• Sickle cell disease(SCD) is a group of inherited blood disorders where red blood cells become stiff, sticky, and C-shaped (like a farm tool called a sickle) instead of round and flexible.
• These misshapen cells die prematurely and can block small blood vessels, leading to chronic anemia, severe pain episodes (pain crises or vaso-occlusive crises), and serious organ damage. 

• Inheritance: A child is born with SCD only if they inherit two copies of the abnormal gene, one from each parent.
• Sickle Cell Trait: People who inherit only one copy of the gene have "sickle cell trait" and are typically carriers who do not experience significant symptoms but can pass the gene to their children.
• High-Risk Groups: The disease is more common in people whose ancestors come from sub-Saharan Africa, the Mediterranean, the Middle East, India, and South and Central America. The high prevalence in these areas is due to the fact that the sickle cell trait offers a degree of protection against severe malaria. 

• Anemia: The rapid breakdown of sickle cells (which last only 10-20 days instead of 90-120 days) leads to a chronic shortage of red blood cells, causing fatigue, paleness, and shortness of breath.
• Pain Crises: Blocked blood flow causes sudden, severe pain in various body parts, such as the chest, back, and limbs. These episodes can last for hours to days and often require a hospital stay.
• Infections: Damage to the spleen increases the risk of serious bacterial infections, particularly in young children.
• Acute Chest Syndrome: A potentially life-threatening condition caused by a blockage in the lungs, resulting in chest pain, fever, and breathing difficulties.
• Stroke: Blocked blood vessels in the brain can lead to stroke, which is a major concern, especially in children.
• Organ Damage: Chronic lack of oxygen can damage nerves and organs over time, including the kidneys, liver, and heart. 

• Medications:
  • Hydroxyurea is a common medication that increases the production of fetal hemoglobin (HbF), which prevents cells from sickling.
  • Other newer drugs like voxelotor and crizanlizumab help red blood cells flow better and reduce pain crises.
  • Pain relievers and daily antibiotics (especially for young children) are also key components of management.

• Supportive Care: Staying hydrated, avoiding extreme temperatures and high altitudes, and getting regular vaccinations can help prevent pain crises and infections.
• Blood Transfusions: Used to treat severe anemia, prevent stroke, or manage other acute complications.
• Curative Options: Bone marrow or stem cell transplants can offer a cure for some patients, though they are complex and require a well-matched donor. Recently approved gene therapies also offer a potential cure for eligible patients. 

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