Health experts in Kenya aim to reduce the number of children born with sickle cell disease — an inherited blood disorder — by more than 12 times through public education and awareness, as the country premiered a guideline for controlling and managing the disease.
The guideline titled Sickle Cell Disease Control and Management and launched recently by the ministry of Health is expected to ensure people living with the condition receive quality and affordable care.
The government has pledged to provide basic treatment medication required by patients like folic acid, hydroxyurea, vaccines, and analgesics for pain management.
Andrew Mulwa, acting director of preventive and promotive health in the Health Ministry, said the guidelines mark a new dawn in the management of sickle cell, which in the past was erratic and relied on individual assessments of health workers across the country.
Sickle cell disease is an inherited blood disorder caused by the presence of abnormal haemoglobin in the red blood cells of affected individuals. Haemoglobin is a protein found in red blood cells that carry oxygen in your body and gives blood its red colour.
Healthy red blood cells are round and move through small blood vessels to carry oxygen to all parts of the body. The United States Centres for Disease Control and Prevention (CDC) said in someone who has sickle cell disease, red blood cells become hard and sticky and look like a C-shaped farm tool called a “sickle.”
Normally, red blood cells deliver oxygen throughout the body, thanks to haemoglobin, bending and adjusting to fit into narrow blood vessels, to deliver oxygenated blood to tissues and organs. Rigid sickle cells with abnormal haemoglobin (haemoglobin-HbS), get stuck in the narrow blood vessels preventing oxygenated blood from reaching tissues and organs, causing anaemia that leaves the patient tired and out of breath and sudden attacks of severe pain called sickle cell crises.
Moreover, without adequate oxygenated blood, tissues and organs such as the spleen, liver and kidneys suffer damage over the years, with potentially life-threatening results.
According to experts, lack of early diagnosis prompted by lack of awareness and proper equipment coupled with the high cost of medication used to manage the condition and /or treat complications arising from it has seen many patients, mostly children, and young adults die prematurely. To remedy the situation, the new guideline puts emphasis on early diagnosis and the measures required to reduce the impact of complications and mortality.
“The rationale for this guideline takes cognisant of the challenge patients with sickle cell disease face and the complexities in the provision of healthcare services thus calling for significant health system changes,” said the 50-page document.
Additionally, experts, most of them haematologists (blood specialists) and paediatricians are calling for aggressive public sensitisation to help reduce the numbers from the currently estimated 6,000 newborns (one in every 150 newborns) to less than 500 instead of the projected 10,000 (one in every 100 newborns) annually by 2050.
“Today (July 29), marks a new dawn for us in the fight against sickle cell. We now have guidelines which we hope will see uniformity and standardisation of care across different disciplines,” said Constance Tenge, professor of paediatrics at Moi University School of Medicine.
It is estimated that out of the 240,000 children who are born with sickle cell in Africa annually, 6,000 are in Kenya.
Fredrick Okinyi, chairman of Sickle Cell Federation of Kenya, pushed for the inclusion of drugs such as hydroxyurea, which is on the list of essential medicines under the National Hospital Insurance Fund (NHIF) cover. Dr Okinyi said the drug currently goes for about Ksh50 ($0.46), an amount that is expensive for some patients who require a daily dosage of three drugs.
New drugs meant to reduce the number of painful crises have been developed. In 2017, the US Food and Drug Administration (FDA) approved a drug known as Endari (also called L-glutamine oral powder), to help reduce complications associated with sickle cell disease. The drug is the first treatment approved for patients with sickle cell disease in almost 20 years and is recommended for patients aged five years upwards.
Two years later, the FDA approved a once-a-month drug called Adakveo® (crizanlizumab-tmca) administered as an infusion through the veins to also reduce the frequency of vaso-occlusive crises in adults and paediatric patients, aged 16 years and older, with sickle cell disease. The drug developer, Norvatis, implemented two clinical trials with crizanlizumab in Ghana and Kenya at the International Cancer Institute (ICI) in 2020.
Scientists have been working on a cure for the disease, with reports indicating that two patients appear to have been cured of sickle cell disease after their own genes were edited with CRISPR-Cas9 technology — a unique technology that enables geneticists and medical researchers to edit parts of the genome by removing, adding or altering sections of the DNA sequence. The two researchers who invented this technology received the Nobel Prize in Chemistry in 2020.