Effect of prior therapy on the clinical activity of imetelstat in patients with transfusion-dependent, ESA-relapsed or -refractory/-ineligible LR-MDS - Summary - MDSpire

Effect of prior therapy on the clinical activity of imetelstat in patients with transfusion-dependent, ESA-relapsed or -refractory/-ineligible LR-MDS

  • By

  • Rami S. Komrokji

  • Valeria Santini

  • Amer M. Zeidan

  • Mikkael A. Sekeres

  • Pierre Fenaux

  • Azra Raza

  • Moshe Mittelman

  • Sylvain Thépot

  • Rena Buckstein

  • Ulrich Germing

  • Yazan F. Madanat

  • María Díez-Campelo

  • David Valcárcel

  • Anna Jonášová

  • Souria Dougherty

  • Sheetal Shah

  • Qi Xia

  • Libo Sun

  • Shyamala Navada

  • Faye Feller

  • Michael R. Savona

  • Uwe Platzbecker

  • December 31, 2025

  • 0 min

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Objective:

To investigate the effects of prior therapies on the clinical activity of imetelstat in patients with transfusion-dependent, ESA-relapsed or -refractory/-ineligible lower-risk myelodysplastic syndromes (LR-MDS), highlighting the significance of these findings in improving treatment strategies.

Key Findings:
  • Median duration of ≥8-week RBC transfusion independence (TI) for the pooled population was 55 weeks (95% CI: 42–70).
  • 36% of ESA-ineligible patients achieved ≥8-week TI, with higher rates in treatment-naive patients (54%).
  • Imetelstat showed clinically meaningful activity after prior ESA, luspatercept, and lenalidomide, but limited activity after prior HMAs.
Interpretation:

Imetelstat demonstrates significant clinical activity in patients with LR-MDS, particularly in those who have not previously received ESA therapy, suggesting its potential as a valuable treatment option in this population and its implications for clinical practice.

Limitations:
  • The analysis is post hoc and may be subject to biases, particularly in patient selection.
  • Overall survival data were not mature at the time of analysis.
Conclusion:

Imetelstat shows promise in treating transfusion-dependent LR-MDS, especially in patients with limited prior therapy exposure, underscoring the need for optimal sequencing of therapies to enhance patient outcomes.

Original Source(s)

Effect of prior therapy on the clinical activity of imetelstat in patients with transfusion-dependent, ESA-relapsed or -refractory/-ineligible LR-MDS

  • by Rami S. Komrokji, Valeria Santini, Amer M. Zeidan, Mikkael A. Sekeres, Pierre Fenaux, Azra Raza, Moshe Mittelman, Sylvain Thépot, Rena Buckstein, Ulrich Germing, Yazan F. Madanat, María Díez-Campelo, David Valcárcel, Anna Jonášová, Souria Dougherty, Sheetal Shah, Qi Xia, Libo Sun, Shyamala Navada, Faye Feller, Michael R. Savona, Uwe Platzbecker

  • December 31, 2025

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