Diagnosis, Treatment and Long-Term Follow Up of Patients with ADA Deficiency: a Single-Center Experience

Journal of Clinical Immunology

Volumn 35, Issue 7, 2015, Pages 624-637

  Baffelli, Renata ^a   Notarangelo, Lucia D ^a   Imberti, Luisa ^a   Hershfield, Michael S ^b   Serana, Federico ^a   Santisteban, Ines ^b   Bolda, Federica ^a   Porta, Fulvio ^a   Lanfranchi, Arnalda ^a  

^a SPEDALI CIVILI   (Italy)

^b DUKE UNIVERSITY MEDICAL CENTER   (United States)

Author keywords

Adenosine deaminase deficiency; allogeneic haematopoietic stem cell transplantation; enzyme replacement therapy with PEG ADA

Indexed keywords

ADENOSINE DEAMINASE; ENZYME ANTIBODY; HEMOGLOBIN; IMMUNOGLOBULIN G;

ADENOSINE DEAMINASE DEFICIENCY; ARTICLE; BRONCHITIS; CHIMERA; CLINICAL ARTICLE; CLINICAL FEATURE; CONTROLLED STUDY; DERMATITIS; ENZYME ACTIVITY; ENZYME REPLACEMENT; ETHNIC GROUP; FAMILY HISTORY; FEMALE; FOLLOW UP; GENE MUTATION; GEOGRAPHY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMOGLOBIN BLOOD LEVEL; HEPATOMEGALY; HUMAN; HYPOTROPHY; INFANT; MALE; MOLECULAR DIAGNOSIS; MUSCLE HYPOTONIA; MUTATIONAL ANALYSIS; PRIORITY JOURNAL; RETROSPECTIVE STUDY; TREMOR; AGAMMAGLOBULINEMIA; COMPARATIVE STUDY; CONSANGUINITY; DEFICIENCY; DNA MUTATIONAL ANALYSIS; GENE THERAPY; GENETICS; ITALY; METABOLISM; MUTATION; PRESCHOOL CHILD; ROMANI (PEOPLE); SEVERE COMBINED IMMUNODEFICIENCY; TREATMENT OUTCOME;

ADENOSINE DEAMINASE; AGAMMAGLOBULINEMIA; CHILD, PRESCHOOL; CONSANGUINITY; DNA MUTATIONAL ANALYSIS; ENZYME REPLACEMENT THERAPY; FEMALE; FOLLOW-UP STUDIES; GENETIC THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; INFANT; ITALY; MALE; MUTATION; RETROSPECTIVE STUDIES; ROMA; SEVERE COMBINED IMMUNODEFICIENCY; TREATMENT OUTCOME;

EID: 84946483122     PISSN: 02719142     EISSN: 15732592     Source Type: Journal    
DOI: 10.1007/s10875-015-0191-z     Document Type: Article

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