Sickle cell success story: Early detection, specialized treatment make difference for 5-year-old

Niyanna Thompson benefits from Children's study
Posted at 8:47 AM, Sep 27, 2018

CINCINNATI - Decades ago, Niyanna Thompson's sickle cell disease might have defined her. Not so now. 

The active, fun-loving 5-year-old was the first patient in a study at Cincinnati Children's Hospital that makes sure she gets very specific doses of the long-used drug, hydroxyurea. In customizing dosing, the goal is to make the drug extra-effective in fighting sickle cells.

Early intervention with the drug -- which Niyanna got -- can prevent damage to internal organs.

“Sharp, rigid sickle cells get stuck in the blood circulation and don't allow delivery of oxygen to parts of the body that need it the most,” explained Dr. Patrick McGann, a pediatric hematologist at Cincinnati Children's.

But not all of the roughly 100,000 Americans living with sickle cell disease have had that early intervention. Acute anemia and serious infections are hallmark symptoms of Sickle Cell, which impacts one in every 365 African-American births.

Why are we talking about it now? Because September is National Sickle Cell Awareness Month. It's one reason Niyanna's mother has this piece of advice for parents-to-be.

“Get tested,” said Devoshay Herbert. “My mom knew she had the trait. My grandmother knew she had the trait. But I didn't till I got tested to realize I had the trait."

Niyanna's father also has it. The mother/father combo resulted in sickle cell disease for their little girl. But Niyanna is doing well despite the diagnosis because they knew early, and have monitored and managed the disease.

“I don't have any concerns about the Sickle Cell especially with the meds she's on,” Herbert said, “and with her levels always looking good, I don't think I have to worry.”

And the picture could get brighter. Cincinnati Children's has a research study looking at a potentially curative gene therapy.