they are still in the early stages of experimentation (Ib-II), but which are significant. The participants received as a first line of treatment a new combination based on two drugs already in use: venetoclax (which is revolutionizing the treatment of acute myeloid leukemia) and azacitadine (a chemotherapy). The treatment was well tolerated and a significant number of patients derived clinical benefit from it.
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December 18, 2023 (changed December 18, 2023 | 07:36)
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