Newly available therapy a turning point for many patients with Alpha-1 antitrypsin deficiency

As a new funded augmentation therapy becomes available to Canadians with Alpha-1 antitrypsin deficiency (A1ATD), physicians are urged to start affected patients on the treatment as soon as possible.“Don’t watch and wait,” was the key message from Dr. Kenneth Chapman, director of the Asthma and Airway centre at the University Health Network, who spoke at the Canadian Thoracic Society’s recent 16th Annual Canadian Respiratory Conference in Toronto. A1ATD is a rare genetic condition associated with an increased risk of emphysema, COPD, cirrhosis and primary liver cancer. Standard therapies and medications to manage COPD are used for A1ATD patients with pulmonary symptoms. In addition, augmentation therapy has been shown to slow disease progression but has been largely unavailable to patients until recently. A significant milestone for A1ATD patients in Canada was reached in March 2024 when Glassia, an alpha-1 proteinase inhibitor augmentation therapy, was made available through Canadian Blood Services (CBS), with costs covered for qualified patients. Previously, augmentation therapy existed but was distributed through the drug product delivery system. Like all blood products, it was expensive and was not covered by most provincial drug plans. Read: What docs should know: Why wildfire smoke is a 'different beast' from other air pollutionAugmentation therapy has been shown to preserve CT scan lung density in patients with A1ATD and significantly lower rates of lung function decline compared to those who did not receive augmentation therapy."Like all other blood products in Canada, if it is medically appropriate, Canadians in need can obtain it based upon this medical need and not their insurance status," said Dr. Chapman, also a professor of medicine at the University of Toronto. "We believe this is a first for augmentation therapy internationally—making it available through the blood supply system and not through the prescription drug system," he added.Start treatmentAugmentation therapy may be considered in non-smoking or ex-smoking patients with COPD (FEV1 25% to 80% predicted) attributable to emphysema and documented A1ATD,” said Dr. Chapman. “The evidence supports consideration of augmentation therapy in patients with FEV-1 of 25% to 80% of predicted and documented A1ATD receiving optimal therapies because of benefits in lung density and mortality.”Dr. Chapman pointed to a large study that showed that patients with severe COPD and A1ATD who received augmentation therapy had significantly better survival compared to those who did not receive augmentation therapy.“Patients lacking adequate amounts of Alpha-1 antitrypsin lose lung function at an accelerated rate. Watching and waiting will lead to the loss of lung function that cannot be recovered. There is no 'catching up' for patients when physicians delay."Read: Can this food help ‘beet’ COPD? Round upDr. Chapman also encouraged practitioners to “round up” the amount of the infusion. The guidelines call for 60 mg per 60 kg. But, Dr. Chapman said, “For a 70-kilogram man, if you prescribe 4,200 mg infusion, and the product is dispensed in 1,000 mg vials, 800 mg will be wasted every week. So round up to 5,000. We hate to see the wasting of this precious resource and we know not only that higher amounts are well-tolerated, but several lines of evidence suggest that higher doses may be more effective."Glassia is a liquid medicine for infusion containing human Alpha 1-Proteinase Inhibitor, which is purified from human blood. The main purpose of infusing Glassia is to increase the levels of the A1AT protein in the blood, which protects the lung tissue by limiting the action of the neutrophil elastase enzyme. Neutrophil elastase can cause damage if its action is not controlled (as is the case in A1ATD).Who should be testedAlthough it is rare, A1ATD is considered to be under-diagnosed. The Canadian Thoracic Society recommends testing for Alpha-1 Antitrypsin Deficiency in:patients diagnosed with COPD before age 65 or with a smoking history of less than 20 “pack years”individuals with a family history of Alpha-1 (along with genetic counselling)adult asthma patients with diagnostic uncertainty (i.e., persistent obstruction on lung function testing)Criteria for treatmentGlassia can be requested for adult patients with severe A1ATD and clinical evidence of emphysema who meet the following criteria:Respirologist has confirmed the diagnosis of severe A1ATD and clinical evidence of emphysema and indicated that the patient would benefit from treatment with A1- PI product.A1ATD, defined as serum A1-PI levels < 57 mg/dL before start of the treatmentclinical evidence of obstruction (FEV1

Newly available therapy a turning point for many patients with Alpha-1 antitrypsin deficiency

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