They often arrive at emergency departments with severe anaemia and abdominal pain, during particularly painful and life-threatening crisis episodes. Thousands of children suffering from severe forms of sickle cell disease are treated every year in the sub-Saharan African hospitals where Médecins Sans Frontières (MSF) teams work. A handful are currently treated with hydroxyurea, a drug that helps prevent severe attacks. In addition to symptomatic treatment, dedicated, more integrated and preventive care is now being developed in Niger, and should be extended to other MSF projects in the region.
Sickle cell disease is the most common hereditary genetic disease in the world, affecting more than 5 million people. 150 million are carriers of this genetic trait, and according to estimates by the World Health Organisation (WHO), 240,000 children with sickle cell anaemia are born every year in Africa. Most of them die before the age of five from complications linked to the disease, such as serious infections, vaso-occlusive crises or respiratory distress.
However, rapid screening techniques and treatments are available to improve their chances of survival and quality of life.
Bone marrow transplants are the only curative treatment for the disease, but recently, new techniques based on gene therapy have produced promising results, for instance in the United States and the United Kingdom.
In Niger, where almost one person in four carries the sickle cell gene, this is a public health issue that is mobilising the health authorities, including the National Reference Centre for Sickle Cell Disease in Niamey, medical players such as MSF, and civil society associations.
Just over 1,000 children suffering from sickle cell disease are currently being treated by MSF and Ministry of Health staff at Madarounfa district hospital, in the Maradi region in the south of the country. "Sickle cell disease affects haemoglobin, which enables red blood cells to carry oxygen to the organs. Sick children generally suffer from anaemia, dizziness, increased risks of infections and sudden, intense episodes of pain, which can vary in location," explains Dr Moussa Souley Abass, MSF medical coordinator in Niger.
These episodes of pain, known as vaso-occlusive crises, occur when the red blood cells deform into a sickle shape, disrupting blood circulation and oxygenation. In the most serious cases, vital organs such as the heart and brain can be affected.
Basic treatment generally focuses on treating anaemia, with folic acid, and preventing infections with antibiotic treatment if necessary, full vaccination, anti-malaria care and deworming.
“Pain management and early identification of complications are very important," continues the medical coordinator. Blood transfusions and other hospital care are regularly required and unfortunately not always sufficient during the most serious crises.
Over the last twenty years, hydroxyurea has been used in many countries. This drug can prevent severe attacks in the most severely affected people, but it is still not widely available in the region, partly because of its cost, especially for life-long illness, and the tests that must be carried out during treatment.
Since 2022, MSF has been providing it to patients with severe forms of the disease in Madarounfa . Little Jidta, aged 4, has been treated since 2021. “At the time, my daughter was receiving folic acid and penicillin," recalls her mother. Despite this, she fell ill very often, with fever, infections and severe anaemia”. They came to hospital every fortnight. From 2023, she was treated with hydroxyurea. “Two months later, we saw a real change," says her mother. “She spent six months without returning to hospital - it was the first time that had happened to us! Today, she's continuing her treatment, she's playing, she's eating better, she's alive!”
A great deal of progress still needs to be made to improve the availability of diagnostic and treatment resources for children with sickle cell disease. MSF teams are continuing their efforts in this area in Niger, as well as in the Central African Republic, Chad, Uganda and Kenya.
‘Treatment with hydroxyurea is accompanied by counselling and therapeutic education sessions and individualised clinical and biological monitoring to check that the patient's body tolerates it well and to adjust it accordingly’, explains Dr Moussa Souley Abass.
Around 40 patients are currently being treated with hydroxyurea at Madarounfa, with medical check-ups every three months when the situation is stable, or more frequently if necessary. ‘We can see that access to this treatment can make a real difference to patients and their families, in terms of survival, crisis episodes and quality of life,’ concludes Dr Moussa Souley Abass.