About WAS

Understanding WAS









Treatment of WAS














Coping with WAS


  • Classification of WAS

    A system of scoring described in 1995 by Zhu et al1 was the first attempt to use a numerical score to describe the severity of the disease based on the symptoms shown by the patient.  The 2009 classification shown below is built upon the classification originally proposed by Dr. Zhu.  It may take up to two years for an infant with a severe WASp mutation to present with the severe WAS characteristics.  Therefore, if the patient has a WAS mutation that is predicted to be severe, but has a clinical scoring of 1-2.5, it is prudent to consider this score as indeterminate until the patient is 2 years old.

    Ochs, H.D. et al 2009 Classification2


        IXLT


        XLT

    Classic

    WAS

    XLN

    Score

    <1

    1

    2

    3

    4

    5

    0

    Thrombocytopenia

     -/+

     +

     +

     +

     +

     +

     -

    Small Platelets

     -

     +

     +

     +

     +

     +

     -

    Eczema

     -

     -

     (+)

     +

     ++

     (+)/+/++

     -

    Immunodeficiency

     -

     -/(+)

     (+)

     +

     +

     (+)/+

     -

    Infections

     -

     -

    (+)

     +

     +/++

     (+)/+/++

     -

    Autoimmunity and/or malignancy

     -

     -

     -

     -

     -

     +

     -

    Congenital Neutropenia

     -

     -

     -

     -

     -

     -

     +


    IXLT- Intermittent XLT

    -/(+) absent or mild, (+) mild, transient eczema or mild transient infections not resulting in sequelae, + persistent, but therapy responsive eczema and recurrent infections requiring antibiotics and often intravenous immunoglobulin prophylaxis, ++ eczema that is difficult to control and severe threatening infections.  Because patients with XLT may develop autoimmune disorders or lymphoma, albeit at a lower rate than those with Classic WAS, progression from a score of 1 or 2 to a 5 is possible for XLT.

    Courtesy:  Biology of Blood and Marrow Transplantation

    Patients who have a score of 5 are sub classified as 5A and 5M 3

    • 5A - Patients with Classic WAS or XLT who have developed one or more autoimmune disorders
    • 5M - Patients with Classic WAS or XLT who have developed a malignancy

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    Citations

    1.   Zhu Q, Zhang M, Blaese RM, Derry JM, Junker A, Francke U, Chen SH, Ochs HD.  The Wiskott_Aldrich Syndrome and X LInked Congenital Thrombocytopenia are caused by mutations in the same gene.   Blood. 1995 Nov 15;86(10):3797-804.  
    2.  Ochs HD, Filipovich AH, Veys P, Cowan MJ, Kapoor N.  Wiskott-Aldrich Syndrome: Diagnosis, Clinical and Laboratory Manifestations, and Treatment.  
    Biol Blood Marrow Transplant. 2008 Jan;15(1 Suppl):84-90.   
    3.  Bosticardo M, Marangoni F, Aiuti A, Villa A, Roncarolo MG.  Recent advances in understanding the pathophysiology of Wiskott-Aldrich syndrome.  Blood. 2009 Apr 7.