Stem cell transplant offers first-ever cure for rare blood disorder

A new stem cell transplant technique that does not require the use of high-dose chemotherapy and radiation successfully cured an adult patient with congenital dyserythropoietic anemia, marking the first documented cure of the condition, according to a recent letter to the editor published in the journal Bone Marrow Transplantation.

CDA is a rare blood disorder that inhibits the body's ability to produce red blood cells. The condition can damage the organs and result in early death. The disorder is typically treated with regular blood transfusions.

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"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, MD, the Michael Reese Professor of Hematology at the University of Illinois at Chicago and the physician who performed the procedure. "This procedure gives some adults the option of a stem cell transplant which was not previously available."

The patient, David Levy, had been receiving regular blood transfusions for more than 30 years to ensure his organs and tissues received enough oxygen. At 32-years-old, Mr. Levy was receiving transfusions every two to three weeks, had an enlarged liver and lost his spleen. He also suffered from fatigue, iron poisoning and heart palpitations. Several institutions denied him the possibility of a stem cell transplant because Mr. Levy's body could not tolerate chemotherapy.

"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," said Dr. Rondelli.

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