Pediatric Blood & Marrow Transplantation Center
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Fanconi Anemia

University of Minnesota Masonic Children's Hospital is the single largest treatment center for patients with FA in the United States. The care provided is individually tailored to meet the unique needs of each patient and family. Our team of expert health care providers who specialize in FA, apply leading-edge research developed by University of Minnesota scientists.

This coordinated care, combined with leading research and long-term follow-up, is one reason the university is a national referral center for diagnosing, monitoring and treating children with FA.

General overview

Fanconi Anemia (FA) is a rare, inherited disease which leads to bone marrow failure. FA prevents bone marrow from making enough new blood cells for the body to work normally. FA also can cause bone marrow to make many faulty blood cells. FA is one of many types of anemia. FA is a type of aplastic anemia. In aplastic anemia, the bone marrow stops making or doesn't make enough of all three types of blood cells.

Low levels of the three types of blood cells can harm many of the body's organs, tissues, and systems. This can lead to serious health problems and an increased risk for developing some cancers, such as leukemia or myelodysplastic syndrome. Although FA is a blood disorder, it also can affect many of your body's organs, tissues, and systems. At least 60 percent of individuals affected by FA are born with at least one physical anomaly. Most often, this includes: short stature, thumb and arm anomalies or other skeletal anomalies.


FA occurs in all racial and ethnic groups and affects men and women equally. Worldwide, the incidence of Fanconi Anemia is around 1/100,000-200,000.

At diagnosis

FA patients who have been referred to us at diagnosis or shortly thereafter are followed carefully by a team of experts specializing in conditions that commonly affect FA patients.

These conditions may include (but are not limited to):

  • physical anomalies
  • heart, intestinal and/or kidney problems
  • hormone deficiencies
  • thyroid insufficiency

Bone marrow tests are performed regularly to monitor any changes in the marrow and blood cells. When bone marrow changes occur, patients’ FA may be initially managed with medications. However, at some point, the only treatment proven to cure the blood system abnormalities associated with FA is through a blood and marrow transplant (BMT), to replace the patient's abnormal stem cells with blood-producing stem cells

Blood and Marrow Transplantation

The decision of when to transplant depends on various factors, including:

  • Blood tests to look for abnormalities in the type and number of blood cells in the body
  • Presence of myelodysplastic syndrome or leukemia
  • Type of stem cell donor available
  • Patient’s overall health

FA patients have unique challenges to transplantation due to higher sensitivity to chemotherapy and radiation along with a high incidence of congenital abnormalities.

Because we have cared for more FA patients than anywhere in the US, our team is committed to finding new ways to improve transplant, limit the side-effects and help our patients live healthy and productive lives.

One example of this commitment is our partnership with reproductive medicine experts to explore options to preserve fertility in FA transplant patients before chemotherapy and radiation are given.

Learn more about the blood and marrow transplant process.

While our entire blood and marrow transplant team is involved in the care of our patients, the following providers lead the care and treatment plans for patients with FA.

Margy MacMillan

John Wagner

We continue to explore novel ways to increase cure rates and improve quality of life for patients with FA. We also collaborate with other researchers throughout the world, keeping our program at the forefront of FA care worldwide. Learn more about current research projects.

FA transplant patients may be eligible for clinical trials, taking advantage of the latest breakthroughs in therapies and treatments. Click here for a list of open trials.

If you would like to gather more information about Fanconi Anemia, please reference the following recommended sources.

Journey Clinic - Pediatric Blood and Marrow Transplantation Center

2450 Riverside Avenue
Minneapolis, MN 55454
612-365-1000 TTY: 612-672-7300

For information about the Adult Blood and Marrow Transplantation program, click here:

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We have been providing innovative care to children in need of a blood and bone marrow transplant for over 50 years. Call us, we're here to help.

(888) 601-0787 or (612) 273-2800

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