Rytelo: a novel option for transfusion-dependent anemia in myelodysplastic syndromes

Rytelo (Imetelstat), approved by the FDA in June 2024, offers a groundbreaking treatment for patients with transfusion-dependent anemia due to lower-risk myelodysplastic syndromes (MDS). Rytelo is a first-in-class telomerase inhibitor that targets telomerase, an enzyme that cancer cells use to maintain their telomeres and continue proliferating. By inhibiting telomerase, Rytelo induces apoptosis in malignant cells in the bone marrow, thus reducing the need for frequent blood transfusions. Administered intravenously every four weeks, clinical trials have shown Rytelo effectively lowers transfusion requirements and enhances patients' quality of life. However, common side effects such as neutropenia and thrombocytopenia require careful monitoring and dose adjustments to manage. Despite these challenges, Rytelo represents a significant advancement in treating transfusion-dependent anemia in MDS, providing a novel therapeutic option that addresses the underlying cause of the disease and improves patient outcomes.