Today, June 19 is World Sickle Cell Disease, SCD Day. In many African countries, it is estimated that 10 to 40 per cent (about every 4th person in Ghana) of the population carries the sickle cell gene.
Ghana, through the gallant efforts of its illustrious son, Prof Ohene Frempong (Senior – old student of Prempeh College and) has been screening newborns as part of a comprehensive program to reduce the burden of SCD since 1995. We now know that 2 to 3 out of every 100 babies born in Ghana have SCD. However, we are only able to screen just about 4 per cent of all babies in the country.
The Newborn Screening identifies a person with SCD right from infancy and ensures early initiation of interventions to prevent both short and long-term complications of the disease, usually by including the children in a comprehensive care program in a clinic. The result is not only an increased survival that is, a relatively longer life but a healthier, happier one: improved quality of life.
Many interventions including pneumococcal immunization, folic acid and zinc supplementation, penicillin prophylaxis and early identification of complications and their treatment, have now ensured that many children globally, with SCD who would hitherto have died during infancy, live to adulthood. However, in Africa with more than 75% of disease burden globally, and 1,000 children born with the disease daily, less than 10 per cent of children born with SCD live to adulthood.
Those who survive continue to experience far too frequent hospitalizations for disease complications, recurrent pain crises affecting quality of life and needing frequent blood transfusions.
Now, whereas penicillin prophylaxis prevents certain deadly infections and folate supplementation support the body with a vital ingredient to replace fragile blood cells lost to SCD effects, none of them actually is able to modify the disease to reduce the frequency of hospitalizations due to illness, transfusions, intensity and frequency of pain and acute chest syndrome (SCD starving the lungs of blood and ‘gas’ leading to sudden death in many instances) crises. The only medicine that has been found to do this and approved by the Food and Drugs Authority, FDA-USA, is HYDROXYUREA or simply, HU.
Although, not all patients with Sickle Cell will benefit from HU, the benefits to even young children as well as adults are so overwhelming that experts recommend its use in ALL children with SCD-SS genotype from the young age of 9 months. HU is a very old medicine which was initially used in the treatment of certain cancers but has been found to be SAFE and EFFECTIVE in persons with SCD-SS. It is commonly used by patients with SCD outside Africa.
In Ghana, it is not routinely used. Many of the few parents whose kids I have offered HU and have agreed to start, have attested to a significant difference in the improved quality of life of their kids who now are able to play more; they are hardly hospitalized; rarely have bone pain crises and do not require as many blood transfusions. These translate to improved feeding habits, longer and less interrupted sleep at night, less money spent on hospital bills, lesser school time missed and parents being able to attend to their jobs.
As a clinician I have seen the effects it has on the blood counts with improved hemoglobin, decreased total white cell count (an increased white cell count is associated with higher mortality in SCD) and increased hemoglobin F. However, many colleagues continue to harbor misconceptions and the few that start do not follow protocols that will ensure that patients derive the utmost benefits from the treatment. I hope we (healthcare professionals) can be ambassadors for hydroxyurea therapy for children and adults with SCD and remember:
- HU is safe for children from the age of 9months
- Offer HU to all kids with SCD SS from the age of 9 months
- HU does not increase the risk of infections among children with SCD-SS
- Always regularly (every 2 to 3 months at least) monitor the blood counts of all patients on HU even if they are doing very well
- HU is very effective when administered once every 24 hours (by the clock, at the same time daily)
- Ensure continuity of care; be guided by a protocol
- In the event of an acute event or illness, you do not need to stop HU
- The KATH Pharmacy’s Manufacturing Unit prepares a stable suspension for kids who cannot take capsules
- For growing children always adjust their doses for their weights; hence the need for regular scheduled visits
- For maximum benefit, dose escalate to maximum tolerated dose with monitoring and/or till hematological toxicity (as per blood counts)
- Patients on HU must continue their penicillin prophylaxis and folate (and zinc) supplementation
Today, inform a parent about HU and how s/he can get help for their kids or relatives with SCD (SS genotype) and do it right: by the protocol.
Author: Dr Lawrence Osei-Tutu
Paediatrician (Specialist, Children’s Blood Disorders and Cancers)
Agogo Presbyterian Hospital, Asante Akyem Agogo