Voxelotor Treatment for Sickle Cell Disease

These instructions are for Children's Hospital of Philadelphia (CHOP) patients with sickle cell disease to learn more about voxelotor.

Important information about voxelotor:

Voxelotor, also known by the brand name OXBRYTA®, is a treatment for sickle cell disease (SCD). It is currently approved for patients 4 years of age and older. Voxelotor is a medicine taken by mouth. You may be able to take this drug even if you are currently taking hydroxyurea. Voxelotor has not been studied in people who are pregnant or breastfeeding. Learn more about voxelotor from the Sickle Cell Disease Coalition.

How voxelotor works:

• Healthy red blood cells contain hemoglobin (Hb), which carries oxygen to all parts of your body.

• Sickle cell disease causes red blood cells to lose their round shape and become sickled, or crescent shaped. This affects how red blood cells carry oxygen.

• Voxelotor decreases the breakdown of red blood cells and makes it easier for hemoglobin to carry oxygen. By increasing the amount of oxygen that hemoglobin carries, voxelotor may prevent red blood cells from sickling.

• Voxelotor helps to reduce the likelihood of long-term complications associated with sickle cell disease, such as organ damage and anemia.

Possible side effects include:

• Diarrhea, nausea and stomach pain – these side effects usually improve or resolve after several weeks. Your team can prescribe medicine to help with these symptoms in the beginning.

• Headaches

• Tiredness

• Rash

• Fever

• Serious allergic reactions such as hives, rash, shortness of breath, swelling of the face can occur with any medicine but are rare with voxelotor.

Patient instructions for voxelotor:

• Voxelotor is available as a standard tablet or a dissolving tablet, depending on your child's age. Talk to your team about which for is best for you.

  • Standard tablets: Do not cut, crush or chew.

  • Dissolving tablets: Pour room temperature clear liquid into a cup, then add tablets into the cup (5 mL of liquid per each tablet). Once tablets start to dissolve, swirl the cup until the tablets break apart, then wait 1-5 minutes before swirling again. Give the dose, then add more liquid to the cup, swirl, and give remaining liquid, repeating until you do not see any parts of the tablet(s) left in the cup.

• The medicine may be taken with or without food.

• If you forget to take a dose of voxelotor, skip that dose and return to your normal dose the next day. Do not take a double dose of this medicine.

• Voxelotor is for long term use. Talk to your healthcare team about how long will be right for you.

• Tell your team about other medicines you take, both prescribed and over the counter.

• Be sure to notify your healthcare team if you have had a previous allergic reaction to any medicines.

General safety tips:

• You and your child should know the names of all the medicines they are taking. Share this information with anyone involved in your child's care.

• Before giving the first dose, read the label. Be sure it is what was prescribed. After a refill, if the medicine looks different to you, ask your pharmacist about it before giving it.

• Always make sure you have enough medicine on hand. Each time you refill your prescription, check to see how many refills are left.

• Always check the label and expiration date before giving any medicine. Do not give expired medicines. Ask your pharmacy what to do with expired or unused medicines. If there is no take-back program, empty them into the trash.

• Store all medicines in their original container away from direct sunlight or heat. Do not store in humid places such as the bathroom. Keep them out of children's reach, locked up if possible.

• If too much or the wrong kind of medicine is taken, call the Poison Control Center at 1-800-222-1222.

Learn more about Oxbryta (voxelator) from the manufacturer, Pfizer.

https://info.chop.edu/3ud1M8G

Please contact your CHOP hematology team with questions or concerns.

Division of Hematology

215-590-3535

Sickle Cell Center

For non-urgent issues, send your team a message in the MyCHOP portal.

Reviewed on June 22, 2023, by Alexandra Kaspin, RN, MSN