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Current Treatment Options for Aplastic Anemia and Myelodysplastic Syndrome

Current Treatment Options for Aplastic Anemia and Myelodysplastic Syndrome

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

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