Alpha1-antitrypsin deficiency (alpha1) is a hereditary disorder that can result in life-threatening liver disease in children and adults or in lung disease in adults. An estimated 100,000 Americans and a similar number in Europe have the severe deficiency, and less than 10% of those predicted to have alpha1 have been diagnosed, according to Alpha-1 Foundation statistics. RT spoke to Paul R. Perreault, Sr, vice president and general manager of North America commercial operations at ZLB Behring, the company that manufactures the alpha1 therapy Zemaira, to gain a better understanding of alpha1 and the treatments available.
Q: Alpha1 is often misdiagnosed as chronic obstructive pulmonary disease (COPD). What do physicians need to know to prevent misdiagnosis? A: First they need to realize that alpha1, according to the American Thoracic Society, is often under-recognized or misdiagnosed. They also need to be aware of the latest guidelines that were released in October 2003 by the American Thoracic Society, which basically set the standards for diagnosis and management of individuals with alpha1-antitrypsin deficiency. There are testing recommendations in the guidelines that propose screening and testing for alpha1 in all COPD patients.
Q: What are some lifestyle changes alpha1 patients can make in order to maintain their health? A: An alpha1 patient should obviously stop smoking if they are smoking. They should avoid areas with high levels of dust and pollen and smoke fumes. They should minimize infection, wash hands. They should not be in contact with a lot of sick people and should exercise daily. They need to find ways to reduce stress, such as relaxation, DVDs, yoga, or whatever. It is important that they also have good medical care and management. The Alpha-1 Foundation has a lot of resources that can help these patients manage their disease and help them with some of their lifestyle changes. We have actually entered into collaboration with AlphaNet, an organization that helps alpha1 patients manage their condition, and with Accredo, the exclusive distributor of our product, to develope a health management system to provide a comprehensive care model for alpha1 patients.
Q: How can augmentation therapy with Zemaira benefit an alpha1 patient? A: For a long time, there was only one product in the category, with frequent supply issues. Zemaira adds another option for patients with alpha1, and with our state-of-the-art manufacturing process, Zemaira is the only alpha1 therapy approved that has such a high level of purity. It is a 98% to 99% pure alpha1 proteinase inhibitor. It offers the same level of lung protection as other augmentation therapies, which is critical to the protection of patients who have lung issues. Zemaira has also been associated with approximately 44% fewer COPD exacerbations. Patients report fewer issues compared to some of the previously available therapies. It is also time efficient, as it has about half the infusion volume of other alpha1 therapies.
Q: What plans does ZLB have in place with regard to new clinical studies for Zemaira? A: We are currently working closely with the US Food and Drug Administration on a protocol to help define endpoints and establish a better understanding of the alpha1 augmentation therapy. We are also planning further studies that will reinforce the clinical benefits of Zemaira within the alpha1 community.