Chidamide therapy with Etoposide and Glucocorticoids for rare immune disorder

The integration of Chidamide with Etoposide and glucocorticoids could revolutionize Hemophagocytic lymphohistiocytosis treatment, offering enhanced efficacy and safety.

Hemophagocytic lymphohistiocytosis (HLH) is a severe, life-threatening condition marked by excessive inflammation and organ failure. Standard treatments often fall short and carry notable side effects. This study assessed the efficacy and safety of Chidamide, a selective histone deacetylase inhibitor, in combination with Etoposide and Glucocorticoids for treating adult HLH patients.

In this open-label, single-center trial, 17 adult patients meeting at least five of the eight HLH-2004 criteria received Chidamide along with standard Etoposide and Glucocorticoids. The overall response rate (ORR) was the main endpoint, whereas the survival rates, safety, and differences in key lab markers were a part of the secondary endpoints.

The ORR was 76.5%, with 17.6% achieving a complete response and 58.8% a partial response. Overall survival rates were 81% at 6 months and 65% at 12 months, while progression-free survival rates were 68% and 55%. Hematologic toxicities were common, but grade 3/4 toxicities were rare. Laboratory indicators such as sCD25, platelets, and liver enzymes significantly improved within 3 weeks of treatment.

Chidamide, when combined with Etoposide and Glucocorticoids, shows high efficacy and a tolerable safety profile in managing HLH. However, future research is needed to optimize treatment protocols and corroborate these findings.