Treating Sickle Cell Disease

Currently, the only cure for sickle cell disease (SCD) is a bone marrow transplant but the procedure is risky and rarely done. Preventing complications is one of the best ways to manage this chronic and unpredictable condition.

Blood Transfusions can be part of living with SCDBlood transfusions may be given to reduce the frequency of sickle cell crises.

Preventing Complications

The goal of managing sickle cell disease (SCD) is to prevent complications by reducing the incidence of sickle cell crises. By reducing the number of sickle cell events, the patient can minimize the potential long term effects of the disease on the body. Managing SCD is a lifelong effort that begins soon after a child is diagnosed. To help reduce the incidence of sickle cell events, physicians often recommend:

Antibiotics: Infants with sickle cell anemia (SCA) may be started on penicillin twice a day beginning at 2 to 3 months and continuing to age 5. Penicillin may be continued after age 5 if the child's spleen has been removed or he or she has a history of sepsis (an overwhelming immune response to infection). 

Folic acid:  Bone marrow needs folic acid to make new red blood cells.

Immunizations:  Along with all the recommended childhood vaccination, children age 2 to 5 should receive the pneumococcal pneumonia vaccine. Children and adults should have vaccinations for meningitis and hepatitis and annual flu vaccinations. 

Hydroxyurea (Hydrea): The only FDA-approved drug for the treatment of SCA in adults (used off-label in children) works by decreasing the number of sickled blood cells and reducing the frequency of sickle cell crises. It is prescribed starting at 9 months and continued throughout adolescence and adulthood.

Blood transfusions: Transfusions may be given to reduce the frequency of sickle cell crises and, in some children and individuals, to lower the risk of stroke and other complications.  (Transcranial Doppler, a test that measures the velocity of blood flow through the brain's blood vessels by means of ultrasound, identifies children at high risk of stroke.)

Treatment for Sickle Cell Crisis

Whether or not a sickle cell crisis requires hospitalization depends on the severity of the pain and the presence of a fever. If there’s no fever and the pain is manageable with ibuprofen (Motrin, Advil, etc) or a prescription pain medicine such as oxycodone/acetaminophen (Percocet), staying well-hydrated and resting may stop the crisis from escalating. However, if the pain increases, or if a fever spikes over 100.4 degrees, a trip to the emergency room may be necessary.

If admitted, treatment may include:

  • Oxygen
  • Intravenous (IV) rehydration to “re-plump”  the sickle blood cells
  • IV morphine for pain
  • IV antibiotics for infection with fever in addition to daily regimen
  • Blood transfusion. Repeated transfusions–20 or more—can sometimes lead to iron overload. If this occurs, you may need chelation therapy, a treatment that helps the body get rid of excess iron.

Prognosis

SCD is a serious condition that requires lifelong management and treatment to prevent and minimize the complications that, over time, may cause irreparable damage to the body. With proper medical care, the average life expectancy is around 50 years with some people living longer, even into their 70s.

At present, the only cure for SCD is a bone marrow transplant, which is also known as a stem cell transplant. Preparation for the procedure involves chemotherapy to destroy the patient’s own bone marrow so that the patient’s body “welcomes” rather than fights the healthy donor marrow. The actual transplant is given through an IV, much like a blood transfusion.   

However, because compatible donors can be scarce and the procedure carries considerable risks, including graft vs. host disease (a serious, life-threatening complication that occurs when the transplanted cells attack cells in the recipient’s body), stem cell transplants are not currently widely done or available. When performed, these transplants are most often done in children with severe symptoms. 

However, a recent clinical trial of stem cell transplantation in adults with SCD, conducted by researchers from NIH’s National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) and National Heart, Lung, and Blood Institute (NHLBI) and published online in the Journal of the American Medical Association, showed some promising results. In the study, 30 patients, age 16 to 65, with severe SCD underwent a stem cell transplant, receiving cells donated by a healthy brother or sister.

The team found that the stem cell transplant reversed the disease in 26 of 30 patients (87%). The patients had normal hemoglobin, fewer hospitalizations, and lower use of opioids to treat pain from the disease. The patients didn’t experience graft-versus-host disease—in which donor cells attack the recipient—after a median follow up of 3.4 years. Fifteen patients successfully stopped immunosuppression medications a year after the transplant. The treatment was unsuccessful in 4 patients, and some complications, such as infections, occurred.

The researchers continue to follow the study subjects.  To find out about other studies go to https://clinicaltrials.gov

 

Updated on: 12/06/16
Continue Reading:
Living with Unpredictable Pain: A Sickle Cell Disease Story
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