Short-Term Efficacy of Enzyme Replacement Therapy in Korean Patients with Fabry Disease
Identifieur interne : 003887 ( Pmc/Checkpoint ); précédent : 003886; suivant : 003888Short-Term Efficacy of Enzyme Replacement Therapy in Korean Patients with Fabry Disease
Auteurs : Jin-Ho Choi [Corée du Sud] ; Young Mi Cho [Corée du Sud] ; Kwang-Sun Suh [Corée du Sud] ; Hye-Ran Yoon [Corée du Sud] ; Gu-Hwan Kim [Corée du Sud] ; Sung-Su Kim [Corée du Sud] ; Jung Min Ko [Corée du Sud] ; Joo Hoon Lee [Corée du Sud] ; Young Seo Park [Corée du Sud] ; Han-Wook Yoo [Corée du Sud]Source :
- Journal of Korean Medical Science [ 1011-8934 ] ; 2008.
Abstract
Fabrazyme has been widely used for treatment of Fabry disease since its approval by the U.S. Food and Drug Administration in 2003. This study was undertaken to assess the short-term efficacy and safety of enzyme replacement therapy (ERT) for Fabry disease in Korea. Eight male patients and three female symptomatic carriers aged 13 to 48 yr were included. Fabrazyme was administered by intravenous infusion at a dose of 1 mg/kg every 2 weeks. Plasma and urine globotriaosylceramide (GL-3) levels, serum creatinine, creatinine clearance, and 24-hr urine protein levels were measured every 3 months. Kidney biopsies, ophthalmologic exams, and pure tone audiometry were performed before and 1 yr after ERT. Kidney function, including serum creatinine, creatinine clearance, and the 24-hr urine protein level, remained stable during ERT. Plasma and urine GL-3 levels were reduced within 3 to 6 months of ERT initiation. Microvascular endothelial deposits of GL-3 were decreased from renal biopsy specimens after 1 yr of treatment. The severity of sensorineural hearing loss and tinnitus did not improve after ERT. ERT is safe and effective in stabilizing renal function and clearing microvascular endothelial GL-3 from kidney biopsy specimen in Korean patients with Fabry disease.
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DOI: 10.3346/jkms.2008.23.2.243
PubMed: 18437007
PubMed Central: 2526436
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wicri:level="1"><nlm:aff id="A1">Department of Pediatrics, Research Institute for Medical Sciences, Chungnam National University Hospital, College of Medicine, Chungnam National University, Daejeon, Korea.</nlm:aff><country xml:lang="fr" wicri:curation="lc">Corée du Sud</country><wicri:regionArea>Department of Pediatrics, Research Institute for Medical Sciences, Chungnam National University Hospital, College of Medicine, Chungnam National University, Daejeon</wicri:regionArea><wicri:noRegion>Daejeon</wicri:noRegion></affiliation></author><author><name sortKey="Cho, Young Mi" sort="Cho, Young Mi" uniqKey="Cho Y" first="Young Mi" last="Cho">Young Mi Cho</name><affiliation wicri:level="1"><nlm:aff id="A2">Department of Pathology, Research Institute for Medical Sciences, Chungnam National University Hospital, College of Medicine, Chungnam National University, Daejeon, Korea.</nlm:aff><country xml:lang="fr" wicri:curation="lc">Corée du Sud</country><wicri:regionArea>Department of Pathology, 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id="A4">Medical Genetics Clinic and Laboratory, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea.</nlm:aff><country xml:lang="fr" wicri:curation="lc">Corée du Sud</country><wicri:regionArea>Medical Genetics Clinic and Laboratory, Asan Medical Center, University of Ulsan College of Medicine, Seoul</wicri:regionArea><placeName><settlement type="city">Séoul</settlement><region type="capital">Région capitale de Séoul</region></placeName></affiliation></author><author><name sortKey="Kim, Gu Hwan" sort="Kim, Gu Hwan" uniqKey="Kim G" first="Gu-Hwan" last="Kim">Gu-Hwan Kim</name><affiliation wicri:level="3"><nlm:aff id="A5">Department of Pediatrics, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea.