Long-term efficacy of hematopoietic stem cell transplantation on brain involvement in patients with mucopolysaccharidosis type II: A nationwide survey in Japan
Hematopoietic stem cell transplantation (HSCT) has not been indicated for patients with mucopolysaccharidosis II (MPS II, Hunter syndrome), while it is indicated for mucopolysaccharidosis I (MPS I) patients
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| Veröffentlicht in: | Molecular genetics and metabolism 2012-11, Vol.107 (3), p.513-520 |
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| creator | Tanaka, Akemi Okuyama, Torayuki Suzuki, Yasuyuki Sakai, Norio Takakura, Hiromitsu Sawada, Tomo Tanaka, Toju Otomo, Takanobu Ohashi, Toya Ishige-Wada, Mika Yabe, Hiromasa Ohura, Toshihiro Suzuki, Nobuhiro Kato, Koji Adachi, Souichi Kobayashi, Ryoji Mugishima, Hideo Kato, Shunichi |
| description | Hematopoietic stem cell transplantation (HSCT) has not been indicated for patients with mucopolysaccharidosis II (MPS II, Hunter syndrome), while it is indicated for mucopolysaccharidosis I (MPS I) patients |
| doi_str_mv | 10.1016/j.ymgme.2012.09.004 |
| format | Article |
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► HSCT showed effectiveness on brain or heart involvements in MPS II patients, when performed before a sign of brain atrophy appear in MRI for brain and before valvular regurgitation appear for heart. ► HSCT showed efficacy on cribriform lesion and ventricular dilatation in MRI of MPS II. ► Cardiac valvular regurgitation was diminished in 32% valves of HSCT-treated patients. ► Urinary GAG in HSCT-treated patients was lower than in ERT-treating patients.</description><identifier>ISSN: 1096-7192</identifier><identifier>EISSN: 1096-7206</identifier><identifier>DOI: 10.1016/j.ymgme.2012.09.004</identifier><identifier>PMID: 23022072</identifier><language>eng</language><publisher>United States: Elsevier Inc</publisher><subject>Activities of Daily Living ; Brain - drug effects ; Brain - enzymology ; Brain - pathology ; Brain efficacy ; Child ; Child, Preschool ; Enzyme Replacement Therapy ; Female ; Glycosaminoglycans - urine ; Health Care Surveys ; Hematopoietic Stem Cell Transplantation ; Humans ; Iduronidase - therapeutic use ; Japan ; Magnetic Resonance Imaging ; Male ; Mitral Valve Insufficiency - enzymology ; Mitral Valve Insufficiency - pathology ; Mitral Valve Insufficiency - prevention & control ; Mucopolysaccharidosis II - enzymology ; Mucopolysaccharidosis II - pathology ; Mucopolysaccharidosis II - therapy ; Mucopolysaccharidosis type II ; Retrospective Studies ; Secondary Prevention ; Survey ; Time ; Treatment Outcome ; Young Adult</subject><ispartof>Molecular genetics and metabolism, 2012-11, Vol.107 (3), p.513-520</ispartof><rights>2012 Elsevier Inc.</rights><rights>Copyright © 2012 Elsevier Inc. All rights reserved.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c392t-b09e443e91128a3c629046525af7b71028e11dc907cdb8c39343d253becbc5843</citedby><cites>FETCH-LOGICAL-c392t-b09e443e91128a3c629046525af7b71028e11dc907cdb8c39343d253becbc5843</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://dx.doi.org/10.1016/j.ymgme.2012.09.004$$EHTML$$P50$$Gelsevier$$H</linktohtml><link.rule.ids>314,780,784,3550,27924,27925,45995</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/23022072$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Tanaka, Akemi</creatorcontrib><creatorcontrib>Okuyama, Torayuki</creatorcontrib><creatorcontrib>Suzuki, Yasuyuki</creatorcontrib><creatorcontrib>Sakai, Norio</creatorcontrib><creatorcontrib>Takakura, Hiromitsu</creatorcontrib><creatorcontrib>Sawada, Tomo</creatorcontrib><creatorcontrib>Tanaka, Toju</creatorcontrib><creatorcontrib>Otomo, Takanobu</creatorcontrib><creatorcontrib>Ohashi, Toya</creatorcontrib><creatorcontrib>Ishige-Wada, Mika</creatorcontrib><creatorcontrib>Yabe, Hiromasa</creatorcontrib><creatorcontrib>Ohura, Toshihiro</creatorcontrib><creatorcontrib>Suzuki, Nobuhiro</creatorcontrib><creatorcontrib>Kato, Koji</creatorcontrib><creatorcontrib>Adachi, Souichi</creatorcontrib><creatorcontrib>Kobayashi, Ryoji</creatorcontrib><creatorcontrib>Mugishima, Hideo</creatorcontrib><creatorcontrib>Kato, Shunichi</creatorcontrib><title>Long-term efficacy of hematopoietic