The 19th June is World Sickle Cell Day and this year, we the Sickle Cell Ambassadors are also educating the general public on Sickle cell disease.
World Sickle Cell Day is a United Nation’s recognised day to raise awareness of sickle cell at a national and international level. On 22nd December 2008, the United Nations General Assembly adopted a resolution that recognises sickle cell disease as a public health problem and “one of the world’s foremost genetic diseases.” The resolution calls for members to raise awareness of sickle cell on June 19th of each year at the national and international levels.
What is Sickle Cell (SCD)?
Sickle cell disorders are a group of illnesses that affect the red blood cells. Sickle cell is a genetic condition, which means it is passed on from your parents and you are born with it; you cannot catch it from other people.
Sickle cell disorders cause your normally round and flexible blood cells to become stiff and sickle shaped, stopping the blood cells, and the oxygen they carry, from being able to move freely around the body and causing pain. This can cause episodes of severe pain. These painful episodes are referred to as sickle cell crisis. These painful episodes are often treated with strong painkillers such as morphine to control the pain.
People with sickle cell are also at risk of complications such as stroke, acute chest syndrome, blindness, bone damage and priapism (a persistent, painful erection of the penis).
Over time people with sickle cell can experience damage to organs such as the liver, kidney, lungs, heart and spleen. Death can also result from complications of the disorder. Management of sickle cell mostly focuses on preventing and managing complications.
The Sickle Cell Trait
Sickle cell trait is inherited when only one of your parents has passed on the sickle gene, and will never develop into sickle cell disorder. You do not have symptoms from sickle cell trait, so it is a good idea to have a blood test to see if you have sickle cell trait. If you have the trait, the majority of red cells in the blood are normal round shaped cells. Some sickle shaped cells may be present under certain conditions.
The trait is not an illness, but if you are planning to have children, then certain factors have to be considered.
If your partner does not have sickle cell trait, then any children you have will not have sickle cell disorder, but they could have the trait (50% chance).
If you and your partner both have the trait, there is a 25% chance that any child conceived may have sickle cell disorder and 50% chance they will have the trait.
The way Forward in Ghana
Novartis Partners with Ghana to Treat SCD, Offering Hydroxyurea and Opening Adakveo Trial.
Ghana is working with Novartis to make hydroxyurea treatment available to people with sickle cell disease, and to open a clinical trial in the African country testing the company’s biologic SCD therapy Adakveo (crizanlizumab), recently approved in United States.
The five-year, public-private partnership was formed in January among the Ministry of Health of Ghana, Ghana Health Service, the Sickle Cell Foundation of Ghana, and the pharmaceutical company.
An estimated 15,000 babies are born with sickle cell disease in Ghana each year, and the country’s leaders are hoping to use a holistic approach to battle SCD, improving disease diagnosis and screening, and patient treatment.
Novartis has already delivered more than 20,000 hydroxyurea treatments being used at 11 centers across Ghana with trained personnel, and is expected to cover patients’ needs for up to a year, officials said in a press release. The company has committed to sending 60,000 additional treatments to be used at others centers the country is opening by the close of 2019.
Ghana is the first African nation to commit to offering the international standard of care to its residents.
“I am proud of this bold partnership, and it is my hope that, through this collaboration, we will help ease the pain and improve the lives of people living with sickle cell disease in our country,” said Alhaji Mahamudu Bawumia, vice president of the Republic of Ghana.“Our collective goal is to reimagine what the future could look like for people with sickle cell disease. Where children do not need to miss out on school or be singled out. Where young adults can have equal opportunity for employment. And where families can flourish and continue to be the bedrock of our civil society,” “In a nutshell, we will make every effort to ‘normalize’ sickle cell disease within the Ghanaian society.”
Commonly used to lower the frequency of pain crises and need for blood transfusions in SCD patients, hydroxyurea was approved last fall by Ghana’s health regulatory agency, granting marketing rights to Novartis.The company has also committed to developing a child-friendly version of this treatment.Hydroxyurea works by increasing levels of a type of hemoglobin called hemoglobin F, or fetal hemoglobin, which helps to prevent anemia and frequent pain crises.
Clinical trials of Adakveo, approved by the the U.S. Food and Drug Administration (FDA) to treat SCD in patients ages 16 and above, are planned to open in Ghana next year, Novartis said in a company press release. A similar trial of crizanlizumab will also open in Kenya.
Adakveo was approved based on results from the yearlong Phase 2 SUSTAIN trial (NCT01895361) in 198 SCD patients with a history of frequent vaso-occlusive crises. Data showed treatment, given each month by intravenous infusion at a 5 mg/kg dose, lowered the annual rate of these pain crises by 45% compared to placebo, and the number of days spent in a hospital each year.
About 80% of SCD patients worldwide are born in sub-Saharan Africa, where it’s reported that more than half die before age 5 due to preventable disease complications.
“Novartis is deeply committed to reimagining medicine for patients in Ghana and across Africa,” said Vas Narasimhan, MD, Novartis CEO. “Building on our long heritage of addressing malaria and leprosy in Africa, we’re excited to help improve the diagnosis and treatment of people with sickle cell here in Ghana.”
Through the partnership, Ghana and Novartis plan to collaborate on field testing and implementing SCD treatment guidelines, and establishing regional centers of excellence. The Sickle Cell Ambassadors will work to get a strong database of sickle cell patients across the country, creating a good link to their medical care givers in the various hospitals. We’ll also help in the implementation of the establishment of sickle cell clinics in at least every district level hospital. We will also work to ensure that newborn screening is in place at those centers, and to use digital technology to monitor and assess patient registration, report real-time data, and to help ensure the safe, widespread roll out of SCD management.
Through this, we will kindly need the help of corporate, public and private companies to assist us in our activities.
Sickle Cell Ambassadors…..Creating Awareness, reducing sickle cell complications.
Sickle cell Ambassadors.