Long-term augmentation therapy with Alpha-
1 Antitrypsin in an MZ-AAT severe persistent asthma.
I. Blanco, H. Canto, J. Flóres, C. Camblor, V. Cárcaba,
F.J. de Serres,S. Janciauskiene, E.F. Bustillo.
A young Caucasian female with severe bronchial
asthma and Alpha1-antitrypsin (AAT) deficiency, MZ
phenotype, experienced a quick and severe limitation of
her physical capacity, which negatively affected her psychological
state and social life, though she was under a
strong antiasthmatic treatment. Given her declining
health status and the significant chronic corticoid administration-
related side-effects (including high reduction of
muscle mass and bone density), a clinical trial with commercial
intravenous AAT was proposed by the patient’s
doctors, and accepted by the Spanish Ministry of Health, although it this therapy was not approved for MZ phenotypes
yet. This new therapy quickly stopped lung function
decline rate, dramatically reduced the number of hospital
admissions of the patient, suppressed the oral administration
of prednisone, reversed the corticosteroid-related
health adverse effects, significantly improving her quality
of life.
Thus, although AAT replacement therapy is not approved
nor indicated for the treatment of bronchial asthma
in MZ patients, its favourable effects observed in this
isolated case support the hypothesis that bronchial asthma
could be due to pathogenic mechanisms related to a protease-
antiprotease imbalance, what which could open new
perspectives for future research on the field
