α1-Proteinase inhibitor (human) in the treatment of hereditary emphysema secondary to α1-antitrypsin deficiency: number and costs of years of life gained.

摘要: Background: α1-Antitrypsin deficiency (α-ATD) is a disorder inherited in an autosomal recessive pattern, with co-dominant alleles known as the protease inhibitor system (Pi). The main function of α1-antitrypsin (α-AT) to protect lungs against powerful elastase released from neutrophil leucocytes. α-ATD typically presents serum α-AT level 80mg/dL), few, if any, adverse effects. Objective: present study was designed discern number years life gained, and expense per year associated use augmentation therapy (α1-proteinase [human]), relative ‘no therapeutic intervention’ persons α-ATD. Methods: Monte Carlo simulation (MCS) used to: (i) estimate gained; (ii) health service expenditures gained for receiving, or not therapy. MCS afforded decision-analytical framework parameterized both stochastic (random) deterministic (fixed) components, yielded fiscal risk-profile each simulated cohort interest (eight total: by sex, smoking status [non-smoker; past (smoker)]; therapy). components employed inquiry were: age-specific body weight, height; mortality; (iii) probability distribution receipt lung transplant, FEV1. age cohort; outlays therapy; transplant; (iv) annual decline FEV1; (v) percent predicted FEV1 (vi) initiation (vii) need transplant (viii) rate infection; (ix) mortality Results are reported payer perspective ($US, costing 2010). Results: Receipt significant increase (p < 0.05) female smokers gaining estimated mean 7.14 (cost year: $US248 361 [95% CI 104 531, 392 190]); non-smokers 9.19 $US160 502 37 056, 283 947)]); male 5.93 $US142 250 48 467, 236 032]); 10.60 $US59 234 20 719, 97 548]). Conclusion: Use sex history tobacco use, at cost comparable that other evidenced-based interventions.