70% of patients with myelodysplastic syndrome develop severe anemia and need recurrent blood transfusions. An international clinical trial co-coordinated by Humanitas Research Hospital has proven the effectiveness of a new therapy that could dramatically improve the quality and life expectancy of people with this disease
The results of the international COMMANDS study – a phase III clinical trial co-coordinated by Humanitas Research Hospital with, among others, the MD Anderson Cancer Center in the United States – have been published in The Lancet. The study tested the effectiveness of a new therapy to treat transfusion-dependent anemia in patients with myelodysplastic syndromes, rare blood cancers affecting between 60,000 and 170,000 only in the USA.
“Traditional therapy to treat anemia in patients with myelodysplastic syndromes (erythropoietin) is unfortunately not effective in about 30% of patients and has a limited efficacy over time in another 30%,” explains Prof. Matteo Della Porta, head of the Leukemia and Myelodysplasia Unit at Humanitas and Full Professor of Hematology in Humanitas University. “After 40 years of research, the study has demonstrated the efficacy of a new drug, luspatercept, both in terms of higher response rate and duration. This success reminds us of the pivotal role of investing in research to bring new and effective solutions to patients’ bedside.”
Preliminary data were presented in Chicago, during the annual American Society of Medical Oncology (ASCO) congress, and at the European Hematology Association (EHA) in Frankfurt.
The Phase III COMMANDS study
Myelodysplastic syndromes differ widely from patient to patient, ranging from indolent, slowly progressing conditions, to ones rapidly progressing towards acute myeloid leukemia. The pathology is caused by damaged bone marrow stem cells, that become ineffective in replenishing the blood tissue. This causes a deficiency of red blood cells, white blood cells and platelets and, consequently, anemia, recurrent infections, and increased risk of bleeding.
The phase 3 clinical recruited 354 people with low-risk myelodysplastic syndrome and transfusion-dependent anemia, a condition that affects about 70% of patients with these syndromes. Results show that 58.5% of participants treated with the new therapy (luspatercept) became independent of blood transfusions, compared with 31.2% of those treated with the standard drug (erythropoietin). In addition, the duration of response was significantly longer for those who received the new therapy.
“Since the vast majority of patients with myelodysplastic syndrome need to have regular blood transfusions, the results are very good news: many people will benefit from an improved quality of life for a prolonged period,” Prof. Della Porta concludes. “Results like these make us proud, because they fulfill one of central goal of the Humanitas Cancer Center: ensuring that patients have access to the most innovative therapies while participating in medical progress.”