Emphysema is a chronic disease of the lungs characterized by shortness of breath that worsens over time. A genetic form of the disease is caused by an inheritable deficiency of the α1 antitrypsin (AAT) protein, leaving the lungs more susceptible to the type of damage that leads to emphysema.
Augmentation therapy is a treatment for the genetic form of emphysema. It involves weekly intravenous injections of purified AAT to boost its levels in the blood and lungs. Although the therapy has been available for over 25 years, it is rarely prescribed to patients because there has been a lack of evidence showing that the treatment actually protects the lungs.
A study led by TWRI clinical researcher Dr. Kenneth Chapman has produced compelling evidence that augmentation therapy does in fact slow the progression of lung damage in people with AAT deficiency. The findings were recently published in the prestigious medical journal The Lancet.
As part of the study, 180 people with emphysema and low blood levels of AAT received either the augmentation therapy or a placebo containing no protein. After two years of treatment, researchers discovered that the participants who received augmentation therapy had less damage to their lungs than those receiving the placebo.
"Patients with AAT deficiency need timely access to diagnosis and treatment to ensure that they receive the best possible care. Augmentation therapy has the potential to add years to a patient's life", says Dr. Chapman of the findings.
This work was supported by CSL Behring and the Toronto General & Western Hospital Foundation.
Intravenous augmentation treatment and lung density in severe a1 antitrypsin deficiency (RAPID): a randomised, double-blind, placebo-controlled trial. Chapman KR, Burdon JG, Piitulainen E, Sandhaus RA, Seersholm N, Stocks JM, Stoel BC, Huang L, Yao Z, Edelman JM, McElvaney NG; RAPID Trial Study Group. The Lancet. 2015 May 27. [Pubmed abstract]