Diagnosis and Treatment of Myelodysplastic Syndromes (MDS)
Myelodysplastic syndromes (MDS) are a group of diseases that cause immature blood cells (called blasts) to accumulate in the bone marrow leading to a shortage of mature blood cells, including red blood cells, white blood cells, and platelets. Furthermore, the mature blood cells that are made can also be defective.
MDS are not cancer themselves, but about 30% of the time the syndromes are a precursor to leukemias such as acute myeloid leukemia (AML) and chrome myelomonocytic leukemia (CMML).
Although MDS can affect people of all ages, the median age of onset is 65. MDS affects less than 15,000 people each year in the United States, and includes the following syndromes:
- 5q syndrome (named after the chromosomal defect that causes the symptoms)
- Hypoplastic MDS
- MDS with myelofibrosis
- MDS with prominent eosinophilia or monocytosis
Causes of MDS
People who have received radiation therapy, chemotherapy with alkylating agents (such as chlorambucil, cyclophosphamide, and melphalan), or who have been exposed to industrial solvents (such as benzene) have a higher risk of developing MDS than people who have not had these exposures. Rarely, genetic disorders are responsible for the disease. Nevertheless, in 60% to 70% of MDS patients, no specific cause can be identified.
Stanford Expertise
The Stanford MDS Center has been recognized by the MDS Foundation as an MDS Center of Excellence. In addition, physicians in the Center participate in developing national treatment guidelines, serve on the boards of MDS organizations, and are actively involved in clinical trials for MDS.
The Stanford MDS Center
The major focus of the MDS Center is:
- Coordination of patient management and scientific exchange with referring physicians
- Patient/family education about MDS
- Individualized prognosis-based treatment approaches
- Quality of life issues in MDS
Management approaches at the MDS Center include use of recently developed National Practice Guidelines based on advances in prognostic evaluation of MDS patients. Differing types of treatment are available based on patients' specific subtype of MDS and related clinical problems.
The Stanford MDS Center provides MDS patients and their physicians access to:
- Current information about MDS
- Current therapeutic approaches
- Clinical trials for MDS
- Knowledge of optimal usage of treatment therapies
- Knowledge of treatment timing and patient selection
In addition to supportive care being provided (e.g., transfusions and antibiotics as needed), treatment options with new agents are available through clinical trials at the MDS Center. The clinical trials through the Stanford MDS Center are comprised of "low intensity" or "high intensity" therapy:
Low intensity therapy:
- Erythropoietin vs. G-CSF plus erythropoietin
- 5 azacytidine
- Anti-thymocyte globulin
- Anti-vascular endothelial growth factor (VEGF) (Avastin)
- Tumor necrosis factor receptor (TNFR) fusion protein (TNFR:Fc, Enbrel)
- Farnesyl transferase inhibitor (FTI) (R115777)
- Thalidomide (Thalomid)
High intensity therapy:
- Anti-CD33 immunotoxin (Mylotarg)
- Bone marrow transplantation
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