Hematopoietic cell transplantation does not prevent myelopathy in X-linked adrenoleukodystrophy: a retrospective study
Background X-linked adrenoleukodystrophy (X-ALD) is a peroxisomal metabolic disorder. Male patients develop adrenocortical insufficiency (80 % before 18 years), a chronic myelopathy (adrenomyeloneuropathy (AMN); all in adulthood), or progressive cerebral demyelination (cerebral ALD; 40 % before 18 y...
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| Veröffentlicht in: | Journal of inherited metabolic disease 2015-03, Vol.38 (2), p.359-361 |
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| container_title | Journal of inherited metabolic disease |
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| creator | van Geel, Björn M. Poll-The, Bwee Tien Verrips, Aad Boelens, Jaap-Jan Kemp, Stephan Engelen, Marc |
| description | Background
X-linked adrenoleukodystrophy (X-ALD) is a peroxisomal metabolic disorder. Male patients develop adrenocortical insufficiency (80 % before 18 years), a chronic myelopathy (adrenomyeloneuropathy (AMN); all in adulthood), or progressive cerebral demyelination (cerebral ALD; 40 % before 18 years). Cerebral ALD is treated with haematopoetic cell transplantation (HCT). It is unknown if AMN still develops in patients with X-ALD that underwent HCT for cerebral ALD in childhood.
Patients and methods
A retrospective observational study was performed by selecting all adult patients with X-ALD in our cohort that underwent HCT in childhood.
Results
This retrospective study found that three out of five patients in our cohort who underwent HCT in childhood developed signs of myelopathy in adulthood.
Conclusion
These data suggest that HCT for cerebral ALD in childhood does not prevent the onset of AMN in X-ALD in adulthood. |
| doi_str_mv | 10.1007/s10545-014-9797-1 |
| format | Article |
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X-linked adrenoleukodystrophy (X-ALD) is a peroxisomal metabolic disorder. Male patients develop adrenocortical insufficiency (80 % before 18 years), a chronic myelopathy (adrenomyeloneuropathy (AMN); all in adulthood), or progressive cerebral demyelination (cerebral ALD; 40 % before 18 years). Cerebral ALD is treated with haematopoetic cell transplantation (HCT). It is unknown if AMN still develops in patients with X-ALD that underwent HCT for cerebral ALD in childhood.
Patients and methods
A retrospective observational study was performed by selecting all adult patients with X-ALD in our cohort that underwent HCT in childhood.
Results
This retrospective study found that three out of five patients in our cohort who underwent HCT in childhood developed signs of myelopathy in adulthood.
Conclusion
These data suggest that HCT for cerebral ALD in childhood does not prevent the onset of AMN in X-ALD in adulthood.</description><identifier>ISSN: 0141-8955</identifier><identifier>EISSN: 1573-2665</identifier><identifier>DOI: 10.1007/s10545-014-9797-1</identifier><identifier>PMID: 25488625</identifier><language>eng</language><publisher>Dordrecht: Springer Netherlands</publisher><subject>Adolescent ; Adrenoleukodystrophy - complications ; Adrenoleukodystrophy - diagnosis ; Adrenoleukodystrophy - surgery ; Adult ; Age Factors ; Biochemistry ; Child ; Child, Preschool ; Disease Progression ; Hematopoietic Stem Cell Transplantation ; Human Genetics ; Humans ; Internal Medicine ; Male ; Medicine ; Medicine & Public Health ; Metabolic Diseases ; Original Article ; Pediatrics ; Retrospective Studies ; Spinal Cord Diseases - diagnosis ; Spinal Cord Diseases - etiology ; Time Factors ; Treatment Outcome ; Young Adult</subject><ispartof>Journal of inherited metabolic disease, 2015-03, Vol.38 (2), p.359-361</ispartof><rights>SSIEM 2014</rights><rights>2015 SSIEM</rights><rights>SSIEM 2015</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c5339-f4222229e35a127b6dd11c7f9398e86db27397628b679753d83b204cc47c96a63</citedby><cites>FETCH-LOGICAL-c5339-f4222229e35a127b6dd11c7f9398e86db27397628b679753d83b204cc47c96a63</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/s10545-014-9797-1$$EPDF$$P50$$Gspringer$$H</linktopdf><linktohtml>$$Uhttps://link.springer.com/10.1007/s10545-014-9797-1$$EHTML$$P50$$Gspringer$$H</linktohtml><link.rule.ids>314,776,780,1411,27901,27902,41464,42533,45550,45551,51294</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/25488625$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>van Geel, Björn M.</creatorcontrib><creatorcontrib>Poll-The, Bwee Tien</creatorcontrib><creatorcontrib>Verrips, Aad</creatorcontrib><creatorcontrib>Boelens, Jaap-Jan</creatorcontrib><creatorcontrib>Kemp, Stephan</creatorcontrib><creatorcontrib>Engelen, Marc</creatorcontrib><title>Hematopoietic cell transplantation does not prevent myelopathy in X-linked adrenoleukodystrophy: a retrospective study</title><title>Journal of inherited metabolic disease</title><addtitle>J Inherit Metab Dis</addtitle><addtitle>J Inherit Metab Dis</addtitle><description>Background
X-linked adrenoleukodystrophy (X-ALD) is a peroxisomal metabolic disorder. Male patients develop adrenocortical insufficiency (80 % before 18 years), a chronic myelopathy (adrenomyeloneuropathy (AMN); all in adulthood), or progressive cerebral demyelination (cerebral ALD; 40 % before 18 years). Cerebral ALD is treated with haematopoetic cell transplantation (HCT). It is unknown if AMN still develops in patients with X-ALD that underwent HCT for cerebral ALD in childhood.
