Real-world study of use patterns and clinical outcomes for patients with myelodysplastic syndrome initiating oral decitabine and cedazuridine or intravenous/subcutaneous hypomethylating agents

Decitabine and cedazuridine (DEC-C) is the only oral hypomethylating agent (HMA) approved for myelodysplastic syndromes (MDS) in the United States. The other HMAs approved for MDS, decitabine and azacitidine, are administered intravenously (IV) or subcutaneously (SC).

We used propensity score matching (PSM) to balance potential confounders. Kaplan–Meier survival analysis was utilized to compare real-world overall survival (rwOS), acute myeloid leukemia (AML)-free survival, and time to next treatment (rwTTNT) among patients treated with oral DEC-C and IV/SC HMAs.

A total of 2101 patients with MDS were included, with 405 treated with oral DEC-C and 1696 treated with IV/SC HMA. After PSM, there were 399 patients in each treatment group; demographic and clinical factors were well balanced. Patients treated with oral DEC-C had a similar median rwOS (22.7 months vs 19.5 months; p = 0.57) and AML-free survival (16.1 months vs 14.3 months; p = 0.10) compared with patients who received IV/SC HMA; however, there were nonsignificant trends in favor of oral DEC-C. The median rwTTNT was significantly longer for the oral DEC-C patients than for the IV/SC HMA patients (9.3 months vs 7.8 months, respectively; p = 0.02).

This real-world study is the largest to date to examine clinical outcomes among MDS patients who initiated oral DEC-C compared to IV/SC HMAs. While study results indicate comparable rwOS and AML-free survival among patients treated with oral DEC-C or with IV/SC HMAs, patients treated with oral DEC-C had a significantly longer rwTTNT. These findings support the use of oral DEC-C as an alternative to parenteral HMA therapy.