</nlm:aff><country xml:lang="fr" wicri:curation="lc">Corée du Sud</country><wicri:regionArea>Department of Pediatrics, Asan Medical Center, University of Ulsan College of Medicine, Seoul</wicri:regionArea><placeName><settlement type="city">Séoul</settlement><region type="capital">Région capitale de Séoul</region></placeName></affiliation></author><author><name sortKey="Kim, Sung Su" sort="Kim, Sung Su" uniqKey="Kim S" first="Sung-Su" last="Kim">Sung-Su Kim</name><affiliation wicri:level="3"><nlm:aff id="A5">Department of Pediatrics, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea.</nlm:aff><country xml:lang="fr" wicri:curation="lc">Corée du Sud</country><wicri:regionArea>Department of Pediatrics, Asan Medical Center, University of Ulsan College of Medicine, Seoul</wicri:regionArea><placeName><settlement type="city">Séoul</settlement><region type="capital">Région capitale de Séoul</region></placeName></affiliation></author><author><name sortKey="Ko, Jung Min" sort="Ko, Jung Min" uniqKey="Ko J" first="Jung Min" last="Ko">Jung Min Ko</name><affiliation wicri:level="3"><nlm:aff id="A6">Biomedical and Pharmaceutical Analysis Laboratory, Department of Analytical Chemistry, School of Pharmacy, Duksung Women's University, Seoul, Korea.</nlm:aff><country xml:lang="fr" wicri:curation="lc">Corée du Sud</country><wicri:regionArea>Biomedical and Pharmaceutical Analysis Laboratory, Department of Analytical Chemistry, School of Pharmacy, Duksung Women's University, Seoul</wicri:regionArea><placeName><settlement type="city">Séoul</settlement><region type="capital">Région capitale de Séoul</region></placeName></affiliation></author><author><name sortKey="Lee, Joo Hoon" sort="Lee, Joo Hoon" uniqKey="Lee J" first="Joo Hoon" last="Lee">Joo Hoon Lee</name><affiliation wicri:level="3"><nlm:aff id="A6">Biomedical and Pharmaceutical Analysis Laboratory, Department of Analytical Chemistry, School of Pharmacy, Duksung Women's University, Seoul, Korea.</nlm:aff><country xml:lang="fr" wicri:curation="lc">Corée du Sud</country><wicri:regionArea>Biomedical and Pharmaceutical Analysis Laboratory, Department of Analytical Chemistry, School of Pharmacy, Duksung Women's University, 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Yoo</name><affiliation wicri:level="3"><nlm:aff id="A6">Biomedical and Pharmaceutical Analysis Laboratory, Department of Analytical Chemistry, School of Pharmacy, Duksung Women's University, Seoul, Korea.</nlm:aff><country xml:lang="fr" wicri:curation="lc">Corée du Sud</country><wicri:regionArea>Biomedical and Pharmaceutical Analysis Laboratory, Department of Analytical Chemistry, School of Pharmacy, Duksung Women's University, Seoul</wicri:regionArea><placeName><settlement type="city">Séoul</settlement><region type="capital">Région capitale de Séoul</region></placeName></affiliation></author></analytic><series><title level="j">Journal of Korean Medical Science</title><idno type="ISSN">1011-8934</idno><idno type="eISSN">1598-6357</idno><imprint><date when="2008">2008</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en"><p>Fabrazyme has been widely used for treatment of Fabry disease since its approval by the U.S. Food and Drug Administration in 2003. This study was undertaken to assess the short-term efficacy and safety of enzyme replacement therapy (ERT) for Fabry disease in Korea. Eight male patients and three female symptomatic carriers aged 13 to 48 yr were included. Fabrazyme was administered by intravenous infusion at a dose of 1 mg/kg every 2 weeks. Plasma and urine globotriaosylceramide (GL-3) levels, serum creatinine, creatinine clearance, and 24-hr urine protein levels were measured every 3 