- 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.