Abstract
Other than bone marrow transplantation, splenectomy is the only effective, long term therapy for the severe thrombocytopenia of Wiscott-Aldrich Syndrome (WAS). Occasional WAS patients demonstrate continued severe thrombocytopenia, either immediately postsplenectomy (p-s) or following an initial improvement in platelet count. These patients may respond to less satisfactory treatment modalities such as steroids or other immunosuppressive agents. We recently evaluated a WAS patient with persistent, severe thrombocytopenia p-s who demonstrated a favorable response to SandoglobulinR (IVIgG). The diagnosis of WAS was based on the following criteria: eczema noted at one month of age, thrombocytopenia and mucous membrane bleeding beginning at two months of age, tiny platelets on peripheral blood smear and a bone marrow aspirate demonstrating normal megakaryocyte numbers and morphology. Bleeding episodes were treated with platelet transfusions until 9 mos. of age at which time splenectomy was performed. Severe thrombocytopenia (platelet count (PC) < 20,000/mm3) persisted p-s requiring continued platelet transfusions. IVIgG, 1gm/kg/day for 3 consecutive days, was given 5 days, It wks, and 7 wks p-s. Post infusion, peak PCs (238,000, 171,000 and 36,000/mm3 respectively) were noted 1-3 days following each course of IVIgG; no platelet transfusions were required during the treatment period. The potential benefits of this modality justify its further investigation in the p-s, WAS patient with persistent thrombocytopenia.
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Bray, G., Albano, E. & Blaese, R. FAVORABLE RESPONSE TO IVIgG IN A WISCOTT-ALDRICH SYNDROME INFANT WITH SEVERE THROMBOCYTOPENIA REFRACTORY TO SPLENECTOMY. Pediatr Res 21 (Suppl 4), 296 (1987). https://doi.org/10.1203/00006450-198704010-00776
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DOI: https://doi.org/10.1203/00006450-198704010-00776