Artigo de Revisão/Review Article
Défice de alfa-1 antitripsina. A experiência do Hospital de Pulido Valente com a terapêutica de reposição
Alpha-1 antitrypsin deficiency. The experience of Pulido Valente Hospital with augmentation therapy
1 Interna do Internato Complementar de Pneumologia, Hospital de Dia de Insuficientes Respiratórios; Unidade de Reabilitação Respiratória, Responsável: Fátima Rodrigues Departamento de Pneumologia do Hospital de Pulido Valente, CHLN, EPE
2 Assistente Hospitalar Graduada de Pneumologia, Hospital de Dia de Insuficientes Respiratórios; Unidade de Reabilitação Respiratória, Responsável: Fátima Rodrigues Departamento de Pneumologia do Hospital de Pulido Valente, CHLN, EPE
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A experiência do Hospital de Pulido Valente com a terapêutica de reposição" "tieneTextoCompleto" => 0 "paginas" => array:1 [ 0 => array:2 [ "paginaInicial" => "473" "paginaFinal" => "482" ] ] "autores" => array:1 [ 0 => array:3 [ "autoresLista" => "Carla Alves Costa, Cristina Santos" "autores" => array:2 [ 0 => array:3 [ "nombre" => "Carla Alves" "apellidos" => "Costa" "referencia" => array:1 [ 0 => array:2 [ "etiqueta" => "<span class="elsevierStyleSup">1</span>" "identificador" => "af0005" ] ] ] 1 => array:3 [ "nombre" => "Cristina" "apellidos" => "Santos" "referencia" => array:1 [ 0 => array:2 [ "etiqueta" => "<span class="elsevierStyleSup">2</span>" "identificador" => "af0010" ] ] ] ] "afiliaciones" => array:2 [ 0 => array:3 [ "entidad" => "Interna do Internato Complementar de Pneumologia, Hospital de Dia de Insuficientes Respiratórios; Unidade de Reabilitação Respiratória, Responsável: Fátima Rodrigues Departamento de Pneumologia do Hospital de Pulido Valente, CHLN, EPE" "etiqueta" => "1" "identificador" => "af0005" ] 1 => array:3 [ "entidad" => "Assistente Hospitalar Graduada de Pneumologia, Hospital de Dia de Insuficientes Respiratórios; Unidade de Reabilitação Respiratória, Responsável: Fátima Rodrigues Departamento de Pneumologia do Hospital de Pulido Valente, CHLN, EPE" "etiqueta" => "2" "identificador" => "af0010" ] ] ] ] "titulosAlternativos" => array:1 [ "en" => array:1 [ "titulo" => "Alpha-1 antitrypsin deficiency. The experience of Pulido Valente Hospital with augmentation therapy" ] ] "pdfFichero" => "main.pdf" "tienePdf" => true "fechaRecibido" => "2008-09-24" "fechaAceptado" => "2008-12-31" "PalabrasClave" => array:2 [ "pt" => array:1 [ 0 => array:4 [ "clase" => "keyword" "titulo" => "Palavras-chave" "identificador" => "xpalclavsec570276" "palabras" => array:3 [ 0 => "lfa-1 antitripsina" 1 => "terapêutica de reposição" 2 => "enfisema" ] ] ] "en" => array:1 [ 0 => array:4 [ "clase" => "keyword" "titulo" => "Key-words" "identificador" => "xpalclavsec570277" "palabras" => array:3 [ 0 => "Alpha-1 antitrypsin" 1 => "augmentation therapy" 2 => "emphysema" ] ] ] ] "tieneResumen" => true "resumen" => array:2 [ "pt" => array:2 [ "titulo" => "Resumo" "resumen" => "<span id="as0005" class="elsevierStyleSection elsevierViewall"><p id="sp0005" class="elsevierStyleSimplePara elsevierViewall">A alfa-1 antitripsina (AAT) é sintetizada pelo fíga-do, com uma semivida plasmática de 4-5 dias. Apresenta acção inibidora das proteases, com particular afinidade para a elastase dos neutrófilos. A sua deficiência está associada a uma menor protecção pulmonar da acção das enzimas dos neutrófilos activados.</p><p id="sp0010" class="elsevierStyleSimplePara elsevierViewall">A deficiência de AAT é uma doença genética resultante da herança de dois alelos deficientes. Dos alelos deficientes, o mais frequente é o Pi*Z, sendo a forma homozigótica Pi*ZZ responsável por níveis séricos mais baixos, habitualmente inferiores a 50<span class="elsevierStyleHsp" style=""></span>mg/dl. O limiar de protecção é 80<span class="elsevierStyleHsp" style=""></span>mg/dl. O ta-bagismo aumenta francamente o risco de enfisema nestes doentes.