Comparing enasidenib to current standard of care therapies for patients with relapsed/refractory acute myeloid leukemia and IDH2 mutations

This study aimed to compare enasidenib (Idhifa) to current standard of care therapies for patients with relapsed/refractory (r/r) acute myeloid leukemia (AML) and an isocitrate dehydrogenase 2 (IDH2) mutation who are ineligible for hematopoietic stem cell transplantation (HSCT). 

This study concluded that enasidenib significantly improved the survival of these patients. 

Up to 20% of AMLs are caused by an abnormality in the IDH2 gene. Enasidenib is a targeted therapy that blocks IDH2 and is approved for use for treating r/r AML in patients with IDH2 mutations. It was unknown if enasidenib is better than standard of care (SOC) therapies for patients with r/r AML with IDH2 mutations who are ineligible for hematopoietic stem cell transplantation (HSCT). Standard of care (SOC) therapy is a treatment that is accepted as the best option for a condition.  

This study analyzed data from 195 patients with r/r AML with IDH2 mutations who received enasidenib and 80 patients who received SOC.  

The average overall survival was 9.26 months with enasidenib and 4.76 months with SOC. Enasidenib was associated with a significantly better survival (by 33%) compared to SOC.   

This study concluded that enasidenib significantly improved the survival of patients with r/r AML and IDH2 mutations when compared to SOC.

This study did not directly compare enasidenib and SOC. The data was based on previous studies and medical records. Further randomized studies are needed.