Doctors at the University of Illinois Hospital & Health Sciences System have cured a patient of a rare blood disorder, congenital dyserythropoietic anemia (CDA), a disorder in which the body does not produce enough red blood cells, causing progressive organ damage and early death. The physicians accomplished this using a technique that avoids the use of high-dose chemotherapy and radiation in preparation for a stem cell transplant.
The one-of-a-kind transplant 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. The Michael Reese Professor of Hematology at the University of Illinois at Chicago, Doctor Damiano Rondelli, says 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.
Rondelli, who is also division chief of hematology and oncology and director of the stem cell transplant program at UI Health stated, “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. This procedure gives some adults the option of a stem cell transplant which was not previously available.”
Illinois resident David Levy suffered with the disorder for more than 30 years. He was having regular blood transfusions and eventually had to drop out of school because the pain was so bad. He said, “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 required transfusions every two to three weeks by the age of 32. He was suffering from iron poisoning, heart palpitations, enlarged liver, fatigue, and had to have his spleen removed. He said, “It was bad. I had been through enough pain. I was angry and depressed, and I wanted a cure. That’s why I started emailing Dr. Rondelli.”
Rondelli says that because of Levy’s range of conditions and inability to tolerate radiation and chemotherapy, several institutions had denied him the possibility of a stem cell transplant. University of Illinois Health’s advances in curing sickle cell patients opened up a new possibility. Rondelli performed Levy’s transplant in 2014. Levy, now 35 said, “The transplant was hard, and I had some complications, but I am back to normal now. I still have some pain and some lingering issues from the years my condition was not properly managed, but I can be independent now. That is the most important thing to me.”