Key result
Monthly alpha 1-antitrypsin augmentation lowers urinary desmosine excretion by ~38% in two severely deficient patients.
Why the study?
It is not known whether augmentation therapy with alpha 1-antitrypsin diminishes degradation of lung elastin in severely deficient persons with lung disease.
Does alpha 1-antitrypsin augmentation therapy reduce urinary desmosine excretion in severely AAT-deficient patients with lung disease?
Case Report (n=2)
Does alpha 1-antitrypsin augmentation therapy reduce urinary desmosine excretion in severely AAT-deficient patients with lung disease?
Alpha 1-antitrypsin augmentation therapy decreases urinary desmosine excretion in severely AAT-deficient patients, suggesting diminished degradation of lung elastin.
Answers are grounded in this paper and cite their sources.
May support AAT augmentation to reduce elastin degradation in PiZ patients; leaves open confirmation in larger trials.
It is hypothesized that emphysema develops in some severely alpha 1-antitrypsin (AAT)-deficient persons because endogenous elastases are not adequately controlled by AAT, and accelerated elastin degradation occurs. It is not known whether augmentation therapy with AAT diminishes degradation of lung elastin in severely deficient persons with lung disease. Two severely deficient, PiZ patients were studied, a 63-year-old never-smoking woman with bronchiectasis and a 41-year-old smoking man with emphysema. Urinary desmosine (DES) was determined before and after augmentation therapy with AAT, 260 mg/kg/month. Mean +/- SEM pretreatment urinary DES was elevated in both patients, 19.7 +/- 0.9 (n = 2) and 10.8 +/- 0.2 (n = 2) micrograms/g creatinine, respectively, compared to normal values of 7.5 +/- 0.3 (n = 22) micrograms/g creatinine. Following augmentation therapy, urinary DES values decreased 40 and 36%, respectively, to 11.9 +/- 0.3 (n = 8) and 6.9 +/- 0.4 (n = 7) microgram/g creatinine (p < 0.05). We conclude that monthly AAT augmentation therapy decreased DES excretion in the urine of these PiZ patients. We speculate that since there was lung disease in both patients, a decrease in degradation of lung elastin is the most likely explanation for this observation.
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Stone et al. (2009) conducted a case report in Severe alpha 1-antitrypsin (AAT) deficiency (n=2). Alpha 1-antitrypsin (AAT) augmentation therapy vs. Pretreatment baseline was evaluated on Urinary desmosine (DES) excretion (36% to 40% decrease, p=<0.05). Monthly augmentation therapy with alpha 1-antitrypsin (260 mg/kg) decreased urinary desmosine excretion by 36% to 40% in two severely deficient PiZ patients with lung disease (p < 0.05).
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