Stem Cell Transplant Cures a Man With Rare Blood Disorder

A new technique in transplanting stem cells has successfully cured an adult patient suffering from a rare blood disorder called congenital dyserythropoietic anemia.

The technique, described in a paper published in the journal Bone Marrow Transplantation, avoids the use of high-dose chemotherapy and radiation 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 Dr. Damiano Rondelli, the Michael Reese Professor of Hematology at the University of Illinois at Chicago, in a press release.

Rondelli said that the procedure will give adult patiets the freedom to have a stem cell transplant that was not available in the past.

David Levy, a 35-year-old resident of Northbrook, Illinois, is the very first adult patient to be cured from congenital dyserythropoietic anemia via stem cell transplant.

Levy has been suffering from the blood disorder for more than 30 years, undergoing regular blood transfusions to ensure his organs and tissues receive enough oxygen.

By 24, the pain from his condition is so severe that he had to withdraw from graduate school. When he was 32, Levy was required to undergo blood transfusions every two to three weeks. During this time, Levy lost his spleen and had an enlarged liver. Aside from fatigue and heart palpitations, Levy is also suffering from iron poisoning due to his regular blood transfusions.

The new stem cell technique was used to Levy before his transplant in 2014. As oppose to usual stem cell transplants, the new technique allows the donor's cells to gradually take over the patient's marrow without using any toxic agents to eliminate the patient's cells before the transplant.

Because of the minimal use of chemotherapy and radiation, the new stem cell technique can be used even in patients with a long history of disease and some organ damage, just like in the case of Levy.