?U N I V E R S I T Y O F M I N N E S O T A A M P L A T Z C H I L D R E N ?S H O S P I T A L
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The Fanconi Anemia team (from left) includes Margaret MacMillan, M.D.; Pat Fidler, R.N., nurse
coordinator; John Wagner, M.D.; and Heather Zierhut, M.S., C.G.C., genetic counselor.
Bench-to-bedside research improves lives
of patients who have Fanconi anemia
Prior to the mid-1970s, most patients with Fanconi anemia (FA) were not expected to
live beyond their teenage years. Although underdiagnosed even today, FA affects about
one in every 300,000 children.
FA is a complex, inherited medical condition
that can affect every organ in the body and
almost always leads to complete bone-marrow
failure. Three decades of increasing success in
treating FA with blood and marrow transplant
(BMT) has changed the once dismal
prognosis; patients are living longer lives
than ever before. With that change, however,
further complications of the disease ? that
tend to manifest in adolescence and adulthood
? have become more prevalent.
Margaret MacMillan, M.D., hematologist/
oncologist, says, ?We continue to make
uofmchildrenshospital.org
September 2009 vol. 3, issue 4
Published bimonthly for health-care professionals?Estimated Read Time: 15 minutes
sequential changes to how we treat these
patients ? beyond transplant ? as we
identify new issues that relate to this complex
condition.? MacMillan, along with John
Wagner, M.D., co-directs the Fanconi Anemia
Comprehensive Care Program (FACCP) at
University of Minnesota Amplatz Children?s
Hospital. ?Our program is one of the
best examples of how clinicians and basic
researchers can work together to change the
practice of medicine,? MacMillan says.
The FACCP research includes ongoing
investigations to understand why children
continues on page 8>>

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