Congenital dyserythropoietic anaemia cured for first time

An innovative technique has been used to successfully cure an adult patient with congenital dyserythropoietic anaemia (CDA) for the first time.

Conducted by the University of Illinois at Chicago, a study was carried out to assess a new  technique that uses a stem cell treatment to address CDA, which is a rare blood disorder that stops the body from producing enough red blood cells.

It allows a donor's own cells to gradually take over the bone marrow without using high-dose chemotherapy and radiation to eliminate a patient's cells prior to the transplant – a process that can be highly dangerous.

As such, it can be used even in patients with a long history of disease and some organ damage. For many adult patients with a blood disorder, treatment options are limited because they are not ill enough to qualify for a risky procedure, or too sick to tolerate the toxic drugs used alongside the transplant.

The method has now been used to cure a 35-year-old American patient who has suffered from the condition for many years, with regular blood transfusions as his only available treatment option.

Dr Damiano Rondelli, professor of haematology at the University of Illinois at Chicago, said: "This procedure gives some adults the option of a stem cell transplant which was not previously available."

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