Aplastic anemia is a disease of bone marrow failure characterized by severe depression of blood counts and an "empty" bone marrow. Untreated patients with granulocyte (an infection fighting cell) of less than 0.5 X109/l, platelet (a clotting cell) counts of less than 20 X 109/l, and reticulocyte (a red blood cell that carries oxygen) counts of less than 1% have an extremely poor prognosis. Untreated, these patients have an 80-90% chance of dying within the first 6 months after diagnosis.Fortunately Aplastic Anemia has become a curable disease. Marrow transplantation from HLA matched relatives or unrelated individuals is the preferred treatment for young patients (under the age of 50 years old), leading to cures in upwards of 80%. (Reference: Storb R et al: Changing trends in marrow transplantation for aplastic anemia. Bone Marrow Transplant 1992: 10 (Suppl 1): 45.). For older individuals or those unable to undergo an allogeneic transplant, high dose immunosuppressive chemotherapy (cyclophosphamide) or immunosuppressive medications (cyclosporine or anti-thymocyte globulin) may be curative.
The Adult Stem Cell- Bone Marrow Transplant Program of Hackensack University Medical Center has active transplant treatment protocols for this disease. For more information about these treatments or to schedule an appointment call (201) 996-5849.
One protocol uses two immunosuppressive medications (cyclophosphamide and anti-thymocyte globulin) followed by infusion of bone marrow from an HLA Matched relative. For more information click here.
Two other protocols are available for patients without HLA matched relatives.
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.