Diamond Blackfan anemia
Treatment & Care
We know how difficult a diagnosis of Diamond Blackfan anemia can be, both for your child and for your whole family. That’s why our physicians are focused on family-centered care: From your first visit, you’ll work with a team of professionals who are committed to supporting all of your family’s physical and psychosocial needs. We’ll work with you to create a care plan that’s best for your child.
Your child’s physician will determine a specific course of treatment based on several factors, including:
- your child's age, overall health and medical history
- the severity of symptoms
- your child's tolerance for specific medications, procedures or therapies
- how your child's doctors expects the disease to progress
- the availability of a suitable stem cell donor
What are the treatments for Diamond Blackfan anemia?
Treatment of Diamond Blackfan anemia may include:
- increases hemoglobin or red blood cells
- taken orally in liquid or pill form every day, every other day or sometimes once a week in divided doses
- side effects may include: upset stomach, increased appetite, weight gain, unstable blood sugars, increased blood pressure, cataracts, decreased height and increased risk for infection
- the majority of patients respond to steroid treatment, and the dose can often be weaned to a low dose that causes few side effects
Red blood cell transfusion
- used when anemia is unusually severe or when steroid therapy is not effective
- red blood cells are delivered through a needle or catheter inserted into a vein
- performed at the hospital or outpatient transfusion center
- takes two to four hours and may be repeated every four to eight weeks
- side effects may include: fever, headache, hives, allergic reaction, infection and iron overload
- patients may eventually develop iron overload and need to take additional medications (chelation therapy) in order to remove this iron from the blood and undergo testing to monitor for heart and liver damage
- A stem cell transplant is the only cure at this time.
- The best results are achieved when the bone marrow comes from an unaffected tissue-compatible sibling. However, a matched sibling donor may not be available in many cases.
- In some cases, a matched unrelated donor may be considered. However, the rate for successful transplant from unrelated donors is lower.
Will my child be OK?
The symptoms of Diamond Blackfan anemia and how well children recover varies significantly depending on the specific genetic mutation your child has and other factors.In more severe cases, DBA can result in life-threatening anemia. Severe cases of DBA require lifelong treatment for anemia and other potential complications. Your child’s physician is available to answer questions about your child’s condition, prognosis and available treatment options.
What is the expected outcome after treatment?
As with any serious medical condition, children’s long-term health can vary greatly from child to child. Prompt medical attention and aggressive therapy are important. The symptoms of Diamond Blackfan anemia and how well children recover varies significantly depending on the specific genetic mutation your child has and other factors.In more severe cases, DBA can result in life-threatening anemia. Severe cases of DBA require lifelong treatment for anemia and other potential complications. Your child’s physician is available to answer questions about your child’s condition, prognosis and available treatment options.
Your child will need lifelong follow-up care to manage symptoms, give therapies such as blood transfusions or monitor your child’s health following a stem cell transplant. New methods are continually being discovered to improve treatment and to decrease side effects of the treatment for the disease.
Resources & support
In addition to providing medical care, we strive to also meet all of your family’s emotional and quality-of-life needs. We know that unfamiliar places, especially hospitals, often create anxiety and fear for a child. We also know this isn’t an easy time or task for parents, who have their own fears and concerns about the situation. Our family support services will help address many of your needs and concerns. Read about our resources and support services.