{"doi":"10.1164/rccm.200209-1035oc","title":"Cost-Effectiveness Analysis of Augmentation Therapy for Severe α1-Antitrypsin Deficiency","abstract":"<jats:title>Abstract</jats:title>\n                  <jats:p>A Markov-based decision model was created to assess the cost-effectiveness of augmentation therapy (Aug) for severe α1-antitrypsin deficiency, comparing strategies of: (1) no Aug, (2) Aug for life, and (3) Aug until FEV1 is below 35% predicted. A hypothetical cohort of 46-year-old patients with FEV1 49% predicted was followed over time using Monte Carlo simulation across five possible health states: (1) FEV1 50 to 79% predicted, (2) FEV1 35 to 49% predicted, (3) FEV1 below 35% predicted, (4) status—post-lung transplantation, and (5) dead. Treatment for life yielded 7.19 quality-adjusted life-years (QALYs) and cost $895,243. Treating until FEV1 is below 35% predicted cost $511,930 and produced 6.64 QALYs. “No Aug” cost $92,091 with 4.62 QALYs. The incremental cost-effectiveness ratio was $207,841/QALY for Aug until FEV1 is below 35% predicted and $312,511/QALY for the “Aug for life” strategy. In all sensitivity analyses, the incremental cost-effectiveness ratio for Aug for life exceeded $100,000. The cost of Aug needed to be reduced from $54,765 to $4,900 for the “Aug for life” strategy to be considered cost-effective. We conclude that, compared with other conventionally used health interventions, Aug is relatively less cost-effective. These results should encourage the development of more clinically and cost-effective therapies for α1-antitrypsin deficiency.</jats:p>","journal":"American Journal of Respiratory and Critical Care Medicine","year":2003,"id":629892,"datarank":0.6456097639806255,"base_score":4.304065093204169,"endowment":4.304065093204169,"self_citation_contribution":0.6456097639806255,"citation_network_contribution":0.0,"self_endowment_contribution":0.6456097639806255,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":73,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":null,"is_data_producer":false,"deposit_databanks":null,"is_oa":false,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":null,"fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":1631516,"name":"Kenneth M. Shermock","orcid":null,"position":1,"is_corresponding":false},{"id":574678,"name":"Mendel E. Singer","orcid":"0000-0002-3331-3340","position":2,"is_corresponding":false},{"id":447157,"name":"James K. Stoller","orcid":"0000-0002-9636-5770","position":3,"is_corresponding":false},{"id":1631515,"name":"Thomas R. Gildea","orcid":null,"position":0,"is_corresponding":false}],"reference_count":0,"raw_metadata":{"has_enrichment":true,"resolved":true,"title":"Cost-Effectiveness Analysis of Augmentation Therapy for Severe α1-Antitrypsin Deficiency","abstract":"<jats:title>Abstract</jats:title>\n                  <jats:p>A Markov-based decision model was created to assess the cost-effectiveness of augmentation therapy (Aug) for severe α1-antitrypsin deficiency, comparing strategies of: (1) no Aug, (2) Aug for life, and (3) Aug until FEV1 is below 35% predicted. A hypothetical cohort of 46-year-old patients with FEV1 49% predicted was followed over time using Monte Carlo simulation across five possible health states: (1) FEV1 50 to 79% predicted, (2) FEV1 35 to 49% predicted, (3) FEV1 below 35% predicted, (4) status—post-lung transplantation, and (5) dead. Treatment for life yielded 7.19 quality-adjusted life-years (QALYs) and cost $895,243. Treating until FEV1 is below 35% predicted cost $511,930 and produced 6.64 QALYs. “No Aug” cost $92,091 with 4.62 QALYs. The incremental cost-effectiveness ratio was $207,841/QALY for Aug until FEV1 is below 35% predicted and $312,511/QALY for the “Aug for life” strategy. In all sensitivity analyses, the incremental cost-effectiveness ratio for Aug for life exceeded $100,000. The cost of Aug needed to be reduced from $54,765 to $4,900 for the “Aug for life” strategy to be considered cost-effective. We conclude that, compared with other conventionally used health interventions, Aug is relatively less cost-effective. These results should encourage the development of more clinically and cost-effective therapies for α1-antitrypsin deficiency.</jats:p>","is_dataset_classified":null,"base_score":4.304065093204169,"endowment":4.304065093204169,"datacite_reuse_total":0,"file_count":0,"downloads":0,"views":0,"has_version_chain":false,"is_dataset":false,"is_oa":false,"pmid":"12574076","pmcid":null,"openalex_id":"https://openalex.org/W2066175645","authors":[],"funders":[],"total_grants":0,"fwci":1.6347,"citation_percentile":0.82968148,"influential_citations":0,"citation_trend":[{"year":2012,"count":3},{"year":2013,"count":3},{"year":2014,"count":3},{"year":2015,"count":6},{"year":2016,"count":7},{"year":2017,"count":1},{"year":2018,"count":5},{"year":2019,"count":4},{"year":2020,"count":2},{"year":2021,"count":2},{"year":2022,"count":2},{"year":2023,"count":1},{"year":2024,"count":1}],"oa_status":"closed","license":"https://academic.oup.com/pages/standard-publication-reuse-rights","oa_locations":[{"url":"https://academic.oup.com/ajrccm/article-pdf/167/10/1387/67350901/ajrccm_167_10_1387.pdf","host_type":"publisher"},{"url":"https://doi.org/10.1164/rccm.200209-1035oc","host_type":"journal"},{"url":"https://pubmed.ncbi.nlm.nih.gov/12574076","host_type":"repository"}],"fields_of_study":["Protease and Inhibitor Mechanisms","Peptidase Inhibition and Analysis","Blood Coagulation and Thrombosis Mechanisms","Cost-Benefit Analysis","Drug Administration Schedule","Female","Humans","Long-Term Care","Male","Markov Chains","Protease Inhibitors","Quality-Adjusted Life Years","Registries","Respiratory Function Tests","Sensitivity and Specificity","Severity of Illness Index","alpha 1-Antitrypsin Deficiency"],"mesh_terms":["Cost-Benefit Analysis","Drug Administration Schedule","Female","Humans","Long-Term Care","Male","Markov Chains","Protease Inhibitors","Registries","Respiratory Function Tests","Sensitivity and Specificity","Severity of Illness Index","Quality-Adjusted Life Years","alpha 1-Antitrypsin Deficiency"],"keywords":["Medicine","Quality-adjusted life year","Incremental cost-effectiveness ratio","Cost effectiveness","Quality of life (healthcare)","Cohort","Psychological intervention","Pediatrics","Intensive care medicine","Internal medicine","Risk analysis (engineering)"],"sdg_mappings":[],"linked_datasets":[],"clinical_trials":[],"software_tools":[],"database_accessions":[],"source":"live","citation_network_status":"fetched"},"created_at":"2026-08-05T19:58:38.779499Z","pmid":null,"pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}