Stem Cell Therapy for Sickle Cell Anemia in Guatemala

This is an inherited type of anemia whereby the red blood cells are not enough to carry enough oxygen throughout the body.

Normal red blood cells are round and flexible and move easily through the blood vessels. However, in sickle cell anemia, red blood cells become rigid and take on a sickle or crescent shape. Because of the irregular shape they can get stuck in the smaller blood vessels and slow or block oxygen and blood flow.

Sickle cell anemia has no cure but can be managed to relieve pain and complications associated with the condition.

It is caused by the presence of hemoglobin S, an abnormal type of hemoglobin (a protein inside red blood cells which carry oxygen). The hemoglobin S causes the red blood cells to become fragile and change shape into the characteristic sickle or crescent. The abnormal cells are unable to deliver adequate oxygen to the tissues. They can also get stuck and interrupt blood flow.

This disease is inherited from both parents. If you get the gene from just one parent, you will have the sickle cell trait but will not experience the symptoms of sickle cell anemia.

Symptoms begin to occur after the age of 4 months. Almost all sickle cell anemia patients suffer from painful episodes called crises that cause pain in the chest, joints, leg and lower back. The crises can last from a few hours to days.

Severe sickle cell anemia is characterized by common symptoms that include:

• Paleness
• Fatigue
• Rapid heart beat
• Jaundice
• Shortness of breath
• Abdominal pain attacks in younger children
• Symptoms due to blocked blood vessels include:
• Poor vision or blindness
• Pianism (painful and prolonged erection)
• Confusion or problems with thinking
• Ulcers on the lower legs
• Other symptoms include:
• Osteomyelitis (Bone infections)
• Pneumonia (lung infection)
• Cholecystitis (gallbladder infection)
• Urinary tract infection (UTI)
• Delayed [puberty
• Painful joints
• Heart or liver failure

The only known cure for sickle cell disease is through bone marrow or stem cell transplants. However, the transplants are rarely done to the risks involved.

Stem cells are produced in the bone marrow and can transform into various types of blood cells. The stem cells from a healthy donor are given to the patient intravenously and they produce healthy blood cells which replace the sickle cells.

The most common and significant risk is graft versus host disease whereby the transplanted cells begin to attack the other cells in the patient’s body.

The stem cell transplant is usually done after treatment with high doses of chemotherapy. The chemotherapy destroys the patient’s bone marrow and makes room for the growth of new bone marrow. The patient is then infused with healthy bone marrow which contains stem cells that will help in forming new blood cells. The new bone marrow resolves the sickle cell disease symptoms.

The bone marrow transplant procedure in its entirety usually requires a hospital stay of 5-6 weeks. Because the procedure may cause serious complications a comprehensive and thorough medical history is done before a child can undergo stem cell transplant procedure to cure sickle cell disease.