Evaluation of tamoxifen as a therapy to augment alpha-1-antitrypsin concentrations in Z homozygous alpha-1-antitrypsin-deficient subjects

M. D. Wewers, M. L. Brantly, M. A. Casolaro, R. G. Crystal

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Tamoxifen, an agent that binds to intracytoplasmic estrogen receptors, was evaluated as a possible means of increasing alpha-1-antitrypsin (α1AT) synthesis and/or secretion and thus α1AT serum levels in subjects with the homozygous form of α1AT deficiency. Administration of tamoxifen (10 mg twice daily) to 30 Z homozygotes for a 30-day period was not associated with adverse reactions. However, although serum α1AT levels increased significantly (p < 0.03), the increase was minor (average pretreatment levels, 32 ± 1 mg/dl; levels at 30 days of therapy, 35 ± 1 mg/dl) and far below the 'threshold' level of 80 mg/dl considered 'protective' against an increased risk for emphysema. Thus, while the concept that increasing α1AT synthesis and/or secretion is a rational goal for treating the Z homozygous form of α1AT deficiency, tamoxifen will not be useful in this regard.

Original languageEnglish
Pages (from-to)401-402
Number of pages2
JournalAmerican Review of Respiratory Disease
Issue number2
Publication statusPublished - 1 Jan 1987


ASJC Scopus subject areas

  • Pulmonary and Respiratory Medicine

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