Ugandans living with sickle cell anaemia could soon a get a reprieve after the Ministry of Health approved the use of hydroxyurea as a treatment for the condition and pledged to make it affordable.
Previously, the drug has been formally approved by government for use only in cancer treatment but while launching findings of clinical trials of the drug in Kampala on Wednesday, Health minister Jane Ruth Aceng said government will now make the drug accessible and asked the National Drug Authority to operationalize the process.
A capsule of hydroxyurea costs between Shs1,000 and Shs1,500 per capsule but Dr Aceng pledged to lower the cost within three months.
“Our teams in the Non Communicable Disease department will work on the treatment policy changes and guidelines,” she said.
According to Dr Robert Opoka, who was lead investigator in the trials, hydroxyurea provided immediate relief to patients by reducing frequencies of blood transfusion and chest pains.
“Children receiving hydroxyurea had significantly increased hemoglobin concentration and fetal hemoglobin decreased leukocytes and reticulocytes,” said Dr Opoka, who is also a lecturer at Makerere University.
A total of 213 children participated in the trial that was held between September 2014 and October 2015.
Researchers randomly assigned the children to either once-daily oral hydroxyurea – 1,000 mg scored tablets and 100 mg dispersible tablets – or a placebo for a full year. All patients also received standard care for sickle cell anemia , including folic acid, penicillin prophylaxis and pneumococcal vaccinations — along with insecticide-treated mosquito nets and antimalaria medication.
According to the study, three children assigned hydroxyurea experienced a total of five malaria episodes, and seven children receiving placebo had a total of seven malaria episodes. All 10 study participants with malaria recovered.
Children receiving hydroxyurea also had lower rates of pain crises and hospitalizations.
Fewer children assigned hydroxyurea experienced a composite of sickle cell anemia-related adverse events that included vaso-occlusive painful crisis, dactylitis, acute chest syndrome, splenic sequestration or blood transfusion.
“Compared with placebo, hydroxyurea did not increase the incidence or severity of malaria events in Uganda,” the study reads in part.
A placebo is a substance with no known medical effects, such as sterile water, saline solution, or a sugar pill. A placebo is a fake treatment that in some cases can produce a very real response.
Sickle cell anemia is a life-threatening inherited disorder characterized by abnormal red blood cells that collect in blood vessels and obstruct blood flow to organs, which may result in severe pain, organ failure, stroke or death. More than 300,000 affected babies are born worldwide each year, mostly in sub-Saharan Africa, where an estimated 50% to 90% of children with sickle cell anemia die by the age of 5 years, often without an established diagnosis.