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</p> Signup and view all the answers

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

    <p>Bradykinin</p> Signup and view all the answers

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

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

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

    <p>Berinert</p> Signup and view all the answers

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

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

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

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

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

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

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

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

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

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

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

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

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

    <p>Donidalorsen</p> Signup and view all the answers

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