months. Kidney biopsies, ophthalmologic exams, and pure tone audiometry were performed before and 1 yr after ERT. Kidney function, including serum creatinine, creatinine clearance, and the 24-hr urine protein level, remained stable during ERT. Plasma and urine GL-3 levels were reduced within 3 to 6 months of ERT initiation. Microvascular endothelial deposits of GL-3 were decreased from renal biopsy specimens after 1 yr of treatment. The severity of sensorineural hearing loss and tinnitus did not improve after ERT. ERT is safe and effective in stabilizing renal function and clearing microvascular endothelial GL-3 from kidney biopsy specimen in Korean patients with Fabry disease.</p></div></front><back><div1 type="bibliography"><listBibl><biblStruct><analytic><author><name sortKey="Kint, Ja" uniqKey="Kint J">JA Kint</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Desnick, Rj" uniqKey="Desnick R">RJ Desnick</name></author><author><name sortKey="Ioannou, Ya" uniqKey="Ioannou Y">YA Ioannou</name></author><author><name sortKey="Eng, Cm" uniqKey="Eng C">CM Eng</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Sheth, Kj" uniqKey="Sheth K">KJ Sheth</name></author><author><name sortKey="Roth, Da" uniqKey="Roth D">DA Roth</name></author><author><name sortKey="Adams, Mb" uniqKey="Adams M">MB Adams</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Peters, Fp" uniqKey="Peters F">FP 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<front><journal-meta><journal-id journal-id-type="nlm-ta">J Korean Med Sci</journal-id><journal-id journal-id-type="publisher-id">JKMS</journal-id><journal-title-group><journal-title>Journal of Korean Medical Science</journal-title></journal-title-group><issn pub-type="ppub">1011-8934</issn><issn pub-type="epub">1598-6357</issn><publisher><publisher-name>The Korean Academy of Medical Sciences</publisher-name></publisher></journal-meta><article-meta><article-id pub-id-type="pmid">18437007</article-id><article-id pub-id-type="pmc">2526436</article-id><article-id pub-id-type="doi">10.3346/jkms.2008.23.2.243</article-id><article-categories><subj-group subj-group-type="heading"><subject>Original Article</subject></subj-group></article-categories><title-group><article-title>Short-Term Efficacy of Enzyme Replacement Therapy in Korean Patients with Fabry Disease</article-title></title-group><contrib-group><contrib contrib-type="author"><name><surname>Choi</surname><given-names>Jin-Ho</given-names></name><xref ref-type="aff" rid="A1"></xref></contrib><contrib contrib-type="author"><name><surname>Cho</surname><given-names>Young Mi</given-names></name><xref ref-type="aff" rid="A2">*</xref></contrib><contrib contrib-type="author"><name><surname>Suh</surname><given-names>Kwang-Sun</given-names></name><xref ref-type="aff" rid="A3">†</xref></contrib><contrib contrib-type="author"><name><surname>Yoon</surname><given-names>Hye-Ran</given-names></name><xref ref-type="aff" rid="A4">‡</xref></contrib><contrib contrib-type="author"><name><surname>Kim</surname><given-names>Gu-Hwan</given-names></name><xref ref-type="aff" rid="A5">§</xref></contrib><contrib contrib-type="author"><name><surname>Kim</surname><given-names>Sung-Su</given-names></name><xref ref-type="aff" rid="A5">§</xref></contrib><contrib contrib-type="author"><name><surname>Ko</surname><given-names>Jung Min</given-names></name><xref ref-type="aff" rid="A6">‖</xref></contrib><contrib contrib-type="author"><name><surname>Lee</surname><given-names>Joo Hoon</given-names></name><xref ref-type="aff" rid="A6">‖</xref></contrib><contrib contrib-type="author"><name><surname>Park</surname><given-names>Young Seo</given-names></name><xref ref-type="aff" rid="A6">‖</xref></contrib><contrib contrib-type="author" corresp="yes"><name><surname>Yoo</surname><given-names>Han-Wook</given-names></name><xref ref-type="aff" rid="A6">‖</xref></contrib></contrib-group><aff id="A1">Department of Pediatrics, Research Institute for Medical Sciences, Chungnam National University Hospital, College of Medicine, Chungnam National University, Daejeon, Korea.