Unique Bone Marrow Transplant Said to Cure Sickle Cell

Technique is safe and effective, say Children's Hospital of Pittsburgh reseachers.

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FRIDAY, Nov. 14 (HealthDay News) -- A unique form of bone marrow transplantation is the only safe and effective cure for sickle cell disease, researchers at Children's Hospital of Pittsburgh report.

Traditional bone marrow transplants rely on heavy doses of chemotherapy prior to transplant in order to destroy a recipient's bone marrow so it won't reject the donated marrow. But that makes patients vulnerable to dangerous complications, something that's viewed as an unnecessary risk, because sickle cell disease typically isn't life-threatening, the researchers said.

This new transplant method relies on reduced intensity conditioning (RIC) regimens, which are less toxic to patients and eliminate life-threatening side effects generally associated with bone marrow transplantation. This means transplants can be offered to patients with severe sickle cell disease.

The researchers at Children's Hospital, part of the University of Pittsburgh Medical Center, reported that six of seven sickle cell patients who received RIC bone marrow transplants in the last decade now have donor marrow and are free of sickle cell disease symptoms.

The report was published in the November issue of the journal Biology of Blood and Marrow Transplantation.

"Bone marrow transplant is the only known cure for sickle cell disease. But doctors have avoided performing them in these patients, because complications from a traditional bone marrow transplant can be life-threatening," Dr. Lakshmanan Krishnamurti, a pediatric hematologist/oncologist and director of the Sickle Cell Program at Children's Hospital, said in a hospital news release. He helped develop RIC bone marrow transplants.

"Through the reduced-intensity approach we developed, the potential for complications is dramatically lessened. This study offers hope for a cure for thousands of patients with severe sickle cell disease," Krishnamurti said.

Sickle cell disease, an inherited blood disorder, affects about 80,000 people in the United States, primarily blacks. The disease can cause agonizing pain, strokes, damage to internal organs, and a shortened life expectancy.

More information

The March of Dimes has more about sickle cell disease.

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