Endari (L-glutamine oral powder) is the first treatment for sickle cell disease to be approved by the Food and Drug Administration in nearly 20 years. It is approved for those ages five years and older to reduce the severe complications of sickle cell disease. Endari, an amino acid, is given orally twice a day. The dose varies by body weight.

Sickle cell disease is a group of genetic variations that cause red blood cells to be misshapen, hard, and sticky. Instead of their normal round shape, the red blood cells of people with sickle cell disease resemble that of a crescent (or sickle). These sickled cells die sooner than normal red blood cells, leading to low levels of circulating red blood cells and anemia. Because of their stickiness, these cells can block blood flow in the circulation and cause ischemia and pain.

Sickle cell disease is most common among those whose ancestors are from sub-Saharan Africa, Spanish-speaking regions in the Western Hemisphere (including South America, the Caribbean, and Central America), Saudi Arabia, India, and Mediterranean countries (Turkey, Greece, and Italy). The exact number of Americans with sickle cell disease is unknown, but the Centers for Disease Control and Prevention estimates that 100,000 are affected by the disease, which occurs in about one out of every 365 African American births and one out of every 16,300 Hispanic American births.

Exactly how Endari achieves its therapeutic effect is not well understood. It is believed to play a role in regulating and preventing oxidative damage to red blood cells. In randomized, double-blind, placebo-controlled clinical trials of subjects ages five to 58 years, those who received Endari over 48 weeks had fewer sickle cell crisis events than those receiving placebo. This was evidenced by fewer hospital visits requiring parenteral narcotics or ketorolac (a nonsteroidal antiinflammatory drug) for pain, fewer hospitalizations for pain, fewer days in the hospital, and fewer occurrences of acute chest syndrome, which can be life threatening. The average cumulative crisis count was reduced by 25% in those receiving Endari, compared with placebo. Adverse effects of Endari noted by more than 10% of patients include constipation, nausea, headache, abdominal pain, cough, pain in the extremities, back pain, and chest pain.

Nurses should teach patients and their families how to mix the drug for administration. The dose should be mixed in either 8 oz. (240 mL) of cold or room temperature liquid (milk, water, or apple juice) or 4 to 6 oz. (0.11 kg to 0.17 kg) of food (applesauce or yogurt). The drug does not need to be completely dissolved, but it must be administered immediately after mixing. For complete prescribing information, go to http://www.accessdata.fda.gov/drugsatfda_docs/label/2017/208587s000lbl.pdf.