</aff><aff id="A2"><label>*</label>Department of Pathology, Research Institute for Medical Sciences, Chungnam National University Hospital, College of Medicine, Chungnam National University, Daejeon, Korea.</aff><aff id="A3"><label>†</label>Department of Pathology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea.</aff><aff id="A4"><label>‡</label>Medical Genetics Clinic and Laboratory, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea.</aff><aff id="A5"><label>§</label>Department of Pediatrics, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea.</aff><aff id="A6"><label>‖</label>Biomedical and Pharmaceutical Analysis Laboratory, Department of Analytical Chemistry, School of Pharmacy, Duksung Women's University, Seoul, Korea.</aff><author-notes><corresp>Address for correspondence: Han-Wook Yoo, M.D. Department of Pediatrics, Asan Medical Center, 388-1 Pungnap-dong, Songpa-gu, Seoul 138-736, Korea. Tel: +82.2-3010-3374, Fax: +82.2-473-3725, <email>hwyoo@amc.seoul.kr</email></corresp></author-notes><pub-date pub-type="ppub"><month>4</month><year>2008</year></pub-date><pub-date pub-type="epub"><day>20</day><month>4</month><year>2008</year></pub-date><volume>23</volume><issue>2</issue><fpage>243</fpage><lpage>250</lpage><history><date date-type="received"><day>22</day><month>3</month><year>2007</year></date><date date-type="accepted"><day>13</day><month>8</month><year>2007</year></date></history><permissions><copyright-statement>Copyright © 2008 The Korean Academy of Medical Sciences</copyright-statement><copyright-year>2008</copyright-year><license license-type="open-access" xlink:href="http://creativecommons.org/licenses/by-nc/3.0"><license-p>This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (<ext-link ext-link-type="uri" xlink:href="http://creativecommons.org/licenses/by-nc/3.0">http://creativecommons.org/licenses/by-nc/3.0</ext-link>) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.</license-p></license></permissions><abstract><p>Fabrazyme has been widely used for treatment of Fabry disease since its approval by the U.S. Food and Drug Administration in 2003. This study was undertaken to assess the short-term efficacy and safety of enzyme replacement therapy (ERT) for Fabry disease in Korea. Eight male patients and three female symptomatic carriers aged 13 to 48 yr were included. Fabrazyme was administered by intravenous infusion at a dose of 1 mg/kg every 2 weeks. Plasma and urine globotriaosylceramide (GL-3) levels, serum creatinine, creatinine clearance, and 24-hr urine protein levels were measured every 3 months. Kidney biopsies, ophthalmologic exams, and pure tone audiometry were performed before and 1 yr after ERT. Kidney function, including serum creatinine, creatinine clearance, and the 24-hr urine protein level, remained stable during ERT. Plasma and urine GL-3 levels were reduced within 3 to 6 months of ERT initiation. Microvascular endothelial deposits of GL-3 were decreased from renal biopsy specimens after 1 yr of treatment. The severity of sensorineural hearing loss and tinnitus did not improve after ERT. ERT is safe and effective in stabilizing renal function and clearing microvascular endothelial GL-3 from kidney biopsy specimen in Korean patients with Fabry disease.