EHA 2024: Ivosidenib for High-Risk MDS Patients Updated Results

Myelodysplastic syndromes (MDS) are a group of diverse bone marrow disorders that affect the ability to produce healthy blood cells. Recently, a new drug called ivosidenib (IVO) has shown promise in treating patients with specific genetic mutations, such as the IDH1 mutation. 

At the European Hematology Association 2024 conference, investigators reported updates on the study that tested the efficacy of ivosidenib in MDS patients with high-risk and low-risk who didn’t respond to erythropoietin, a red blood cell-stimulating agent. 

What Are the 2024 Updates for Ivosidenib?

Ivosidenib (IVO) is an oral medication that specifically targets the mutation in the IDH1 gene (IDH1m). It has been approved for use in AML and MDS patients with the IDH1 mutation.

The IDIOME trial enrolled 48 patients with different risk levels of MDS, and a great percentage of them responded well to therapy:

• High-risk MDS who didn’t respond to previous azacitidine treatment: 63.6% achieved a response.
• High-risk MDS who received IVO as a first-line treatment: 78.3% achieved a response
• Low-risk MDS who did not respond to previous erythropoietin treatment: 60% of patients achieved complete remission 

Ivosidenib was generally well tolerated. The most common side effect was differentiation syndrome (various symptoms caused by a large, rapid release of immune substances from cancer cells that are affected by the medication), which was reversible in all cases. A total of nine patients reported side effects.

The study concluded that ivosidenib is effective for patients with MDS. For high-risk MDS patients, especially those who haven’t received previous treatment, Ivosidenib showed high response rates and prolonged overall survival. Investigators reported that 78% of patients remained transfusion independent 56 days after therapy, making it a potential first-line treatment option. 

Although this study has a small patient sample, it shows that ivosidenib might be a favorable treatment option. Discuss with your healthcare provider whether this therapy could be suitable for you, especially if you are looking for alternatives after other treatments have not worked.

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Sources: 

• IVOSIDENIB MONOTHERAPY IN IDH1 MUTATED MYELODYSPLASTIC SYNDROME, FINAL RESULTS OF THE IDIOME TRIAL, A GFM STUDY
• Ivosidenib Monotherapy Is Effective in Patients with IDH1 Mutated Myelodysplastic Syndrome (MDS): The Idiome Phase 2 Study By the GFM Group