Hematopoietic Stem Cell Transplantation for Progressive Combined Immunodeficiency and Lymphoproliferation in Activated PI3K Syndrome Type 1.

Journal of Allergy and Clinical Immunology（2019）

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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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