Hematopoietic Stem Cell Transplantation for Progressive Combined Immunodeficiency and Lymphoproliferation in Activated PI3K Syndrome Type 1.
Journal of Allergy and Clinical Immunology(2019)
摘要
Patients with APDS1 showed variable clinical manifestations. Life-threatening progressive combined immunodeficiency and massive lymphoproliferation were common indications for HSCT. Fludarabine-based RIC-HSCT ameliorated clinical symptoms, but transplant-related complications were frequent, including graft failure.
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关键词
Activated phosphatidylinositol-3-OH kinase δ syndrome type 1,PIK3CD,hematopoietic stem cell transplantation,combined immunodeficiency,T-cell receptor excision circle,kappa-deleting recombination excision circle,IgG2
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