{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,22]],"date-time":"2025-10-22T03:01:15Z","timestamp":1761102075672},"reference-count":32,"publisher":"FapUNIFESP (SciELO)","issue":"suppl 1","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Cad. Sa\u00fade P\u00fablica"],"published-print":{"date-parts":[[2013]]},"abstract":"<jats:p>A mucopolissacaridose tipo II (MPS II) \u00e9 uma doen\u00e7a gen\u00e9tica de amplo espectro cl\u00ednico, caracterizada por defici\u00eancia da enzima iduronato-2sulfatase. Revis\u00e3o sistem\u00e1tica avaliou a efic\u00e1cia e seguran\u00e7a da terapia de reposi\u00e7\u00e3o enzim\u00e1tica (TRE) com idursulfase (IDS) na MPS II. As bases de dados PubMed\/MEDLINE, Embase, LILACS e Biblioteca Cochrane foram pesquisados at\u00e9 30 de novembro de 2012. Apenas cinco estudos preencheram os crit\u00e9rios de inclus\u00e3o (ensaios cl\u00ednicos randomizados - ECRs, ECRs abertos ou s\u00e9ries de caso prospectivas, incluindo cinco ou mais pacientes e avaliando desfechos relevantes). Metan\u00e1lise foi realizada para capacidade vital for\u00e7ada (CVF; valores absolutos e em %) e para a dist\u00e2ncia percorrida no teste da caminhada dos seis minutos, com mudan\u00e7as significativas em ambas as vari\u00e1veis; tamb\u00e9m foi encontrado risco aumentado de rea\u00e7\u00f5es leves relacionadas \u00e0 infus\u00e3o e de desenvolvimento de anticorpos IgG \u00e0 IDS. Em face dos dados apresentados neste estudo, conclui-se que a TRE com IDS \u00e9 segura e tem benef\u00edcio potencial em MPS II, mas estudos adicionais s\u00e3o necess\u00e1rios.<\/jats:p>","DOI":"10.1590\/0102-311x00017613","type":"journal-article","created":{"date-parts":[[2013,11,11]],"date-time":"2013-11-11T16:58:24Z","timestamp":1384189104000},"page":"s45-s58","source":"Crossref","is-referenced-by-count":4,"title":["Efic\u00e1cia e seguran\u00e7a da terapia com idursulfase em pacientes com mucopolissacaridose tipo II, com e sem compara\u00e7\u00e3o com placebo: revis\u00e3o sistem\u00e1tica e metan\u00e1lise"],"prefix":"10.1590","volume":"29","author":[{"given":"Taciane","family":"Alegra","sequence":"first","affiliation":[{"name":"Universidade Federal do Rio Grande do Sul,  Brasil"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Dauana Pitano","family":"Eizerik","sequence":"additional","affiliation":[{"name":"Universidade Federal do Rio Grande do Sul,  Brasil"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Caio C\u00e9sar Silva de","family":"Cerqueira","sequence":"additional","affiliation":[{"name":"Universidade Federal do Rio Grande do Sul,  Brasil"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tiago V.","family":"Pereira","sequence":"additional","affiliation":[{"name":"Hospital Alem\u00e3o Oswaldo Cruz,  Brasil; Universidade de S\u00e3o Paulo,  Brasil"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Al\u00edcia Dorneles","family":"Dornelles","sequence":"additional","affiliation":[{"name":"Universidade Federal do Rio Grande do Sul,  Brasil"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ida Vanessa Doederlein","family":"Schwartz","sequence":"additional","affiliation":[{"name":"Universidade Federal do Rio Grande do Sul,  Brasil; Universidade Federal do Rio Grande do Sul,  Brasil; Universidade Federal do Rio Grande do Sul,  Brasil"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"530","reference":[{"key":"ref1","series-title":"The metabolic and molecular bases of inherited disease","first-page":"3421","volume-title":"The mucopolysaccharidoses","author":"Neufeld EF","year":"2001"},{"key":"ref2","doi-asserted-by":"crossref","first-page":"63","DOI":"10.1111\/j.1651-2227.2007.00212.x","article-title":"A clinical study of 77 patients with mucopolysaccharidosis type II","volume":"96","author":"Schwartz IV","year":"2007","journal-title":"Acta Paediatr Suppl","ISSN":"http:\/\/id.crossref.org\/issn\/0803-5326","issn-type":"print"},{"key":"ref3","series-title":"GeneReviews","volume-title":"Mucopolysaccharidosis type II","author":"Scarpa M","year":"2007"},{"key":"ref4","doi-asserted-by":"crossref","first-page":"76","DOI":"10.1111\/j.1651-2227.2008.00661.x","article-title":"Enzyme replacement therapy with idursulfase in patients with mucopolysaccharidosis type II","volume":"97","author":"Wraith JE","year":"2008","journal-title":"Acta Paediatr Suppl","ISSN":"http:\/\/id.crossref.org\/issn\/0803-5326","issn-type":"print"},{"key":"ref5","doi-asserted-by":"crossref","first-page":"732","DOI":"10.1007\/s10545-009-1275-9","article-title":"Clinical and biochemical studies in mucopolysaccharidosis type II carriers","volume":"32","author":"Schwartz IVD","year":"2009","journal-title":"J Inherit Metab Dis","ISSN":"http:\/\/id.crossref.org\/issn\/0141-8955","issn-type":"print"},{"key":"ref6","first-page":"50","article-title":"Are MPS II heterozygotes actually asymptomatic: A study based on clinical and biochemical data, X-inactivation analysis and imaging evaluations","volume":"155A","author":"de Camargo Pinto LL","year":"2001","journal-title":"Am J Med Genet A"},{"key":"ref7","doi-asserted-by":"crossref","first-page":"S8","DOI":"10.1590\/S0021-75572008000500003","article-title":"Tratamento de erros inatos do metabolismo","volume":"84","author":"Schwartz IV","year":"2008","journal-title":"J Pediatr (Rio J)","ISSN":"http:\/\/id.crossref.org\/issn\/0021-7557","issn-type":"print"},{"key":"ref8","series-title":"Cochrane handbook for systematic reviews of interventions version 5.0.2","author":"Higgins JPT","year":"2011"},{"key":"ref9","doi-asserted-by":"crossref","first-page":"2","DOI":"10.1136\/ebm.11.1.2-a","article-title":"An emerging consensus on grading recommendations","volume":"11","author":"Guyatt G","year":"2006","journal-title":"Evid Based Med","ISSN":"http:\/\/id.crossref.org\/issn\/1356-5524","issn-type":"print"},{"key":"ref10","doi-asserted-by":"crossref","first-page":"924","DOI":"10.1136\/bmj.39489.470347.AD","article-title":"GRADE: an emerging consensus on rating quality of evidence and strength of recommendations","volume":"336","author":"Guyatt GH","year":"2008","journal-title":"BMJ","ISSN":"http:\/\/id.crossref.org\/issn\/0959-8138","issn-type":"print"},{"key":"ref11","doi-asserted-by":"crossref","first-page":"380","DOI":"10.1016\/j.jclinepi.2010.09.011","article-title":"GRADE guidelines: a new series of articles in the Journal of Clinical Epidemiology","volume":"64","author":"Guyatt GH","year":"2011","journal-title":"J Clin Epidemiol","ISSN":"http:\/\/id.crossref.org\/issn\/0895-4356","issn-type":"print"},{"key":"ref12","doi-asserted-by":"crossref","first-page":"401","DOI":"10.1016\/j.jclinepi.2010.07.015","article-title":"GRADE guidelines: 3. 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