TY - JOUR
T1 - Assessment of Patient Perspectives and Barriers to Self-Infusion of Augmentation Therapy for Alpha-1 Antitrypsin Deficiency During the COVID-19 Pandemic
AU - Colello, Jacob
AU - Ptasinski, Anna
AU - Zhan, Xiang
AU - Kaur, Sundeep
AU - Craig, Timothy
N1 - Funding Information:
We would like to thank the participants of the study. No industry funding or sponsorship was received for this study or publication of this article. This project was supported by NIH/NCATS Colorado CTSA Grant Number UL1 TR002535. Its contents are the authors’ sole responsibility and do not necessarily represent official NIH views. No Rapid Service Fee was received by the journal for the publication of this article. All named authors meet the International Committee of Medical Journal Editors (ICMJE) criteria for authorship for this article, take responsibility for the integrity of the work as a whole, and have given their approval for this version to be published. JC: study design, survey implementation, data input, and manuscript drafting. AP: manuscript drafting and editing. SK: manuscript editing and gathering of literature references. XZ: primary statistician, head of statistical analysis. TC: concept design, supervision, manuscript editing, project administration. Jacob Colello, Xiang Zhan, and Anna Ptasinski have nothing to disclose. Sundeep Kaur has changed their affiliation since the completion of this study. They are now affiliated with Zucker School of Medicine at Hofstra/Northwell Residency Program at North Shore University Hospital and Long Island Jewish Medical Center. Timothy Craig speaks and does research for CSL Behring, Grifols and Takeda, and is the Director of Penn State’s Clinical Resource Center for the Alpha-1 Foundation. The study protocol was approved on July 3, 2020 by the Human Subjects Protection Office of Pennsylvania State University Institutional Review Board before study initiation. The study was carried out in accordance with the principles embodied in the Helsinki Declaration of 1995 (as revised in 2013). All participants provided verbal informed consent before the survey was conducted. The datasets generated during and/or analyzed during the current study are available from the corresponding author on reasonable request.
Funding Information:
No industry funding or sponsorship was received for this study or publication of this article. This project was supported by NIH/NCATS Colorado CTSA Grant Number UL1 TR002535. Its contents are the authors’ sole responsibility and do not necessarily represent official NIH views. No Rapid Service Fee was received by the journal for the publication of this article.
Publisher Copyright:
© 2022, The Author(s).
PY - 2022/3
Y1 - 2022/3
N2 - Introduction: Alpha-1 antitrypsin (AAT) deficiency is an autosomal co-dominant genetic condition that predisposes individuals to pulmonary and hepatic disease, and in severe cases is treated with augmentation by intravenous infusion. Our aim was to assess patient reluctance to transition to self-administered augmentation of alpha-1-antitrypsin, during the pandemic of SARS-CoV-2. Methods: A phone questionnaire was administered to 22 patients with severe alpha-1-antitrypsin deficiency who were currently receiving AAT augmentation therapy. Inclusion criteria included patients ≥ 18 years old, diagnosed with AATD, and receiving intravenous AAT protein augmentation therapy. Information was gathered regarding demographics, perspectives on transitioning to self-administered treatment, and anxiety and depression prevalence. Results were collected anonymously using REDCap. Joint and marginal statistical analysis was done to quantify links between participants’ willingness to transition to self-infusion and correlations with sex, age, years of therapy, anxiety, and depression. Results: Of 22 patients, 14 were male and eight were female. Ages ranged from 36 to 79 years, with an average of 62.5. Genotypes were ZZ (14), MZ (3), and SZ (2) among others. Average length of intravenous augmentation was 9.5 years. The majority, 16 participants, were aware self-infusion was an option. Eight participants were willing to consider transitioning to self-infusion if trained and educated. Eight patients reported that fear of COVID-19 transmission influenced their decision-making. Above-normal anxiety, and depression scores, were found in four, and six patients, respectively. Neither sex, age, years of treatment, anxiety, or depression were found to be associated with willingness to consider self-infusion therapy. Conclusions: Although there are many reasons AATD patients may benefit from AAT self-infusion, including decreased exposure to SARS-CoV-2, the majority preferred home nurse-infused therapy.
AB - Introduction: Alpha-1 antitrypsin (AAT) deficiency is an autosomal co-dominant genetic condition that predisposes individuals to pulmonary and hepatic disease, and in severe cases is treated with augmentation by intravenous infusion. Our aim was to assess patient reluctance to transition to self-administered augmentation of alpha-1-antitrypsin, during the pandemic of SARS-CoV-2. Methods: A phone questionnaire was administered to 22 patients with severe alpha-1-antitrypsin deficiency who were currently receiving AAT augmentation therapy. Inclusion criteria included patients ≥ 18 years old, diagnosed with AATD, and receiving intravenous AAT protein augmentation therapy. Information was gathered regarding demographics, perspectives on transitioning to self-administered treatment, and anxiety and depression prevalence. Results were collected anonymously using REDCap. Joint and marginal statistical analysis was done to quantify links between participants’ willingness to transition to self-infusion and correlations with sex, age, years of therapy, anxiety, and depression. Results: Of 22 patients, 14 were male and eight were female. Ages ranged from 36 to 79 years, with an average of 62.5. Genotypes were ZZ (14), MZ (3), and SZ (2) among others. Average length of intravenous augmentation was 9.5 years. The majority, 16 participants, were aware self-infusion was an option. Eight participants were willing to consider transitioning to self-infusion if trained and educated. Eight patients reported that fear of COVID-19 transmission influenced their decision-making. Above-normal anxiety, and depression scores, were found in four, and six patients, respectively. Neither sex, age, years of treatment, anxiety, or depression were found to be associated with willingness to consider self-infusion therapy. Conclusions: Although there are many reasons AATD patients may benefit from AAT self-infusion, including decreased exposure to SARS-CoV-2, the majority preferred home nurse-infused therapy.
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U2 - 10.1007/s41030-022-00182-z
DO - 10.1007/s41030-022-00182-z
M3 - Article
C2 - 35067906
AN - SCOPUS:85123473298
SN - 2364-1754
VL - 8
SP - 95
EP - 103
JO - Pulmonary Therapy
JF - Pulmonary Therapy
IS - 1
ER -