Three studies in four nations seek to prove value of effective, inexpensive treatments

Here in the US, most children born with sickle cell disease can expect to live at least into their 40s or 50s. But in Africa, the vast majority of children born with this disease die before they reach age 5.

It doesn’t have to be that way, says Russell Ware, MD, PhD, the new Director of Hematology at Cincinnati Children’s and Executive Co-Director of the Cancer and Blood Diseases Institute. Simply expanding the use of simple blood tests and inexpensive treatments could give hundreds of thousands of African children a chance to grow up.

“If you look at all of the births of children with sickle cell, less than 1 percent are in the United States. More than 75 percent of the children with sickle cell are born in Africa. So if we really want to make an impact on this disease, we need to go outside the United States.”

Ware joined Cincinnati Children’s in July 2013, bringing with him years of experience working with sickle cell and other blood diseases. Two years ago, he helped launch a pilot screening and treatment program in Angola that has shown promising results. Here, he expects to accelerate that effort with three new projects to begin in 2014.

Uganda Sickle Surveillance Study (US3): Using funds from our Cancer and Blood Diseases Institute, Ware will launch an 18-month mapping study with the Ugandan Ministry of Health. The project will analyze blood sample cards collected from babies born to HIV-infected mothers to identify the burden and distribution of sickle cell throughout Uganda to help launch sickle cell screening programs.

Novel use Of Hydroxyurea in an African Region with Malaria (NOHARM): Ware is co-principal investigator for a two-year, placebo-controlled clinical trial in Uganda to evaluate whether hydroxyurea treatment for sickle cell disease makes children more susceptible to malaria. Up to 200 children will be involved. Ware is working with Chandy John, MD, MS, a malaria expert at the University of Minnesota, and a team in Uganda on this Doris Duke Charitable Foundation-funded project.

Realizing Effectiveness Across Continents with Hydroxyurea (REACH): This Phase I/II clinical trial will involve up to 450 children in Angola, Kenya and the Democratic Republic of Congo to determine safe and effective dose levels of hydroxyurea in regions with high rates of malaria and malnourishment.  Ware is working with colleagues at The Hospital for Sick Children in Toronto on this study.

The potential to make a difference against sickle cell in Africa is large because much of the medical care for the disease is not complicated or expensive, Ware says. If diagnosed early in life, babies can be placed on life-saving penicillin and receive all childhood immunizations. As they grow, many could be treated with hydroxyurea, a well-established medicine in the US, but rarely used in Africa.

”It is taken once a day by mouth and it costs about $1 a day,” Ware says. “Once we get the right dosing and show that it’s safe, I think it will catch on and save many, many lives.”