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Human α1-proteinase inhibitor (Respreeza®) in α1-antitrypsin deficiency emphysema: a profile of its use in the EU

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Abstract

Augmentation therapy with human α1-proteinase inhibitor (Respreeza®) is an effective and generally well tolerated treatment option for patients with severe α1-antitrypsin deficiency. In the 2-year RAPID trial, intravenous Respreeza 60 mg/kg once weekly was associated with a trend towards reduced mean annual rate of lung density loss (as measured by computed tomography) at total lung capacity (TLC) and functional residual capacity (FRC) combined, and at FRC alone, with a significant reduction seen at TLC alone. The continued efficacy of Respreeza in slowing the progression of emphysema was demonstrated in a 2-year open-label extension of the RAPID trial. Although lung density loss was slowed when treatment was initiated later in the disease course, lung density loss prior to treatment was not recovered.

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Acknowledgements

The manuscript was updated from Drugs & Therapy Perspectives 2016;32(10):422–7 [29], and was reviewed by: J. Burdon, St Vincent’s Private Hospital, Melbourne, VIC, Australia; K. R. Chapman, Asthma & Airway Centre, University Health Network (Toronto Western Hospital), Toronto, ON, Canada; C. E. Green, Institute of Inflammation and Ageing, University of Birminghan, Birmingham, UK; R. A. Stockley, Lung Investigation Unit Medicine—University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital, Birmingham, UK. During the peer review process, the marketing-authorization holder of Respreeza® was also offered an opportunity to provide a scientific accuracy review of their data. Changes resulting from comments received were made on the basis of scientific and editorial merit.

Additional information for this Adis Drug Q&A can be found at https://doi.org/10.6084/m9.figshare.8282129.

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    Hannah A. Blair

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Correspondence to Hannah A. Blair.

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H. A. Blair is an employee of Adis International Ltd/Springer Nature, is responsible for the article content and declares no conflicts of interest.

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The preparation of this review was not supported by any external funding.

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Blair, H.A. Human α1-proteinase inhibitor (Respreeza®) in α1-antitrypsin deficiency emphysema: a profile of its use in the EU. Drugs Ther Perspect 35, 363–368 (2019). https://doi.org/10.1007/s40267-019-00648-2

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