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Pathology · Immunodeficiency: Wiskott-Aldrich syndrome
Sample itemA 1-year-old boy has eczema, petechiae and recurrent otitis media. His platelet count is 30,000 per microlitre, and the platelets are small on the smear. Which defect explains this triad?
Original practice item, not a recalled exam question. Nothing here is recorded.
The answer and the reasoning
B. Mutation of the WAS gene affecting actin cytoskeleton organisation in haematopoietic cells
Wiskott-Aldrich syndrome is X-linked, with defective WAS protein that regulates actin cytoskeleton, giving microthrombocytopenia (small platelets), eczema and recurrent infections. Typically IgM is low while IgA and IgE are raised. Patients are prone to autoimmunity and lymphoma and need transplantation for definitive treatment.
Option by option
A. Mutation of GP Ib-IX-V with giant dysfunctional platelets
GP Ib-IX-V mutation causes Bernard-Soulier syndrome with giant platelets and no eczema or immunodeficiency.
B. Mutation of the WAS gene affecting actin cytoskeleton organisation in haematopoietic cells (correct)
Correct. WAS protein deficiency produces the classic triad with small platelets.
C. Mutation of BTK with absent B cells
BTK mutation causes agammaglobulinaemia without eczema or thrombocytopenia.
D. Deficiency of ADAMTS13 with platelet microthrombi
ADAMTS13 deficiency causes thrombotic thrombocytopenic purpura, a microangiopathic haemolytic anaemia.
Source note: Original NextPrep sample item — pending expert review. This item is original practice material awaiting independent expert review, so confirm important facts in a standard textbook.
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