Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID

Claire Booth1,2, H Bobby Gaspar1,21Centre for Immunodeficiency, Molecular Immunology Unit, UCL Institute of Child Health, London, UK; 2Dept of Clinical Immunology, Great Ormond Street Hospital NHS Trust, London, UKAbstract: Adenosine deaminase deficiency (ADA) is a rare, inherited disorder of purine...

Descripción completa

Guardado en:

Detalles Bibliográficos
Autores principales:	Claire Booth, H Bobby Gaspar
Formato:	article
Lenguaje:	EN
Publicado:	Dove Medical Press 2009
Materias:	Medicine (General) R5-920
Acceso en línea:	https://doaj.org/article/951c4df78fd143a5b421a262ed2ac31b
Etiquetas:	Agregar Etiqueta Sin Etiquetas, Sea el primero en etiquetar este registro!

id	oai:doaj.org-article:951c4df78fd143a5b421a262ed2ac31b
record_format	dspace
spelling	oai:doaj.org-article:951c4df78fd143a5b421a262ed2ac31b2021-12-02T00:13:54ZPegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID1177-54751177-5491https://doaj.org/article/951c4df78fd143a5b421a262ed2ac31b2009-06-01T00:00:00Zhttp://www.dovepress.com/pegademase-bovine-peg-ada-for-the-treatment-of-infants-and-children-wi-a3286https://doaj.org/toc/1177-5475https://doaj.org/toc/1177-5491Claire Booth1,2, H Bobby Gaspar1,21Centre for Immunodeficiency, Molecular Immunology Unit, UCL Institute of Child Health, London, UK; 2Dept of Clinical Immunology, Great Ormond Street Hospital NHS Trust, London, UKAbstract: Adenosine deaminase deficiency (ADA) is a rare, inherited disorder of purine metabolism characterized by immunodeficiency, failure to thrive and metabolic abnormalities. A lack of the enzyme ADA allows accumulation of toxic metabolites causing defects of both cell mediated and humoral immunity leading to ADA severe combined immune deficiency (SCID), a condition that can be fatal in early infancy if left untreated. Hematopoietic stem cell transplant is curative but is dependent on a good donor match. Other therapeutic options include enzyme replacement therapy (ERT) with pegademase bovine (PEG-ADA) and more recently gene therapy. PEG-ADA has been used in over 150 patients worldwide and has allowed stabilization of patients awaiting more definitive treatment with hematopoietic stem cell transplant. It affords both metabolic detoxification and protective immune function with patients remaining clinically well, but immune reconstitution is often suboptimal and may not be long lived. We discuss the pharmacokinetics, immune reconstitution, effects on systemic disease and side effects of treatment with PEG-ADA. We also review the long-term outcome of patients receiving ERT and discuss the role of PEG-ADA in the management of infants and children with ADA-SCID, alongside other therapeutic options.Keywords: adenosine deaminase deficiency, PEG-ADA, enzyme replacement therapy, severe combined immune deficiency (SCID) Claire BoothH Bobby GasparDove Medical PressarticleMedicine (General)R5-920ENBiologics: Targets & Therapy, Vol 2009, Iss default, Pp 349-358 (2009)
institution	DOAJ
collection	DOAJ
language	EN
topic	Medicine (General) R5-920
spellingShingle	Medicine (General) R5-920 Claire Booth H Bobby Gaspar Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID
description	Claire Booth1,2, H Bobby Gaspar1,21Centre for Immunodeficiency, Molecular Immunology Unit, UCL Institute of Child Health, London, UK; 2Dept of Clinical Immunology, Great Ormond Street Hospital NHS Trust, London, UKAbstract: Adenosine deaminase deficiency (ADA) is a rare, inherited disorder of purine metabolism characterized by immunodeficiency, failure to thrive and metabolic abnormalities. A lack of the enzyme ADA allows accumulation of toxic metabolites causing defects of both cell mediated and humoral immunity leading to ADA severe combined immune deficiency (SCID), a condition that can be fatal in early infancy if left untreated. Hematopoietic stem cell transplant is curative but is dependent on a good donor match. Other therapeutic options include enzyme replacement therapy (ERT) with pegademase bovine (PEG-ADA) and more recently gene therapy. PEG-ADA has been used in over 150 patients worldwide and has allowed stabilization of patients awaiting more definitive treatment with hematopoietic stem cell transplant. It affords both metabolic detoxification and protective immune function with patients remaining clinically well, but immune reconstitution is often suboptimal and may not be long lived. We discuss the pharmacokinetics, immune reconstitution, effects on systemic disease and side effects of treatment with PEG-ADA. We also review the long-term outcome of patients receiving ERT and discuss the role of PEG-ADA in the management of infants and children with ADA-SCID, alongside other therapeutic options.Keywords: adenosine deaminase deficiency, PEG-ADA, enzyme replacement therapy, severe combined immune deficiency (SCID)
format	article
author	Claire Booth H Bobby Gaspar
author_facet	Claire Booth H Bobby Gaspar
author_sort	Claire Booth
title	Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID
title_short	Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID
title_full	Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID
title_fullStr	Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID
title_full_unstemmed	Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID
title_sort	pegademase bovine (peg-ada) for the treatment of infants and children with severe combined immunodeficiency (scid
publisher	Dove Medical Press
publishDate	2009
url	https://doaj.org/article/951c4df78fd143a5b421a262ed2ac31b
work_keys_str_mv	AT clairebooth pegademasebovinepegadaforthetreatmentofinfantsandchildrenwithseverecombinedimmunodeficiencyscid AT hbobbygaspar pegademasebovinepegadaforthetreatmentofinfantsandchildrenwithseverecombinedimmunodeficiencyscid
_version_	1718403876196974592

Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID

Ejemplares similares