immunology allergic disorders
Wiskott Aldrich Syndrome
Treatment of Wiskott Aldrich Syndrome
Supportive care using prophylactic immune globulin, antibiotics, andacyclovir For symptomatic thrombocytopenia, platelet transfusion and rarely splenectomy Hematopoietic stem cell transplantation Gene therapy Treatment of Wiskott-Aldrich syndrome is prophylactic antibiotics and Replacement of missing immune components to prevent recurrent bacterial infections, The most well-established cure is Hematopoietic Stem Cell Transplantation , but gene therapy is under study. A recent small study of lentiviral hematopoietic stem/progenitor cell gene therapy in 8 patients with Wiskott-Aldrich syndrome followed for up to 7.6 years demonstrated resolution of severe infections and eczema and a decrease in autoimmune and bleeding disorders in all of the patients ( Without aggressive intervention with transplantation or gene therapy, most patients die by age 15; however, some patients survive into adulthood. 1.Magnani A, Semeraro M, Adam F, et al: Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome [published correction appears inNat Med2022 Oct;28(10):2217].Nat Med28(1):71–80, 2022. doi:10.1038/s41591-021-01641-x 