Lung transplant outcomes for recipients with alpha-1 antitrypsin deficiency, by use of alpha-1 antitrypsin augmentation therapy
Background: For patients with alpha-1 antitrypsin (AAT) deficiency, AAT augmentation therapy can be an important part of care. However, for those who require a lung transplant (LT), there is currently only limited information to guide the use of AAT augmentation therapy post-LT. Methods: We identifi...
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Elsevier
2025-02-01
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| Series: | JHLT Open |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S2950133424001514 |
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| author | Atharv V. Oak, MEng Jessica M. Ruck, MD, PhD Alfred J. Casillan, MD, PhD Armaan F. Akbar, BS Ramon A. Riojas, MD, PhD Pali D. Shah, MD Jinny S. Ha, MD, MHS Sara Strout, PharmD Allan B. Massie, PhD Dorry L. Segev, MD, PhD Christian A. Merlo, MD, MPH Errol L. Bush, MD |
| author_facet | Atharv V. Oak, MEng Jessica M. Ruck, MD, PhD Alfred J. Casillan, MD, PhD Armaan F. Akbar, BS Ramon A. Riojas, MD, PhD Pali D. Shah, MD Jinny S. Ha, MD, MHS Sara Strout, PharmD Allan B. Massie, PhD Dorry L. Segev, MD, PhD Christian A. Merlo, MD, MPH Errol L. Bush, MD |
| author_sort | Atharv V. Oak, MEng |
| collection | DOAJ |
| description | Background: For patients with alpha-1 antitrypsin (AAT) deficiency, AAT augmentation therapy can be an important part of care. However, for those who require a lung transplant (LT), there is currently only limited information to guide the use of AAT augmentation therapy post-LT. Methods: We identified all LT recipients from 2011-2021 in the Scientific Registry of Transplant Recipients with an AAT deficiency diagnosis. We categorized recipients by use of AAT augmentation therapy post-LT and compared their baseline characteristics using Fisher’s exact test and Wilcoxon rank-sum tests. We used Kaplan-Meier analyses and estimated the average treatment effect (ATE) of post-LT AAT augmentation therapy on mortality and all-cause graft failure (ACGF). The ATE measures the observed effect we would see if everyone in the population received the intervention as opposed to just a subset. Results: Among the 447 recipients with AAT deficiency, 109 used AAT augmentation therapy pre-LT, of which 32 (29.4%) continued post-LT. Recipients who used augmentation therapy post-LT were younger (56.5 [53-59.75] vs 57 [53.75-63], p = 0.04) and had shorter ischemia time (mean 311 vs 363 minutes, p = 0.03) than those who did not. The age-adjusted ATE estimate of post-LT augmentation therapy use on time to death and ACGF was +1.69 and +1.48 years, respectively. Post-LT augmentation therapy use was associated with a mortality reduction in the top quartile bilirubin subgroup (p = 0.02, log-rank test). Conclusions: In our study, the use of augmentation therapy post-LT was associated with improved survival. Confirmatory prospective studies should be considered to inform post-LT AAT therapy guidelines. |
| format | Article |
| id | doaj-art-e21b3b937d094698b595d0099d603dc6 |
| institution | Kabale University |
| issn | 2950-1334 |
| language | English |
| publishDate | 2025-02-01 |
| publisher | Elsevier |
| record_format | Article |
| series | JHLT Open |
| spelling | doaj-art-e21b3b937d094698b595d0099d603dc62025-02-09T05:02:01ZengElsevierJHLT Open2950-13342025-02-017100201Lung transplant outcomes for recipients with alpha-1 antitrypsin deficiency, by use of alpha-1 antitrypsin augmentation therapyAtharv V. Oak, MEng0Jessica M. Ruck, MD, PhD1Alfred J. Casillan, MD, PhD2Armaan F. Akbar, BS3Ramon A. Riojas, MD, PhD4Pali D. Shah, MD5Jinny S. Ha, MD, MHS6Sara Strout, PharmD7Allan B. Massie, PhD8Dorry L. Segev, MD, PhD9Christian A. Merlo, MD, MPH10Errol L. Bush, MD11Department of Surgery, Johns Hopkins University School of Medicine, Baltimore, MarylandDepartment of Surgery, Johns Hopkins University School of Medicine, Baltimore, MarylandDepartment of Surgery, Johns Hopkins University School of Medicine, Baltimore, MarylandDepartment of Surgery, Johns Hopkins University School of Medicine, Baltimore, MarylandDepartment of Surgery, Johns Hopkins University School of Medicine, Baltimore, MarylandDepartment of Medicine, Johns Hopkins University School of Medicine, Baltimore, MarylandDepartment of Surgery, Johns Hopkins University School of Medicine, Baltimore, MarylandDepartment of