MYELODYSPLASTIC SYNDROMES

The myelodysplastic syndromes are a group of marrow diseases characterized by progressive decline in the blood counts and potential conversion to acute leukemia. These diseases are more common in the elderly and may be related to prior chemical or radiation damage.

Most patients first discover their disease when they have recurrent infections, bleeding or extreme fatigue. As the bone marrow fills up with abnormal cancer cells (known as blasts) or the bone marrow becomes more abnormal (dysplastic), the normal bone marrow cells decrease in number. Thus patients may have low numbers of red blood cells (the cells that carry oxygen and nutrients), low numbers of platelets (the cells that help with clotting), and low numbers of normal white blood cells (the cells that fight infections).

The prognosis of these diseases is quite variable. Refractory anemia (with or without ringed sideroblasts) is a slowly progressive disease with survivals often in the range of 7-10 years. Refractory anemia with blasts (excess or blasts in transition) are much more aggressive diseases in which patients are at risk of immediate infection and bleeding complications and diseases which may progress to leukemia in under 2 years.

Treatment of the myelodysplastic diseases has been quite difficult. Many patients are too old to withstand aggressive therapy such as chemotherapy. Transfusions of blood and antibiotic treatments are common. In younger patients, combination chemotherapy (which medications such as topotecan and cytarabine) may result in temporary remissions.

Allogeneic bone marrow transplantation has been a curative treatment in these diseases. The effectiveness of this treatment is determined, in large part, by the number of blasts at the time of transplantation and the age of the patient. In one report 54% of patients with early stage disease were disease free at 4 years. By contrast, among patients with blasts at the time of transplant, over 50% had relapsed. (Anderson JE et al: Blood 1993; 82:677).

The Adult Stem Cell- Bone Marrow Transplant Program of Hackensack University Medical Center has active transplant treatment protocols for Myelodysplastic Syndromes. For more information on this treatment or to schedule an appointment call (201) 996-5849.

ALLOGENEIC TRANSPLANT OPTIONS

For patients with Tissue-matched (HLA-matched) brother/sister bone marrow donors allogeneic transplantation treatment protocols may be appropriate. One trial uses high-dose cyclophosphamide combined with either total body irradiation or high-dose busulfan. Following this aggressive conditioning therapy, bone marrow (or peripheral blood stem cells) from an HLA-matched donor is infused. Graft-versus-host disease prevention is accomplished with combinations of tacrolimus and methotrexate. This very aggressive curative treatment requires a one-month hospitalization.

UNRELATED TRANSPLANT TREATMENT OPTIONS

For patients lacking a family member marrow donor, the Hackensack University Medical Center Transplant Programs are participating in several unrelated transplant protocols.

The National Marrow Donor Program has enlisted over 3 million volunteer bone marrow donors. If an HLA matched unrelated donor can be located, a transplant using high-dose cyclophosphamide and total body irradiation can be performed.

Hackensack University Medical Center is also participating in a multicenter trial evaluating the use of expanded Cord Blood Cells in the treatment of cancers and blood diseases. This trial also extends the treatment options for patients without HLA Matched relative donors. For more information click here and also review ex-vivo expansion in the stem cell laboratory section.

AUTOLOGOUS TRANSPLANT OPTIONS

High-dose treatments using autologous rescue are under development. For more information call (201) 996-5849.

NON-TRANSPLANT OPTIONS

The physicians at the Northern New Jersey Cancer Center are experienced in the diagnosis and treatment of these diseases. One study is evaluating the value of growth factors (erythropoietin and filgrastim) in MDS. For more information on these trials call (201) 996-5900.