AATD Augmentation Adherence, Infusion Burden, and Plasma Supply

Our experts open "AATD Augmentation Adherence, Infusion Burden, and Plasma Supply" with hard numbers on how patients actually use weekly therapy.

Dr. Haumschild asks Dr. Sandhaus what data and experience show about persistence and adherence with weekly IV infusions, and how infusion burden affects quality of life, employment, and caregivers.

Dr. Sandhaus says patient education is the most important factor. AlphaNet evaluated one augmentation company's data and found that fewer than 60 percent of patients prescribed therapy ever received a first infusion. He believes this reflects patients' delay in accepting weekly infusions. Among those who started, only about half received 50 of 52 infusions in the first year, a threshold that allows for vacations.

The data convinced the company to use AlphaNet's services. Once patients enrolled in the disease management program, which includes frequent telephone calls early and monthly calls later, both prescription conversion and adherence rose above 90 percent. His group sets expectations by telling patients they will not feel better after starting. A year later, they can look back and say it was a good year.

Dr. Haumschild then raises the sustainability of plasma-derived therapy, noting that donation fell during COVID. Dr. Barjaktarevic calls the approach relatively rudimentary but effective, and a lifeline for many patients. He then lists its flaws. The treated population is the sickest of the sick, and the right dose is unknown. Alpha-1 antitrypsin fluctuates as an acute phase reactant, while the protein has a short half-life.

A full weekly dose keeps patients in the normal range for about three days. For the rest of the week, levels sit below the lower limit of normal, so patients receive only partial coverage. Therapy is expensive, and each dose requires multiple donors. This raises ethical questions and infection risk, including reactions in immunodeficient patients. He concludes that the field must ask whether it can do better.

Up next, in "Evidence for AATD Augmentation: FEV1 Limits, CT Density, Unmet Needs," Dr. Kuhn and Dr. Sandhaus take on the missing FEV1 evidence and argue that clinicians may be measuring the wrong thing.