Last week, I started a column on the new medicine called crizanlizumab that is on the market for people living with sickle cell in Europe and the US for now. I mentioned that there are other alternatives available too, for example, L-Glutamine Vexelotor and Hydroxyurea. Let’s remind ourselves of these other tablets and co that are in use and available. Below is the continuation from last week and conclusion.
How is and how does L-Glutamine work?
L-glutamine (pronounced “L-gloo-ta-meen,”), or ENDARI® is a treatment for sickle cell disease that may help patients age five years and older (If you don’t know the type of SCD you or the person receiving care has, ask your doctor). Share the information in this fact sheet with your health care team to decide whether this drug might be a good choice for you or the person receiving care. You may be able to take this drug even if you are currently taking hydroxyurea but consult with your health care team. L-glutamine has not been studied in women who are pregnant or breastfeeding.
L-glutamine is an amino acid. The body naturally produces amino acids to help prevent and fight against infection, injury, and stress. However, people with SCD might need additional L-glutamine because they experience more pain and infection than what the body can handle on its own.
For individuals living with SCD, red blood cells are distressed, which makes them stiff, sticky, and sickle-shaped. This causes damage that blocks blood flow in a process called vaso-occlusion, which can cause pain crises. L-glutamine helps stop or slow down this damage and works to lower the number of pain crises.
L-glutamine is taken in powder form and is intended for long-term use.
Each dose normally is mixed in eight ounces of cold or room temperature beverage or four to six ounces of food.
In a study looking at the effectiveness of L-glutamine, researchers found that participants assigned to take L-glutamine had fewer pain crises than those on the placebo (a harmless substance that looks like the treatment but is specifically designed to do nothing).
After 48 weeks, the L-glutamine group had fewer crisis events than the placebo group (median of 3 instead of 4 events). People who took L-glutamine went to the hospital less often than the other group (median of two instead of three hospitalisations).
L-glutamine may help decrease other sickle cell complications. Please discuss further with your health care team. Some of the side effects of L-glutamine (occurring in at least one in 10 people — same as placebo) include constipation, nausea, headache, stomach pain, cough, leg or arm pain, back pain, and chest pain.
What is Voxelotor and how does it work?
Voxelotor (pronounced “vox-EL-o-tor”), or OXBRYTA® is a treatment for sickle cell disease (SCD) that may help people aged 12 years and older (If you don’t know the type of SCD you or the person receiving care has, ask your doctor). Share the information in this fact sheet with your health care team to decide whether this drug might be a good choice for you or the person receiving care. You may be able to take this drug even if you are currently taking hydroxyurea but consult with your health care team. Voxelotor has not been studied in women who are pregnant or breastfeeding.
Healthy red blood cells contain hemoglobin (Hb), which carries oxygen to all parts of your body.
For individuals living with SCD, red blood cells lose their round shape and become sickled, or crescent-shaped. The sickled red blood cells break apart in a process called hemolysis.
Voxelotor decreases hemolysis and strengthens hemoglobin’s carrying of oxygen.
Voxelotor is given as a pill. The typical dose is three pills (1,500 mg) by mouth daily, with or without food but patients should consult with their health care team. Voxelotor tablets should not be crushed.
Voxelotor is for long-term use. Talk to your health care team about how long will be right for you or the person receiving care.
During a clinical study known as the HOPE study, 50 per cent of participants between the ages of 12 and 64 years were found with increased Hb levels (i.e. in two to four weeks), whereas only seven per cent of those given the placebo (a harmless substance that looks like the treatment but is specifically designed to do nothing) had increased Hb levels.
After 24 weeks, the group given voxelotor also showed fewer hemolysis events than the placebo group. Side effects of voxelotor (occurring in at least one in 10 people – same as placebo) include headache, diarrhea, stomach pain, nausea, tiredness, rash, or fever.
What is hydroxyurea and how does it work?
But here’s some good news: a medicine called hydroxyurea (“hi-drox-ee-ure-EE-a”) can help adults and children with sickle cell disease. This medicine can help you have fewer pain crises — and even live longer. Hydroxyurea has helped many people and it could help you.
Hydroxyurea is a medicine that doctors have used to treat people with sickle cell disease since the 1980s. The Food and Drug Administration (FDA) approved it for treating adults with sickle cell disease in 1998. In 2017, the FDA approved it to treat children with sickle cell disease. Hydroxyurea is also used to treat cancer. But doctors use a lower dose (amount) to treat sickle cell disease than to treat cancer.
How can hydroxyurea help with sickle cell disease?
Hydroxyurea reduces the problems that sickle cell disease causes. People with sickle cell disease who take hydroxyurea have fewer: • Pain crises • Episodes of acute chest syndrome • Blood transfusions • Hospital stays Hydroxyurea can also prevent or slow down damage to your organs.
Hydroxyurea makes your red blood cells bigger. It helps them stay rounder and more flexible — and makes them less likely to turn into a sickle shape. The medicine does this by increasing a special kind of hemoglobin called hemoglobin F. Hemoglobin F is also called fetal hemoglobin because newborn babies have it. When you have higher levels of hemoglobin F, your red blood cells are less likely to cause problems.
If you would like to get in touch with me about this article or about sickle cell, do so, via email: [email protected] and do visit my blog: www.howtolivewithsicklecell.co.uk. The e-copy of my book on sickle cell – How To Live With Sickle Cell is available for purchase on www.toladehinde.com and if you want to purchase a paperback version, it is available on Amazon.