The ASH Warm-Up: Expert Perspectives on Evolving MDS Treatment and Care

The field of myelodysplastic syndromes (MDS) is advancing rapidly, and a recent PeerView educational session held just before the 66th American Society for Hematology (ASH) annual conference provided valuable insights into the latest developments in MDS care.

This session, titled Shifting to Higher Standards in MDS: Custom and Innovative Care to Improve Outcomes for Lower- and Higher-Risk Disease, brought together world-renowned experts to discuss current MDS diagnosis and treatment strategies. While the event was designed for healthcare professionals (HCPs), the information shared has important implications for patients and caregivers.

Here’s a breakdown of the key takeaways to help you better understand the evolving landscape of MDS care.

Key Highlights for Patients Living with MDS

Diagnosing MDS: Why It Matters

Accurate diagnosis and risk assessment are essential for determining the best treatment plan for MDS. One of the first steps is identifying whether a patient has lower-risk or higher-risk MDS.

Additionally, understanding your mutational status—especially mutations like IDH1 and IDH2—can open the door to targeted therapies. If you’re unsure about your genetic profile, talk to your doctor about testing.

For more on MDS genetics, check out our webinar with Dr. Chandhok. What is genetic testing? What are the most important mutations in MDS? How can these mutations affect your prognosis or your treatment options? Create a free HealthTree account below to watch our webinar recording and answer all these questions.

What is Genetic Testing in MDS? 

Treatment Updates for Lower-Risk MDS

1. Luspatercept as a First-Line Option

Luspatercept (Reblozyl, BMS) is now a key first-line treatment for patients with lower-risk MDS who experience anemia and are transfusion-dependent. It has shown promising results in reducing the need for blood transfusions.

2. Imetelstat for Relapsed Patients

For patients who don’t respond or are not eligible to receive erythropoiesis-stimulating agents (ESAs), imelelstat (Rytelo, Geron) is now available as a second-line option. Data from the IMerge study shows that 83% of patients who achieve transfusion independence within the first eight weeks of treatment experience long-lasting responses.

Treating Patients with Higher-Risk MDS

1. Stem Cell Transplantation: Still an Effective Option

Stem cell transplantation remains the most effective treatment for higher-risk MDS, offering the potential for longer survival. However, experts emphasized the need for research on safer transplant processes with fewer complications, such as graft-versus-host disease (GvHD).

2. New Trials with Venetoclax are Aiming for an All-Oral Treatment

The combination of decitabine/cedazuridine (Inqovi, Taiho Oncology) with venetoclax is an exciting development in the move toward an all-oral regimen for MDS. Early data shows promising response rates, but this approach requires careful management of side effects.

One of the key highlights from Dr. Garcia-Manero was that venetoclax for MDS is different from what most doctors are used to when treating patients with acute leukemia. For example:

• • Venetoclax dosing in MDS differs from its use in acute myeloid leukemia (AML).
  • Tumor lysis syndrome (TLS) prophylaxis is less critical in MDS compared to when treating AML patients, but it should still be considered.
  • Dose adjustments are needed when combining venetoclax with antifungal medications like azoles.

3. Targeted Therapies for IDH1 Mutated MDS

Ivosidenib (Tibsovo, Servier) is a rising preferred targeted therapy for patients with IDH1 mutations who have relapsed from previous therapies. Ongoing studies are exploring its use also for newly diagnosed patients.

4. Game-Changer Clinical Trials Are on the Horizon

MDS experts shared their excitement for the VERONA trial (NCT04401748), which investigates the combination of venetoclax with azacitidine for newly diagnosed high-risk MDS patients. Results are eagerly awaited, with experts expressing optimism about the potential for higher cure rates in the near future.

Key Takeaways for Patients and Caregivers

• Personalized care matters: A detailed assessment of your risk status and genetic profile is essential for tailoring the best treatment plan.
• Explore all options: Don’t hesitate to ask your doctor about clinical trials or new therapies that may be available to you.
• Stay informed: Advances in combination therapies and targeted treatments are paving the way for better outcomes for all patients living with MDS.

Looking Ahead

The PeerView session ended on an optimistic note, with experts like Dr. Guillermo Garcia-Manero expressing hope that we may soon cure 50-60% of patients with higher-risk MDS.

As the field of MDS care continues to advance, patients and caregivers are encouraged to stay engaged, ask questions, and explore all available resources.

Stay tuned for more updates from ASH 2024 as we break down the latest research and what it means for you. Visit our HealthTree Conference Coverage page for more insights!

Sources: 

• Study Of Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Disease Activity In Adult Participants With Newly Diagnosed Higher-Risk Myelodysplastic Syndrome (Verona)