• Folic Acid, essential for producing red blood cells since the lifespan for red blood cells is short in sickle cell disease.
• Antibiotics, usually penicillin, are commonly given to infants and young children, as well as adults, to help prevent infections.
• Immunization, such as Haemophilus influenza, pneumococcal, meningococcal, hepatitis B, and influenza.
• Pain relief medication ranging from nonprescription ibuprofen (NSAIDs) and acetaminophen to opiods (prescription medication) are given to control pain.
• Hydroxyurea (Droxia, Hydrea) is prescribed for patients with moderate-to-severe sickle cell disease to help reduce the frequency of pain episodes and acute chest syndrome by stimulating production of HbF. HbF known as fetal hemoglobin is present in fetus and small infants and most of it disappears by early childhood in sickle cell disease. Fetal hemoglobin helps to block the sickling action of red blood cells.
• For severe pain crisis: intravenous hydration, intravenous narcotics, NSIADs, and oxygen are administered at your doctor’s office, in the hospital, or in your home by a home health care agency.
• Endari (amino acid L-glutamine) is indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients 5 years of age and older. Administer Endari orally, twice per day at the dose based on body weight. Mix Endari immediately before ingestion with 8 oz. (240 mL) of cold or room temperature beverage, such as water, milk or apple juice, or 4 oz. to 6 oz. of food such as applesauce or yogurt. Complete dissolution is not required prior to administration.
• ADAKVEO^® (crizanlizumab-tmca) is indicated to reduce the frequency of vaso-occlusive crises (VOCs) in adults and pediatric patients, aged 16 years and older, with sickle cell disease. Crizanlizumab 5 mg/kg is administered by IV infusion over a period of 30 minutes at Weeks 0 and 2, then every 4 weeks
• Oxbryta (voxelotor) works on hemoglobin S to interfere with the first step of the process that causes red blood cells to sickle (polymerization). Because Oxbryta impacts this very first step, it helps to prevent sickling and hemolysis (the breakdown of red blood cells). Hemolysis can lead to anemia (having too few red blood cells). Oxbryta helps hemoglobin do its job, that is, helping red blood cells deliver oxygen throughout the body. OXBRYTA is a prescription medicine used for the treatment of sickle cell disease in adults and children 12 years of age and older.  It is not known if OXBRYTA is safe and effective in children below 12 years of age.
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Normal hemoglobin levels for patients with sickle cell disease are around 7-8 g/dL. Doctors will try to keep the hemoglobin level no higher than 10 g/dL after transfusion. Transfusions may be used either as treatment for specific episodes or as chronic transfusion therapy to prevent life-threatening complications, but carries its own risks.

 

Episodic Transfusions are needed in the following situations:

• To manage sudden severe events, including acute chest syndrome, stroke, widespread infection (septicemia), and multi-organ failure.
• To manage severe anemia, usually caused by splenic sequestration (dangerously enlarged spleen) or aplasia (halting of red blood cell production, most often caused by parvovirus). Transfusions are generally not required for mild or moderate anemia.
• Before major surgeries, but generally not required for minor surgeries.

 

Chronic Transfusions are used for

• Stroke prevention for first or recurrent strokes. Regular blood transfusions every 3-4 weeks can reduce the risk of a stroke in high-risk children.
• Pulmonary hypertension and chronic lung disease
• Heart failure
• Chronic kidney failure and severe anemia
• To reduce episodes of pain and acute chest syndrome

With chronic blood transfusions; iron overload (where the extra iron from the transfused blood cells deposit into the organs and leads to damage), alloimmunization (a condition in which the body’s immune system attacks transfused blood), and exposure to bloodborne pathogens can occur. Still, data from large-scale research trials suggest that the risks of certain situations, including strokes, outweigh the risks associated with transfusions.

 

Kinds of Transfusions

• Simple Transfusion -It involves the infusion of donor blood to restore blood volume levels and oxygen flow. It is used for moderately severe anemia, severe fatigue, and nonemergency situations when there is a need for increased oxygen. It is also used for acute chest syndrome
• Exchange Transfusion –It involves drawing out the patient’s blood while exchanging it with donor red blood cells. It can be done manually or automatically with erythrocytapheresis. Exchange transfusions may be used when there is any evidence that the patient’s condition is deteriorating. It prevents stroke and may be used in patients with severe acute chest syndrome. It reduces the risk of iron overload in patients who require chronic transfusion therapy. Studies suggest that it can improve oxygenation and reduce hemoglobin S levels.

