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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

2021-11Journal article Open access
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dc.contributor.authorHerth, Felix JF
dc.contributor.authorSandhaus, Robert A
dc.contributor.authorTurner, Alice M
dc.contributor.authorSucena, Maria
dc.contributor.authorWelte, Tobias
dc.contributor.authorGreulich, Timm
dc.date.accessioned2023-10-31T11:44:39Z
dc.date.available2023-10-31T11:44:39Z
dc.date.issued2021-11
dc.description.abstractAlpha 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.pt_PT
dc.description.versioninfo:eu-repo/semantics/publishedVersionpt_PT
dc.identifier.citationHerth FJF, Sandhaus RA, Turner AM, Sucena M, Welte T, Greulich T. 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. Int J Chron Obstruct Pulmon Dis. 2021;16:2983-2996. doi:10.2147/COPD.S325211pt_PT
dc.identifier.doi10.2147/COPD.S325211pt_PT
dc.identifier.issn1176-9106
dc.identifier.issn1178-2005
dc.identifier.urihttp://hdl.handle.net/10400.16/2867
dc.language.isoengpt_PT
dc.peerreviewedyespt_PT
dc.publisherDove Medical Presspt_PT
dc.relation.publisherversionhttps://www.dovepress.com/alpha-1-antitrypsin-therapy-in-patients-with-alpha-1-antitrypsin-defic-peer-reviewed-fulltext-article-COPDpt_PT
dc.rights.urihttp://creativecommons.org/licenses/by-nc/4.0/pt_PT
dc.subjectAlpha 1 Antitrypsinpt_PT
dc.subjectAlpha 1 Antitrypsin deficiencypt_PT
dc.subjectCOVID-19pt_PT
dc.subjectefficacypt_PT
dc.subjectself-administrationpt_PT
dc.titleAlpha 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 Pandemicpt_PT
dc.typejournal article
dspace.entity.typePublication
oaire.citation.conferencePlaceNew Zealandpt_PT
oaire.citation.endPage2996pt_PT
oaire.citation.startPage2983pt_PT
oaire.citation.titleInternational Journal of Chronic Obstructive Pulmonary Diseasept_PT
oaire.citation.volumeVolume 16pt_PT
person.familyNameCampos Miranda Duarte Sucena
person.givenNameMaria
person.identifier.ciencia-id5216-37E8-6966
person.identifier.orcid0000-0002-0115-7080
rcaap.rightsopenAccesspt_PT
rcaap.typearticlept_PT
relation.isAuthorOfPublication4968b345-427f-4b1c-8eae-60d6e3d77fe2
relation.isAuthorOfPublication.latestForDiscovery4968b345-427f-4b1c-8eae-60d6e3d77fe2

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