Fanconi Anemia

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Treatment Options

Fanconi anemia can cause a wide range of medical problems. There is no cure for this disease, but many medical therapies, procedures, and surgeries can now treat the symptoms and make life better and longer.

Over the course of an FA patient’s life, many different treatments are often needed. No two FA patients develop the same problems. That’s why patients and their families need to develop a close working relationship with a multidisciplinary treatment center that knows them well and is ready to tackle any problem that arises.

A pediatric hematologist, for example, will assess and treat any problems with blood cells in the bone marrow. (Examples of specific treatments are described below.)

Other problems may require attention from other specialists. For example:

  • a pediatric orthopedic surgeon can help correct a malformation of the arms or legs
  • a pediatric heart surgeon can fix a hole in the heart wall (ventricular septal defect)
  • a gastroenterologist can help with digestive problems that affect normal growth.

All told, children with FA may need access to several different specialists and unique therapies during their lifetime.

Only a large team of specialists can ensure that FA patients receive this whole range of appropriate care and avoid risks. The Fanconi Anemia Center is one of the few places in the U.S. where families dealing with FA can receive this type of comprehensive care and get access to the newest treatments.

Our specialists include bone marrow transplantation, cardiology, endocrinology, gastroenterology, genetics, gynecology, hand/arm surgeons, hearing specialists, hematology, immunology, infectious disease, nephrology, nutrition, oncology,  orthopedics, otolaryngology, pulmonology, psychiatry, social work, surgery, and urology.

Tailored Treatment 

Every patient with FA is different. The age of diagnosis can be age two or age 10 or even higher. The medical problems can vary widely, so can the severity and rate of progression of these problems. Treatment must be individualized based on the patient’s own condition.

Every patient coming to the FA Center gets a comprehensive evaluation, testing, and review of past medical history. Based on review by our full team of specialists, an individualized long-term care plan for treatment is mapped out. A schedule for monitoring and check-ups is also important. For example, since FA patients are at risk for certain cancers they need to be monitored by their physician. And because FA patients have a defect in their DNA repair mechanism, they need to be educated about how to minimize unnecessary exposure to DNA-damaging agents.