ASCO 2021 | Establishing the clinical benchmark of azacitidine in patients with MDS

Jacqueline Garcia, MD, Dana-Farber Cancer Institute, Boston, MA, discusses the aims and findings of a recently published systematic review of outcomes for patients with higher-risk myelodysplastic syndromes (MDS) treated with azacitidine in clinical trials. The pivotal AZA-001 trial (NCT00071799), which provided the supporting evidence for the use of frontline azacitidine in high-risk MDS, achieved a median overall survival (mOS) of 24 months. However, the results achieved in subsequent trials have been highly variable and real-world survival data is consistently shorter than that seen in AZA-001. The lack of a clear clinical benchmark has made it challenging to determine the contribution of novel therapies added to azacitidine. To address this, the results from 237 studies were pooled which found a mOS of around 18 months. The study also explored potential surrogate endpoints but a robust parameter that correlated with OS was not found. This interview took place at the American Society of Clinical Oncology (ASCO) 2021 Virtual Meeting.