Patients and methods
A retrospective observational study was performed by selecting all adult patients with X-ALD in our cohort that underwent HCT in childhood.
Results
This retrospective study found that three out of five patients in our cohort who underwent HCT in childhood developed signs of myelopathy in adulthood.
Conclusion
These data suggest that HCT for cerebral ALD in childhood does not prevent the onset of AMN in X-ALD in adulthood.</description><subject>Adolescent</subject><subject>Adrenoleukodystrophy - complications</subject><subject>Adrenoleukodystrophy - diagnosis</subject><subject>Adrenoleukodystrophy - surgery</subject><subject>Adult</subject><subject>Age Factors</subject><subject>Biochemistry</subject><subject>Child</subject><subject>Child, Preschool</subject><subject>Disease Progression</subject><subject>Hematopoietic Stem Cell Transplantation</subject><subject>Human Genetics</subject><subject>Humans</subject><subject>Internal Medicine</subject><subject>Male</subject><subject>Medicine</subject><subject>Medicine & Public Health</subject><subject>Metabolic Diseases</subject><subject>Original Article</subject><subject>Pediatrics</subject><subject>Retrospective Studies</subject><subject>Spinal Cord Diseases - diagnosis</subject><subject>Spinal Cord Diseases - etiology</subject><subject>Time Factors</subject><subject>Treatment Outcome</subject><subject>Young Adult</subject><issn>0141-8955</issn><issn>1573-2665</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2015</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><sourceid>BENPR</sourceid><recordid>eNqFkV9r1jAYxYMo7nX6AbyRgDfeVJOm-eedbOomG7tR8C6kzfO6bm1Sk_Qd_famdIoMxNwkkN95ck4OQi8peUsJke8SJbzhFaFNpaWWFX2EdpRLVtVC8MdoVy5opTTnR-hZSjeEEK04f4qOat4oJWq-Q4czGG0OU-gh9x3uYBhwjtanabA-29wHj12AhH3IeIpwAJ_xuMAQJpuvF9x7_L0aen8LDlsXwYcB5tvglpRjmK6X99jiCOWcJuhyfwCc8uyW5-jJ3g4JXtzvx-jbp49fT86qi6vP5ycfLqqOM6arfVOvSwPjltayFc5R2sm9ZlqBEq6tJdNS1KoVJT9nTrG2Jk3XNbLTwgp2jN5sc6cYfs6Qshn7tIa0HsKcDBW86EV5rKCvH6A3YY6-uFspxaSiqikU3aiuREoR9maK_WjjYigxaylmK8WUvzdrKYYWzav7yXM7gvuj-N1CAeQG3PUDLP-faL6cX54SxnVR1psyFZH_AfEv0__08wszEKkL</recordid><startdate>201503</startdate><enddate>201503</enddate><creator>van Geel, Björn M.</creator><creator>Poll-The, Bwee Tien</creator><creator>Verrips, Aad</creator><creator>Boelens, Jaap-Jan</creator><creator>Kemp, Stephan</creator><creator>Engelen, Marc</creator><general>Springer Netherlands</general><general>Blackwell Publishing Ltd</general><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>7QP</scope><scope>7TK</scope><scope>7X7</scope><scope>7XB</scope><scope>88E</scope><scope>8AO</scope><scope>8FI</scope><scope>8FJ</scope><scope>8FK</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>BENPR</scope><scope>CCPQU</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>K9.</scope><scope>M0S</scope><scope>M1P</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>PRINS</scope><scope>7X8</scope></search><sort><creationdate>201503</creationdate><title>Hematopoietic cell transplantation does not prevent myelopathy in X-linked adrenoleukodystrophy: a retrospective study</title><author>van Geel, Björn M. ; Poll-The, Bwee Tien ; Verrips, Aad ; Boelens, Jaap-Jan ; Kemp, Stephan ; Engelen, Marc</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c5339-f4222229e35a127b6dd11c7f9398e86db27397628b679753d83b204cc47c96a63</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2015</creationdate><topic>Adolescent</topic><topic>Adrenoleukodystrophy - complications</topic><topic>Adrenoleukodystrophy - diagnosis</topic><topic>Adrenoleukodystrophy - surgery</topic><topic>Adult</topic><topic>Age Factors</topic><topic>Biochemistry</topic><topic>Child</topic><topic>Child, Preschool</topic><topic>Disease Progression</topic><topic>Hematopoietic Stem Cell Transplantation</topic><topic>Human Genetics</topic><topic>Humans</topic><topic>Internal Medicine</topic><topic>Male</topic><topic>Medicine</topic><topic>Medicine & Public Health</topic><topic>Metabolic Diseases</topic><topic>Original Article</topic><topic>Pediatrics</topic><topic>Retrospective Studies</topic><topic>Spinal Cord Diseases - diagnosis</topic><topic>Spinal Cord Diseases - etiology</topic><topic>Time Factors</topic><topic>Treatment Outcome</topic><topic>Young Adult</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>van Geel, Björn M.