Treatment for Alpha-1 Antitrypsin Deficiency: Does Augmentation Therapy Work?

Affiliations

Editorial

Mark Brantly. Am J Respir Crit Care Med. 2023.

. 2023 Nov 1;208(9):948-949.

doi: 10.1164/rccm.202309-1585ED.

¹ Division of Pulmonary, Critical Care and Sleep Medicine University of Florida College of Medicine Gainesville, Florida.

No abstract available

Comment on

Augmentation Therapy for Severe Alpha-1 Antitrypsin Deficiency Improves Survival and Is Decoupled from Spirometric Decline-A Multinational Registry Analysis.
Fraughen DD, Ghosh AJ, Hobbs BD, Funk GC, Meischl T, Clarenbach CF, Sievi NA, Schmid-Scherzer K, McElvaney OJ, Murphy MP, Roche AD, Clarke L, Strand M, Vafai-Tabrizi F, Kelly G, Gunaratnam C, Carroll TP, McElvaney NG. Fraughen DD, et al. Am J Respir Crit Care Med. 2023 Nov 1;208(9):964-974. doi: 10.1164/rccm.202305-0863OC. Am J Respir Crit Care Med. 2023. PMID: 37624745 Free PMC article.

1. Abboud RT, Ford GT, Chapman KR. Emphysema in alpha1-antitrypsin deficiency: does replacement therapy affect outcome? Treat Respir Med . 2005;4:1–8. - PubMed
1. Greene CM, Marciniak SJ, Teckman J, Ferrarotti I, Brantly ML, Lomas DA, et al. α1-Antitrypsin deficiency. Nat Rev Dis Primers . 2016;2:16051. - PubMed
1. Blanco I, Diego I, Castañón C, Bueno P, Miravitlles M. Estimated worldwide prevalence of the PI*ZZ alpha-1 antitrypsin genotype in subjects with chronic obstructive pulmonary disease. Arch Bronconeumol . 2023;59:427–434. - PubMed
1. Tonelli AR, Brantly ML. Augmentation therapy in alpha-1 antitrypsin deficiency: advances and controversies. Ther Adv Respir Dis . 2010;4:289–312. - PubMed
1. Wewers MD, Casolaro MA, Sellers SE, Swayze SC, McPhaul KM, Wittes JT, et al. Replacement therapy for alpha 1-antitrypsin deficiency associated with emphysema. N Engl J Med . 1987;316:1055–1062. - PubMed