Myelodysplastic Syndromes

Myelodysplastic Syndromes

Signs and Symptoms
Diagnosis
Treatment

Treatment

lFor most patients, myelodysplastic syndromes (MDS) is considered incurable and the goals of treatment are to make patients feel better. Some patients do not need any initial treatment. However, for those who do need therapy, many treatments are available to ease their symptoms. Patients with anemia often benefit from red blood cell transfusions, which can lead to several years of good functioning life. Other options include weekly injections of red blood cell hormones such as erythropoietin (EPO, Procrit), thalidomide, vitamin B6 or low-dose chemotherapy. Patients with RA (anemia alone) often can live five to 10 years with these supportive care measures. Patients with infections due to a low white blood cell count may be given the hormone G-CSF (Neupogen) and antibiotic therapy.

Patients with more advanced forms of MDS require more aggressive therapy. These patients often have more leukemia cells in their bone marrow or have abnormal cytogenetics, and they usually live only one to two years. Treatment options include low-dose or aggressive chemotherapy as well as experimental treatments. The goals of therapy are to improve symptoms, prolong survival and to improve the quality of the remaining life. Patients who are young enough may be eligible for an allogeneic stem cell transplant, which uses stem cells from a relative or unrelated donor. Allogeneic stem cell transplantation is the only curative therapy possible. Unfortunately, most patients with MDS are too old or too debilitated to undergo allogeneic stem cell transplantation.

Evaluation

• A general history and physical examination
• Multiple blood tests
• A bone marrow aspiration and biopsy procedure

Patients with advanced MDS may qualify for a number of experimental therapies including stem cell transplantation. Whether stem cell transplantation is an appropriate therapy for an individual patient depends on a number of factors including age, MDS subtype, current symptoms and the availability of a donor. Patients are encouraged to consult with a stem cell transplantation doctor to learn more about MDS, possible treatments and stem cell transplantation.

Stem Cell Transplantation

The only know curative treatment for MDS is an allogeneic stem cell transplant, also called a bone marrow transplant. At UCSF, there are five different allogeneic transplantation protocols available for patients with MDS. Some are only available for younger patients (those under 55 years old), but others are available for patients up to age 70 or even 75. These experimental protocols are aimed at evaluating the safety and effectiveness of allogeneic transplantation in older patients with MDS. With allogeneic transplantation it is expected that 20 percent to 30 percent of patients will die early of treatment-related complications, but that a minority of patients will be cured. Results seem to be better when the transplant is performed at an earlier stage of MDS and before the disease progresses to acute myeloid leukemia (AML). Transplantation is being investigated in patients who have a related donor as well as with donors found through the National Marrow Donor Program (NMDP).

Another option is an autologous stem cell transplant, where patients donate their own stem cells for transplantation. Some results suggest that a small percentage of patients (perhaps up to 30 percent) may benefit from high-dose chemotherapy and re-infusion of their own stem cells. The advantage of this type of transplant is that it does not require a donor. The disadvantage is that abnormal stem cells may be re-infused and cause MDS to develop again after the transplantation procedure.

Investigational Therapies

An experimental chemotherapy drug called 5-Azacytidine has shown very promising results as a treatment for MDS. It is given by injection seven days each month for four to six months. It generally is well tolerated, even by older and sicker patients. The main side effect is mild nausea, which is easy to control. This low-dose chemotherapy medication is available at UCSF Medical Center through the National Cancer Institute (NCI) on a compassionate use basis.

An experimental therapy utilizing a high-dose form of Vitamin D also is available for patients with transfusion-dependent MDS. This multi-center trial (being tested across the United States) is currently open to volunteers. Please contact the Hematology Practice at (415) 353-2421 to learn more about this trial.

Reviewed by health care specialists at UCSF Medical Center.
Last updated May 8, 2007