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Wiskott-Aldrich syndrome (WAS) is a very rare, serious and potentially life-threatening disorder that almost always affects boys. It causes a child to have a poorly functioning immune system – the body’s “defense center” against infection – and difficulty producing platelets , the blood cells that keep bleeding under control.
When a child has WAS, he typically faces:
In addition, children with WAS are at elevated risk for developing:
How Boston Children’s Hospital approaches Wiskott-Aldrich syndrome
Boston Children’s has a long history of caring for children with complex disorders of the blood and immune system. Clinicians in Dana-Farber/Boston Children's Cancer and Blood Disorder Center and our Division of Allergy and Immunology are regarded as international leaders in understanding and treating rare conditions like Wiskott-Aldrich syndrome.
The only known cure for WAS is a stem cell transplant (using as bone marrow, peripheral blood or umbilical cord blood from a healthy suitably tissue matched donor). Once introduced into your child’s bloodstream, the stem cells can develop into normal immune cells and platelets. The Stem Cell Transplant Center at Dana-Farber/Boston Children’s – one of the first and most renowned pediatric transplant centers in the nation – performs over 90 stem cell transplants each year. Our specialists not only conduct groundbreaking stem cell research and devise exciting new treatments; they also have a unique understanding of the special needs of the youngest, smallest patients.
In addition, Dana-Farber/Boston Children’s has opened one of the first gene therapy clinical trials for children with WAS in the world and the only one in the United States. The clinical trial for WAS – which transplants genetically modified stem cells from a child’s own bone marrow or blood cells – may signify a promising new approach to fighting and defeating the disease.
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