Three months after risky stem cell transplant, young patient shows no signs of sickle cell

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

Caitlyn is only the third person to receive a cord blood transplant 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 Caitlyn's 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 hasn't sunk in -- even after seeing Caitlyn's red blood cell counts at levels they had never seen.

"It's hard to see beyond to think, 'Oh my gosh, maybe she'll never have sickle cell in her life. We haven't 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 hospital's 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.

"It's 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 body's immune system so it won't 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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Three months after risky stem cell transplant, young patient shows no signs of sickle cell