First patient cured of rare blood disorder

David Levy, shown here with his sister, is the first adult patient cured of CDA. Credit: David Levy

Using a technique that avoids the use of high-dose chemotherapy and radiation in preparation for a stem cell transplant, physicians at the University of Illinois Hospital & Health Sciences System have documented the first cure of an adult patient with congenital dyserythropoietic anemia. CDA is a rare blood disorder in which the body does not produce enough red blood cells, causing progressive organ damage and early death.

The technique is unique, because it allows a donor’s cells to gradually take over a patient’s without using toxic agents to eliminate a patient’s prior to the transplant.

Dr. Damiano Rondelli, the Michael Reese Professor of Hematology at the University of Illinois at Chicago, says the protocol can be used even in patients with a long history of disease and some because of the minimal use of chemotherapy.

“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 Rondelli, who is also division chief of hematology and oncology and director of the stem cell transplant program at UI Health.

“This procedure gives some adults the option of a stem cell transplant which was not previously available.”

For more than 30 years, Northbrook, Illinois, resident David Levy’s only course of treatment for CDA was regular blood transfusions to ensure his organs and tissues received enough oxygen. Levy was 24 when the pain became so severe he had to withdraw from graduate school.

“I spent the following years doing…

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