Impact of discontinuing 5 years of enzyme replacement treatment in a cohort of 6 adults with hypophosphatasia: A case series

Asfotase alfa is a human recombinant enzyme replacement therapy for hypophosphatasia. We describe 6 adults who were treated with asfotase alfa for 61–68 months in a clinical trial (NCT01163149), after which asfotase alfa was discontinued for 15–48 months. The patients experienced clinical deteriorat...

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Detalles Bibliográficos
Autores principales: Rockman-Greenberg, Cheryl, Josse, Robert, Francis, Mira, Mhanni, Aziz
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Case Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9463174/
https://www.ncbi.nlm.nih.gov/pubmed/36097602
http://dx.doi.org/10.1016/j.bonr.2022.101617
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author Rockman-Greenberg, Cheryl
Josse, Robert
Francis, Mira
Mhanni, Aziz
author_facet Rockman-Greenberg, Cheryl
Josse, Robert
Francis, Mira
Mhanni, Aziz
author_sort Rockman-Greenberg, Cheryl
collection PubMed
description Asfotase alfa is a human recombinant enzyme replacement therapy for hypophosphatasia. We describe 6 adults who were treated with asfotase alfa for 61–68 months in a clinical trial (NCT01163149), after which asfotase alfa was discontinued for 15–48 months. The patients experienced clinical deterioration and, when treatment was restarted, showed improvement. Patients with hypophosphatasia should be closely monitored if asfotase alfa is stopped as clinical decline is likely. Clinical practice guidelines are needed.
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spelling pubmed-94631742022-09-11 Impact of discontinuing 5 years of enzyme replacement treatment in a cohort of 6 adults with hypophosphatasia: A case series Rockman-Greenberg, Cheryl Josse, Robert Francis, Mira Mhanni, Aziz Bone Rep Case Report Asfotase alfa is a human recombinant enzyme replacement therapy for hypophosphatasia. We describe 6 adults who were treated with asfotase alfa for 61–68 months in a clinical trial (NCT01163149), after which asfotase alfa was discontinued for 15–48 months. The patients experienced clinical deterioration and, when treatment was restarted, showed improvement. Patients with hypophosphatasia should be closely monitored if asfotase alfa is stopped as clinical decline is likely. Clinical practice guidelines are needed. Elsevier 2022-08-30 /pmc/articles/PMC9463174/ /pubmed/36097602 http://dx.doi.org/10.1016/j.bonr.2022.101617 Text en © 2022 The Authors. Published by Elsevier Inc. https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).
spellingShingle Case Report
Rockman-Greenberg, Cheryl
Josse, Robert
Francis, Mira
Mhanni, Aziz
Impact of discontinuing 5 years of enzyme replacement treatment in a cohort of 6 adults with hypophosphatasia: A case series
title Impact of discontinuing 5 years of enzyme replacement treatment in a cohort of 6 adults with hypophosphatasia: A case series
title_full Impact of discontinuing 5 years of enzyme replacement treatment in a cohort of 6 adults with hypophosphatasia: A case series
title_fullStr Impact of discontinuing 5 years of enzyme replacement treatment in a cohort of 6 adults with hypophosphatasia: A case series
title_full_unstemmed Impact of discontinuing 5 years of enzyme replacement treatment in a cohort of 6 adults with hypophosphatasia: A case series
title_short Impact of discontinuing 5 years of enzyme replacement treatment in a cohort of 6 adults with hypophosphatasia: A case series
title_sort impact of discontinuing 5 years of enzyme replacement treatment in a cohort of 6 adults with hypophosphatasia: a case series
topic Case Report
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9463174/
https://www.ncbi.nlm.nih.gov/pubmed/36097602
http://dx.doi.org/10.1016/j.bonr.2022.101617
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