Expert Perspectives on IDH Inhibitors in AML Treatment

EP: 1.Overview of Pathophysiology of and Progression of AML

EP: 2.Risk Classification Systems in AML

EP: 3.Key Patient Factors to Consider in AML Treatment Decisions

EP: 4.Provider Perspectives on Biggest Unmet Needs in AML Care

EP: 5.Social Determinants to AML Treatment Approach

EP: 6.Latest NCCN Guidelines on the Standard of Care for Patients With AML

EP: 7.Patient Eligibility for Transplant, Induction, and Consolidation in AML Treatment

EP: 8.Venetoclax and the Impact on Management of Older Patients With AML

EP: 9.Combination Therapies in AML Treatment Approach

EP: 10.Tracking the Shifting Treatment Landscape of AML

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EP: 11.Expert Perspectives on IDH Inhibitors in AML Treatment

EP: 12.Managing Toxicities in Patients With AML

Patients with specific IDH1 or IDH2 mutations have access to targeted inhibitors that represent some of the most effective treatments available in modern acute myeloid leukemia (AML) care, with the potential for exceptional survival benefits. While these mutations affect only 5% to 15% of AML patients, those who carry them can achieve remarkable outcomes with IDH inhibitor combinations. The AGILE trial demonstrated that patients with IDH1 mutations receiving ivosidenib plus azacitidine experienced median overall survival improvements of over 21 months compared to azacitidine alone, representing one of the most significant advances in AML treatment.

Treatment decisions involving IDH inhibitors require rapid genetic testing results, as patients and families are typically anxious to begin therapy immediately after diagnosis. The challenge lies in balancing the need for quick treatment initiation against waiting for mutation results that could identify patients who would benefit most from these highly effective but mutation-specific therapies. Some health care providers start patients on standard combinations and later modify treatment based on genetic results, while others advocate for waiting the few days needed for mutation analysis to optimize initial therapy selection.

Patients face important considerations regarding treatment sequencing, as some evidence suggests the order in which therapies are given may impact long-term outcomes. Those who receive venetoclax first may have reduced responses to subsequent IDH inhibitor therapy, while patients treated with IDH inhibitors initially may maintain sensitivity to venetoclax upon disease progression. For patients with IDH mutations who are not transplant candidates, the choice of initial therapy becomes particularly crucial, as it may influence the effectiveness of subsequent treatment options and overall survival duration.