Eight Month Old Boy Needs Bone Marrow Transplant to Treat WAS Syndrome

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Wiskott-Aldrich syndrome (WAS) is an inherited immune system disorder that can lead to frequent infections and problems with bleeding too easily. This rare disorder occurs only in males, affecting about 4 out of every 1 million boys born in the United States. The only treatment that can cure WAS is a bone marrow or cord blood transplant (also called a BMT).

As with eight month old Kyler Musgrove, WAS appears most commonly in children. AOL Health tells his story this morning, reporting that Kyler’s was diagnosed when he was only days old. His mother Jill noticed he was still bleeding from his circumcision two days after coming home from the hospital. This resulted in a workup which revealed Kyler’s extremely low blood platelet count. To be certain of the diagnosis, a doctor will also do a genetic test to look for a mutation of the gene that is linked to WAS.

WAS attacks platelets, T Cells and B Cells, interfering with the blood clotting as well as the body’s normal protections against infection.

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Common signs and symptoms of WAS usually appear soon after birth or within the first year of life:

  • Bleeding easily because of too few normal platelets. This may include tiny red spots under the skin (petechiae), bruises, blood in bowel movements, bleeding gums and nose bleeds.
  • Frequent infections because of too few B cells and T cells. Common infections are ear and sinus infections and pneumonia.
  • Eczema (an itchy rash) of the skin.

The only treatment that can cure WAS is a bone marrow or cord blood transplant (also called a BMT). A bone marrow or cord blood transplant replaces the abnormal cells in the bone marrow with healthy blood-forming cells from a family member or unrelated donor or cord blood unit. The healthy cells can come from the bone marrow or peripheral (circulating) blood of an adult donor or from the blood collected from the umbilical cord after a baby is born. The transplanted blood-forming cells will make normal platelets, T cells and B cells for the body.

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How to Be a Bone Marrow Donor

Sources
St Jude Children's Hospital
National Marrow Donor Program

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