Myelodysplastic Syndromes Treatment
UC San Diego: An MDS Center of Excellence
Moores Cancer Center is an MDS Center of Excellence designated by the Myelodysplastic Syndromes Foundation. It is one of just six in California and the only one in San Diego County. As a Center of Excellence, we provide expertise in all aspects of MDS care, including:
- Experienced hematopathologists to ensure an accurate diagnosis
- Physicians dedicated to the personalized care of patients with MDS and their families
- State-of-the-art genetic testing to individualize treatment options
- Opportunities to participate in therapeutic clinical trials and MDS research programs
- Access to advanced bone marrow stem cell transplantation techniques
- Warm and knowledgeable support staff members on our MDS care team
Researchers and physicians have recently gained a better understanding of how myelodysplastic syndromes (MDS) develop at the genetic level. This has led to a rapid translation into the clinic. Researchers from UC San Diego have identified gene mutations that can help establish a diagnosis, predict response to treatment, and affect survival rates of patients with MDS. Working with UC San Diego's Center for Personalized Cancer Therapy, we now use genetic sequencing to evaluate our patients and determine which treatments might be best suited for them personally.
Dr. Bejar meets with a patient at Moores Cancer Center.
Rafael Bejar, MD, PhD, is a physician/scientist who both cares for patients and runs a research laboratory dedicated to studying MDS. He is an expert on diagnosing and treating these disorders, and is passionate about discovering ways to personalize the care of MDS patients. His papers and research efforts have led to tests for mutations that help doctors predict a patient's prognosis and individualize their treatment based on the results.
Peter Curtin, MD, is part of UC San Diego's Blood and Marrow Transplantation (BMT) Program, also located at Moores Cancer Center. If your care requires blood and marrow transplant, we have the facilities and an outstanding BMT team who coordinates closely with Dr. Bejar to provide your treatment. Dr. Curtin has more than 20 years' interest and experience in caring for patients with MDS and related disorders.
Diagnosing and Treating MDS
MDS can be difficult to diagnose, and an accurate diagnosis is crucial for proper treatment. Our dedicated hematopathologists, Elizabeth Broome, MD, and Huan-You Wang, MD, PhD, focus solely on blood and bone marrow disorders and have decades of experience between them. We employ state-of-the-art genetic testing to help make an accurate diagnosis and better predict how your disease is likely to behave.
A bone marrow aspirate and biopsy is typically required to make the diagnosis. Your doctor will combine this information with a careful personal history, physical exam, and blood tests to determine your disease risk and best treatment options.
Moores Cancer Center now uses genetic testing to analyze your cancer type and tailor treatments specifically for you.
See Center for Personalized Cancer Therapy.
Our treatment is customized for you based on your type of MDS. Some types, considered "low-risk," may cause mild to moderate anemia or other issues. Other types, considered "high-risk," can evolve more quickly or develop into acute leukemia.
- Treatment options for low-risk MDS are designed to maximize quality of life. These interventions include:
- Active observation without therapy
- Blood transfusions
- Growth factor therapy to increase red blood cell production (usually erythropoietin or granulocyte colony-stimulating factor)
- Lenalidomide (Revlimid) for patients with chromosome 5q deletion
- Immune suppression for patients with features of aplastic anemia
- Supportive drug therapy, which may include antibiotics to fight infections or medications to help reduce the need for transfusions
- Treatment for high-risk MDS may include:
Because of the active MDS research program at UC San Diego, your doctor may also suggest a clinical trial to test newer, more promising treatments.
Appointments may be available in Hillcrest, La Jolla, or Encinitas.
Dr. Bejar's research laboratory focuses on understanding the genetic changes that drive the development and progression of blood cancers like MDS. The goal is to translate discoveries into new treatments. Previously, our researchers helped characterize the range of genetic changes in MDS patients by studying bone marrow samples from over 400 patients. They identified mutations in several genes that, when present, are associated with a poorer prognosis than we could otherwise predict with a standard clinical scoring system. This finding has helped develop tumor sequencing tests to better evaluate patients with MDS.
Currently we are investigating how acquired mutations in MDS patients are associated with their response to treatment with drugs like azacitidine and decitabine. We hope to discover genes that physicians can test for mutations in order to select the best treatment options for their patients with MDS. These studies should also give us insight into the mechanisms of drug resistance and how we may be able to overcome them.
In the future, we plan to implement clinical sequencing of bone marrow specimens from patients with MDS and related disorders. Our plan is to study how best to use this information to personalize patients' treatments and overall care.
For more general information about MDS, see the tabs below:
Myelodysplastic syndromes (MDS) are diseases of the blood and bone marrow. In a healthy person, bone marrow stem cells divide to create daughter cells that produce all of the mature cells found in our blood: red blood cells (which carry oxygen), white blood cells (which fight infection), or platelets (which form blood clots to stop bleeding).
In people with MDS, the blood stem cells have become abnormal and have difficulty producing mature blood cells. These abnormal cells either die in the bone marrow or soon after they enter the blood, leading to a lack of healthy cells and potential health complications including infection, anemia or bleeding.
There are several subtypes of MDS defined by features of cell sin the blood and bone marrow. They include:
- Refractory anemia
- Refractory anemia with ringed sideroblasts
- Refractory anemia with excess blasts
- Refractory anemia with excess blasts in transformation
- Refractory cytopenia with multilineage dysplasia
- Myelodysplastic syndrome associated with an isolated del(5q) chromosome abnormality
- Unclassifiable myelodysplastic syndrome
- MDS and myeloproliferative overlap syndromes
The symptoms of MDS are often vague and can be caused by unrelated disorders. These symptoms may include:
- Shortness of breath
- Weakness or fatigue
- Pale skin
- Easy bruising or bleeding
- Flat, pinpoint spots under the skin caused by bleeding
- Fever or frequent infections
MDS usually afflicts people in their 60s or older. Exposure to chemotherapy, radiation therapy, or certain chemicals can predispose people to get MDS, but in most patients, there is no identifiable factor responsible for their disease.