Corticosteroid Therapy (Prednisone, Prednisolone)
In Diamond Blackfan anemia (DBA), the bone marrow (the center of the bone where blood cells are made) does not make enough red blood cells. A special protein in red blood cells called hemoglobin carries oxygen to all of the organs in the body. When the number of red blood cells is low, the organs in the body do not get the oxygen they need.
Corticosteroids can help people with DBA make more red blood cells. They are powerful drugs used to treat many conditions. Corticosteroids are not the same type of drugs that athletes and other people take to increase muscle – those are called anabolic steroids.
This therapy is often one of the first types of treatments doctors recommend for people with DBA. Doctors do not know exactly why corticosteroids work, but about 80% of people with DBA who take them produce more red blood cells.
About Corticosteroid Therapy
The goal of corticosteroid therapy is to take only what the person needs to keep the hemoglobin (a protein in red blood cells that carries oxygen to all of the organs in the body) at a healthy level (about 10 g/dL).
Doctors usually prescribe a certain dose of corticosteroids for several weeks. If the hemoglobin in the body increases, then the dose may be decreased slowly. If the number of red blood cells drops too far, the dose might be increased. When the number of red blood cells is at a healthy level, the dose will be kept the same or slowly reduced.
Over time, the body might not respond to the corticosteroids and they will not work as well. If this happens, the doctor might increase the dose or suggest a different type of treatment such as blood transfusions or stem cell transplantation.
When changing the dose of corticosteroids, the doctor will check the amount of hemoglobin in the body using a blood test called a CBC (complete blood count) and reticulocyte count (this tells if the body is trying to make its own red blood cells).
If corticosteroids are going to work, the number of red blood cells usually increases in 2 to 4 weeks.
Corticosteroids have both short-term and long-term side effects. Low doses of corticosteroids may be helpful with minimal side effects, but use of high dose corticosteroids for a long time can cause serious side effects. People taking steroids must be watched carefully for side effects. Other treatments, such as red cell transfusions, should be considered if a patient requires high corticosteroid doses or if serious side effects develop.
Possible side effects of short-term corticosteroid use:
- Upset stomach
- Increased blood sugar
- Increased hunger
- Behavior changes, trouble sleeping, irritability
- Increased risk of pneumonia, thrush (white coating in the mouth), and other infections
- Weight gain, salt and water retention
- High blood pressure
- Increased fat on the face (rounded face), upper back, and belly
- Stretch marks on the skin, acne, poor wound healing, increased and unusual hair growth
Possible side effects of long-term use (3 months or longer):
- All short-term side effects
- Poor growth in children (can be severe)
- Brittle bones (bones break easily, problems with hips and shoulder joints)
- Muscle weakness
- Eye problems
Tips for Success
- Take steroids exactly as prescribed by your doctor.
- Take steroids in the morning (if prescribed once a day), and take them with food to prevent an upset stomach.
- If you miss a dose of medicine, take it as soon as possible. If it is almost time for the next dose, skip the missed dose and return to the regular schedule. Do not take a double dose or extra doses.
- Tell your doctor if you get sick. Your blood count might drop from the stress of being sick, and you might need extra doses of medicine or other treatment.
- Always check with your doctor before changing the dose or stopping this medicine. It can be dangerous to stop corticosteroids suddenly. Your doctor may want to slowly reduce the amount you’re taking until you stop completely.
- Talk with your doctor about any concerns. Make a list of your questions. Do your research so you can help make decisions about your treatment.
- Centers for Disease Control and Prevention
National Center on Birth Defects and Developmental Disabilities
Division of Blood Disorders
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Atlanta, GA 30333
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