Pharmacy, Johns Hopkins University School of Medicine, Baltimore, MarylandDepartment of Surgery, New York University Grossman School of Medicine and Langone Health, New York, New York; Department of Population Health, New York University Grossman School of Medicine and Langone Health, New York, New YorkDepartment of Surgery, New York University Grossman School of Medicine and Langone Health, New York, New York; Department of Population Health, New York University Grossman School of Medicine and Langone Health, New York, New York; Scientific Registry of Transplant Recipients, Minneapolis, MinnesotaDepartment of Medicine, Johns Hopkins University School of Medicine, Baltimore, MarylandDepartment of Surgery, Johns Hopkins University School of Medicine, Baltimore, Maryland; Corresponding author: Errol L. Bush, MD, Advanced Lung Disease and Lung Transplant Program, Department of Surgery, Johns Hopkins Medical Institutions, Blalock 240, 600 North Wolfe Street, Baltimore, MD 21287.Background: For patients with alpha-1 antitrypsin (AAT) deficiency, AAT augmentation therapy can be an important part of care. However, for those who require a lung transplant (LT), there is currently only limited information to guide the use of AAT augmentation therapy post-LT. Methods: We identified all LT recipients from 2011-2021 in the Scientific Registry of Transplant Recipients with an AAT deficiency diagnosis. We categorized recipients by use of AAT augmentation therapy post-LT and compared their baseline characteristics using Fisher’s exact test and Wilcoxon rank-sum tests. We used Kaplan-Meier analyses and estimated the average treatment effect (ATE) of post-LT AAT augmentation therapy on mortality and all-cause graft failure (ACGF). The ATE measures the observed effect we would see if everyone in the population received the intervention as opposed to just a subset. Results: Among the 447 recipients with AAT deficiency, 109 used AAT augmentation therapy pre-LT, of which 32 (29.4%) continued post-LT. Recipients who used augmentation therapy post-LT were younger (56.5 [53-59.75] vs 57 [53.75-63], p = 0.04) and had shorter ischemia time (mean 311 vs 363 minutes, p = 0.03) than those who did not. The age-adjusted ATE estimate of post-LT augmentation therapy use on time to death and ACGF was +1.69 and +1.48 years, respectively. Post-LT augmentation therapy use was associated with a mortality reduction in the top quartile bilirubin subgroup (p = 0.02, log-rank test). Conclusions: In our study, the use of augmentation therapy post-LT was associated with improved survival. Confirmatory prospective studies should be considered to inform post-LT AAT therapy guidelines.http://www.sciencedirect.com/science/article/pii/S2950133424001514alpha-1 antitrypsinemphysemalung transplantaugmentation therapysurvival outcomes |
| spellingShingle | Atharv V. Oak, MEng Jessica M. Ruck, MD, PhD Alfred J. Casillan, MD, PhD Armaan F. Akbar, BS Ramon A. Riojas, MD, PhD Pali D. Shah, MD Jinny S. Ha, MD, MHS Sara Strout, PharmD Allan B. Massie, PhD Dorry L. Segev, MD, PhD Christian A. Merlo, MD, MPH Errol L. Bush, MD Lung transplant outcomes for recipients with alpha-1 antitrypsin deficiency, by use of alpha-1 antitrypsin augmentation therapy JHLT Open alpha-1 antitrypsin emphysema lung transplant augmentation therapy survival outcomes |
| title | Lung transplant outcomes for recipients with alpha-1 antitrypsin deficiency, by use of alpha-1 antitrypsin augmentation therapy |
| title_full | Lung transplant outcomes for recipients with alpha-1 antitrypsin deficiency, by use of alpha-1 antitrypsin augmentation therapy |
| title_fullStr | Lung transplant outcomes for recipients with alpha-1 antitrypsin deficiency, by use of alpha-1 antitrypsin augmentation therapy |
| title_full_unstemmed | Lung transplant outcomes for recipients with alpha-1 antitrypsin deficiency, by use of alpha-1 antitrypsin augmentation therapy |
| title_short | Lung transplant outcomes for recipients with alpha-1 antitrypsin deficiency, by use of alpha-1 antitrypsin augmentation therapy |
| title_sort | lung transplant outcomes for recipients with alpha 1 antitrypsin deficiency by use of alpha 1 antitrypsin augmentation therapy |
| topic | alpha-1 antitrypsin emphysema lung transplant augmentation therapy survival outcomes |
| url | http://www.sciencedirect.com/science/article/pii/S2950133424001514 |
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