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Sickle cell anemia is an inherited genetic disorder affecting the red blood cells. The disease is, in most cases, not curable, and required life-long medical attention. New treatments are being developed to manage the symptoms of the disease.

Existing treatments of sickle cell anemia are usually aimed at avoiding crises and relieving the symptoms of pain as well as preventing any complications. Regular visits to hematologist are required to check the blood count and monitor one’s overall health.

Existing Treatments

1.    Blood and Marrow Stem Cell Transplant: This treatment may cure a small percentage of people. For this procedure to work, the stem cells used for the transplant should closely match those of the affected person and should be from a family member who doesn’t have sickle cell anemia, thus limiting the patients who may find a donor.

The transplant process is risky and may lead to serious side effects or even death. The decision to give this treatment depends upon how severe an individual’s disease is.

Researchers are also looking for sources of bone marrow stem cells like blood from baby’s umbilical cord etc. Though these studies offer promising steps towards treatment of this disease, they may pose the risks in finding a donor as well as body rejecting the transplant, which may result in life threatening complications.

2.      Medications:

  • Antibiotics: Children suffering from sickle cell anemia may be required to take antibiotics like penicillins until the age of five years to prevent infections like pneumonia.
  • Hydroxyurea: This drug stimulates the production of fetal hemoglobin (a type of hemoglobin that prevents the formation of sickle cells and is found in newborns). But there is a concern that if this drug is taken for a long term, it may cause tumors or leukemia.
  • Pain Killers: They are usually prescribed during the periods of crises. 

3.      Vaccinations:

Though vaccinations in childhood are important for preventing infections in all children, they are even more important for children with sickle cell anemia since even the smallest infection can prove to be severe in them.

4.      Blood transfusions:

In blood transfusions, red blood cells from donated blood are given to the affected person intravenously. This increases the normal blood cell count in the body, relieves anemia and decreases the risk of stroke. But this process may carry some risk of increasing the iron levels in the body, which may cause a risk of heart, liver or other organ damage.

So sickle cell patients who undergo regular blood cell transfusions need to take medication to reduce excess iron levels in their body.

Experimental Treatments

1.      Gene Therapy:

Gene therapy is being investigated as a possible treatment for sickle cell anemia by exploring the option of inserting a normal gene into the bone marrow which may cause the body to make normal hemoglobin. Researchers are also studying the possibility of "turning off" the sickle hemoglobin gene or the defective gene and reactivating another gene that makes the red blood cells behave normally.

To date, no human trials have been done to test this approach.

2.      New Drugs:

Several medicines and drugs are being studied for the treatment of this disease. These include:

  • Decitabine: This drug helps the body to make fetal hemoglobin which will further prevent red blood cells from sickling and improves anemia. This medicine can be used instead of hydroxyurea or may be added to hydroxyurea.
  • Adenosine A2a receptor agonists: These medicines reduce complications related to pain in patients already suffering from sickle cell anemia.
  • 5-HMF: It is a natural compound that gets attached to the red blood cells and increases their oxygen absorption, thus preventing them from sickling.
  • Statins: These medications improve the blood flow through the blood vessels.

3.     Nitric oxide: This is a gaseous compound that works as a signal messenger in the body. Nitric oxide reduces the stickiness of the red blood cells and keeps the blood vessels open. People suffering from sickle cell anemia usually have low levels of nitric oxide in their blood. Studies on nitric oxide have shown a mixed result so far but a treatment, if developed, may prevent sickle cells from clumping together.

Sickle cell anemia is an inherited genetic condition and a life-long disease. There is a lot of on-going research on the treatment methods for this disease with the hope that these studies will provide a way to predict the severity of the condition as well as provide a better treatment for the affected patients.

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  • 305(9):893-902.Photo courtesy of euthman via Flickr: www.flickr.com/photos/euthman/5610746554
  • Photo courtesy of Nicolas Alejandro Street Photography via Flickr: www.flickr.com/photos/nalejandro/13200868333

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