Glossary

Acute attacks

Sudden attacks of hereditary angioedema.

Clinical trials

Studies that evaluate the effectiveness and safety of medications or medical devices before they are approved for use by the general public.

Ecallantide

A protein made in yeast cells; ecallantide is the active ingredient in KALBITOR.

HAE

Abbreviation for hereditary angioedema.

Hereditary Angioedema

A rare, hereditary disease of the immune system that causes attacks of sudden swelling in various parts of the body.

KALBITOR

A prescription medicine used to treat sudden attacks of hereditary angioedema in patients 16 years of age and older. KALBITOR is not a cure for HAE.

KALBITOR Access

KALBITOR Access is a program that provides support services to hereditary angioedema patients, healthcare professionals and caregivers. KALBITOR Access services include health insurance coordination, financial assistance for qualified patients and treatment location set up.

Laryngeal attack

An HAE attack that affects the larynx (throat); this can block or close the airway, leading to difficulty breathing and, potentially, death.

Larynx

The part of the throat that contains the vocal cords and is used for breathing, swallowing, and talking.

Mean Symptom Complex Severity

Patient reported measurement of efficacy. Also known as MSCS score, a point-in-time measure of symptom severity. At baseline, 4 hours, and 24 hours, patients rated the severity on a categorical scale (0 = normal, 1 = mild, 2 = moderate, 3 = severe) for symptoms at each affected anatomical location. Ratings were averaged to obtain the MSCS score. A decrease in MSCS score reflected an improvement in symptoms.

MSCS

Mean Symptom Complex Severity. MSCS score is a point-in-time measure of symptom severity. At baseline, 4 hours, and 24 hours, patients rated the severity on a categorical scale (0 = normal, 1 = mild, 2 = moderate, 3 = severe) for symptoms at each affected anatomical location. Ratings were averaged to obtain the MSCS score. A decrease in MSCS score reflected an improvement in symptoms.

Plasma kallikrein

A protein in the body that triggers the release of a substance that may lead to hereditary angioedema attack symptoms.

Subcutaneous

Under the skin.

TOS

Treatment Outcome Score, patient reported measurement of efficacy. TOS is a measure of symptom response to treatment. At 4 hours and 24 hours, patient assessment of response, characterized by their change from baseline in symptom severity and collected by anatomic site of attack involvement, was recorded on a categorical scale (significant improvement [100], improvement [50], same [0], worsening [-50], significant worsening [-100]). The response at each anatomic site was weighted by baseline severity, and then the weighted scores across all involved sites were averaged to calculate the TOS. A TOS value >0 reflected an improvement in symptoms from baseline.

Treatment Outcome Score

Patient-reported measurement of efficacy. Also known as TOS, a measure of symptom response to treatment. At 4 hours and 24 hours, patient assessment of response, characterized by their change from baseline in symptom severity and collected by anatomic site of attack involvement, was recorded on a categorical scale (significant improvement [100], improvement [50], same [0], worsening [-50], significant worsening [-100]). The response at each anatomic site was weighted by baseline severity, and then the weighted scores across all involved sites were averaged to calculate the TOS. A TOS value >0 reflected an improvement in symptoms from baseline.

Trigger

In hereditary angioedema, something that causes an attack.