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Sickle cell disease is the most common inherited blood disorder in the United States and affects millions of people worldwide. New treatments for sickle cell disease are needed to prevent and control serious complications of the disease, the FDA said.1. What is sickle cell disease?
According to the FDA's Centers for Disease Control and Prevention, sickle cell disease affects millions of people worldwide. More than 3 million Americans, including about 231,000 African-Americans, carry sickle cell trait - the gene that causes the disease to be passed from parents to children. A child born with sickle cell disease inherits genes from both parents. Babies born in the United States are usually screened for the inherited sickle cell disorder at birth.People with the disease have “sickle” or abnormally shaped red blood cells, which get stuck in small blood vessels, blocking the flow of blood and oxygen to major organs in the body. These blockages can cause severe pain, organ damage, or even a stroke. Other complications include: Ease of infection, fatigue, and growth retardation.
Sickle cell is a chronic disease of varying degrees of severity. Most people with the disease will have a shortened life expectancy.
Tế bào hình liềm là bệnh mãn tính với nhiều mức độ nghiêm trọng khác nhau
2. Is sickle cell disease curable?
Previously, sickle cell patients had only one treatment option with the drug hydroxyurea. This drug was approved by the FDA in 1998 and is intended for adults only. The drug helps red blood cells keep their normal round shape and flexibility, thereby reducing complications. Common side effects of hydroxyurea include:Low red and white blood cell counts; Gastrointestinal symptoms; Anorexia . In July 2017, the FDA approved Endari (L-glutamine oral powder) to reduce acute complications of sickle cell disease, such as reducing the frequency of sudden and severe attacks of pain. This product is intended for patients 5 years of age and older. This is the first new treatment after nearly 20 years of approval of the old drug. Common Endari side effects include:
Constipation ; Nausea; Headache; Stomachache; Cough; Extremity pain; Backache; Chest pain. In December 2017, the FDA approved hydroxyurea to reduce the frequency of painful attacks and the need for blood transfusions in patients 2 years of age and older. This is the first time the FDA has approved the use of hydroxyurea for children with moderate to severe disease and frequent relapses.
But overall, the treatments for sickle cell disease are still limited. Example:
Bone marrow or stem cell transplant may be an option for young patients with severe sickle cell disease. However, this method has many disadvantages, including expensive procedures, the risk of serious and possibly life-threatening side effects. Transplants also require finding a matching bone marrow or stem cell donor. This is quite difficult so most people cannot apply this measure. Blood transfusions can treat anemia - a common complication of sickle cell disease. Patients will receive regular and long-term blood transfusions to prevent complications. But according to experts, some patients who are treated for sickle cell disease do not have any relief from their symptoms, even the disease continues to progress. Therefore, there is a great need for better therapies.
Truyền máu có thể điều trị thiếu máu - một biến chứng phổ biến của tế bào hình liềm
3. FDA encourages new treatments
3.1. Working with stakeholders FDA continues to work with stakeholders — including patients, academics, and companies developing treatments — to improve treatment options for those who have to live. in common with sickle cells.More and more new products are in the making and researchers are discovering new treatments for sickle cell disease. These potential treatments are in various stages of development, including clinical trials. (Clinical trials are voluntary studies in people, designed to answer specific questions about the safety and effectiveness of potential new treatments, or to study new ways to use them.) use existing treatments.)
Because sickle cell is a global problem, FDA encourages the pharmaceutical industry to develop treatments internationally. Now, companies are conducting clinical trials not only in the United States, but also in Europe and Africa.
3.2. Meet Patients FDA has also met with patients to learn more about their experiences with sickle cells. Understanding the patient's perspective is important in developing new therapies, including:
What symptoms bother them most; What do they want the treatments to focus on? Previously, patients reported that the most important symptoms for them were acute and chronic pain, stroke, fatigue and sleep disturbances. Patients also say they are willing to participate in clinical trials to find new treatments.
FDA Department of Hematology considers the development of new treatments for sickle cell disease a priority. The agency will continue to work to reduce obstacles to new product development, because patients living with the devastating consequences of this chronic condition deserve attention.
FDA cũng đã gặp gỡ các bệnh nhân để tìm hiểu thêm về kinh nghiệm của họ với tế bào hình liềm
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Reference source: fda.gov
