Recommendations for the Implementation of the Self-Administration of Alpha-1 Antitrypsin

Abstract

Purpose

Administration of exogenous alpha-1 antitrypsin (AAT) is the only specific therapy for the management of pulmonary morbidity in patients with AAT deficiency. It requires weekly or biweekly intravenous infusions, which may impact patient independence and quality of life. Self-administration of AAT therapy is an alternative to reduce the burden for patients who require AAT therapy. We presented herein experts’ recommendations for the implementation of a program for the self-administration of AAT.

Methods

This project was conducted using a modified nominal group technique and was undertaken in two online meetings involving the participation of 25 experts: specialists in pulmonology (n=17), nurses (n=5) and hospital pharmacists (n=3).

Results

The following issues were discussed, and several recommendations were agreed upon on the following topics: a) patient profile and clinical evaluation, establishing selection criteria that should include clinical as well as social criteria; b) role of health care professionals, suggested roles for specialists in pulmonology, nurses, and hospital pharmacists; c) training by the nurse, including recommendations before initiating the training and the content of the training sessions; and d) logistic issues and follow-up, adherence, and patient support.

Conclusion

We expect this proposal to increase awareness of this therapeutic alternative and facilitate the implementation of self-administration programs, thus contributing to optimizing the patient experience with AAT therapy. Further research on the outcomes of these programs, especially from the patient perspective, will also help to improve their design and implementation.

Keywords:

• alpha-1 antitrypsin deficiency
• disease burden
• augmentation therapy
• self-administration

Abbreviations

AAT, alpha-1 antitrypsin; HCP, health care professionals; REDAAT, Spanish Network on AAT deficiency.

Data Sharing Statement

Not applicable. All the relevant information is presented with the manuscript.

Ethics Approval and Informed Consent

This project did not involve the participation of patients. Due to its nature, following the general Spanish regulations on biomedical research (ie Law 14/2007, of July 3, on Biomedical Research/La Ley 14/2007, de 3 de julio, de Investigación biomédica), this project did not require the evaluation of an Ethics Committee. The participating experts were informed about the nature of the project and agreed to participate by signing a contract.

Acknowledgments

The authors thank Sonia Pisa and Sofía Barreiro (Ampersand Consulting, Barcelona, Spain) for the coordination of the project and methodological support and Fernando Rico-Villademoros (COCIENTE S.L., Madrid, Spain) for editorial assistance. This support and assistance was funded by CSL Behring España.

Author Contributions

All authors made a significant contribution to the work reported, whether that is in the conception, study design, execution, acquisition of data, analysis and interpretation, or in all these areas; took part in drafting, revising or critically reviewing the article; gave final approval of the version to be published; have agreed on the journal to which the article has been submitted; and agree to be accountable for all aspects of the work.

Disclosure

MT-D: has received consulting fees from CSL Behring and Grifols; payment or honoraria for lectures, presentations, speaker bureaus, manuscript writing or educational events from CSL Behring and Grifols; and support for attending meetings and/or travel from CSL Behring, Grifols and Chiesi. JLL-C: has received honoraria during the last 3 years for lecturing, scientific advice, participation in clinical studies or writing for publications from AstraZeneca, Bial, Boehringer Ingelheim, Chiesi, CSL Behring, Ferrer, Gebro, GlaxoSmithKline, Grifols, Menarini, Megalabs, Novartis and Rovi. MCR: has received speaker fees from AstraZeneca, Bial, Chiesi, CSL Behring, GlaxoSmithKline, Menarini, and Grifols; and consulting fees from GlaxoSmithKline, CSL Behring and Bial. CM-M: has received speaker fees and/or consulting fees and/or support to attend congresses from Astra-Zeneca, Boehringer-Ingelheim, Chiesi, CSL Behring, GlaxoSmithKline, Grifols and Menarini. APC: has received payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events from CSL Behring. MB: has received speaker fees from Grifols, Menarini, CSL Behring, GSK, Boehringer Ingelheim and consulting fees from GSK, Novartis, CSL Behring and Boehringer Ingelheim. FJC-G: has received speaker honorarium from GlaxoSmithKline, Chiesi, Boehringer Ingelheim, Mundipharma, Menarini, Pfizer, Novartis, Esteve, Teva Pharmaceutical, Ferrer, Rovi, Roche, Astra Zeneca, Bial, Actelion, Alter, CSL Behring, Faes Farma, Alter, Grifols, Sanofi Genzyme and Gebro Pharma; consulting honorarium from Chiesi, Boehringer Ingelheim, Teva Pharmaceutical, Astra Zeneca, Bial, CSL Behring and Sanofi Genzyme; and travel grants from GlaxoSmithKline, Chiesi, Boehringer Ingelheim, Teva Pharmaceutical, Astra Zeneca and CSL Behring. FC-M: has received speaker fees from AstraZeneca, Bial, Boehringer Ingelheim, Chiesi, GlaxoSmithKline, Novartis, Sanofi, Vertex, CSL Behring and Grifols; and consulting fees from AstraZeneca, Chiesi, GlaxoSmithKline, CSL Behring, and Grifols. JMH-P: Speaker honorarium from Grifols, CSL Behring, Astra-Zeneca, GSK, Bial laboratory, Teva laboratory and FAES Farma. Advisory honorarium from CSL Behring. Travel grants from Grifols and CSL Behring. LL-A: has received speaker honorarium from CSL Behring and Grifols SA; research grants from Grifols SA; consulting honorarium from CSL Behring; and travel grants from CSL Behring and Grifols SA. CMR: has received speaker honorarium from Astra Zeneca, CSL Behring CSL, Chiesi, Gebro, GSK, Mundipharma, TEVA and Sanofi; research grants from AstraZeneca, GSK and TEVA; and consulting and advisory honorarium from Astra Zeneca, CSL Behring, Chiesi, Mundipharma and TEVA. FJM: has received speaker fees and/or consulting fees and/or support to attend congresses from Astra-Zeneca, Boehringer-Ingelheim, Chiesi, CSL Behring, GlaxoSmithKline, Grifols, Menarini, Novartis, Sanofi Aventis and Teva. J-BM-R: has received speaker fees and/or consulting fees and/or support to attend congresses from CSL Behring and Grifols. RS: has received speaker fees and/or consulting fees and/or support to attend congresses from CSL Behring, Astra Zeneca, Chiesi, GlaxoSmithKline, Grifols, Teva, Novartis and Menarini. MO-P: has received speaker honorarium from Biogen, Novartis, Roche, Genzyme Sanofi and Takeda. JPQG: Advisory board member for CSL Behring. MM: has received speaker fees from AstraZeneca, Boehringer Ingelheim, Chiesi, Cipla, Menarini, Rovi, Bial, Kamada, Takeda, Sandoz, Zambon, CSL Behring, Specialty Therapeutics, Janssen, Grifols and Novartis; consulting fees from AstraZeneca, Atriva Therapeutics, Boehringer Ingelheim, Chiesi, GlaxoSmithKline, Bial, Gebro Pharma, CSL Behring, Inhibrx, Laboratorios Esteve, Ferrer, Menarini, Mereo Biopharma, Verona Pharma, Spin Therapeutics, ONO Pharma, pH Pharma, Palobiofarma SL, Takeda, Novartis, Sanofi and Grifols; and research grants from Grifols. RAR, MABG, LD-C, PGM, CM-G, IP, MRR-S-E and BT-G: have no conflict of interest.

Additional information

Funding

This project was funded by CSL Behring España.