Post-FDA approval of imetelstat for MDS, several treatment implementation gaps remain

A study assessed real-world usage of imetelstat following its FDA approval for the treatment of lower-risk myelodysplastic syndromes (MDS) and observed that the treatment induced clinically meaningful improvements in hemoglobin levels; however, imetelstat was initiated during later lines of therapy and may be better suited for earlier use.

Jessy Thomas, PharmD, of Geron Corporation, and colleagues presented the findings at the 2026 SOHO Annual Meeting.

The authors noted that early real-world findings for imetelstat are limited, “particularly as community practices gained familiarity with patient selection and cytopenia management,” thus they sought to analyze trends in the field.

Using a multicenter chart abstraction approach, 30 patients with MDS who were treated with imetelstat at US community oncology practices between July 2024 and April 2025 were included in the assessment.

Patients were older, with 77% aged ≥75 years, and heavily pretreated. Most patients received imetelstat in later lines of care, with 83% receiving three or more therapies prior to imetelstat.

Median baseline hemoglobin was 7.7 g/dL. Among 24 evaluable patients, median hemoglobin increased by 2.25 g/dL; however, the authors found that median treatment duration was short, at 8.9 weeks, with the longest duration being 57.0 weeks. Median peak hemoglobin was 9 g/dL.

Nearly a third of patients (27%) had incomplete information available regarding baseline transfusion burden despite having documented transfusion dependence, which the authors said underscores a real-world documentation challenge.

Thrombocytopenia and neutropenia were common in imetelstat-treated patients and often led to treatment discontinuation. However, the authors noted that these early discontinuations suggest a limited real-world experience with cytopenia management, as alternative approaches could have included dose modifications or treatment interruption and reinitiation prior to discontinuation.

In summary, the authors highlighted the following areas as notable gaps related to imetelstat implementation:

• Suboptimal line of therapy selection
• Inconsistent transfusion documentation
• Limited familiarity with proactive cytopenia management

“Earlier use and optimized cytopenia management may improve real-world outcomes” associated with imetelstat, the authors concluded.

The study was funded by Geron Corporation.

Reference

Thomas J, Sura S, Whitesell M, Charles M, Sweet B, Zackon I. Real-world outcomes with early adoption of imetelstat in patients with transfusion-dependent myelodysplastic syndromes: insights from a United States community oncology chart review. Abstract MDS-775. Presented at the 2026 Society of Hematologic Oncology Annual Meeting; September 9-12, 2026; Houston.

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