Myelodysplastic syndromes (MDS) are a diverse group of cancers that result in bone marrow failure and may develop into more advanced cancers such as acute myeloid leukemia. Yale researchers have recently published the results of multiple ongoing clinical trials evaluating the efficacy of treatment regimens for both low and high-risk MDS.
A phase 2 placebo-controlled trial evaluated the role of sabatolimab, a novel immunotherapy, and a hypomethylating agent in individuals with high-risk MDS. They found that this treatment combination did not improve the response rate or progression-free survival compared to placebo. However, these results will be followed up by an ongoing phase 3 trial, evaluating the efficacy of sabatolimab and hypomethylating agents in a broader patient population.
A phase 3 placebo-controlled clinical trial was also conducted evaluating the role of imetelstat, a telomerase inhibitor, in individuals with lower-risk MDS who are dependent on red blood cell transfusions. They found that the primary endpoint of independence from red blood cell transfusions was met in those treated with imetelstat, with significant improvement in hemoglobin levels compared with placebo.
To learn more, read the studies: Sabatolimab plus hypomethylating agents in previously untreated patients with higher-risk myelodysplastic syndromes (STIMULUS-MDS1): a randomised, double-blind, placebo-controlled, phase 2 trial and Imetelstat in patients with lower-risk myelodysplastic syndromes who have relapsed or are refractory to erythropoiesis-stimulating agents (IMerge): a multinational, randomised, double-blind, placebo-controlled, phase 3 trial.
Zeidan, A. M., Ando, K., Beyne Rauzy, O., Turğut, M., Wang, M.-C., Cairoli, R., Hou, H., Kwong, Y., Arnán, M., Meers, S., Pullarkat, V., Santini, V., Malek, K., Kiertsman, F., Niolat, J., Marques Ramos, P., Menssen, H. D., Fenaux, P., Miyazaki, Y., & Platzbecker, U. (2024). Sabatolimab plus hypomethylating agents in previously untreated patients with higher-risk myelodysplastic syndromes (STIMULUS-MDS1): a randomised, double-blind, placebo-controlled, phase 2 trial. The Lancet Haematology, 11(1), e38–e50. https://doi.org/10.1016/s2352-3026(23)00333-2
Platzbecker, U., Santini, V., Fenaux, P., Sekeres, M. A., Savona, M. R., Madanat, Y. F., Díez-Campelo, M., Valcárcel, D., Illmer, T., Jonášová, A., Bělohlávková, P., Sherman, L., Berry, T., Dougherty, S., Shah, S., Xia, Q., Sun, L., Wan, Y., Huang, F., Ikin, A., Navada, S., Feller, F., Komrokji, R., Zeidan, A. M. (2023). Imetelstat in patients with lower-risk myelodysplastic syndromes who have relapsed or are refractory to erythropoiesis-stimulating agents (IMerge): a multinational, randomised, double-blind, placebo-controlled, phase 3 trial. The Lancet. https://doi.org/10.1016/s0140-6736(23)01724-5