 

Complications of Blood Transfusions

• Iron overload increases risk for damage to the liver, heart, and other organs. Ferritin levels should be monitored. Liver biopsy accurately determines whether excess iron levels are present.
• An immune reaction may occur in response to donor blood. In such cases, the patient develops antibodies that target and destroy the transfused cells. This reaction, which can occur 5 – 20 days after a transfusion, can result in severe anemia, and in some cases it can be life-threatening. It can be generally prevented with careful screening and matching of donor blood groups before the transfusion.
• Hyperviscosity may occur due to a in increased concentration of red blood cells causing the blood to become thick and sticky. The patient is at risk for high blood pressure, strokes, altered mental status, and seizures. Careful monitoring can prevent this condition.

 

Treatment of iron overload

Chelation therapy is used to remove excess iron stores in the body. The drug deferoxamine (Desferal) is commonly used during such therapy. Unfortunately, deferoxamine has some severe side effects and must be used with a intravenous pump for at least 12 hours each day. Many patients do not continue treatment. Deferasirox (Exjade) is another drug used for the treatment of transfusion-related iron overload in patients age 2 and older. It is taken once a day by mouth. Patients mix the pills in liquid and drink the mixture. Newer medications are being developed around the world.

 

JADENU® (deferasirox) tablets for oral use and JADENU® Sprinkle (deferasirox) granules are indicated for the treatment of chronic iron overload due to blood transfusions (transfusional hemosiderosis) in patients 2 years of age and older.

JADENU and JADENU Sprinkle are indicated for the treatment of chronic iron overload in patients 10 years of age and older with non-transfusion-dependent thalassemia (NTDT) syndromes, and with a liver iron concentration (LIC) of at least 5 milligrams of iron per gram of liver dry weight (mg Fe/g dw), and a serum ferritin >300 mcg/L.

 

Bone Marrow or Stem Cell Transplantation

Sickle cell disease can often be treated by transplantation of the hematopoietic (blood-forming) stem cells contained within bone marrow. When successful, the donor’s bone marrow replaces that of the patient’s and can then make normal red blood cells that do not sickle. Bone marrow transplants work best when the patient has a brother or sister who is well-matched for the major tissue markers (HLA antigens match). From several clinical trials reported world-wide, approximately 85-90% of patients with sickle cell receiving a bone marrow transplant from a matched sibling will essentially be cured of their sickle cell disease. However, a small number (5-10%) will reject the transplant and have their own bone marrow return and the sickle cell disease not be eliminated. Unfortunately, some patients (5-10%) may die from the procedure, from either side-effects of the strong chemotherapy drugs given before the transplant to eliminate the patient’s own bone marrow, infections, or a reaction of the donor’s immune cells that are contained within their bone marrow against the tissues of the patient (graft versus host disease). The results can either work well and be an improvement in quality of life for the patient or lead to serious complications and be life-threatening. Thus, the decision to undergo a bone marrow transplant for sickle cell disease is a complex one, involving medical, family, personal, and ethical considerations.

Most patients with sickle cell disease do not have a matched sibling to serve as bone marrow donor. Clinical trials have been performed looking at the potential to use alternative donors, including unrelated adult donors or umbilical cord blood units frozen from infants born. While these unrelated donor transplants can be successful, there are higher risks for complications, especially graft versus host disease, since the patient and the donor may differ for more tissue markers than between family members.

 

Other New Investigational Treatments

Nitric Oxide, Arginine, Gene therapy, Decitabine, Adenosine A2a receptor agonists, 5-HMF

Information from our site is referenced from the following websites:

http://www.nlm.nih.gov/medlineplus/ency/article/000527.htm

http://www.nhlbi.nih.gov/health/dci/Diseases/Sca/SCA_Treatments.html

http://www.marchofdimes.com/baby/birthdefects_sicklecell.html

http://emedicine.medscape.com/article/1014514-overview

 

Disclaimer The Hina Patel Foundation for Sickle Cell Disease website is designed for educational purposes only. The information provided should not be used for diagnosing or treating. Seek medical advice from a physician if you have any health problems.