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...
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| Veröffentlicht in: | Archivum Immunologiae et Therapiae Experimentalis 2015-02, Vol.63 (1), p.65-71 |
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| creator | Machaczka, Maciej Kämpe Björkvall, Cecilia Wieremiejczyk, Joanna Paucar Arce, Martin Myhr-Eriksson, Kristina Klimkowska, Monika Hägglund, Hans Svenningsson, Per |
| 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. |
| doi_str_mv | 10.1007/s00005-014-0308-8 |
| format | Article |
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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.</description><identifier>ISSN: 0004-069X</identifier><identifier>ISSN: 1661-4917</identifier><identifier>EISSN: 1661-4917</identifier><identifier>DOI: 10.1007/s00005-014-0308-8</identifier><identifier>PMID: 25205209</identifier><language>eng</language><publisher>Basel: Springer Basel</publisher><subject>Adult ; Biomedical and Life Sciences ; Biomedicine ; Body Weight ; Chemokines, CC - metabolism ; Enzyme Replacement Therapy ; Female ; Gaucher Disease - drug therapy ; Glucosylceramidase - administration & dosage ; Glucosylceramidase - supply & distribution ; Glucosylceramidase - therapeutic use ; Hemoglobins - metabolism ; Hexosaminidases - blood ; Humans ; Immunology ; Male ; Middle Aged ; Original ; Original Article ; Pharmacology/Toxicology ; Platelet Count ; Surveys and Questionnaires ; Sweden ; Time Factors ; Treatment Outcome ; Young Adult</subject><ispartof>Archivum Immunologiae et Therapiae Experimentalis, 2015-02, Vol.63 (1), p.65-71</ispartof><rights>The Author(s) 2014</rights><rights>L. Hirszfeld Institute of Immunology and Experimental Therapy, Wroclaw, Poland 2015</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c545t-d7483fbbc67a06797ee144c37fb6f3f22370c608ba3f739f6d044557324ed4e3</citedby><cites>FETCH-LOGICAL-c545t-d7483fbbc67a06797ee144c37fb6f3f22370c608ba3f739f6d044557324ed4e3</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://link.springer.com/content/pdf/10.1007/s00005-014-0308-8$$EPDF$$P50$$Gspringer$$Hfree_for_read</linktopdf><linktohtml>$$Uhttps://link.springer.com/10.1007/s00005-014-0308-8$$EHTML$$P50$$Gspringer$$Hfree_for_read</linktohtml><link.rule.ids>230,314,550,776,780,881,27901,27902,41464,42533,51294</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/25205209$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink><backlink>$$Uhttps://urn.kb.se/resolve?urn=urn:nbn:se:uu:diva-245273$$DView record from Swedish Publication Index$$Hfree_for_read</backlink><backlink>$$Uhttp://kipublications.ki.se/Default.aspx?queryparsed=id:130459019$$DView record from Swedish Publication Index$$Hfree_for_read</backlink></links><search><creatorcontrib>Machaczka, Maciej</creatorcontrib><creatorcontrib>Kämpe Björkvall, Cecilia</creatorcontrib><creatorcontrib>Wieremiejczyk, Joanna</creatorcontrib><creatorcontrib>Paucar Arce, Martin</creatorcontrib><creatorcontrib>Myhr-Eriksson, Kristina</creatorcontrib><creatorcontrib>Klimkowska, Monika</creatorcontrib><creatorcontrib>Hägglund, Hans</creatorcontrib><creatorcontrib>Svenningsson, Per</creatorcontrib><title>Impact of Imiglucerase Supply Shortage on Clinical and Laboratory Parameters in Norrbottnian Patients with Gaucher Disease Type 3</title><title>Archivum Immunologiae et Therapiae Experimentalis</title><addtitle>Arch. Immunol. Ther. Exp</addtitle><addtitle>Arch Immunol Ther Exp (Warsz)</addtitle><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.</description><subject>Adult</subject><subject>Biomedical and Life Sciences</subject><subject>Biomedicine</subject><subject>Body Weight</subject><subject>Chemokines, CC - metabolism</subject><subject>Enzyme Replacement Therapy</subject><subject>Female</subject><subject>Gaucher Disease - drug therapy</subject><subject>Glucosylceramidase - administration & dosage</subject><subject>Glucosylceramidase - supply & distribution</subject><subject>Glucosylceramidase - therapeutic use</subject><subject>Hemoglobins - metabolism</subject><subject>Hexosaminidases - blood</subject><subject>Humans</subject><subject>Immunology</subject><subject>Male</subject><subject>Middle Aged</subject><subject>Original</subject><subject>Original