stem cell transplantation on brain involvement in patients with mucopolysaccharidosis type II: A nationwide survey in Japan</title><title>Molecular genetics and metabolism</title><addtitle>Mol Genet Metab</addtitle><description>Hematopoietic stem cell transplantation (HSCT) has not been indicated for patients with mucopolysaccharidosis II (MPS II, Hunter syndrome), while it is indicated for mucopolysaccharidosis I (MPS I) patients <2years of age and an intelligence quotient (IQ) of ≥70. Even after the approval of enzyme replacement therapy for both of MPS I and II, HSCT is still indicated for patients with MPS I severe form (Hurler syndrome). To evaluate the efficacy and benefit of HSCT in MPS II patients, we carried out a nationwide retrospective study in Japan. Activities of daily living (ADL), IQ, brain magnetic resonance image (MRI) lesions, cardiac valvular regurgitation, and urinary glycosaminoglycan (GAG) were analyzed at baseline and at the most recent visit. We also performed a questionnaire analysis about ADL for an HSCT-treated cohort and an untreated cohort (natural history). Records of 21 patients were collected from eight hospitals. The follow-up period in the retrospective study was 9.6±3.5years. ADL was maintained around baseline levels. Cribriform changes and ventricular dilatation on brain MRI were improved in 9/17 and 4/17 patients, respectively. Stabilization of brain atrophy was shown in 11/17 patients. Cardiac valvular regurgitation was diminished in 20/63 valves. Urinary GAG concentration was remarkably lower in HSCT-treated patients than age-matched untreated patients. In the questionnaire analysis, speech deterioration was observed in 12/19 patients in the untreated cohort and 1/7 patient in HSCT-treated cohort. HSCT showed effectiveness towards brain or heart involvement, when performed before signs of brain atrophy or valvular regurgitation appear. We consider HSCT is worthwhile in early stages of the disease for patients with MPS II.
► HSCT showed effectiveness on brain or heart involvements in MPS II patients, when performed before a sign of brain atrophy appear in MRI for brain and before valvular regurgitation appear for heart. ► HSCT showed efficacy on cribriform lesion and ventricular dilatation in MRI of MPS II. ► Cardiac valvular regurgitation was diminished in 32% valves of HSCT-treated patients. ► Urinary GAG in HSCT-treated patients was lower than in ERT-treating patients.</description><subject>Activities of Daily Living</subject><subject>Brain - drug effects</subject><subject>Brain - enzymology</subject><subject>Brain - pathology</subject><subject>Brain efficacy</subject><subject>Child</subject><subject>Child, Preschool</subject><subject>Enzyme Replacement Therapy</subject><subject>Female</subject><subject>Glycosaminoglycans - urine</subject><subject>Health Care Surveys</subject><subject>Hematopoietic Stem Cell Transplantation</subject><subject>Humans</subject><subject>Iduronidase - therapeutic use</subject><subject>Japan</subject><subject>Magnetic Resonance Imaging</subject><subject>Male</subject><subject>Mitral Valve Insufficiency - enzymology</subject><subject>Mitral Valve Insufficiency - pathology</subject><subject>Mitral Valve Insufficiency - prevention & control</subject><subject>Mucopolysaccharidosis II - enzymology</subject><subject>Mucopolysaccharidosis II - pathology</subject><subject>Mucopolysaccharidosis II - therapy</subject><subject>Mucopolysaccharidosis type II</subject><subject>Retrospective Studies</subject><subject>Secondary Prevention</subject><subject>Survey</subject><subject>Time</subject><subject>Treatment Outcome</subject><subject>Young