Article taken from The St. Louis American, written by Sandra Jordan
The fact that Gabby Carter of Cape Girardeau, Missouri can go to school and play just about like any other child her age is nothing short of extraordinary.
She received a cord blood stem cell transplant last summer at St. Louis Children’s Hospital. It was her best chance at a cure for the debilitating complications of sickle cell anemia.
With this hereditary blood disorder, normally round red blood cells, that contain the iron-rich hemoglobin protein that carries oxygen throughout the body, are sickle-shaped. Those irregularly shaped cells become stiff and sticky and tend to block blood flow in the vessels, organs and limbs, resulting in oxygen restriction and painful crisis episodes, strokes, organ damage and ultimately, an early end of the lives of its sufferers.
Shalini Shenoy, M.D., director of pediatric stem cell transplantation program at St. Louis Children’s Hospital and professor of pediatrics at Washington University in St. Louis, said all indications pointed to Gabby not having a good outcome if she had not received the life-saving procedure.
“She was on chronic transfusion therapy for multiple complications, the most severe of them was involvement of her central nervous system; she was at risk for future strokes and debilitating disease and early mortality,” Dr. Shenoy said. “And the other organs that she’s had trouble with, as many patients do – the lungs and pain crises. These are not just a risk for death, but a risk for a very poor quality of life as these kids grow older.”
After Gabby’s transplant, periodic blood tests after the transplant showed less and less of Gabby’s own sickled cells as the newly transplanted stem cells made normal cells.
“We access the number of donor cells in her system so we do that from a blood test or we can do that from a sample of the bone marrow,” Shenoy explained. “We periodically do both along the way and Gabby’s testing has come back consistently as having engrafted, which means that the donor cells have settled in and are now making normal donor blood, as opposed to her own.”
Gabby experienced some setbacks in her recovery, including graft-versus- host disease, where the newly implanted cells began to attack her body following her cord blood stem cell bone marrow transplant. During Gabby’s illness and her recovery, her “village” was in action. Clippard Elementary School held prayer vigils, classmates raised gasoline money for her family to get back and forth to St. Louis and her teacher tutored her after school.
Gabby’s mom, Debbie Carter, watched her daughter become reinvented, from an isolated child with frequent bouts of fatigue and debilitating pain to an active 7-year-old.
The stem cell transplant also changed Gabby’s blood type. She now shares the same blood type as her mother.
Although they remain mindful about potential germ exposure, the Carters are readjusting their lives from being in crisis mode at a moment’s notice to just being normal. Carter said the transplant was worth it to see Gabby cured of sickle cell disease and getting her personality back to being “all things sparkly.”
“It’s taken a bit [of time]for her to get that confidence back, but that school – because everybody knows her, everybody gives her that attention, she is slowly coming back to herself,” Carter said.
While Gabby was the beacon of hope in St. Louis during transplant and recovery, students at Gabby’s school began wearing uniforms. Some minor bedazzling (with permission from the principal) brought back the flair for the colorful young fashionista.
“You know, Gabby sparkles every day,” Carter said. “I asked would it be okay. I said I wouldn’t go overboard, but I needed to add ‘her’ back in. It’s been a long time without her being able to be herself.”
“That makes her happy and they are okay with that.”
Sickle cell diseases affect millions of people around the world and it is found most commonly among African, Indian, Mediterranean, Middle-eastern, Caribbean, and Latin populations.
If a child inherits one sickle gene from one parent and a normal gene from the other, the child has the sickle cell trait. If both parents have the sickle cell trait, there is a one in four chance their child will also have sickle cell anemia. In Missouri, all newborn infants receive sickle cell screening.
Dr. Shenoy said parents of children, in particular should contact their child’s hematology teams to keep up advances in current research that could benefit their child because there is no one place to look for the data.
She describes three main areas of ongoing research underway to treat sickle cell anemia. It includes, 1) drug therapy to lessen the severity of pain or prevent it from happening; 2) cell transplantation to cure sickle cell disease, and 3) gene therapy, which involves taking out a patient’s cell and removing the sickled gene and inserting a normal gene to see if it will permanently make normal hemoglobin.
“I think these are all exciting areas of research and I think they are promising and I think what they will serve to do is will improve the course of the disease as we know it now.”
For Gabby and her mother, Debbie Carter, are hoping to participate in this year’s annual Sickle Cell Stroll, which takes place at 9:30 a.m. on Saturday, Sept. 7 at Hope Plaza on the campus of Washington University School of Medicine, just east of St. Louis Children’s Hospital.
For more information on the event, visit www.sicklecellstroll.com.
Click here for success stories from M.A.Z.E. parents who have banked their baby’s cord blood.