A Bone Marrow Disease With a Brighter Prognosis

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Advances in stem cell research and anti-rejection drugs have meant that transplantations from unrelated donors also are becoming more successful, Paquette said.

One promising avenue of treatment involves transplantation using stem cells harvested from the umbilical cord of new mothers. "The cells can be cryopreserved [frozen] and saved, then given to unrelated donors," Paquette said. "It's quite encouraging."

For these patients, again, speed is of the essence. "The data show the earlier you do a transplant, the better the outcome," Paquette said.

Patients whose transplants fail, or for whom transplantation is not an option, often receive successful immunosuppressive therapy with agents like anti-thymocyte globulin and cyclosporine. Response rates typically range from 70 percent to 80 percent, according to the Aplastic Anemia & MDS International Foundation Inc.

Blood transfusions from matched donors also are used to keep blood counts high and help relieve symptoms, although they are not an effective long-term treatment.

"Whether we cure the disease or not, patients are getting better across the board," Maciejewski said. "We now can maintain life, keep these patients alive longer."

More information

To learn more, visit the Aplastic Anemia & MDS International Foundation Inc.