Five Things To Know: New Therapy for Hereditary Angioedema

Monoclonal y-shaped antibodies

Researchers from the Massachusetts General Hospital Division of Rheumatology, Allergy and Immunology recently came out with a study published in New England Journal of Medicine. Here are five things to know:

  1. Hereditary angioedema (HAE) is a rare but serious condition passed down through families. HAE affects the blood vessels and is caused by a low level or improper function of a protein called the C1 inhibitor.
  2. HAE causes rapid swelling, particularly in tissues in the face, hands, gastrointestinal tract and airway. The uncontrolled swelling not only restricts movement, sometimes for several days, but also can be life-threatening when the airway is involved. While it’s important to treat the swelling during an HAE attack, preventing attacks would be beneficial for many patients. However, current FDA-approved preventive treatments have limitations.
  3. HAE patients participating in this clinical trial (37 total) received either two shots of an antibody called lanadelumab 14 days apart, or a placebo. The strength of the shot varied from 30 to 400 mg and results were analyzed for the 8-50 days following treatment.
  4. Researchers found that from day 8 to day 50, the 300-mg and 400-mg groups had 100% and 88% fewer HAE attacks, respectively, than the placebo group. All patients in the 300-mg group and 82% (9 of 11) in the 400-mg group were attack-free, as compared with 27% (3 of 11) in the placebo group. Reports of pain at the injection site and headache were similar across all groups, and no serious side effects were reported, supporting the safety of the treatment.
  5. “If this kind of efficacy is seen in the larger phase 3 trial, which is now underway here at Mass General and many other sites, this could significantly improve the quality of life for patients with HAE,” says Aleena Banerji, MD, corresponding and a lead author of the NEJM. 

Learn more about this study here.

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