Article</subject><subject>Pharmacology/Toxicology</subject><subject>Platelet Count</subject><subject>Surveys and Questionnaires</subject><subject>Sweden</subject><subject>Time Factors</subject><subject>Treatment Outcome</subject><subject>Young Adult</subject><issn>0004-069X</issn><issn>1661-4917</issn><issn>1661-4917</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2015</creationdate><recordtype>article</recordtype><sourceid>C6C</sourceid><sourceid>EIF</sourceid><sourceid>BENPR</sourceid><sourceid>D8T</sourceid><recordid>eNp1kl2LEzEUhgdR3Fr9Ad5IwBsvHM3nZOZGWLq6FooKW8S7kEnPtFlnkjHJuPTSf25K67IrbAjk433OOcnhLYqXBL8jGMv3EechSkx4iRmuy_pRMSNVRUreEPm4mGU1K1Xz46x4FuN1PjFB-NPijAqK82xmxZ_lMGqTkO_QcrDbfjIQdAR0NY1jv0dXOx-S3gLyDi1666zRPdJug1a69UEnH_bomw56gAQhIuvQFx9C61NyVrssJQsuRXRj0w5d6snsIKALG-FQY70fAbHnxZNO9xFenNZ5sf70cb34XK6-Xi4X56vSCC5SuZG8Zl3bmkpqXMlGAhDODZNdW3Wso5RJbCpct5p1kjVdtcGcCyEZ5bDhwOZFeUwbb2CcWjUGO-iwV15bdbr6mXegBGkOjZoXbx_kL-z3c-XDVk2TolxQyTL-4YhndoCNyb8Our8XdV9xdqe2_rfitG4EIznBm1OC4H9NEJMabDTQ99qBn6IiVS5E66rBGX39H3rtp-By8w4UZ4wK0WSKHCkTfIwButvHEKwO9lFH-6hsH3Wwj6pzzKu7v7iN-OeXDNBTV7LkthDulH4w6199o9I8</recordid><startdate>20150201</startdate><enddate>20150201</enddate><creator>Machaczka, Maciej</creator><creator>Kämpe Björkvall, Cecilia</creator><creator>Wieremiejczyk, Joanna</creator><creator>Paucar Arce, Martin</creator><creator>Myhr-Eriksson, Kristina</creator><creator>Klimkowska, Monika</creator><creator>Hägglund, Hans</creator><creator>Svenningsson, Per</creator><general>Springer Basel</general><general>Springer Nature B.V</general><scope>C6C</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>3V.</scope><scope>7T5</scope><scope>7U9</scope><scope>7X7</scope><scope>7XB</scope><scope>88E</scope><scope>8AO</scope><scope>8FE</scope><scope>8FH</scope><scope>8FI</scope><scope>8FJ</scope><scope>8FK</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>AZQEC</scope><scope>BBNVY</scope><scope>BENPR</scope><scope>BHPHI</scope><scope>CCPQU</scope><scope>DWQXO</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>GNUQQ</scope><scope>H94</scope><scope>HCIFZ</scope><scope>K9.</scope><scope>LK8</scope><scope>M0S</scope><scope>M1P</scope><scope>M7P</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>PRINS</scope><scope>7X8</scope><scope>5PM</scope><scope>ADTPV</scope><scope>AOWAS</scope><scope>DF2</scope><scope>D8T</scope><scope>ZZAVC</scope></search><sort><creationdate>20150201</creationdate><title>Impact of Imiglucerase Supply Shortage on Clinical and Laboratory Parameters in Norrbottnian Patients with Gaucher Disease Type 3</title><author>Machaczka, Maciej ; Kämpe Björkvall, Cecilia ; Wieremiejczyk, Joanna ; Paucar Arce, Martin ; Myhr-Eriksson, Kristina ; Klimkowska, Monika ; Hägglund, Hans ; Svenningsson, Per</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c545t-d7483fbbc67a06797ee144c37fb6f3f22370c608ba3f739f6d044557324ed4e3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2015</creationdate><topic>Adult</topic><topic>Biomedical and Life Sciences</topic><topic>Biomedicine</topic><topic>Body Weight</topic><topic>Chemokines, CC - 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Immunol. Ther. Exp</stitle><addtitle>Arch Immunol Ther Exp (Warsz)</addtitle><date>2015-02-01</date><risdate>2015</risdate><volume>63</volume><issue>1</issue><spage>65</spage><epage>71</epage><pages>65-71</pages><issn>0004-069X</issn><issn>1661-4917</issn><eissn>1661-4917</eissn><abstract>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. 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| subjects | Adult Biomedical and Life Sciences Biomedicine Body Weight Chemokines, CC - metabolism Enzyme Replacement Therapy Female Gaucher Disease - drug therapy Glucosylceramidase - administration & dosage Glucosylceramidase - supply & distribution Glucosylceramidase - therapeutic use Hemoglobins - metabolism Hexosaminidases - blood Humans Immunology Male Middle Aged Original Original Article Pharmacology/Toxicology Platelet Count Surveys and Questionnaires Sweden Time Factors Treatment Outcome Young Adult |
| title | Impact of Imiglucerase Supply Shortage on Clinical and Laboratory Parameters in Norrbottnian Patients with Gaucher Disease Type 3 |
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