Wiskott - Aldrich Syndrome (was)

Dr. Ira Shah
Consultant Pediatrician, Nanavati Hospital and B.J.Wadia Hospital for Children, Mumbai India
First Created: 02/05/2001  Last Updated: 08/01/2015

Patient Education

What is Wiskott - Aldrich syndrome?

Wiskott-Aldrich syndrome is also called as WAS. It is a primary immunodeficiency that affects T-lymphocytes, B-lymphocytes as well as blood cells that control bleeding called platelets.

What are the symptoms of Wiskott - Aldrich syndrome?

Symptoms of WAS vary from patient to patient. Symptoms of the classic form of WAS are bleeding, eczema, and recurrent infections. Patients with WAS have increased risk of certain cancers such as lymphoma & leukemia and increased risk of certain autoimmune disorders.

How is Wiskott Aldrich syndrome inherited?

WAS is caused by mutations in the WASP gene that is located on X-chromosome. As a result, the Wiskott-Aldrich syndrome protein (WASP) is not produced. If some amount of WASP is produced then, the clinical symptoms will be mild. Since the gene is located on X-chromosome, WAS is seen in males and boys and not in girls.

How is the diagnosis of Wiskott-Aldrich syndrome made?

A reduced number of platelets with small size platelets (blood examination shows low MPV) is a hallmark of the disease. The functions of T-lymphocytes & B-lymphocytes are deficient and patients have a low level of antibodies to blood group antigens, fail to produce antibodies to the pneumococcal polysaccharide vaccine. The diagnosis confirmed by demonstrating low WASP in blood or by the presence of a mutation in the WASP gene.

What is the treatment of Wiskott-Aldrich syndrome?

Immunoglobulin replacement therapy helps to prevent infections. Intravenous immunoglobulin is preferred rather than subcutaneous immunoglobulin as the latter may cause bleeding. Eczema is treated with bath oils and moisturizing creams. In case of bleeding, platelet transfusion may be required. Splenectomy (removal of the spleen) may be done in patients with recurrent bleeding and very low platelet counts to improve the platelet count. The curative therapy is bone marrow transplantation or cord blood stem cell transplant.

What vaccines should be avoided in patients with Wiskott-Aldrich syndrome?

Boys with WAS should not receive live vaccines such as MMR, Measles, Chickenpox, and BCG and the oral polio vaccine.

Can Wiskott-Aldrich syndrome by diagnosed in the fetus?

If the mutation causing WAS is known in a given family, the prenatal diagnosis in a male fetus in form of checking the mutation in the amniotic cells or chorionic villus cells can be done to see if the fetus is carrying the defective gene or not. Girls are not affected.


Wiskott - Aldrich Syndrome (WAS) Wiskott - Aldrich Syndrome (WAS) https://www.pediatriconcall.com/show_article/default.aspx?main_cat=immunodeficiencies&sub_cat=wiskott-aldrich-syndrome-was&url=wiskott-aldrich-syndrome-was-patient-education 2015-08-01
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