May 18, 2010 - Myelodysplastic Syndromes (MDS): An Update on Diagnosis and Treatment
- MDS and its subtypes
- Diagnostic tests for MDS
- Current and emerging treatment options
- Potential benefits of patient participation in clinical trials
- Possible treatment side effects and the importance of follow-up care
Stephen D. Nimer, M.D.
Chief, Hematology Service
Vice Chairman for Faculty Development
Department of Medicine
Alfred P. Sloan Chair
Memorial Sloan-Kettering Cancer Center
Professor of Medicine and Pharmacology
Weill Cornell Medical College
New York, NY
There are numerous therapeutic options for patients who need treatment. Current drug studies are showing survival benefits even among older patients with additional investigational therapies on the horizon.
Dr. Nimer provides an introductory overview of MDS and its subtypes. He explains how MDS is diagnosed and reviews treatment options for managing the disease and its complications. Dr. Nimer also answers audience questions.
- Doctors know very little about what triggers MDS. It's unclear why some patients remain stable for many years and don't need treatment while others have more progressive illness or develop leukemia.
- Patients with MDS have a problem with the production of mature blood cells. Most often, patients have low red cell counts (anemia), low white cell counts (leukopenia or neutropenia) or low platelet counts (thrombocytopenia).
- A bone marrow examination is needed to diagnose MDS. Doctors look for abnormalities in the blood cells and platelets, the percentage of blast cells (cells in the earliest stage of development) and genetic irregularities.
- The type of chromosomal abnormalities present in cells helps determine treatment approach.
- Treatment for patients with anemia may include growth factors, lenalidomide (Revlimid®), red cell transfusions or no therapy at all, depending on symptoms.
- Patients who have transfusion-dependent MDS, more advanced MDS or very low blood counts may be treated with the hypomethylating agents 5-azacitidine (Vidaza®) or decitabine (Dacogen®). Patients with very low blood counts should also be considered for stem cell transplantation.
- Revlimid® is used to treat some MDS patients
- MDS treatment helps reduce the possibility of progression to leukemia.
- Both 5-azacitidine and decitabine provide great benefit and are very well tolerated, even when used with older MDS patients. Patients who are told they are too old for these treatments should seek a second opinion. Results of one study showed that people with advanced MDS treated with 5-azacitidine survived nearly twice as long as patients who received supportive care (transfusions, antibiotics), intensive chemotherapy or low-dose cytarabine (ara-C).
- Investigational therapies are on the horizon, including those combining already-approved treatments.
- Allogeneic stem cell transplantation is potentially curative for MDS.
- It's important for doctors to monitor patients receiving red cell transfusions for iron overload. Drugs are available to help remove excess iron from the body.
Questions Asked by the MDS Community
- If a treatment doesn't work, does it suggest a misdiagnosis?
- Is it better to receive Vidaza every four weeks instead of every six weeks?
- Can patients safely stop taking Vidaza for a short period?
- About how many cycles does it take for Vidaza to begin working?
- Why should patients with low platelet counts not take vitamin E or do strenuous exercise?
- Are outcomes data available for patients with certain chromosome abnormalities?
- Can treatment for one type of cancer trigger an MDS diagnosis?
- What are the treatment options for patients with a very low platelet count?
- What are unclassified types of MDS?
- What are overlap syndromes and how should they be treated?
- What is a myeloproliferative (MPD)-like disease?
- What is graft versus host disease?
- Is it possible for disease to return after stem cell transplantation?
- What does a median number mean?
- Does a decline in hemoglobin suggest treatment may be losing efficacy?
- If MDS becomes leukemia, does the patient have leukemia only or both leukemia and MDS?
- What are the possible outcomes for patients whose MDS develops into leukemia?
Sponsors and Supporters
This program was sponsored by The Leukemia & Lymphoma Society and was supported by a grant from Celgene Corporation.