Ivosidenib combined with azacitidine for patients with mutant IDH1 acute myeloid leukemia

This study aimed to investigate the safety and effectiveness of the combination of ivosidenib (Tibsovo) and azacitidine (Vidaza) in patients with acute myeloid leukemia (AML) with isocitrate dehydrogenase 1 (IDH1) mutation (permanent change). 

This study concluded that this combination was well tolerated and provided good responses in these patients.  

Ivosidenib is an inhibitor of the mutant IDH1 enzyme which is found in some types of AML. Some preclinical studies have suggested that adding azacitidine to ivosidenib improves its effectiveness. Azacitidine is a chemotherapy used for AML treatment.  

It was unknown if the combination of ivosidenib and azacitidine was safe and effective in patients with mutant IDH1 AML. 

This study involved 23 patients with newly diagnosed mutant IDH1 AML ineligible for intensive induction chemotherapy. Patients were treated with ivosidenib once daily continuously and azacitidine on days 1 to 7 in 28-day cycles. The average treatment duration was 15.1 months.  

Treatment-related severe side effects occurred in more than 10% of patients included neutropenia (low level of white blood cells) in 22% and anemia (low level of red blood cells) in 13%. Other side effects included electrocardiogram abnormalities and low platelet levels.  

The overall response rate (ORR) was 78.3% and the complete remission rate was 60.9%. After an average follow-up of 16 months, the average duration of response had not been reached. The 12-month survival estimate was 82%. 

This study concluded that ivosidenib plus azacitidine was well tolerated and provided deep and durable responses were deep and durable for patients with AML and IDH1 mutation. 

This study included a small number of patients. Also, there was no control group. More studies are needed.