Adult</subject><issn>1096-7192</issn><issn>1096-7206</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2012</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNqFkc2KFDEUhQtRnHH0CQTJ0k2VuUn9RXAxDP60NLjRdUilbk2nqSRlku6hnmZe1fT0jEuFQA7knHPJ_YriLdAKKLQf9tVqby1WjAKrqKgorZ8Vl0BFW3aMts-fNAh2UbyKcU8pQCPql8UF45Qx2rHL4n7r3W2ZMFiC02S00ivxE9mhVckv3mAymsSElmicZ5KCcnGZlUsqGe9IPkNQxhHjjn4-okWXsiZLfs4ykjuTdsQedO6a16i03qlgRh9NJGldkGw2H8k1cQ9td2ZEEg_hiOup47talHtdvJjUHPHN431V_Pry-efNt3L74-vm5npbai5YKgcqsK45CgDWK65bJmjdNqxRUzd0QFmPAKMWtNPj0OcMr_nIGj6gHnTT1_yqeH_uXYL_fcCYpDXx9GXl0B-iBNaxDnoh4P9WYKwFzgXNVn626uBjDDjJJRirwiqByhNEuZcPEOUJoqRCZog59e5xwGGwOP7NPFHLhk9nA-aNHA0GGXVet8bRBNRJjt78c8Af8tqxbA</recordid><startdate>201211</startdate><enddate>201211</enddate><creator>Tanaka, Akemi</creator><creator>Okuyama, Torayuki</creator><creator>Suzuki, Yasuyuki</creator><creator>Sakai, Norio</creator><creator>Takakura, Hiromitsu</creator><creator>Sawada, Tomo</creator><creator>Tanaka, Toju</creator><creator>Otomo, Takanobu</creator><creator>Ohashi, Toya</creator><creator>Ishige-Wada, Mika</creator><creator>Yabe, Hiromasa</creator><creator>Ohura, Toshihiro</creator><creator>Suzuki, Nobuhiro</creator><creator>Kato, Koji</creator><creator>Adachi, Souichi</creator><creator>Kobayashi, Ryoji</creator><creator>Mugishima, Hideo</creator><creator>Kato, Shunichi</creator><general>Elsevier Inc</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>7X8</scope><scope>7QO</scope><scope>7TK</scope><scope>8FD</scope><scope>FR3</scope><scope>P64</scope><scope>RC3</scope></search><sort><creationdate>201211</creationdate><title>Long-term efficacy of hematopoietic stem cell transplantation on brain involvement in patients with mucopolysaccharidosis type II: A nationwide survey in Japan</title><author>Tanaka, Akemi ; Okuyama, Torayuki ; Suzuki, Yasuyuki ; Sakai, Norio ; Takakura, Hiromitsu ; Sawada, Tomo ; Tanaka, Toju ; Otomo, Takanobu ; Ohashi, Toya ; Ishige-Wada, Mika ; Yabe, Hiromasa ; Ohura, Toshihiro ; Suzuki, Nobuhiro ; Kato, Koji ; Adachi, Souichi ; Kobayashi, Ryoji ; Mugishima, Hideo ; Kato, Shunichi</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c392t-b09e443e91128a3c629046525af7b71028e11dc907cdb8c39343d253becbc5843</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2012</creationdate><topic>Activities of Daily Living</topic><topic>Brain - drug effects</topic><topic>Brain - enzymology</topic><topic>Brain - pathology</topic><topic>Brain efficacy</topic><topic>Child</topic><topic>Child, Preschool</topic><topic>Enzyme Replacement Therapy</topic><topic>Female</topic><topic>Glycosaminoglycans - urine</topic><topic>Health Care Surveys</topic><topic>Hematopoietic Stem Cell Transplantation</topic><topic>Humans</topic><topic>Iduronidase - therapeutic use</topic><topic>Japan</topic><topic>Magnetic Resonance Imaging</topic><topic>Male</topic><topic>Mitral Valve Insufficiency - enzymology</topic><topic>Mitral Valve Insufficiency - pathology</topic><topic>Mitral Valve Insufficiency - prevention & control</topic><topic>Mucopolysaccharidosis II - enzymology</topic><topic>Mucopolysaccharidosis II - pathology</topic><topic>Mucopolysaccharidosis II - therapy</topic><topic>Mucopolysaccharidosis type II</topic><topic>Retrospective Studies</topic><topic>Secondary Prevention</topic><topic>Survey</topic><topic>Time</topic><topic>Treatment Outcome</topic><topic>Young Adult</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Tanaka, Akemi</creatorcontrib><creatorcontrib>Okuyama, Torayuki</creatorcontrib><creatorcontrib>Suzuki, Yasuyuki</creatorcontrib><creatorcontrib>Sakai, Norio</creatorcontrib><creatorcontrib>Takakura, Hiromitsu</creatorcontrib><creatorcontrib>Sawada, Tomo</creatorcontrib><creatorcontrib>Tanaka, Toju</creatorcontrib><creatorcontrib>Otomo, Takanobu</creatorcontrib><creatorcontrib>Ohashi, Toya</creatorcontrib><creatorcontrib>Ishige-Wada, Mika</creatorcontrib><creatorcontrib>Yabe, Hiromasa</creatorcontrib><creatorcontrib>Ohura, Toshihiro</creatorcontrib><creatorcontrib>Suzuki, Nobuhiro</creatorcontrib><creatorcontrib>Kato, Koji</creatorcontrib><creatorcontrib>Adachi, Souichi</creatorcontrib><creatorcontrib>Kobayashi, Ryoji</creatorcontrib><creatorcontrib>Mugishima, Hideo</creatorcontrib><creatorcontrib>Kato, Shunichi</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><collection>Biotechnology Research Abstracts</collection><collection>Neurosciences Abstracts</collection><collection>Technology Research Database</collection><collection>Engineering Research Database</collection><collection>Biotechnology and BioEngineering Abstracts</collection><collection>Genetics Abstracts</collection><jtitle>Molecular genetics and