Sickle Cell Anemia Has Multiple Treatment Options

Anemia is a condition in which the human body lacks enough healthy red blood cells to carry adequate oxygen to the body and fight infections.

Not receiving adequate oxygen to the body can cause chest pain, cold feet and hands, dizziness, fatigue, headaches, and shortness of breath.¹ Red blood cells in the body contain hemoglobin, which is an iron rich protein that gives blood the red color, and these cells transport oxygen from the lungs to the whole body and carbon dioxide back to the lungs.

There are various types of anemia, with different causes. Some are long-term, and some are short-term, and they can range from mild to severe. These include aplastic, hemolytic, iron deficiency, sickle cell, and vitamin deficiency anemia and thalassemia. The causes include blood loss, the body destroying red blood cells, and genetic disorders.

Risk factors for these patients may include a diet that lacks essential nutrients, intestinal disorders, menstruation, pregnancy, family history and possibly age. Many of these anemias could be prevented simply by replenishing the critical nutrients, utilizing foods rich in iron, folate, vitamin B 12 and vitamin C.

In sickle cell disease, which is a genetic disorder, the red blood cells are shaped like sickles or crescent moons, instead of healthy round red blood cells, which cause them to be rigid, sticky, rigid get stuck in small blood vessels, preventing the flow of oxygen to the body.² Normally, red blood cells die in about 120 days. However, sickle cells die much faster, in about 10 to 20 days, which leaves a shortage of red blood cells in the body, causing the disease. Most patients who carry the disease experience a delay in growth or puberty, deep vein thrombophlebitis and pulmonary embolisms, splenic frequent infections, leg ulcers, pain, sequestration, strokes, leg ulcers, and vision problems.

Like other diseases, sickle cell anemia has its own history. In about 1910, young dental student Walter Clement Noel complained to James Herrick, MD, a cardiologist, about pain episodes and symptoms of anemia. Further evaluation by a resident of Herrick’s led to evaluation of the patient’s red blood cell, which he later described as “having the shape of a sickle.” The role played by deoxygenation was discovered in the 1920s by Hahn and Gillespie. The hereditary nature of the disease was suspected but not demonstrated until 1949 by James V. Neel, MD. The association with hemoglobin was discovered by Linus Pauling and Harvey Itano in 1951 and the actual amino acid substitution by Vernon Ingram in 1956. Thus, the 100th anniversary marks the discovery of this ancient disease from Africa by western medicine and naming of the disease for a simple agricultural implement, sickle.³

Sickle cell disease affects about 100,000 Americans, including about 1 of every 365 African-Americans and about 1 of every 16,300 Latino Americans, and is estimated to cost $1.1 billion in annual health care costs in the United States.⁴

Fortunately, these patients have multiple treatment options. Adakveo, which is given as an infusion, is the first and only FDA-approved once-monthly medication to reduce pain in patients with sickle cell anemia. Other options that ease pain are Endari (L-glutamine oral powder) and hydroxyurea (Droxia, Hydrea, Siklos), as well as OTC medications, such as aspirin and ibuprofen.

One recent novel treatment is Oxbryta (voxelotor). This drug, unlike others, prevents red blood cells from forming the sickle shape and binding together. This process reduces the destruction of some red blood cells, which in turn lowers the risk of anemia and in these patients improves blood flow to the organs.

Oxbryta is recommended to be taken at dosage of 1500 mg once daily, with or without food. If the patient has a history of hepatic impairment, then the dosage recommended is 1000 mg daily. The tablets for this medication come in 500-mg strengths.

This drug was extensively studied in the HOPE trial, a double-blind, multicenter, placebo-controlled randomized trial of 274 patients who receive Oxbryta 1500 mg, 900 mg, or a placebo. In this phase 3 trial, Oxbryta significantly increased hemoglobin levels and reduced the markers of hemolysis.⁵

With the introduction of Oxbryta, sickle cell anemia is again become the focus of research into other drugs, which may indicate a better and brighter future for those who suffer from this disease.

Saro Arakelians, PharmD, is the general manager and pharmacist in charge at BioScrip Infusion in Burbank, California.

REFERENCES

• Mayo Clinic. Anemia. mayoclinic.org/diseases-conditions/anemia/symptoms-causes/syc-20351360. Updated August 16, 2019. Accessed May 11, 2020.
• Mayo Clinic. Sickle cell anemia. mayoclinic.org/diseases-conditions/sickle-cell-anemia/symptoms-causes/syc-20355876. Updated January 30, 2020. Accessed May 11, 2020.
• Winter WP. A brief history of sickle cell disease. sicklecell.howard.edu/ABriefHistoryofSickleCellDisease.htm. Accessed May 11, 2020.
• CDC. Sickle cell disease (SCD). cdc.gov/ncbddd/sicklecell/data.html. Updated October 21, 2019. Accessed May 11, 2020.
• Vichinsky E, Hoppe CC, Ataga KI, et al. A phase 3 randomized trial of voxelotor in sickle cell disease. N Engl J Med. 2019;381(6):509-519. doi: 10.1056/NEJMoa1903212.