</creatorcontrib><creatorcontrib>Poll-The, Bwee Tien</creatorcontrib><creatorcontrib>Verrips, Aad</creatorcontrib><creatorcontrib>Boelens, Jaap-Jan</creatorcontrib><creatorcontrib>Kemp, Stephan</creatorcontrib><creatorcontrib>Engelen, Marc</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>ProQuest Central (Corporate)</collection><collection>Calcium & Calcified Tissue Abstracts</collection><collection>Neurosciences Abstracts</collection><collection>Health & Medical Collection</collection><collection>ProQuest Central (purchase pre-March 2016)</collection><collection>Medical Database (Alumni Edition)</collection><collection>ProQuest Pharma Collection</collection><collection>Hospital Premium Collection</collection><collection>Hospital Premium Collection (Alumni Edition)</collection><collection>ProQuest Central (Alumni) (purchase pre-March 2016)</collection><collection>ProQuest Central (Alumni Edition)</collection><collection>ProQuest Central UK/Ireland</collection><collection>ProQuest Central</collection><collection>ProQuest One Community College</collection><collection>Health Research Premium Collection</collection><collection>Health Research Premium Collection (Alumni)</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>Health & Medical Collection (Alumni Edition)</collection><collection>Medical Database</collection><collection>ProQuest One Academic Eastern Edition (DO NOT USE)</collection><collection>ProQuest One Academic</collection><collection>ProQuest One Academic UKI Edition</collection><collection>ProQuest Central China</collection><collection>MEDLINE - Academic</collection><jtitle>Journal of inherited metabolic disease</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>van Geel, Björn M.</au><au>Poll-The, Bwee Tien</au><au>Verrips, Aad</au><au>Boelens, Jaap-Jan</au><au>Kemp, Stephan</au><au>Engelen, Marc</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Hematopoietic cell transplantation does not prevent myelopathy in X-linked adrenoleukodystrophy: a retrospective study</atitle><jtitle>Journal of inherited metabolic disease</jtitle><stitle>J Inherit Metab Dis</stitle><addtitle>J Inherit Metab Dis</addtitle><date>2015-03</date><risdate>2015</risdate><volume>38</volume><issue>2</issue><spage>359</spage><epage>361</epage><pages>359-361</pages><issn>0141-8955</issn><eissn>1573-2665</eissn><abstract>Background
X-linked adrenoleukodystrophy (X-ALD) is a peroxisomal metabolic disorder. Male patients develop adrenocortical insufficiency (80 % before 18 years), a chronic myelopathy (adrenomyeloneuropathy (AMN); all in adulthood), or progressive cerebral demyelination (cerebral ALD; 40 % before 18 years). Cerebral ALD is treated with haematopoetic cell transplantation (HCT). It is unknown if AMN still develops in patients with X-ALD that underwent HCT for cerebral ALD in childhood.
Patients and methods
A retrospective observational study was performed by selecting all adult patients with X-ALD in our cohort that underwent HCT in childhood.
Results
This retrospective study found that three out of five patients in our cohort who underwent HCT in childhood developed signs of myelopathy in adulthood.
Conclusion
These data suggest that HCT for cerebral ALD in childhood does not prevent the onset of AMN in X-ALD in adulthood.</abstract><cop>Dordrecht</cop><pub>Springer Netherlands</pub><pmid>25488625</pmid><doi>10.1007/s10545-014-9797-1</doi><tpages>3</tpages><oa>free_for_read</oa></addata></record> |
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| ispartof | Journal of inherited metabolic disease, 2015-03, Vol.38 (2), p.359-361 |
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| language | eng |
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| source | MEDLINE; Wiley Online Library Journals Frontfile Complete; SpringerLink Journals |
| subjects | Adolescent Adrenoleukodystrophy - complications Adrenoleukodystrophy - diagnosis Adrenoleukodystrophy - surgery Adult Age Factors Biochemistry Child Child, Preschool Disease Progression Hematopoietic Stem Cell Transplantation Human Genetics Humans Internal Medicine Male Medicine Medicine & Public Health Metabolic Diseases Original Article Pediatrics Retrospective Studies Spinal Cord Diseases - diagnosis Spinal Cord Diseases - etiology Time Factors Treatment Outcome Young Adult |
| title | Hematopoietic cell transplantation does not prevent myelopathy in X-linked adrenoleukodystrophy: a retrospective study |
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