Real-World Outcomes of Hypomethylating Agents for Higher-Risk Myelodysplastic Syndromes: Report From the Nationwide...

Hypomethylating agents (HMAs) are the standard of care for higher-risk myelodysplastic syndromes (MDSs); however, their accessibility remains limited. This retrospective multicenter cohort study aimed to evaluate the real-world overall survival (OS) of HMAs compared to best-available treatments (BATs) in newly diagnosed higher-risk MDS in Thailand. Higher-risk MDS was defined by a Revised International Prognostic Scoring System (IPSS-R) score > 3.5. Data were extracted from the MDS registry of multicenter hospitals in Thailand from 2013 to 2023. Patients who underwent hematopoietic stem cell transplantation were excluded. A total of 155 patients were included, with a median age of 70 years and a male predominance. The median follow-up time was 36 months. Of these, 86 patients (55.5%) received BATs, while 69 patients (44.5%) were treated with HMAs. HMAs were found to significantly improve OS compared to BATs (log-rank test: p = 0.03), with a median OS of 15 months versus 6 months, respectively. Multivariable Cox regression analysis revealed that HMAs were associated with a decreased risk of death (hazard ratio: 0.58, 95% CI, 0.38-0.89, p = 0.01), while age and high- and very high-risk IPSS-R were identified as poor prognostic factors. This study underscores the real-world effectiveness of HMAs for higher-risk MDS in Thailand.