Tuesday, June 19, 2018

Another sickle cell treatment option now available to Ugandans

By Esther Nakkazi

Today, 19th June is world sickle cell day and from this year hydroxyurea, arguably the most significant breakthrough in sickle cell treatment ever is now on the list of essential drugs in Uganda.

That means it is now more available in drug shops and that brings the price down to a third of the original price, to Ushs 3,000 per tablet from Ushs 10,000 although this does not necessarily mean its affordable and it is not yet available in public health facilities.

As well it does not mean that it will be prescribed to everybody who’s eligible as Ugandan doctors like elsewhere in the world may not necessarily want to prescribe hydroxyurea because it is given in the most maximum tolerated doses and requires continuous blood tests.

Hydroxyurea was approved for sickle cell treatment in 1998 by the FDA and was originally and it still is a cancer drug which increases healthy forms of oxygen-carrying hemoglobin, resulting in less organ damage and fewer pain crises, transfusions, and emergencies for sickle cell patients.

Specialists doctors who are supposed to treat sickle cell disease, hematologists, are hard to come by so most patients are seen by clinicians except for a few dedicated doctors like Prof. Christopher Ndugwa, a paediatrician now known to many as the ‘Uganda grandfather of sickle cell disease’. He has trained about 80 percent of the doctors who treat sickle cell disease in Uganda.

Sickle cell is a multi-organ disease. When patients get an attack they go through a vaso-occlusive crisis in which sickle-shaped red blood cells clog the vessels and cut off oxygen to joints and organs. The inadequate blood supply triggers excruciating pain, damages vital organs and causes a stroke.

Sickle cell disease has been declared a major public health problem for sub-Saharan Africa by the World Health Organization.

The intensity of the disease was unknown until a study was done which prompted action and institution of policy. Now after 20 years since its approval, the now called wonder drug to Ugandans is no longer scarce and here are some efforts that led to policy, treatment changes and action.

In 2014, the Ministry of Health carried out a survey to profile the sickle cell trait and sickle cell disease across Uganda. To date, Uganda is the only African country with current national prevalence data, which was also been published in a leading medical journal, the Lancet.

The survey found scary statistics; at 15,000 to 20,000 babies are born with sickle cell disease every year in Uganda and 80 percent of them die before their 5th birthday. It further documented a high sickle cell burden with a national trait average of 13.3 percent and a disease burden of 0.73 percent.

The research earned Uganda a reward. It was nominated to host the 6th International Symposium on Sickle Cell Disease (REDAC 2016). Mass screening, patient management, early testing, pre-marital counseling and sensitization campaigns were created.

On 16th March 2017, the Minister of Health, Dr. Jane Aceng presented a Ministerial Statement to parliament about the situation of sickle cell in Uganda. The shocked parliamentarians pledged to support it in terms of allocations of funds to the budget, creating awareness and policy.

They requested that equipment be available to screen at birth for sickle cells in all regional hospitals and a budget be allocated so that funding for sickle cell treatment is not left to donors as was the practice.

Aceng informed them that the Ministry of Health had in fact already set up a national programme to screen newborn babies and children below two years in high prevalence districts and a National Sickle Cell reference laboratory with the capacity to run 8,000 samples at ago was operational.

Aceng also appealed to the Buganda kingdom to collaborate with the Ministry and create awareness. The study showed that Buganda was one of the high burden regions with a prevalence of 20 percent and disease burden above 1.5 percent.

The Kingdom of Buganda agreed to provide support to which they accepted to use Sickle Cell Anaemia treatment drives as a theme in the Kabaka Birthday Run for the next three years.

As such the 2017 edition of the Kabaka’s birthday run launched by the Katikkiro of Buganda Charles Peter Mayiga, he equated the lack of awareness to the early HIV days. He said people referred to sicklers as 'offsprings of parents with bad blood or those that are cursed'. That year the funds from the birthday run went to support sickle cell.

More efforts continued like lobbying from civil society organizations like HEPS Uganda and now finally hydroxyurea is on the table in Uganda. However, elsewhere more treatment options are becoming available.

Endari, a nutritional supplement which has been shown to relax the stiff, sickle-shaped red blood cells of people with the disease is now the newest drug on the market. Another treatment option that still needs to go through clinical trials is CRISPR or gene editing therapy. This can be used to edit the sickle mutation in blood stem cells so they produce more fetal hemoglobin, which can reduce the severity of the disease.

It would be interesting to know if Ugandans would participate in the CRISPR sickle cell clinical trials if they got here. But all we know there is hope after all more treatment options are on the way.

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