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New treatment for Sickle Cell Anemia

October 6, 2009 3:16:21 PM PDT
Sickle cell anemia is the most common inherited blood disorder in the United States, affecting as many as 80,000 people. Instead of having round-shaped red blood cells, people with the disease have cells that look like half-moons, or sickles, causing pain, organ damage, and in severe cases, death. Doctors are now using a new intervention that may cure more patients. The Jones brothers are as "tight" as three siblings can be, but 16-year-old A.J. and 10-year old Austin share a special bond.

"I am sort of like my brother," Austin told Ivanhoe. "I have half of him in me."

When Austin was 5, he had a bone marrow transplant to cure him of sickle cell anemia. Both his brothers were matches, but A.J. was the oldest.

"They were small, like 11 and 9, so they didn't fully understand all that they were wanting to do," Sarah Jones, Austin and A.J.'s mom, told Ivanhoe. "But their attitude was yeah, we want him to get better. We don't want him to die."

Lakshmanan Krishnamurti, M.D., a pediatric hematologist at the Children's Hospital of Pittsburgh of UPMC, pioneered bone marrow transplantation that cuts down the need for toxic chemotherapy. It's called reduced intensity conditioning, or RIC.

In the past, patients would receive heavy doses of chemo before a transplant to wipe out their existing marrow. With RIC, Dr. Krishnamurti reduces the chemo dose by using drugs that suppress the immune system. This reduces the risk of rejection without destroying as much marrow.

"You can cut down the chemotherapy and rely instead on some of the donor's immune system," Dr. Krishnamurti said.

Bone marrow transplants are the only cure for sickle cell, but typically it's a last resort because of the risks chemo poses. Doctors hope the new approach makes transplants available to more kids.

"A child with sickle cell disease with a transplant is basically a regular kid," Dr. Krishnamurti said.

Before his transplant, Austin was prone to infection. Cold weather or cold water could spark a crisis. These days, no activity is out of reach.

"The outcome is amazing," mom Sarah said. "I never take that for granted."

Six of seven young sickle cell patients who have undergone RIC bone marrow transplants in Pittsburgh show no signs of the disease.

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