</p></abstract><kwd-group><kwd>α-galactosidase A</kwd><kwd>Enzyme Replacement Therapy</kwd><kwd>Fabry Disease</kwd><kwd>Globotriaosylceramide</kwd><kwd>Lysosomal Storage Diseases</kwd></kwd-group></article-meta></front><floats-group><fig id="F1" position="float"><label>Fig. 1</label><caption><p>Renal function of patients during enzyme replacement therapy. Serum creatinine <bold>(A)</bold>, creatinine clearance <bold>(B)</bold>, and 24-hr urine protein levels <bold>(C)</bold> remained stable without aggravation or improvement (<italic>p</italic>>0.05). Data are presented as mean±SD.</p></caption><graphic xlink:href="jkms-23-243-g001"></graphic></fig><fig id="F2" position="float"><label>Fig. 2</label><caption><p>Sequential changes in the GL-3 concentration in plasma <bold>(A)</bold> and urine <bold>(B)</bold> during enzyme replacement therapy.</p></caption><graphic xlink:href="jkms-23-243-g002"></graphic></fig><fig id="F3" position="float"><label>Fig. 3</label><caption><p>Pathologic features before <bold>(A, C)</bold> and after <bold>(B, D)</bold> ERT. No significant changes were identified the glomerulus and tubulointerstitium on histologic examination. However, the vascular endothelial GL-3 depositions (arrows) were markedly decreased after the ERT (from patient 1; A and B, PAS staining at ×400 magnification; C and D, toluidine blue staining of semithin sections at ×1,000 magnification).</p></caption><graphic xlink:href="jkms-23-243-g003"></graphic></fig><table-wrap id="T1" position="float"><label>Table 1</label><caption><p>Clinical characteristics of patients with Fabry disease</p></caption><graphic xlink:href="jkms-23-243-i001"></graphic><table-wrap-foot><fn><p>ERT, enzyme replacement therapy; GL-3, globotriaosylceramide.</p></fn></table-wrap-foot></table-wrap><table-wrap id="T2" position="float"><label>Table 2</label><caption><p>Clinical and demographic profiles of the Korean Fabry registry</p></caption><graphic xlink:href="jkms-23-243-i002"></graphic><table-wrap-foot><fn><p>ERT, enzyme replacement therapy; MRI, magnetic resonance imaging.</p></fn></table-wrap-foot></table-wrap><table-wrap id="T3" position="float"><label>Table 3</label><caption><p>Histology and ultrastructural studies of kidney pathologic findings of patients with Fabry disease; Changes in individual scores for globotriaosyleramide (GL-3) deposits of kidney specimens from baseline to after 1 yr of infusion</p></caption><graphic xlink:href="jkms-23-243-i003"></graphic></table-wrap></floats-group></pmc><affiliations><list><country><li>Corée du Sud</li></country><region><li>Région capitale de Séoul</li></region><settlement><li>Séoul</li></settlement></list><tree><country name="Corée du Sud"><noRegion><name sortKey="Choi, Jin Ho" sort="Choi, Jin Ho" uniqKey="Choi J" first="Jin-Ho" last="Choi">Jin-Ho Choi</name></noRegion><name sortKey="Cho, Young Mi" sort="Cho, Young Mi" uniqKey="Cho Y" first="Young Mi" last="Cho">Young Mi Cho</name><name sortKey="Kim, Gu Hwan" sort="Kim, Gu Hwan" uniqKey="Kim G" first="Gu-Hwan" last="Kim">Gu-Hwan Kim</name><name sortKey="Kim, Sung Su" sort="Kim, Sung Su" uniqKey="Kim S" first="Sung-Su" last="Kim">Sung-Su Kim</name><name sortKey="Ko, Jung Min" sort="Ko, Jung Min" uniqKey="Ko J" first="Jung Min" last="Ko">Jung Min Ko</name><name sortKey="Lee, Joo Hoon" sort="Lee, Joo Hoon" uniqKey="Lee J" first="Joo Hoon" last="Lee">Joo Hoon Lee</name><name sortKey="Park, Young Seo" sort="Park, Young Seo" uniqKey="Park Y" first="Young Seo" last="Park">Young Seo Park</name><name sortKey="Suh, Kwang Sun" sort="Suh, Kwang Sun" uniqKey="Suh K" first="Kwang-Sun" last="Suh">Kwang-Sun Suh</name><name sortKey="Yoo, Han Wook" sort="Yoo, Han Wook" uniqKey="Yoo H" first="Han-Wook" last="Yoo">Han-Wook Yoo</name><name sortKey="Yoon, Hye Ran" sort="Yoon, Hye Ran" uniqKey="Yoon H" first="Hye-Ran" last="Yoon">Hye-Ran Yoon</name></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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