metabolism</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Tanaka, Akemi</au><au>Okuyama, Torayuki</au><au>Suzuki, Yasuyuki</au><au>Sakai, Norio</au><au>Takakura, Hiromitsu</au><au>Sawada, Tomo</au><au>Tanaka, Toju</au><au>Otomo, Takanobu</au><au>Ohashi, Toya</au><au>Ishige-Wada, Mika</au><au>Yabe, Hiromasa</au><au>Ohura, Toshihiro</au><au>Suzuki, Nobuhiro</au><au>Kato, Koji</au><au>Adachi, Souichi</au><au>Kobayashi, Ryoji</au><au>Mugishima, Hideo</au><au>Kato, Shunichi</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Long-term efficacy of hematopoietic stem cell transplantation on brain involvement in patients with mucopolysaccharidosis type II: A nationwide survey in Japan</atitle><jtitle>Molecular genetics and metabolism</jtitle><addtitle>Mol Genet Metab</addtitle><date>2012-11</date><risdate>2012</risdate><volume>107</volume><issue>3</issue><spage>513</spage><epage>520</epage><pages>513-520</pages><issn>1096-7192</issn><eissn>1096-7206</eissn><abstract>Hematopoietic stem cell transplantation (HSCT) has not been indicated for patients with mucopolysaccharidosis II (MPS II, Hunter syndrome), while it is indicated for mucopolysaccharidosis I (MPS I) patients <2years of age and an intelligence quotient (IQ) of ≥70. Even after the approval of enzyme replacement therapy for both of MPS I and II, HSCT is still indicated for patients with MPS I severe form (Hurler syndrome). To evaluate the efficacy and benefit of HSCT in MPS II patients, we carried out a nationwide retrospective study in Japan. Activities of daily living (ADL), IQ, brain magnetic resonance image (MRI) lesions, cardiac valvular regurgitation, and urinary glycosaminoglycan (GAG) were analyzed at baseline and at the most recent visit. We also performed a questionnaire analysis about ADL for an HSCT-treated cohort and an untreated cohort (natural history). Records of 21 patients were collected from eight hospitals. The follow-up period in the retrospective study was 9.6±3.5years. ADL was maintained around baseline levels. Cribriform changes and ventricular dilatation on brain MRI were improved in 9/17 and 4/17 patients, respectively. Stabilization of brain atrophy was shown in 11/17 patients. Cardiac valvular regurgitation was diminished in 20/63 valves. Urinary GAG concentration was remarkably lower in HSCT-treated patients than age-matched untreated patients. In the questionnaire analysis, speech deterioration was observed in 12/19 patients in the untreated cohort and 1/7 patient in HSCT-treated cohort. HSCT showed effectiveness towards brain or heart involvement, when performed before signs of brain atrophy or valvular regurgitation appear. We consider HSCT is worthwhile in early stages of the disease for patients with MPS II.
► HSCT showed effectiveness on brain or heart involvements in MPS II patients, when performed before a sign of brain atrophy appear in MRI for brain and before valvular regurgitation appear for heart. ► HSCT showed efficacy on cribriform lesion and ventricular dilatation in MRI of MPS II. ► Cardiac valvular regurgitation was diminished in 32% valves of HSCT-treated patients. ► Urinary GAG in HSCT-treated patients was lower than in ERT-treating patients.</abstract><cop>United States</cop><pub>Elsevier Inc</pub><pmid>23022072</pmid><doi>10.1016/j.ymgme.2012.09.004</doi><tpages>8</tpages></addata></record> |
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| ispartof | Molecular genetics and metabolism, 2012-11, Vol.107 (3), p.513-520 |
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| subjects | Activities of Daily Living Brain - drug effects Brain - enzymology Brain - pathology Brain efficacy Child Child, Preschool Enzyme Replacement Therapy Female Glycosaminoglycans - urine Health Care Surveys Hematopoietic Stem Cell Transplantation Humans Iduronidase - therapeutic use Japan Magnetic Resonance Imaging Male Mitral Valve Insufficiency - enzymology Mitral Valve Insufficiency - pathology Mitral Valve Insufficiency - prevention & control Mucopolysaccharidosis II - enzymology Mucopolysaccharidosis II - pathology Mucopolysaccharidosis II - therapy Mucopolysaccharidosis type II Retrospective Studies Secondary Prevention Survey Time Treatment Outcome Young Adult |
| title | Long-term efficacy of hematopoietic stem cell transplantation on brain involvement in patients with mucopolysaccharidosis type II: A nationwide survey in Japan |
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