New Landscapes in the Management of Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia: Oral Decitabine

Latest Insights and Therapeutic Advances in Myelodysplastic Neoplasms

“…In such a setting, the approval of eltrombopag for thrombocytopenic patients, in line with the recently published results of the phase III-controlled trial [85], will provide meaningful and substantial progress in this difficult-to-treat setting. Again, ongoing research on venetoclax and other emerging agents, such as oral HMAs as the potential backbone for several treatment combinations [119,120], is shaping the landscape of MDS management, offering potential useful therapeutic alternatives for HR patients, which continue to represent an unmet clinical need. Indeed, the safety and activity of the first oral combination of decitabine plus cedazuridine and venetoclax in patients with HR-MDS has been reported [120] with an ORR of 95%.…”

“…Indeed, the safety and activity of the first oral combination of decitabine plus cedazuridine and venetoclax in patients with HR-MDS has been reported [120] with an ORR of 95%. Therefore, this entirely oral treatment combination, other than providing highly effective therapeutic activity, might be paradigm-changing for HR-MDS patients, avoiding the inconvenient parenteral administration route, reducing travel times, and providing other obvious subjective benefits for family and caregivers [119,120]. In addition, attempts to modulate the immune system and inflammatory pathways, inducing defects in HSC progenitor cells, may lead to the development of immunotherapeutic interventions, such as CAR-T, vaccines, and immune checkpoint inhibitors, mitigating immunological dysregulation and altered inflammation in MDS [8,121].…”