A new drug improves anemia and reduces the need for transfusions in patients with myelodysplastic syndromes – breaking latest news

For the first time in 40 years there is a new treatment, more effective than those available so far, to treat anemia in patients with myelodysplastic syndromes (also called myelodysplasias), rare blood cancers diagnosed each year at approx three thousand Italians, mostly over seventy. Almost everyone suffers from anemia to such a level as to force them to continue transfusions, with a very negative impact not only on the quality of life, but also on survival. The good news comes from the results of the COMMANDS study, presented in Chicago al annual meeting of the American Association of Medical Oncology (Asco) and expected in the plenary session, the main one, of the conference of the European Hematology Association (EHA) which has just begun in Frankfurt. To explain to Courier the importance of this novelty Matteo Della Porta, director of the Leukemia Unit at the Humanitas Clinical Institute in Milan, which will exhibit the final data of the trial in Germany on 10 June, when they will be published simultaneously in the prestigious scientific journal The Lancet.

Professor Della Porta, what is so relevant about this study?

Erythropoietin has been the treatment of choice for anemia for decades in myelodysplasia patients, but about 30% of patients are either ineligible for it or refractory, i.e. do not benefit from it, and on another 30% it has a very limited (less than one year). After 40 years of research, luspatercept is the first drug to demonstrate superiority over erythropoietin in both response rate (i.e., it works in more people) and duration of care across all subtypes of low-grade myelodysplastic syndrome. risk.

What exactly are myelodysplastic syndromes?

It is a heterogeneous group of rare blood pathologies that until a few years ago were considered incurable, while today they can begin to benefit from therapies that significantly improve the lives of patients. These are very different subtypes, ranging from “sluggish” conditions (that is, slowly progressing conditions that make no difference on the life expectancy of the person concerned) to cases that progress rapidly towards acute myeloid leukemia. It always causes damage to the bone marrow stem cells which fail to produce an adequate amount of functional blood cells and this leads to a shortage of white blood cells, red blood cells and/or platelets.

This is where the most relevant complications arise, especially in the initial stages of the disease: anemia, thrombocytopenia and recurrent infections. What are the therapies?

Up until a few years ago, the treatment of patients was based almost exclusively on supportive therapy which aimed to mitigate the symptoms, especially related to anemia (present in almost all patients), while today more and more effective therapies have arrived which improve in important way the life of the sick. The drugs to be administered are different and are prescribed on the basis of certain criteria that take into consideration the specific subtype of syndrome from which the person suffers, how it evolves, the reactions and the quality of life. We are also beginning to better understand who is most at risk of acute leukemia and therefore a candidate for a transplant.

The COMMANDS study, however, concerns patients at low risk of leukemia evolution. What are the most important results you have arrived at?

The Phase 3 trial recruited 354 people with low-risk MDS who were transfusion dependent because of anemia. The participants, aged 74 on average, were divided into two groups and treated for at least 24 weeks: 178 received an injection of luspatercept (the innovative medicine already approved in Italy) every three weeks, the other 176 patients were treated with standard therapy based on erythropoietin, always intravenously, once a week. The results show that luspatercept nearly doubled the number of people made independent of blood transfusions: 86 participants (58.5%) compared to 48 (31.2%) treated with erythropoietin. Furthermore, the duration of the response to the treatment was significantly longer for those who received the new drug which has a targeted action on the medulla, where it stimulates the differentiation of red blood cells. It does not present significant side effects and is therefore well tolerated: a very important fact because we are referring to a population of patients who are often elderly and frail.

What are the most immediate consequences for the sick?

About 70% of patients with low-risk myelodysplasia and about as many need transfusions, so this is good news for a great many people. Based on these results, luspatercept could become the new first-line therapy, the standard one. The advantages for the sick are numerous, starting from the fact that they will have to go to the hospital less frequently for treatment. And then everyday life improves and survival lengthens, given that severe anemia (i.e. the one that requires red blood cell transfusions) is the most important clinical symptom, which causes a significant reduction in functional capacity and quality of life of the patient and causes a reduction in life expectancy (especially by contributing to an increased risk of other cardiac diseases and mortality).