LOS ANGELES—Myelodysplastic syndrome and aplastic anemia are both diseases of bone marrow failure and are characterized by reticulocytopenic anemia, with variable neutropenia and thrombocytopenia. Other than that, the two diseases are substantially different from each other. Tools for diagnosing and managing these two complex diseases were updated by Ronald L. Paquette, MD, associate professor of medicine at the University of California Medical Center in Los Angeles.
Aplastic Anemia
Patients with aplastic anemia (AA) are usually quite ill and symptomatic when they present, predominantly because of pancytopenia. They may have fatigue or dyspnea on exertion, severe infections, petechiae, purpura, and/or mucosal bleeding. These patients can have profound anemia. "It’s not uncommon for them to come in with a hemoglobin of 5 g/dL," Dr. Paquette reported.
Diagnosis is by bone marrow aspiration and biopsy. "Usually the bone marrow is nearly completely empty and no dysplastic features are seen. This is a disease that really is autoimmune in nature, with the patient’s own immune system attacking bone marrow precursors and essentially wiping them out," Dr. Paquette said.
Disease criteria are:
- absolute neutrophil count < 500/µL;
- absolute reticulocyte count < 20,000/µL; and
- platelet count < 20,000/µL.
Patients with one criterion have moderate AA; those with two criteria have severe disease; and patients with three criteria, including a neutrophil count < 200/µL, have very severe disease.
Very Effective Therapies
