Approved Treatments for Sickle Cell Disease


Adakveo (crizanluzumab) is administered as an injection into the bloodstream to treat painful vaso-occlusive crisis events caused by sickle cell disease. It contains a monoclonal antibody that blocks the protein in the blood vessels that binds to sickle cells, causing pain and inflammation when the sickle cells block blood flow. By blocking this P-selectin protein, Adakveo prevents this adhesion molecule from starting the process that leads to blood vessel occlusion, inflammation, and pain, and helps to maintain normal blood flow.

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Endari (L-glutamine) is an oral therapy that works to increase the amount of free glutamine circulating in the blood. Sickle cells can take up this free glutamine and use it to generate antioxidant molecules. These new antioxidants can help neutralize the oxidative stress in sickle cells, which can allow them to regain the flexibility necessary to travel through blood vessels and capillaries, carrying oxygen to tissues throughout the body.

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Hydroxyurea is a medicine used to reduce the frequency of pain crises and the need for blood transfusions in patients with sickle cell disease. Hydroxyurea works by increasing the levels of hemoglobin F (HbF), found at high levels in early stages of development. Compared with the adult form of hemoglobin, HbF is more effective in binding and carrying oxygen around the body. The increase of HbF production stimulated by hydroxyurea prevents the symptoms of anemia from developing.

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Ketamine is a medication used for anesthesia, generally for surgery or other medical procedures. It also can be used to treat acute pain in sickle cell disease patients. The precise mechanism of action of ketamine is not known, but it is thought to cause anesthesia (a treatment that prevents patients from feeling pain) primarily through the blockage of the N-methyl-D-aspartate receptors, which are normally involved in the transmission of nerve signals, learning, and memory.

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Common pain medications for sickle cell disease include nonsteroidal anti-inflammatory drugs, or NSAIDs, which are therapies that reduce pain, fever, and inflammation. Some are available only by prescription, but many are over-the-counter medications. They work by inhibiting an enzyme called cyclooxygenase (COX), which has two forms: COX-1 and COX-2. NSAIDs fall into two categories: those that affect both COX-1 and COX-2 and those that primarily affect COX-2. Patients should work with their healthcare provider to determine which NSAID to use.

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Oxbryta is an oral therapy that targets the abnormal hemoglobin that causes sickle cell disease. It prevents hemoglobin from sticking together, which destabilizes the large aggregates, or clumps of sickled cells that block blood vessels and prevent blood flow. This should restore normal red blood cell function and oxygen delivery, reducing disease caused by the loss of red blood cells. It should also help lower the risk of vaso-occlusive pain crises caused by sickle cells blocking small blood vessels.

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Opioid Narcotics

Moderate and severe pain in sickle cell disease can often be managed with opioid narcotics, or opioids. Opioids are substances that relieve and manage many different kinds of pain. They act by binding to opioid receptors that are located on the surface of nerve cells and inhibiting the transmission of pain signals. In patients with sickle cell disease, opioids can be used to treat both acute and chronic pain.

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