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Doctors have cured a patient with rare blood disorder in which the patient's body did not produce enough red blood cells that could have caused progressive organ damage and early death.
Physicians from the University of Illinois Hospital and Health Sciences System cured an adult patient with congenital dyserythropoietic anemia (CDA) using a technique that avoids the use of high-dose chemotherapy and radiation in preparation for a stem cell transplant.
The technique allows a donor's cells to gradually take over a patient's bone marrow without using toxic agents to eliminate a patient's cells prior to the transplant.
"For many adult patients with a blood disorder, treatment options have been limited because they are often not sick enough to qualify for a risky procedure, or they are too sick to tolerate the toxic drugs used alongside a standard transplant," said Damiano Rondelli, Professor at the University of Illinois.
He said the protocol can be used even in patients with a long history of disease and some organ damage because of the minimal use of chemotherapy.
"This procedure gives some adults the option of a stem cell transplant which was not previously available," Rondelli added.
David Levy, 35, experienced a severe pain at the age 24 and had to withdraw from graduate school.
"I spent the following years doing nothing -- no work, no school, no social contact --because all I could focus on was managing my pain and getting my health back on track," Levy said.
Levy required transfusions every two to three weeks until three years ago and had lost his spleen and suffered from fatigue and other complications.
"The use of this transplant protocol may represent a safe therapeutic strategy to treat adult patients with many types of congenital anemias -- perhaps the only possible cure," Rondelli said in a letter published in the journal Bone Marrow Transplantation.
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