</p><p id="sp0015" class="elsevierStyleSimplePara elsevierViewall">O objectivo da terapêutica de reposição é a manuten-ção de níveis séricos de AAT acima do limiar protector, retardando a progressão da doença.</p><p id="sp0020" class="elsevierStyleSimplePara elsevierViewall">Os autores apresentam a experiência do Hospital de Dia de Insuficientes Respiratórios do Hospital de Pulido Valente, de cinco doentes com enfisema por deficiência de AAT, fazendo reposição endovenosa se-manal com prolastina®.</p><p id="sp0025" class="elsevierStyleSimplePara elsevierViewall">Foi efectuada uma avaliação clínica, funcional respiratória e radiológica dos doentes entre 2003 e 2007. Verificou-se estabilidade clínica e radiológica e menor declínio anual de FEV<span class="elsevierStyleInf">1</span> após início do tratamento.</p><p id="sp0030" class="elsevierStyleSimplePara elsevierViewall">A reposição com prolastina® é um tratamento de custos elevados, havendo falta de estudos aleatorizados e controlados que demonstrem a sua eficácia clínica. A evidência do benefício é baseada em estudos observacionais. A nossa <span class="elsevierStyleBold">experiência é positiva, com benefícios clínicos, funcionais e radiológicos</span>. Apesar de estar descrita na literatura uma redução da mortalida-de, ainda não foi possível fazer essa inferência na nossa pequena amostra.</p><p id="sp0035" class="elsevierStyleSimplePara elsevierViewall"><span class="elsevierStyleBold">Rev Port Pneumol 2009; XV (3): 473-481</span></p></span>" ] "en" => array:2 [ "titulo" => "Abstract" "resumen" => "<span id="as0010" class="elsevierStyleSection elsevierViewall"><p id="sp0040" class="elsevierStyleSimplePara elsevierViewall">Alpha-1 antitrypsin (AAT) is synthesised in the liver and has half-life of 4-5 days. AAT has antiprotease activity, with particular affinity for neutrophil elastase. Its deficiency leads to a lack of effective lung protection against activated neutrophil enzymes.</p><p id="sp0045" class="elsevierStyleSimplePara elsevierViewall">Deficiency of AAT is a genetic disorder that occurs as a result of the inheritance of two protease inhibitor deficient alleles. Of the deficient alleles, Pi*Z is the most common, and the homozygous form Pi*ZZ results in the lowest serum levels, usually below 50<span class="elsevierStyleHsp" style=""></span>mg/ dl. The “protective threshold” is 80<span class="elsevierStyleHsp" style=""></span>mg/dl. Smoking increases the risk of emphysema.</p><p id="sp0050" class="elsevierStyleSimplePara elsevierViewall">The current goal of augmentation therapy is to raise the plasma levels, above protective threshold and slow disease progression.</p><p id="sp0055" class="elsevierStyleSimplePara elsevierViewall">The authors present the experience of the Day Care Hospital of the Pulido Valente Hospital with five male patients presenting emphysema due to AAT deficiency, receiving weekly intravenous treatment with Prolastin®. We performed a clinical, respiratory functional and radiological evaluation between 2003 and 2007.</p><p id="sp0060" class="elsevierStyleSimplePara elsevierViewall">The results point to a slower progression of the disease, with clinical and radiological stability and a reduced rate of FEV<span class="elsevierStyleInf">1</span> decline.</p><p id="sp0065" class="elsevierStyleSimplePara elsevierViewall">Augmentation therapy is an expensive treatment and its use is lacking supportive evidence of efficacy by randomized controlled clinical trials. Evidence that it confers benefits is based on observational studies. <span class="elsevierStyleBold">Our experience is positive, showing clinical, radiological and functional benefits</span>. The literature available points to a decrease in mortality, but we could not affirm so in our small population.