PMID- 33328731 OWN - NLM STAT- MEDLINE DCOM- 20210625 LR - 20210625 IS - 1178-2005 (Electronic) IS - 1176-9106 (Print) IS - 1176-9106 (Linking) VI - 15 DP - 2020 TI - Improving the Lives of Patients with Alpha-1 Antitrypsin Deficiency. PG - 3313-3322 LID - 10.2147/COPD.S276773 [doi] AB - Alpha-1 Antitrypsin Deficiency (AATD) is a rare genetic condition that predisposes patients to lung and liver disease and is often underdiagnosed due to incomplete diagnosis of chronic obstructive pulmonary disease (COPD) and asthma. Improvements in physician awareness have been made, but better strategies for both diagnosis and management are still required. The only current disease-modifying therapy for AATD is the infusion of the missing Alpha-1 Antitrypsin (AAT) protein, which can slow progression of emphysema. However, AAT treatment can impact patient freedom and quality of life due to the need for weekly intravenous infusions. A symposium was held to discuss patient-centric aspects of care that have impact on the lives of patients with AATD, including exacerbations of their lung disease, self-administration of intravenous AAT therapy and pulmonary rehabilitation. Intravenous self-infusion of drugs is an established treatment strategy for patients with a variety of conditions and can improve patient quality of life, freedom and mental well-being. Experience from these areas show that patients typically manage their treatment well and without complications. When applied to AATD, training patients to self-infuse therapy can be successful, but formal guidelines would be beneficial. In addition to pharmacological intervention, individualized pulmonary rehabilitation, exercise and educational programs can encourage health-enhancing patient behavior and further improve patient quality of life. However, differences in skeletal muscle adaptations to pulmonary rehabilitation exercise regimens have been observed between patients with AATD and non-AATD COPD, highlighting the need to develop training programs specifically designed for patients with AATD. CI - (c) 2020 Sandhaus et al. FAU - Sandhaus, Robert A AU - Sandhaus RA AD - Division of Pulmonary, Critical Care and Sleep Medicine, National Jewish Health, Denver, CO, USA. FAU - Strange, Charlie AU - Strange C AUID- ORCID: 0000-0002-8109-8067 AD - Division of Pulmonary and Critical Care Medicine, Medical University of South Carolina, Charleston, SC, USA. FAU - Zanichelli, Andrea AU - Zanichelli A AD - Department of Internal Medicine, Luigi Sacco Hospital, University of Milan, AAST Fatebenefratelli Sacco, Milan, Italy. FAU - Skalvoll, Karen AU - Skalvoll K AD - Team Alpha-1 Athlete, Olsvik, Norway. FAU - Koczulla, Andreas Rembert AU - Koczulla AR AUID- ORCID: 0000-0002-5884-3006 AD - Department of Medicine, Pulmonary and Critical Care Medicine and Pulmonary Rehabilitation, and Head of Teaching Hospital Schoen Klinik BGL, Philipps-University Marburg, Marburg, Germany. AD - German Center for Lung Research (DZL), Germany Teaching Hospital, Paracelsus Medical University, Salzburg, Austria. FAU - Stockley, Robert A AU - Stockley RA AUID- ORCID: 0000-0003-3726-1207 AD - University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital Birmingham, Birmingham, UK. LA - eng PT - Journal Article PT - Review DEP - 20201210 PL - New Zealand TA - Int J Chron Obstruct Pulmon Dis JT - International journal of chronic obstructive pulmonary disease JID - 101273481 RN - 0 (alpha 1-Antitrypsin) SB - IM MH - Humans MH - *Pulmonary Disease, Chronic Obstructive/diagnosis/drug therapy MH - *Pulmonary Emphysema/diagnosis/drug therapy/etiology MH - Quality of Life MH - Treatment Outcome MH - alpha 1-Antitrypsin MH - *alpha 1-Antitrypsin Deficiency/diagnosis/drug therapy PMC - PMC7735792 OTO - NOTNLM OT - alpha-1 antitrypsin OT - chronic obstructive pulmonary disease OT - exacerbations OT - pulmonary rehabilitation OT - quality of life OT - self-administration COIS- Dr Robert A Sandhaus and Dr Charlie Strange are medical directors of AlphaNet, a not-for profit disease management company for AATD. Dr Robert A Sandhaus reports participation in advisory boards for Grifols, CSL Behring, AstraZeneca, Mereo BioPharma, and Inhibrx for which he receives reimbursement of travel expenses. Dr Charlie Strange consults on AATD and/or asthma and COPD with AstraZeneca, CSL Behring, Dicerna, Grifols, and Vertex. He has grants paid to MUSC from Adverum, CSA Medical, CSL Behring, Grifols, MatRx, Novartis, Nuvaira, Pulmonx, Takeda, and Vertex. Dr Andrea Zanichelli has received meeting sponsorship from and performed clinical trial research funded by BioCryst, CSL Behring, Shire, Pharming Dyax Corporation, and acts as a consultant for CSL Behring and Shire. Karen Skalvoll reports personal fees from CSL Behring which was paid to Team Alpha-1 Athlete. Dr A Rembert Koczulla reports personal fees from CSL Behring, outside the submitted work. Dr Robert A Stockley serves on the advisory boards for several pharmaceutical companies with an interest in AATD, including CSL Behring, Mereo BioPharma, Vertex, Z Factor and Kamada, and has received non-commercial funding from CSL Behring, Grifols, Takeda and Mereo BioPharma. The authors report no other conflicts of interest in this work. EDAT- 2020/12/18 06:00 MHDA- 2021/06/29 06:00 PMCR- 2020/12/10 CRDT- 2020/12/17 05:47 PHST- 2020/08/12 00:00 [received] PHST- 2020/11/06 00:00 [accepted] PHST- 2020/12/17 05:47 [entrez] PHST- 2020/12/18 06:00 [pubmed] PHST- 2021/06/29 06:00 [medline] PHST- 2020/12/10 00:00 [pmc-release] AID - 276773 [pii] AID - 10.2147/COPD.S276773 [doi] PST - epublish SO - Int J Chron Obstruct Pulmon Dis. 2020 Dec 10;15:3313-3322. doi: 10.2147/COPD.S276773. eCollection 2020.