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200 Ugandan patients for sickle cell drug trial

By Vision Reporter

Added 16th September 2014 12:56 PM

At least 200 patients are to take part in a clinical trial for a more effective drug used to treat sickle cell anaemia.

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By Taddeo Bwambale

At least 200 patients are to take part in a clinical trial for a more effective drug used to treat sickle cell anaemia.
The drug known as Hydroxyuria has been widely used in the US and Europe to manage acute pain and reduce the need for blood transfusion in children with sickle cell disease.
Sickle cell disease is an inherited blood disorder in which the body creates sickle-shaped red blood cells, affecting blood flow in cells and organs.
It is acquired when both parents have the sickle cell trait gene.  Sickle cell patients present with anaemia, acute pain, body swellings and persistent infections.
Clinical trials for the drug are expected to start in October at Mulago Hospital. Details of the clinical trial seen by New Vision show that only children aged between 12 and 47 months are eligible to take part in the one-year clinical trial.
Dr Phillip Kasirye, the head of the Sickle Cell Clinic at Mulago Hospital told New Vision that at least 300 patients would be screened to select patients for the in clinical trials.
“We have completed the approval process with the National Council for Science and Technology and are scheduled to start the trial,” Kasirye told New Vision in an interview on Monday.
It is the first study on the safety and efficacy of the drug in malaria endemic regions of sub-Saharan Africa which has the highest share of sickle cell patients globally.
The trial will be conducted by a team of health experts from Makerere University, Mulago Hospital, Cincinnati Children's Hospital Medical Center, University of Minnesota-Clinical and Translational Science Institute and Doris Duke Charitable Foundation.
Kasirye, who is also a member of the research team, said the clinical trial would determine the effectiveness of the drug where patients have Malaria and its possible side effects.
If the drug is proved to be effective, health experts will recommend its inclusion on Uganda’s list of essential drugs, thereby increasing its access to sickle cell patients.
The drug is available in a few pharmacies and costs about sh2,500 per pill taken daily for life.
Its inclusion on the list of essential drugs is expected to help lower the cost and increase its reach to more patients.
Rising burden
In Uganda, over 25,000 babies are born with sickle cell disease every year and majority of them die before the age of five. 
According to Kasirye, the sickle cell clinic at Mulago receives at least 50 new patients every month, pointing to a rising trend. The clinic alone has over 10,600 registered patients.
The same research team is conducting the Uganda Sickle Cell surveillance study (US3), the first countrywide survey on the prevalence of sickle cell disease in Uganda.
Normal red blood cells (L) and sickle red blood cells. (Wellcome Trust, UK)
Partial findings from the study released in June show that 12% of Ugandans (one out of every ten Ugandans) carries a gene that causes sickle cell disease.
It shows that Northern Uganda has the highest prevalence of the sickle cell trait at 18.2%, followed by the Eastern region at 14.6%, Central region (12%) and Western region (4.1%).
The findings are based on analysis of blood samples from 23,000 new born babies across the country tested for the sickle cell gene at the Central Public Health Laboratories in Kampala.
Prof Christopher Ndugwe, a consultant on sickle cell treatment and member of the team conducting the clinical trial said the drug was very effective in managing sickle cell anaemia.
“It helps to reduce the rate at which red blood cells form the sickle shape and complications that cause acute pain and infection,” Ndugwe explained.
There is no traditional cure for sickle cell disease and advanced treatment options are very expensive. Bone marrow transplant and stem cell procedures cost up to $400,000 (Sh1b).
Currently, standard treatment accorded to sickle cell patients includes treating infections, giving preventive malaria treatment and folic acid to boost red blood cells, with regular check-ups.
To prevent the spread of sickle cell disease, health experts strongly advise couples to test for the sickle cell trait before marriage before deciding to have children.
Regional problem
In Kenya and Tanzania, sickle cell disease has been prevalent around the coastal towns, Ndugwe said, although he said there were no recent studies on the cases.
Globally, over 300,000 babies are born with blood disorders including sickle cell anaemia and majority of the cases are in Africa, according to the World Health Organisation (WHO).
About 5% of the world’s population has the sickle cell gene, and studies have linked the gene to Malaria-endemic regions.
The WHO country profile on sickle cell diseases shows that the prevalence rate in some parts of Uganda is as high as 45%.
In Cameroon, Democratic Republic of Congo, Gabon, Ghana and Nigeria, prevalence ranges between 20% and 30% while in North Africa and Southern Africa has between 1% and 2%. 


200 Ugandan patients for sickle cell drug trial

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