</p><p id="sp0070" class="elsevierStyleSimplePara elsevierViewall"><span class="elsevierStyleBold">Rev Port Pneumol 2009; XV (3): 473-481</span></p></span>" ] ] "lecturaRecomendada" => array:1 [ 0 => array:3 [ "vista" => "all" "titulo" => "<span class="elsevierStyleSectionTitle" id="st0025">Bibliografia</span>" "seccion" => array:1 [ 0 => array:2 [ "vista" => "all" "bibliografiaReferencia" => array:16 [ 0 => array:3 [ "identificador" => "bb0005" "etiqueta" => "1." "referencia" => array:1 [ 0 => array:2 [ "contribucion" => array:1 [ 0 => array:2 [ "titulo" => "Alpha-1 antitrypsin deficiency – Clinical aspects and management" "autores" => array:1 [ 0 => array:2 [ "etal" => false "autores" => array:2 [ 0 => "K. Thomas" 1 => "W. 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"referencia" => array:1 [ 0 => array:2 [ "contribucion" => array:1 [ 0 => array:2 [ "titulo" => "Replacement therapy for alpha-1 antitrypsin deficiency associated with emphysema" "autores" => array:1 [ 0 => array:2 [ "etal" => false "autores" => array:7 [ 0 => "M. Wewers" 1 => "M.A. Casolaro" 2 => "S.E. Sellers" 3 => "S.C. Swazye" 4 => "K.M. McPhaul" 5 => "J.T. Wittes" 6 => "R.G. Crystal" ] ] ] ] ] "host" => array:1 [ 0 => array:2 [ "doi" => "10.1056/NEJM198704233161704" "Revista" => array:6 [ "tituloSerie" => "N Engl J Med" "fecha" => "1987" "volumen" => "316" "paginaInicial" => "1055" "paginaFinal" => "1062" "link" => array:1 [ 0 => array:2 [ "url" => "https://www.ncbi.nlm.nih.gov/pubmed/3494198" "web" => "Medline" ] ] ] ] ] ] ] ] 13 => array:3 [ "identificador" => "bb0070" "etiqueta" => "14." "referencia" => array:1 [ 0 => array:2 [ "contribucion" => array:1 [ 0 => array:2 [ "titulo" => "Treatment of alpha-1-antitrypsin deficiency" "autores" => array:1 [ 0 => array:2 [ "etal" => false "autores" => array:2 [ 0 => "S.M. Rovner" 1 => "J.K. Stoller" ] ] ] ] ] "host" => array:1 [ 0 => array:1 [ "WWW" => array:2 [ "link" => "www.uptodate.com" "fecha" => "2008" ] ] ] ] ] ] 14 => array:3 [ "identificador" => "bb0075" "etiqueta" => "15." "referencia" => array:1 [ 0 => array:2 [ "contribucion" => array:1 [ 0 => array:2 [ "titulo" => "High-resoluion computed tomography scanning in α-1 antitrypsin deficiency: relationship to lung function and health status" "autores" => array:1 [ 0 => array:2 [ "etal" => false "autores" => array:5 [ 0 => "L.J. Dowson" 1 => "P.J. Guest" 2 => "S.L. Hill" 3 => "R.L. Holder" 4 => "R.A. Stockley" ] ] ] ] ] "host" => array:1 [ 0 => array:1 [ "Revista" => array:6 [ "tituloSerie" => "Eur Respir J" "fecha" => "2001" "volumen" => "17" "paginaInicial" => "1097" "paginaFinal" => "1104" "link" => array:1 [ 0 => array:2 [ "url" => "https://www.ncbi.nlm.nih.gov/pubmed/11491150" "web" => "Medline" ] ] ] ] ] ] ] ] 15 => array:3 [ "identificador" => "bb0080" "etiqueta" => "17." "referencia" => array:1 [ 0 => array:2 [ "contribucion" => array:1 [ 0 => array:1 [ "autores" => array:1 [ 0 => array:2 [ "etal" => false "autores" => array:1 [ 0 => "Prolastin®, human alpha-1 antitrypsin" ] ] ] ] ] "host" => array:1 [ 0 => array:1 [ "Libro" => array:3 [ "titulo" => "Product Monograph" "fecha" => "2007" "editorial" => "Talecris Biotherapeutics GmbH" ] ] ] ] ] ] ] ] ] ] ] ] "idiomaDefecto" => "pt" "url" => "/08732159/0000001500000003/v1_201509151500/S0873215915301471/v1_201509151500/pt/main.assets" "Apartado" => array:4 [ "identificador" => "43356" "tipo" => "SECCION" "pt" => array:2 [ "titulo" => "Artigos de revisão" "idiomaDefecto" => true ] "idiomaDefecto" => "pt" ] "PDF" => "https://static.elsevier.es/multimedia/08732159/0000001500000003/v1_201509151500/S0873215915301471/v1_201509151500/pt/main.pdf?idApp=UINPBA00004E&text.app=https://journalpulmonology.org/" "EPUB" => "https://multimedia.elsevier.es/PublicationsMultimediaV1/item/epub/S0873215915301471?idApp=UINPBA00004E" ]
Article information
ISSN: 08732159Original language: Portuguese
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