Corticosteroid therapy includes taking a prescription steroid medication, Prednisone or Prednisolone, to make the patient’s bone marrow create red blood cells. To date, it is not understood how or why steroids cause the bone marrow to make red blood cells. The goal of steroid therapy is to allow the patient’s bone marrow to make a sufficient amount of red blood cells to ensure the patient maintains a hemoglobin level of 9 gm/dl or higher.
When beginning a steroid trial, patients are typically started on dose of steroids that is the equivalent of approximately 2 milligrams of medication per kilogram of patient weight per day. Initially, the dose is typically split between a morning and evening dose. For example, if a patient weighs 10 kilograms (approximately 20 pounds), the patient would start with an initial dose of 20 milligrams of steroid medication per day and would take 10 milligrams in the morning and 10 milligrams in the evening.
If the patient is going to respond to steroids, there is usually an increase in the hemoglobin or reticulocyte count within two to four weeks.
Patients will typically stay at this high dose for several weeks. This initial dose of steroids is high and therefore patients cannot stay on this high dose for long periods of time. Different doctors may approach the weaning process differently. It is important for patients and families to remember that the weaning process should be conducted slowly and methodically. This can be frustrating, but it is necessary. The DBA Nurse (1-877-DBA-NURSE) can help answer questions related to the weaning process; however you must continue to follow your doctor’s recommendations.
Generally, a sustainable, long term dose of steroids is the equivalent of 0.5 milligrams per kilogram per day or 1.0 milligrams per kilogram every other day, or lower. These are only general guidelines. It is important to remember that each individual patient must be assessed individually, with the steroid therapy goal being to allow the DBA patient to make their own red blood cells while having as “normal” of a life as possible without side effects.
Statistics about steroids
According to the DBAR, 79% of patients initially responded to steroids; 17% of patients were steroid non-responsive; and 4% of patients were never treated with steroids. As of 2008, 38.7% of patients are on steroids. Although 79% of patients initially respond to steroids, some patients will stop taking steroids for a variety of reasons, including too high of a dose, to address growth issues or because of other side effects.
Parents and patients are often very concerned about the side effects of steroids. Patients may react differently and may have none, any, or all of the following short-term side effects:
- Upset stomach (take with food or milk)
- Increased blood sugar in the body- diabetes
- Increased hunger
- Behavior changes, such as fussiness, inability to sleep, irritability
- Increased risk of infections, including pneumonia, thrush (a white coating in the mouth), and other yeast infections
- Weight gain, salt and water retention
- High blood pressure
- Increased fat on face (rounded or moon face), upper back and belly
- Stretch marks on the skin, acne, poor wound healing, increased and unusual hair growth
Possible long-term (taking steroids for more than three months) side effects even in low doses,may include all of the short-term side effects listed above, in addition to:
- Poor growth in children (can be severe)
- Brittle bones or problems with hip or shoulder joints
- Muscle weakness
- Problems with eyes
When patients are on steroids, there are certain other medications that may be taken to avoid or treat certain side effects.
- Antibiotics: When patients are on a high dose of steroids, it is recommended that they take a prophylactic (preventative) dose of antibiotics (Bactrim). This will help ensure the patient does not contract certain types of pneumonia, including pneumocystis pneumonia, also referred to as PCP pneumonia.
- Anti-fungal: Anti-fungal medications are helpful for treating a yeast-based diaper rash or thrush, only taken as needed- not preventative.
- Acid reflux medication: It is recommended that patients take an acid reflux medication to prevent stomach problems, while on steroids. Some common brand names of proton pump inhibitors include: Nexium, Prevacid, Prilosec, Pepcid, Zantac.
- Vaccines: Patients taking high doses of steroids should not get live vaccines, such as chicken pox (varicella) and Measles, Mumps and Rubella (MMR). If given vaccines while on steroids, they may not build an adequate immune response and will need to be repeated in the future.
Complimentary Medical Care
The overall health of a DBA patient on long-term steroids should be carefully monitored. Recommended medical monitoring includes:
- Annual vision check
- Twice a year dental visits. Some DBA patients report high levels of tooth decay and tooth loss.
- Ask about getting a baseline dexascan, which is a special x-ray used to determine the strength of the bones. Monitor future bone loss. This is usually done over the age of 5 years, or when the child can stay still without anesthesia.
- Ask whether a referral to an endocrinologist is warranted. An endocrinologist is a doctor who specializes in the endocrine system in the body. An endrocrinologist may be particularly helpful if there are issues with growth and development while taking steroids, or if diabetes becomes an issue. An endocrinologist can also help if steroids have been taken for long periods and you would like to stop.
- Get a flu shot every fall. Do not take the nasal spray flu vaccine, as this contains live viruses. Everyone in the patient’s home should also receive a flu vaccine.
- Avoid people with viral infections, such as measles or chicken pox.
Effect of puberty
Puberty is a time of considerable chemical and physical body changes. These changes may affect the patient’s responsiveness or unresponsiveness to steroids. This is a time when DBA patients should be carefully monitored.
Effect of pregnancy
Women taking steroids who become pregnant should be carefully monitored. Steroid responsive pregnant women may require an increased steroid dose or even transfusion therapy during pregnancy. The potential for steroid toxicity on the mother and the unborn child should be carefully monitored.
For more information on taking corticosteroids, consider reading, “Coping with Prednisone,” by Eugenia Zukerman and Julie R. Ingelfinger. This book published in 2007 and is usually available at your local bookstore.