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Cincinnati Children's Helping To Eliminate Sickle Cell Symptoms

Jun 25, 2019

Increasingly, sickle cell anemia patients in the U.S. are moving away from blood transfusions and instead using a medicine called Hydroxyurea to control their disease and eliminate the symptoms. Cincinnati Children's Hospital Medical Center researchers are perfecting a technique that determines the correct dosage for each patient in one day.

Doctors say this is important because in some places it can take as long as a year to get the dosage right, and in the meantime, patients suffer. Some kids require double the dose of others.

The process at Cincinnati Children's involves rapidly analyzing a few drops of blood at intervals of 15, 60 and 180 minutes during one day. Researchers track how fast the drug levels rise and fall over time. In May, Cincinnati Children's published results of the method it tested in a clinical trial in The American Journal of Hematology.

There were no sickle cell complications in the 50 patients studied using this rapid testing.

"So instead of starting everybody at the same dose and tweaking the dose over the course of six or 12 months, we start each patient at the right dose," says Dr. Patrick McGann. "And through this study at Children's, we show that the results of this study are significantly better than the standard dose." (The standard dose is by the patient's weight.)

Children's is one of 11 hospitals where a study is underway comparing the standard Hydroxyurea dosing method with weight to Children's rapid testing.

In the future, McGann hopes to find a way to use simpler, more affordable technology in Africa. That's where many children suffer from sickle cell and often die before their fifth birthday because they haven't been diagnosed.

Dr. Luke Smart, a clinical fellow at Cincinnati Children's and a doctor who used to work in Tanzania, wants to increase the use of Hydroxyurea in sub-Saharan Africa. "It's hard to access for this indication. And really, it's not widely available on any drug formulary for sickle cell disease. So that would be the next step in trying to make it available to people.

Smart says even a dollar a day is expensive for this drug because many people live on a dollar a day there.