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.

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.)

Blood Transfusions can be part of living with SCDBlood transfusions may be given to reduce the frequency of sickle cell crises. (Source: 123RF)

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 medication 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 a 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.

Other promising treatments for sickle cell disease include:

Gene therapy: While treatments may be far off, researchers are exploring whether inserting a normal gene into the bone marrow of sickle cell anemia patients will result in normal hemoglobin, according to the Mayo Clinic. Scientists are exploring the possibility of turning off the defective gene while reactivating another gene responsible for the production of “fetal hemoglobin,” or a type of hemoglobin found in newborns that prevent sickle cells from forming.

Endari (L-glutamine oral powder): In July 2017, FDA approved Endari for the treatment of pain complications associated with sickle cell disease. Patients who were treated with Endari experienced fewer hospital visits for pain treated with a parenterally administered narcotic or ketorolac (sickle cell crises), on average, compared to patients who received a placebo, fewer hospitalizations for sickle cell pain, and fewer days in the hospital. Patients who received Endari also had fewer occurrences of acute chest syndrome (a life-threatening complication of sickle cell disease) compared with patients who received a placebo.

oral THU-decitabine: This promising medication has shown increased hemoglobin and improved the markers for hemolysis, the rupture or destruction of red blood cells, in an early study by colleagues at the Cleveland Clinic. Further studies are planned for the near future.

Crizanlizumab: In patients with sickle cell disease, the medication crizanlizumab resulted in a significantly lower rate of sickle cell-related pain crises (vaso-occlusive pain) than placebo and was associated with a low incidence of side effects. Michael DeBaun, MD, MPH, professor of pediatrics and medicine and director of the Vanderbilt-Meharry Center for Excellence in SCD at Vanderbilt University Medical Center in Nashville, TN says that it will be “opening the door to alternative strategies for decreasing vaso-occlusive pain events in individuals with SCD,” in a review for the American Society of Hematology. An October 2018 study confirmed these results.

Nitric oxide: People with sickle cell anemia have low levels of nitric oxide, a gas that helps keep blood vessels open and reduces the stickiness of red blood cells, in their blood. Treatment with inhaled nitric oxide might prevent sickle cells from clumping together, but studies have shown little benefit so far, according to the Mayo Clinic.

Medical Marijuana/Cannabis: A small, anonymous survey conducted by researchers at Yale University found that approximately 42% of sickle cell disease (SCD) patients have used marijuana for its medicinal benefits within the past two years. The results encourage a need to study the effects of marijuana on sickle cell disease specifically, as well as considerations into whether sickle cell disease should be included on the list of conditions that can be legally treated with medical marijuana in more states. As of November 2018, only Connecticut, Ohio, and Pennsylvania include SCD as an approved condition for cannabis treatment.

-Additional reporting by Steven Aliano, November 2018

Updated on: 11/01/18
Continue Reading:
Living with Unpredictable Pain: A Sickle Cell Disease Story
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