What is the best medication for sickle cell

Blood Transfusion

Some sickle cell patients need blood transfusions (simple or exchange) because of severe anemia, before surgery or to help control pain. Emergency blood transfusion is used to treat stroke and acute chest syndrome. Patients who have had a stroke may receive chronic transfusion therapy (blood transfusions every 4 to 6 weeks) to prevent a second stroke and other SCD complications.

Blood transfusions have potential complications including too much iron over time or transfusion reactions like hives and other symptoms of an allergic response. A person’s immune system can also develop antibodies that can make finding matching blood difficult. Blood transfusions also carry a very low risk for infections such as HIV and hepatitis. Any possible complications are checked closely and will be addressed as needed. 

If your provider suggests a blood transfusion, you will need blood drawn. You will also need to sign a consent. In the outpatient setting, a new consent is needed each year. Transfusions are given in the Infusion Clinic on the first floor of the Specialty Clinics building at Froedtert Hospital.

Hydroxyurea to Treat Sickle Cell Disease

Hydroxyurea (Hydrea) is the only approved medication to treat sickle cell disease. The medication works by several mechanisms, but primarily by increasing the fetal hemoglobin in your blood which in turn results in less sickle cell hemoglobin. Studies have shown having fewer sickle cells can help decrease pain, and patients who take hydroxyurea live longer.

• If a woman becomes pregnant while taking hydroxyurea, the clinic staff should be contacted. Hydroxyurea is usually stopped during pregnancy.
• While taking hydroxyurea, patients need to have monthly blood tests to monitor the medication. This medication can change the blood cell values, and the dose may need to be stopped temporarily.

Additional Medications to Treat Sickle Cell Disease Symptoms

The following is a list of common medications that are used to help treat and manage SCD and its symptoms.

• Acetaminophen (Tylenol): For fevers and/or mild pain
• Deferasirox (Exjade): Treatment for iron overload due to blood transfusion
• Deferoxamine (Desferal): If unable to tolerate exjade. This medication is given subcutaneous under the skin over 8 hours, 5-7 days a week.
• Gabapentin (Neurontin): For the nerve pain. Works well for numbness and tingling
• Hydromorphone (Dilaudid): For severe pain
• Ibuprofen (Advil, Motrin): Anti-inflammatory for mild pain and headaches
• Ketorolac (Toradol): For mild pain
• Morphine: For severe pain. Remember, narcotics cause constipation.
• Oxycodone (Percocet/Endocet): For moderate pain
• Polyethylene Glycol (Miralax): To prevent and/or treat constipation
• Senna (Senokot): Stool softener
• Tramadol (Ultram): For mild-moderate pain

Supporting Care

Sickle cell disease can affect many organ systems. Therefore, it can cause a variety of symptoms and complications. Many times, a patient will need different types of therapies to treat these symptoms and complications. Sometimes, a patient will need to work with other medical specialists. 

Hydroxyurea

Hydroxyurea is a once-daily medication which may decrease the chances of acute pain events and hospitalizations and may prevent organ damage in adults and children with sickle cell disease. It also helps decrease the amount of anemia a patient experiences.

Consistent use of hydroxyurea provides a protective effect by increasing the amount of fetal hemoglobin (Hemoglobin F) contained in red blood cells. Cells with more Hemoglobin F are less likely to sickle.

Side effects of hydroxyurea, although rare, may include nausea, gastrointestinal irritation, and headache.  Contact your physician immediately if side effects occur.

Hydroxyurea therapy requires regular laboratory monitoring for problems. Neutropenia (low white blood count), thrombocytopenia (low platelet count), or decreased reticulocyte count (without an associated increase in hemoglobin) may mean the dose is too high. If this happens, the daily dose may be reduced or stopped for a period to allow your blood counts to recover. Once this occurs, medication may be restarted at the same or a lower dose.

EndariTM

What is L-glutamine (EndariTM)?

In July 2017, L-glutamine was approved by the FDA for people with sickle cell disease (SCD) ages 5 years and older.

• L-glutamine is a natural amino acid that our bodies make. It is also found in meat or plants such as red cabbage, beans, and raw spinach.
• The Emmaus Company has studied this treatment in patients with SCD since the late 1990s. L-glutamine is manufactured from the sugar cane plant and is purified to pharmaceutical grade as brand name EndariTM. This makes it different from the L-glutamine capsules that are on the shelves of the supplement stores as these cannot be guaranteed for purity.

How do you take L-glutamine?

L-glutamine is a powder. It must be mixed well in 8 ounces of a cold beverage, yogurt, or applesauce. It is taken twice a day and the number of packets depends on your weight:

• A person less than 66 pounds needs to take 1 packet twice a day.
• A person 66 to 123 pounds needs to take 2 packets twice a day.
• A person over 143 pounds needs to take 3 packets twice a day.

How can L-glutamine benefit sickle cell patients?

A study carried out in 31 centers in the United States led to FDA approval. Patients on the study could continue taking hydroxyurea. (Niihara Y, et al. A Phase 3 Trial of L-Glutamine in Sickle Cell Disease. N Engl J Med 2018; 379:226-235)

• Patients had fewer pain crises in a year (an average of 3 a year for those on the study vs. 4 a year for those not on the study). 
• Patients had fewer episodes of acute chest syndrome.
• Patients taking EndariTM had fewer hospital visits, and their hospital stays lasted a shorter time.
• Patients experienced longer times between their pain crises.

What else should I know about L-glutamine?

• L-glutamine can be used in addition to hydroxyurea.
• The most common side effects reported with L-glutamine use are nausea, constipation and cough.
• L-glutamine should not be used in people who have problems with their liver or kidney function.

How does L-glutamine work?

• The way L-glutamine works is not yet fully understood.
• Sickle red blood cells (RBCs) are destroyed more quickly than healthy RBCs. This destruction happens in part because of natural products called reactive oxygen species.
• The NAD (nicotinamide adenine dinucleotide) system can protect our RBCs against this destruction.
• L-glutamine helps protect RBCs by supporting the NAD system. L-glutamine may also make RBCs less sticky, which could reduce the number of painful crises.  

How can I learn more about L-glutamine?

• Speak with your Hematologist about L-glutamine (EndariTM). Your sickle cell care team will help you decide if you need one or both of the two preventive medications for sickle cell care (hydroxyurea and EndariTM) and if your liver and kidney function are healthy enough for you to take EndariTM.
• Your insurance company will review your records and approve the EndariTM on a case by case basis with the help of your Hematologist.

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