After three years of luspatercept treatment, about half of patients reduce their need for transfusions by 50% and 12% gain independence for more than two months. These two of the new results of the Believe study, presented in Vienna in the prestigious showcase of Eha 2022, the congress of the European Hematology Association. The new drug, reimbursed by AIFA in 2021, makes it possible to reduce the need for blood by more than 30%, with a great improvement in the quality of life, underlines a note.
Beta-thalassemia is a genetic disease transmitted by two asymptomatic parents or healthy carriers, which affects about 7 thousand people in Italy, 5 thousand in the most serious form, the ‘major’. It requires continuous transfusions, every 2-3 weeks and for life, with the risk of incurring iron accumulations that can damage the heart, liver and pancreas. For this reason, iron chelating drugs must be taken, which in turn can cause side effects. The international Believe study, already published in the ‘New England Journal of Medicine’, showed that, on more than 300 patients with transfusion dependent thalassemia, 70% thanks to luspatercept achieved an initial reduction of 33% in transfusion requirements. The data is presented today at a virtual press conference, sponsored by Celgene now part of Bristol Myers Squibb.
“We know more than 350 different mutations of the beta-globin gene that cause the disease, but from a clinical point of view, thalassemias are classified into transfusion-dependent forms (thalassemia major) and non-transfusion-dependent forms (intermediate forms) – he says Maria Domenica Cappelliniformer director of the internal medicine operating unit, head of the Rare Diseases Center, coordinator of the rare diseases unit at the Irccs Cà Grande Foundation, Maggiore Policlinico hospital in Milan -. The major affects about 80% of patients, is the most severe and requires continuous transfusion therapy, while the intermediate forms require periodic checks and occasional transfusions at particular times.such as pregnancy, surgery or infections “.
“In the most severe form – continues – land manifestations of the disease occur as early as infants, with very low hemoglobin levels, enlarged liver and spleen, and slowed growth. The intermediates can occur later in time and with less severe symptoms. The main cause of severe anemia and clinical manifestations is ineffective erythropoiesis, a consequence of the lack of production of the globin chains that form hemoglobin. The new drug reduces it: consequently the anemia also decreases. It has a tolerable safety profile and represents a prospect of enormous interest as an alternative to traditional therapy of transfusions and iron chelation “.
“After 3 years of therapy – explains Cappellini – i50% of patients achieved a 50% reduction in transfusions, in each 12-week interval, compared with 40% of patients who achieved this endpoint after 1 year. 12% of patients achieved transfusion independence for more than 8 weeks after 3 years, compared with 10.7% after 1 year. Obviously, the reduction in hospital access has an important impact on the quality of life “.
“Luspatercept had also given excellent results during phase two of the clinical trial – he declares Silverio Perrotta, head of the Up Hematology and Pediatric Oncology of the University of Campania ‘L. Vanvitelli ‘, head of the Center for the Diagnosis and Treatment of Hemoglobinopathies and of the Ern-EuroBloodNet Center – ma the data we have now allows us to ensure its long-term security. The Believe group of patients who received placebo joined those on treatment, for a total of 315 patients who received the drug. Among them, who have continued the treatment for three years, only 7% stopped it due to side effects, ranging from bone pain, manageable with mild pain relievers, to thromboembolic events. In this last case, however, it should be noted that the patients were already at risk, because they had undergone the removal of the spleen “.
Luspatercept, highlights the hematologist, “does not seem to have significantly increased this risk. Not only that, it also seems to have beneficial effects on malleolar ulcers, a common complication, which in some cases even heals with the drug, and on the bones, with an improvement in the osteoporosis. It also improves the problem of iron overload caused by transfusions by reducing the intake of iron chelators. It is therefore capable of significantly changing the life of people suffering from beta-thalassemia, with a significant reduction in periods of absence from work and a minor impact on family duties Today the indications are for adults, but there is already a trial underway for approval in the pediatric range, with the age of 18: a great proof of its safety. In many Italian regions there are still difficulties in prescribing transfusion-dependent adult patients, but it will soon be available throughout the P aese “.
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