Wiskott-Aldrich Syndrome (WAS)

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Treatment Options

If your child’s doctor suspects that he/she may have WAS, they will initially use antibiotic, antifungal, or antiviral medications to treat any active infections that your child may have. They will also start Intravenous Immune Globulin (IVIG) to try and protect your child from getting some of the infections that they are susceptible to. These measures can temporarily keep WAS patients healthy but ultimately, most patients with the more severe form of WAS require bone marrow transplantation (BMT), also known as hematopoietic stem cell transplantation.

BMT provides them with a functioning immune system that is capable of protecting them from infections. SCT also replaces the cells that make abnormal platelets so the bleeding problems associated with WAS can be resolved as well. It is a cure for the disease and is highly effective in the majority of patients. 

Gene therapy to correct the genetic mutation in WAS is just beginning to be utilized. At this point, it is still considered an investigational treatment option but further studies are underway to improve this approach. 

Our doctors are very experienced in the care of patients with WAS and are well versed in the use of these therapies.  Many of them are performing leading-edge research related to immune deficiency diseases including WAS and are considered to be national and international experts in this field.