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Impact of Imiglucerase Supply Shortage on Clinical and Laboratory Parameters in Norrbottnian Patients with Gaucher Disease Type 3
A viral contamination of the production plant producing imiglucerase (Cerezyme™) resulted in an unpredicted worldwide shortage of global supplies during 2009–2010. The aim of the study was to describe the effects of dose reduction of enzyme replacement therapy (ERT) in adults with Norrbottnian form...
Autores principales: | , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer Basel
2014
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4289531/ https://www.ncbi.nlm.nih.gov/pubmed/25205209 http://dx.doi.org/10.1007/s00005-014-0308-8 |
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author | Machaczka, Maciej Kämpe Björkvall, Cecilia Wieremiejczyk, Joanna Paucar Arce, Martin Myhr-Eriksson, Kristina Klimkowska, Monika Hägglund, Hans Svenningsson, Per |
author_facet | Machaczka, Maciej Kämpe Björkvall, Cecilia Wieremiejczyk, Joanna Paucar Arce, Martin Myhr-Eriksson, Kristina Klimkowska, Monika Hägglund, Hans Svenningsson, Per |
author_sort | Machaczka, Maciej |
collection | PubMed |
description | A viral contamination of the production plant producing imiglucerase (Cerezyme™) resulted in an unpredicted worldwide shortage of global supplies during 2009–2010. The aim of the study was to describe the effects of dose reduction of enzyme replacement therapy (ERT) in adults with Norrbottnian form of Gaucher disease type 3 (N-GD3). There were ten adults with N-GD3 treated with imiglucerase in the county of Norrbotten in June 2009. Analyzed variables included plasma chitotriosidase activity and concentration of CCL18/PARC, whole blood hemoglobin concentration (Hb) and platelet count (PLT), as well as patients’ body weight, subjective complaints and health status measured by the EuroQoL-5D questionnaire. The median duration of ERT shortage lasted for 14 months (10–20 months). The median percentage reduction of imiglucerase dose was 36 % (26–59 %). Hb decreased in four patients, PLT decreased in three patients, chitotriosidase increased in three patients (max. +22 % of baseline), and CCL18/PARC increased in six patients (+14 % to +57 %). The body weight was moderately decreased in one patient. No new bone events were noted. Self-assessment of individual patient’s health status was stable in all but one patient. Our results suggest that moderate reduction of ERT dosage lasting for relatively short period of time can lead to worsening in biomarkers of adults with N-GD3. However, this worsening is infrequently translated to clinical worsening of patients. It is possible that CCL18/PARC has a higher sensitivity than chitotriosidase in monitoring of ERT dosing in GD3. |
format | Online Article Text |
id | pubmed-4289531 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2014 |
publisher | Springer Basel |
record_format | MEDLINE/PubMed |
spelling | pubmed-42895312015-01-15 Impact of Imiglucerase Supply Shortage on Clinical and Laboratory Parameters in Norrbottnian Patients with Gaucher Disease Type 3 Machaczka, Maciej Kämpe Björkvall, Cecilia Wieremiejczyk, Joanna Paucar Arce, Martin Myhr-Eriksson, Kristina Klimkowska, Monika Hägglund, Hans Svenningsson, Per Arch Immunol Ther Exp (Warsz) Original Article A viral contamination of the production plant producing imiglucerase (Cerezyme™) resulted in an unpredicted worldwide shortage of global supplies during 2009–2010. The aim of the study was to describe the effects of dose reduction of enzyme replacement therapy (ERT) in adults with Norrbottnian form of Gaucher disease type 3 (N-GD3). There were ten adults with N-GD3 treated with imiglucerase in the county of Norrbotten in June 2009. Analyzed variables included plasma chitotriosidase activity and concentration of CCL18/PARC, whole blood hemoglobin concentration (Hb) and platelet count (PLT), as well as patients’ body weight, subjective complaints and health status measured by the EuroQoL-5D questionnaire. The median duration of ERT shortage lasted for 14 months (10–20 months). The median percentage reduction of imiglucerase dose was 36 % (26–59 %). Hb decreased in four patients, PLT decreased in three patients, chitotriosidase increased in three patients (max. +22 % of baseline), and CCL18/PARC increased in six patients (+14 % to +57 %). The body weight was moderately decreased in one patient. No new bone events were noted. Self-assessment of individual patient’s health status was stable in all but one patient. Our results suggest that moderate reduction of ERT dosage lasting for relatively short period of time can lead to worsening in biomarkers of adults with N-GD3. However, this worsening is infrequently translated to clinical worsening of patients. It is possible that CCL18/PARC has a higher sensitivity than chitotriosidase in monitoring of ERT dosing in GD3. Springer Basel 2014-09-10 2015 /pmc/articles/PMC4289531/ /pubmed/25205209 http://dx.doi.org/10.1007/s00005-014-0308-8 Text en © The Author(s) 2014 https://creativecommons.org/licenses/by/4.0/ Open AccessThis article is distributed under the terms of the Creative Commons Attribution License which permits any use, distribution, and reproduction in any medium, provided the original author(s) and the source are credited. |
spellingShingle | Original Article Machaczka, Maciej Kämpe Björkvall, Cecilia Wieremiejczyk, Joanna Paucar Arce, Martin Myhr-Eriksson, Kristina Klimkowska, Monika Hägglund, Hans Svenningsson, Per Impact of Imiglucerase Supply Shortage on Clinical and Laboratory Parameters in Norrbottnian Patients with Gaucher Disease Type 3 |
title | Impact of Imiglucerase Supply Shortage on Clinical and Laboratory Parameters in Norrbottnian Patients with Gaucher Disease Type 3 |
title_full | Impact of Imiglucerase Supply Shortage on Clinical and Laboratory Parameters in Norrbottnian Patients with Gaucher Disease Type 3 |
title_fullStr | Impact of Imiglucerase Supply Shortage on Clinical and Laboratory Parameters in Norrbottnian Patients with Gaucher Disease Type 3 |
title_full_unstemmed | Impact of Imiglucerase Supply Shortage on Clinical and Laboratory Parameters in Norrbottnian Patients with Gaucher Disease Type 3 |
title_short | Impact of Imiglucerase Supply Shortage on Clinical and Laboratory Parameters in Norrbottnian Patients with Gaucher Disease Type 3 |
title_sort | impact of imiglucerase supply shortage on clinical and laboratory parameters in norrbottnian patients with gaucher disease type 3 |
topic | Original Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4289531/ https://www.ncbi.nlm.nih.gov/pubmed/25205209 http://dx.doi.org/10.1007/s00005-014-0308-8 |
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