Alpha-1 Antitrypsin Replacement Improves Walking Capacity in Subjects With Severe Deficiency.

This article focuses on evaluating the clinical and functional effects of alpha-1 antitrypsin replacement therapy in subjects with severe alpha-1 antitrypsin deficiency, a genetic disorder linked to pulmonary emphysema and chronic obstructive pulmonary disease (COPD). In a retrospective study of sev...

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Publicado en:Respiratory Care Vol. 71; no. 5; pp. 496 - 501
Autores principales: Marchese, Alessandra, Frizzelli, Annalisa, De Simoni, Alessandro, Bondarenko, Olha, Pisi, Roberta, Aluia, Agnese, Pelà, Giovanna, Ledda, Roberta Eufrasia, Caramori, Gaetano, Chetta, Alfredo, Aiello, Marina
Formato: research tables/charts Journal Article
Publicado: Mary Ann Liebert, Inc. May2026
Acceso en línea:Ver este registro en EBSCOhost
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        atl: Alpha-1 Antitrypsin Replacement Improves Walking Capacity in Subjects With Severe Deficiency.
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          Marchese, Alessandra
          Frizzelli, Annalisa
          De Simoni, Alessandro
          Bondarenko, Olha
          Pisi, Roberta
          Aluia, Agnese
          Pelà, Giovanna
          Ledda, Roberta Eufrasia
          Caramori, Gaetano
          Chetta, Alfredo
          Aiello, Marina
        affil: Drs. Marchese, Frizzelli, De Simoni, Bondarenko, Pisi, Aluia, Prof. Caramori, Professors Chetta and Aiello are affiliated with Division of Pneumology, Department of Medicine and Surgery, University of Parma, Parma, Italy.
      sug:
        subj:
          alpha 1-Antitrypsin Deficiency Therapy
          Walking
          Genotype
          Lung Physiology
          Disease Exacerbation
          Dyspnea
          Outcomes (Health Care)
          Human Italy
          Italy
          Male
          Female
          Adult
          Middle Age
          Aged
          Retrospective Design
          Record Review
          Descriptive Statistics
          Paired T-Tests
          Data Analysis Software
          Adult: 19-44 years
          Middle Aged: 45-64 years
          Aged: 65+ years
          Male
          Female
      ab: This article focuses on evaluating the clinical and functional effects of alpha-1 antitrypsin replacement therapy in subjects with severe alpha-1 antitrypsin deficiency, a genetic disorder linked to pulmonary emphysema and chronic obstructive pulmonary disease (COPD). In a retrospective study of seven patients with both common and rare severe genotypes, at least 12 months of weekly intravenous augmentation therapy significantly improved quality of life, dyspnea (breathlessness), and six-minute walk test (6MWT) distance, while reducing the frequency of bronchial exacerbations. However, no significant changes were observed in standard lung function parameters or arterial blood gases. The study highlights potential benefits of replacement therapy on physical fitness and symptom relief but notes limitations including a small sample size and lack of a control group, suggesting the need for further research.
      pubtype: Academic Journal
      doctype:
        research
        tables/charts
        Journal Article
      ougenre: Article
    language: English
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