Adverse Events of Menin Inhibitors in AML: What Nurses Should Know

Differentiation syndrome is the principal, class-defining toxicity of menin inhibitors—and early recognition followed by prompt corticosteroid therapy typically prevents serious organ complications, Ghayas C. Issa, MD, MS, said.

Menin inhibitors are emerging as an important therapeutic class for patients with acute myeloid leukemia (AML) harboring NPM1 mutations or KMT2A rearrangements (KMT2Ar). With new therapies come new considerations for safety, and understanding the adverse events (AEs) associated with these agents is critical for oncology nurses and advanced practice providers.

In an interview with Oncology Nursing News at the 2025 SOHO Annual Meeting, Issa, a medical oncologist at The University of Texas MD Anderson Cancer Center in Houston, Texas, outlined the key safety signals seen with menin inhibitors. According to Issa, differentiation syndrome is a class-defining toxicity and must be recognized early. The syndrome occurs when leukemia cells release proteins that can trigger inflammation in organs such as the lungs, kidneys, or heart. If left unchecked, it can lead to life-threatening complications. However, Issa emphasized that prompt initiation of corticosteroids typically prevents serious outcomes.

Other AEs linked to menin inhibitors include cytopenias and mild gastrointestinal symptoms, such as nausea. Still, Issa noted that compared with many other AML therapies, these agents generally demonstrate a more favorable tolerability profile.

For nurses and APPs, early recognition and intervention remain central to optimizing outcomes for patients on menin inhibitors.

Transcript

The class adverse effect that is seen with any of the menin inhibitors that are in clinic now is differentiation syndrome, which is a reaction that’s caused by these inhibitors attacking the leukemia, and in the process, some of those leukemia cells make proteins that may irritate the lungs or the kidneys or the heart. In dangerous scenarios, this could shut down the lungs and cause death.

It’s very important to recognize differentiation syndrome, because physicians would start steroids, and in almost all the cases, by starting steroids early, we can prevent any complications from this syndrome. The other adverse effects that could happen with menin inhibitors could be affecting blood counts to a certain degree, or having some mild adverse effects, such as nausea or some effects on the stomach, but if you compare them to other treatments we use for acute myeloid leukemia, these medicines have very few adverse effects in general.

This transcript has been edited for clarity and conciseness.