Prognosis

Attenuated WAS

The long-term outlook is believed to be good. Long-term complications include a risk of severe bleeding and development of autoimmunity.[13]

Severe WAS

The long-term outlook in the absence of curative bone marrow transplant has improved with the introduction of prophylactic therapy. Data are limited but suggest that survival into the third decade is unlikely.[13] Severe bleeding, especially intracranial bleeding, serious infections, and autoimmunity are major causes of morbidity and mortality.[1][13][6][32] In addition, hematologic malignancies (lymphoma and myelodysplastic syndromes) complicate 10% to 20% of cases.[13][6]

Bone marrow transplantation can be curative. Five-year survival rates have improved significantly over the past decade and now approach 90% for both matched sibling donors and fully matched unrelated donors. Transplant using mismatched family donors is generally avoided owing to historically poor survival rates (50% to 55%), although outcomes in this group are also improving.[33][34][35][36]

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