Girl, 10, shows no sign of sickle cell after stem cell transplant in St. Louis

After spending three months undergoing and recovering from a risky stem cell transplant, 10-year-old Caitlyn Hill left St. Louis Childrens Hospital last week without any signs of her sickle cell disease.

Caitlyn is only the third person to receive a cord blood transplant from an unrelated donor to treat sickle cell as part of a nationwide study involving Washington University School of Medicine, where researchers are leading the way in finding easier and safer ways to treat non-cancerous diseases with bone marrow and stem cell transplants.

For Caitlyns parents, who for months lived with the fear their young daughter could suffer a stroke, the idea that she could no longer suffer symptoms from the inherited blood disorder hasnt sunk in even after seeing Caitlyns red blood cell counts at levels they had never seen.

Its hard to see beyond to think, Oh my gosh, maybe shell never have sickle cell in her life. We havent totally grasped that, said Lena Hill, 38, of Iowa City, Iowa. We feel really blessed we had Dr. Shenoy to cure our child of the disease.

Dr. Shalini Shenoy is the director of the hospitals Pediatric Stem Cell Transplant Program and a pioneer in developing the protocol for transplanting grafts from unrelated donors without using massive amounts of damaging chemotherapy and radiation opening the door to the rapidly evolving field of using transplants to treat non-cancerous diseases like sickle cell.

Its moving the field forward in a way that has not successfully been done before, Shenoy said.

Bone marrow transplants are typically used as a last-line of defense against cancer, especially leukemia. But in the past 10 years, non-cancerous diseases have gone from making up about 15 percent of all pediatric bone marrow transplants to 35 percent, Shenoy said.

The field emerged in the mid-90s, when the transplants were found to be successful for patients suffering severe sickle cell disease, but only if their tissue matched a sibling donor.

Transplants from unrelated donors were found to be too toxic requiring too much chemotherapy and radiation to wipe out a bodys immune system so it wont reject the donor cells. Efforts to use smaller doses of the chemicals called reduced intensity resulted in patients bodies rejecting the transplants.

Shenoy and researchers at Washington U., however, continued testing the reduced-intensity regimen. Our approach was different, so we persisted, she said.

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Girl, 10, shows no sign of sickle cell after stem cell transplant in St. Louis