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2.
J Pediatr ; 177: 316-320, 2016 10.
Article in English | MEDLINE | ID: mdl-27514238

ABSTRACT

Adenosine deaminase 2 deficiency is an autoinflammatory disease, characterized by various forms of vasculitis. We describe 5 patients with adenosine deaminase 2 deficiency with various hematologic manifestations, including pure red cell aplasia, with no evidence for vasculitis.


Subject(s)
Adenosine Deaminase/deficiency , Intercellular Signaling Peptides and Proteins/deficiency , Adenosine Deaminase/genetics , Child , Child, Preschool , Female , Humans , Infant , Intercellular Signaling Peptides and Proteins/genetics , Male , Metabolism, Inborn Errors/diagnosis , Metabolism, Inborn Errors/genetics , Phenotype
3.
J Pediatr ; 145(5): 710-2, 2004 Nov.
Article in English | MEDLINE | ID: mdl-15520787

ABSTRACT

Purine nucleoside phosphorylase deficiency is a primary immunodeficiency syndrome characterized by the triad of recurrent infection, neurologic dysfunction, and autoimmunity. This patient presented atypically with few infections and normal T-cell function. Progressive lymphopenia, ataxia, and developmental delay led to diagnosis. Umbilical cord blood transplantation corrected the immunodeficiency.


Subject(s)
Cord Blood Stem Cell Transplantation , Immunologic Deficiency Syndromes/surgery , Purine-Nucleoside Phosphorylase/deficiency , Developmental Disabilities/enzymology , Developmental Disabilities/etiology , Humans , Immunologic Deficiency Syndromes/complications , Immunologic Deficiency Syndromes/enzymology , Infant , Lymphopenia/enzymology , Lymphopenia/etiology , Male
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