In the past ten years, there have been significant developments in the management of sickle cell disease. Several more therapeutic drugs have been licensed by the FDA as therapies for sickle cell anemia since research (BABYHUG) suggested hydroxyurea therapy for young kids with the condition.
Treatment for this illness is also available thanks to advancements in stem cell transplantation, and substantial developments in gene therapy promise even more restorative possibilities. Accessibility issues and toxicity reduction remain issues with these therapeutic methods, nevertheless. Let’s examine the state of sickle cell disease treatment today.
The Pain Crisis in the Disease
The characteristic sign of sickle cell disorder is pain crises. They result from sickle-shaped blood cells blocking arteries, which reduces blood flow and oxygenates tissue. This results in severe pain that is incapacitating and can linger for hours or even weeks. In addition to blocking the arteries that provide blood to the organs, sickled cells frequently lead to organ damage, which leads to other long-term issues.
The kidney, heart, lung, bone, and brain all frequently show the first indications of organ damage in young adults and teenagers with sickle cell disease. Inevitably, as people with sickle cell disease age, organ damage commonly worsens, and organ damage’s start, accumulation, and advancement are all strongly related to early mortality.
The Changing Treatment for the Disease
A breakthrough in lowering the incidence of pain crises was hydroxyurea. However, the early use of the medication probably also slows and, in some circumstances, stops organ damage. By boosting the amount of fetal hemoglobin within red blood cells, prevents blood sickling and decreases white blood cells to lessen inflammation.
The sickled cells are treated in several ways by recent medicines. The medications, such as crizanlizumab, glutamine, and voxelotor, are also demonstrating their value. Glutamate and crizanlizumab lessen the number of pain episodes, while voxelotor enhances anemia. In addition, if a patient receives combination therapy, it seems as though these advantages are in addition to the advantages of hydroxyurea therapy.
The sickled cells in the blood absorb L-glutamine, which causes antioxidants to be released, reducing the blood cells’ degree of stress. A molecule found on blood platelets and another on the inner walls of blood vessels are the targets of crizanlizumab’s binding activity. A recognized driver for the sickling process is the molecular structure of P-selectin, and crizanlizumab prevents P-selectin from functioning by blocking the cellular connections that give rise to it. Voxelotor aids hemoglobin in retaining more oxygen and prevents sickling by allowing blood cells to maintain their regular shape.
Even while hydroxyurea has a significant role in lowering pain episodes as well as mortality, it is insufficient on its own. Hydroxyurea treatment for sickle cell disease does not eliminate problems, and some patients even have organ damage. Fortunately, recently authorized treatments outperform hydroxyurea in terms of advantages and do not have any negative effects. We are now looking at combo therapy. The objective is to more accurately pinpoint, for each patient, the most potent combination therapy that will both lessen his or her pain and, more crucially, safeguard his or her organ function.