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For Immediate Release

Children’s Hospital of Pittsburgh Successful in Rare Transplant to Cure Patient with Sickle Cell Disease

Children’s physician has pioneered approach that could lead to a cure for more patients

For the first time in the tri-state region, doctors at Children’s Hospital of Pittsburgh have successfully performed a bone marrow transplant on a young child suffering from sickle cell disease, using an approach that may lead to the cure of more people with the disease.

Bone marrow transplants are the only known cure for sickle cell disease, but the procedure has rarely been performed because side effects from the transplants, such as infections and graft-versus-host disease, can be fatal.

Hematologist Lakshmanan Krishnamurti, MD, last month performed the first successful bone marrow transplant on a patient with sickle cell disease at Children’s Hospital, using a rare approach known as reduced-intensity transplant. The goal of this approach is to make bone marrow transplant more widely available as a cure to patients with sickle cell disease by eliminating the life-threatening side effects of transplant.

Austin Jones, 5, son of Anthony and Sarah Jones of Indiana, Pa., underwent the reduced-intensity transplant on Friday, Aug. 8, 2003. He has been recovering at Children’s and tests show that his body has accepted the marrow donated by his 11-year-old brother, Anthony Jr.

“Our goal with Austin was to weaken his bone marrow with immunosuppressive drugs, not destroy it with chemotherapy, prior to transplant. By doing this, we were able to rid him of sickle cell disease and prevent the harmful side effects of transplant,” said Dr. Krishnamurti, director of the Comprehensive Hemoglobinopathy Program at Children’s. “Our success with Austin is encouraging. We hope that we can provide a cure for more of the 1,000 children born with sickle cell disease each year in the United States.”

Only approximately 210 bone marrow transplants for sickle cell disease have been performed worldwide in the last two decades, in part because of the dangerous, potentially life-threatening side effects like fever and infection.

Traditionally, bone marrow transplants require heavy doses of chemotherapy prior to transplant in order to destroy the bone marrow so it will not reject the donated marrow. But with their bone marrow destroyed, transplant recipients are vulnerable to life-threatening complications.

However, the reduced-intensity transplants only weaken the recipient’s bone marrow by relying on lower levels of chemotherapy and high doses of immunosuppressive drugs. This lessens the chance of these life-threatening complications.

Dr. Krishnamurti was the first physician in the world to perform a reduced-intensity bone marrow transplant in a patient with sickle cell disease while at the University of Minnesota in 1999. Since then, there have been only approximately 10-15 reduced-intensity bone marrow transplants performed in the world, including Austin’s transplant, according to Dr. Krishnamurti.

“Austin’s life has revolved around excruciating pain, life-threatening complications and long stays at the hospital,” Anthony Jones said. “It seems like we’ve spent the majority of his life in doctors’ offices or hospitals. This bone marrow transplant will give Austin a chance to be a normal 5 year old, and to grow to be an old man.”

Sickle cell disease is an inherited blood disorder that affects approximately 80,000 people in the United States, primarily African-Americans. There are nearly 250,000 African-Americans in western Pennsylvania and about 250 pediatric patients with sickle cell disease at Children’s.

Sickle cell disease can cause excruciating pain, damage to organs including the heart, kidneys and lungs, and a shortened life expectancy. Sickle cell is characterized by defective hemoglobin, a protein in red blood cells that carries oxygen to the body’s tissues.

For more information about sickle cell disease or the Sickle Cell Program at Children’s, visit Children’s Web site at www.chp.edu.

Contacts:
Marc Lukasiak, 412-692-5016, Marc.Lukasiak@chp.edu
Melanie Tush Finnigan, 412-692-5016,Melanie.Finnigan@chp.edu

Last Update
February 20, 2008
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Last Update
February 20, 2008
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