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Alpha 1 Antitrypsin Therapy in Patients with Alpha 1 Antitrypsin Deficiency: Perspectives from a Registry Study and Practical Considerations for Self-Administration During the COVID-19 Pandemic.
Herth, Felix J F; Sandhaus, Robert A; Turner, Alice M; Sucena, Maria; Welte, Tobias; Greulich, Timm.
Afiliación
  • Herth FJF; Department of Pneumology and Critical Care Medicine, University of Heidelberg, Heidelberg, Germany.
  • Sandhaus RA; Division of Pulmonary, Critical Care and Sleep Medicine, National Jewish Health, Denver, CO, USA.
  • Turner AM; Institute of Applied Health Research, University of Birmingham, Birmingham, England.
  • Sucena M; Pulmonology Department, Centro Hospitalar Universitário do Porto, Porto, Portugal.
  • Welte T; Department of Pulmonary and Infectious Diseases, Hannover Medical School, Hannover, Germany.
  • Greulich T; Department of Internal Medicine and Pneumology, University Hospital Marburg, Marburg, Germany.
Int J Chron Obstruct Pulmon Dis ; 16: 2983-2996, 2021.
Article en En | MEDLINE | ID: mdl-34754184
Alpha 1 Antitrypsin deficiency (AATD) is a hereditary condition characterized by low serum Alpha 1 Antitrypsin (AAT) levels and a predisposition towards early-onset emphysema. Infusion of AAT is the only disease-modifying therapy that can sufficiently raise plasma AAT levels above the putative protective threshold and reduce the decline in lung density loss. Several randomized controlled trials (RCTs) and registry studies support the clinical efficacy of AAT therapy in slowing the progression of AATD-related emphysema and improving survival outcomes. The COVID-19 pandemic has prompted physicians to develop additional strategies for delivering AAT therapy, which are not only more convenient for the patient, but are "COVID-19 friendly", thereby reducing the risk of exposing these vulnerable patients. Intravenous (IV) self-administration of AAT therapy is likely to be beneficial in certain subgroups of patients with AATD and can remove the need for weekly hospital visits, thereby improving independence and well-being. Increasing the awareness of self-administration in AATD through the development of formal guidelines and training programs is required among both physicians and patients and will play an essential role, especially post-COVID-19, in encouraging physicians to consider self-administration for AATD in suitable patients. This review summarizes the benefits of AAT therapy on the clinical endpoints of mortality and quality of life (QoL) and discusses the benefits of self-administration therapy compared with conventional therapy administered by a healthcare professional. In addition, this review highlights the challenges of providing AAT therapy during the COVID-19 pandemic and the potential considerations for its implementation thereafter.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Deficiencia de alfa 1-Antitripsina / Enfermedad Pulmonar Obstructiva Crónica / COVID-19 Tipo de estudio: Clinical_trials / Diagnostic_studies / Guideline Aspecto: Patient_preference Límite: Humans Idioma: En Revista: Int J Chron Obstruct Pulmon Dis Año: 2021 Tipo del documento: Article País de afiliación: Alemania Pais de publicación: Nueva Zelanda
Texto completo
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XML
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Deficiencia de alfa 1-Antitripsina / Enfermedad Pulmonar Obstructiva Crónica / COVID-19 Tipo de estudio: Clinical_trials / Diagnostic_studies / Guideline Aspecto: Patient_preference Límite: Humans Idioma: En Revista: Int J Chron Obstruct Pulmon Dis Año: 2021 Tipo del documento: Article País de afiliación: Alemania Pais de publicación: Nueva Zelanda