Low Risk vs. High Risk MDS

Jul 11, 2024

Low Risk vs. High Risk MDS

Introduction and Overview

  • Presenter: Dr. Luskin (MD from University of Pennsylvania; Residency at Brigham and Women's Hospital; Fellowship in Hematology Oncology at University of Pennsylvania; Member of Adult Leukemia Program at Dana Farber)
  • Moderator: Lee Clark
  • Sponsors: Celgene, Takeda, and Jaz Pharmaceuticals
  • Audience: Patients, families, caregivers

Understanding Myelodysplastic Syndrome (MDS)

  • MDS is a bone marrow disease affecting blood production.
  • MDS characteristics: Ineffective hematopoiesis, risk of progression to AML (acute myeloid leukemia), low blood cell counts (cytopenias).
  • MDS affects the myeloid lineage of blood cells (red blood cells, white blood cells, platelets).

Diagnosis of MDS

  • Usually diagnosed through bone marrow biopsy and blood tests.
  • Signs: Dysplasia (abnormal cell appearance), abnormal chromosomes, excess blasts (immature cells).
  • Evaluations are important to rule out other causes of low blood counts.

Who Gets MDS?

  • Age-related: Median age of diagnosis is mid-70s.
  • Incidence: 10,000 to 40,000 cases annually in the US (possibly underreported).
  • Risk Factors: Age, prior chemotherapy or radiation, environmental toxins, familial predisposition, prior diagnosis of aplastic anemia.

Symptoms and Complications

  • Common symptoms: Often asymptomatic initially; can include anemia, easy bruising/bleeding, infections.
  • Long-term issues: Risk of progression to AML, progressive bone marrow failure.

Prognosis and Risk Assessment

  • Risk determined by IPSS and IPSS-R scoring systems (blood counts, blast percentage, genetic abnormalities).
  • Low vs. High Risk: Low-risk MDS progresses slowly; high-risk MDS progresses more quickly and aggressively.

Treatment Options

Low-Risk MDS Treatment

  • Supportive care: Managing symptoms, transfusions for low blood counts, erythropoiesis-stimulating agents (ESAs), lenalidomide for specific genetic abnormalities (del 5q).
  • Transfusions: Red blood cells and platelets; risks include iron overload.
  • Monitoring: Regular blood tests to detect changes in disease status.

High-Risk MDS Treatment

  • Chemotherapy: Hypomethylating agents like azacitidine and decitabine to improve blood counts and delay progression to AML.
  • Stem cell transplant: Considered the only curative option but involves high risks.

Future Directions and Clinical Trials

  • Emphasis on the need for new and better treatments.
  • Encouraging participation in clinical trials to advance understanding and treatment options.

Closing Remarks

  • Importance of personalized care: Understanding each patient’s unique disease and overall health for tailored treatment recommendations.
  • Questions and monitoring: Continuous engagement with healthcare providers to manage disease and treatment effects effectively.
  • Educational resources: Patients are encouraged to stay informed and involved in their health management.

Questions and Answers Section

  • Monitoring and Treatment Timelines: Establishing a monitoring plan with physicians.
  • Challenges in MDS Management: Addressing patient-specific scenarios, including medication effectiveness and MDS-related genetic abnormalities.
  • Role of Clinical Trials: Importance of exploring and participating in ongoing research efforts.

Additional Remarks

  • Importance of comprehensive evaluation of newly diagnosed patients.
  • Risk of AML in MDS patients and the need for continuous disease monitoring.