alpha1-Antitrypsin augmentation therapy for PI*MZ heterozygotes: a cautionary note.
The use of IV augmentation therapy with plasma-derived alpha1-antitrypsin (AAT) has become the standard of care for the treatment of pulmonary disease associated with the severe genetic deficiency of AAT. The Medical and Scientific Advisory Committee of the Alpha-1 Foundation has become aware that physicians are prescribing this expensive blood product for the treatment of individuals with a single abnormal AAT gene, primarily the PI*MZ genotype. We are aware of no evidence that such therapy is effective in this patient population. The most important therapeutic interventions in such patients remain smoking cessation and elimination of other risk factors for lung disease. This commentary discusses the treatment of AAT deficiency and the concerns regarding treatment of PI*MZ individuals. We conclude that clinicians should avoid prescribing augmentation therapy for this heterozygote population.
Sandhaus
Robert A
RA
National Jewish Medical and Research Center; Denver, CO. Electronic address: rasandhaus@alphaone.org.
Turino
Gerard
G
St. Luke's-Roosevelt Hospital Center, Columbia University College of Physicians & Surgeons, New York, NY.
Stocks
James
J
University of Texas Health Sciences Center at Tyler, Tyler, TX.
Strange
Charlie
C
Medical University of South Carolina, Charleston, SC.
Trapnell
Bruce C
BC
Cincinnati Children's Hospital Medical Center, University of Cincinnati School of Medicine, Cincinnati, OH.
Silverman
Edwin K
EK
Channing Laboratory, Brigham and Women's Hospital, Boston, MA.
Everett
Sarah E
SE
Medical and Scientific Advisory Committee, Alpha-1 Foundation, Miami, FL.
Stoller
James K
JK
Cleveland Clinic Foundation, Cleveland, OH.
D003362:Cost-Benefit Analysis; D006579:Heterozygote; D006801:Humans; D008173:Lung Diseases, Obstructive / Q000188:drug therapy* / Q000209:etiology; D010818:Practice Patterns, Physicians'; D015842:Serine Proteinase Inhibitors / Q000191:economics / Q000627:therapeutic use*; D012720:Severity of Illness Index; D000515:alpha 1-Antitrypsin / Q000191:economics / Q000627:therapeutic use*; D019896:alpha 1-Antitrypsin Deficiency / Q000150:complications / Q000188:drug therapy* / Q000235:genetics*
D015842:Serine Proteinase Inhibitors; D000515:alpha 1-Antitrypsin