Single-center phase 2 trial of first-line sintilimab plus decitabine for higher-risk myelodysplastic syndromes

The optimal first-line treatment for patients with higher-risk myelodysplastic syndromes (MDS) is not known. A phase 2 trial at Peking University People’s Hospital (Beijing, China) evaluated the first-line combination of sintilimab (anti-PD-1) and decitabine for these patients. A total of 54 patients were enrolled and treated, with 25 having very high-risk MDS. Among 53 evaluable patients, the overall response rate was 77.4% (n=41), including 26.4% (n=14) with complete response. The median event-free survival was 23 months, and the median overall survival was not reached. Treatment was generally well tolerated, with hematologic toxicities being the most common adverse events. Biomarker analysis highlighted a negative correlation between T cell exhaustion markers, particularly TIM-3 and PD-1, and overall response rate. The authors conclude that the first-line combination of sintilimab an decitabine showed promising efficacy for higher-risk MDS, with a favorable safety profile. The potential predictive value of T cell exhaustion biomarkers might help to identify the possibly benefiting population.