It is a simple but harsh reality that children with sickle cell disease living in Haiti will have more pain, worse quality of life, and are less likely to survive childhood because they don’t have access to the treatments routinely provided to children with sickle cell disease in the United States and other developed nations.
Two doctors from Akron Children’s are hoping to change that by bringing a drug that costs less than $1 a day to these children.
Dr. Prasad Bodas, a pediatric hematologist-oncologist, and Dr. Nicholas McGregor, a hematology-oncology fellow in Akron Children’s Showers Family Center for Childhood Cancer and Blood Disorders, are working with Dr. Emmeline Lerebours at St. Damien Hospital, outside of Port-au-Prince, on a clinical trial to see if the drug hydroxyurea can have the same outcomes in Haiti as it has in the U.S.
“It’s not a question of efficacy – we know hydroxyurea works – we just need to learn that it can be an acceptable and feasible treatment for a population that does not have the same access to health care because of distance, a lack of transportation, cost or other factors,” said Dr. McGregor.
The study, which now has approximately 30 children enrolled, could potentially have a huge impact on the lives of thousands – if not hundreds of thousands – of Haitians in the years to come.
“In Haiti, investigators estimate that about 1 out of every 180 children born have sickle cell disease, which is more than twice the incidence among African-Americans,” said Dr. McGregor.
Sickle cell disease is an inherited blood disorder where red blood cells contort into a sickle or crescent shape. The cells die early, leaving a shortage of healthy red blood cells and blocking blood flow, which causes pain often known as a “sickle cell crisis.”
Hydroxyurea helps boost the levels of fetal hemoglobin, which, in turn, lowers the concentration of abnormal hemoglobin S molecules that cause the red blood cells to distort into the sickle shape.
Diagnosing and treating sickle cell disease is vastly different for the average child in Haiti compared to the United States.
In the United States, parents typically learn shortly after birth that their baby has sickle cell disease through newborn screening. They are started on a regimen of penicillin to lower the risk of infection and oftentimes, within the first year, begin to take hydroxyurea.
In Haiti, without newborn screening, children are typically diagnosed with sickle cell when they become sick enough to seek care at the hospital.
In the 1960s the average life expectancy for those with sickle cell disease in the United States was under 15 years. Advances in treatment, including care provided in comprehensive sickle cell disease programs like the one at Akron Children’s, have increased life expectancy today to the mid-50s. Unfortunately, children in Haiti do not have access to many of these advances. Hydroxyurea is one of the more recent and most successful improvements in the care of sickle cell disease, and the low cost and ease of use make it ideal for low income settings such as Haiti.
With this study, said Dr. Bodas, we hope to demonstrate that Haitian parents will accept the drug, which can be taken as a pill or liquid, and will bring their children for treatment, and that clinicians will see the same benefits of hydroxyurea in Haiti that we see in the United States.
“Ultimately,” said Dr. Bodas, “our goal is to demonstrate feasibility of hydroxyurea in Haiti, so that the national government and other organizations can adopt the drug as standard care for sickle cell disease.”
Drs. Bodas and McGregor began the study in July 2014, following a trip by Dr. Bodas to St. Damien the month before with Dr. Jeff Kempf, director of Global Health for Akron Children’s. Dr. Kempf led the effort to forge an official affiliation between Akron Children’s and St. Damien, and other initiatives between the 2 hospitals have focused on pediatric cardiology, emergency medicine and critical care.
The physicians were awarded a grant of $6,000 from the American Academy of Pediatrics for the sickle cell study. Other funding for the 3-year study is coming from the Global Health Department, the Showers Center, and a grant request has also been submitted to the hospital’s Foundation.
Pediatric emergency medicine fellow, Dr. Danielle Paulin, will present initial data from her efforts to describe sickle cell disease patients in Haiti at the American Society of Pediatric Hematology and Oncology meeting in May, and Drs. Bodas and McGregor plan to present further updates about the hydroxyurea study in November at the American Society of Hematology Annual Meeting.
Dr. McGregor, who just returned from a trip to Haiti last week, said his involvement with the study has become a deeper confirmation of his career choice.
“I chose pediatrics as my focus in medicine because I have the opportunity to help some of the most vulnerable among us – our children,” he said. “Because of their severe poverty, the children with sickle cell disease in Haiti are as vulnerable as anywhere in the world – and yet by addressing this one gap in their care, we think we can make a difference in their everyday life.”