Oral decitabine and cedazuridine for the treatment of myelodysplastic syndromes: an integrated review of clinical, economic and patient-centered evidence

Abstract

Introduction: Myelodysplastic syndromes/neoplasms (MDS) are a heterogenous group of myeloid cancers that impose a substantial negative impact on patient health-related quality of life. As MDS predominately affects older individuals, who are especially susceptible to the debilitating nature of the disease and its burdensome symptoms, treatment decisions should consider therapeutic value from multiple perspectives to balance clinical efficacy with patient-centered outcomes. This comprehensive approach is known as relative medical value.

Areas covered: Although improved outcomes have been observed with hypomethylating agents (HMAs) in patients with higher-risk MDS, parenteral administration of HMA requires frequent infusion clinic visits and is associated with substantial time burden, especially in older patients, impacting treatment persistence. Suboptimal use of HMAs in MDS may lead to poorer outcomes and higher healthcare costs, underscoring the need for patient-centered treatment options that improve persistence.

Expert opinion: Oral decitabine and cedazuridine (DEC-C) is an approved treatment for higher-risk MDS and may reduce patient burden associated with parenteral HMA therapy. Oral DEC-C has the potential to improve treatment persistence and clinical outcomes and reduce healthcare resource utilization and costs. This review integrates the available clinical, patient-centered, and economic evidence on the relative medical value of oral DEC-C treatment for MDS.

Keywords: Myelodysplastic syndromes; hypomethylating agents; myelodysplastic neoplasms; oral decitabine and cedazuridine; relative medical value.