203 Symposium: Hereditary Angioedema: Cardinal Features
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Questions and Answers

What is the cardinal feature of Hereditary Angioedema (HAE)?

  • Recurrent attacks of subcutaneous swelling (correct)
  • Recurrent attacks of muscle weakness
  • Recurrent attacks of joint pain
  • Recurrent attacks of visual disturbances

Which body sites have a predilection for swelling in Hereditary Angioedema?

  • Hands, feet, face, and genitals (correct)
  • Abdomen and chest
  • Head and neck
  • Back and shoulders

What is the inheritance pattern of Hereditary Angioedema?

  • Autosomal recessive
  • Autosomal dominant (correct)
  • X-linked dominant
  • Mitochondrial

Which type of HAE is characterized by low levels of C1 inhibitor protein?

<p>Type 1 (C)</p> Signup and view all the answers

What is the major mediator of swelling in C1 inhibitor deficiency?

<p>Bradykinin (D)</p> Signup and view all the answers

What triggers an increased risk of attacks in patients with HAE?

<p>Surgery and infections (D)</p> Signup and view all the answers

What is the recommended strategy for treating all abdominal attacks and any airway attacks in HAE patients?

<p>Self-administration of medication (D)</p> Signup and view all the answers

Which on-demand treatment option for HAE involves a slow intravenous injection?

<p>Berinert (A)</p> Signup and view all the answers

'Kallikrein' is involved in which pathway that leads to swelling in C1 inhibitor deficiency?

<p>'Bradykinin' pathway (D)</p> Signup and view all the answers

'C1 inhibitor deficiency' results in increased activity of which mediator responsible for swelling?

<p>'Bradykinin' (C)</p> Signup and view all the answers

Which treatment option in the text has a short half-life, high cost, and intravenous formulation, limiting its use?

<p>Cinryze (D)</p> Signup and view all the answers

What is the effect of Lanadelumab on attack frequency compared to placebo?

<p>90% reduction (D)</p> Signup and view all the answers

Which drug is described as a small molecule inhibitor of pre-kallikrein and the only licensed oral prophylaxis option?

<p>Berotralstat (C)</p> Signup and view all the answers

What is the most common indication for the use of C1 inhibitor concentrate injections in the text?

<p>Pregnancy (D)</p> Signup and view all the answers

Which treatment option is described as having gastro-intestinal side-effects in some patients and requires several weeks to reach full effect?

<p>Deucrictibant (B)</p> Signup and view all the answers

What is the dosing frequency for Berotralstat, the small molecule inhibitor of pre-kallikrein?

<p>Once daily (C)</p> Signup and view all the answers

Which drug mentioned in the text is an anti-sense inhibitor of pre-kallikrein and is in phase 3?

<p>Donidalorsen (B)</p> Signup and view all the answers

What is the effect of Berotralstat on attack frequency compared to placebo?

<p>40% reduction (C)</p> Signup and view all the answers

What is the common side effect of Deucrictibant according to the text?

<p>Gastrointestinal issues (B)</p> Signup and view all the answers

Which drug treatment option mentioned is seeking approval according to the text?

<p>Donidalorsen (C)</p> Signup and view all the answers

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