NTDT Patient Management
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Questions and Answers

What is the primary goal of luspatercept in treating anemia in NTDT?

  • To increase hepcidin production
  • To decrease the transfusion of red blood cells (correct)
  • To reduce iron overload
  • To increase the lifespan of erythroid cells

    • What was the primary endpoint met in the BEYOND trial?

  • Reduction in iron overload
  • Decrease in tiredness and weakness
  • Increase in hemoglobin level of ≥2 g/dL
  • Increase in hemoglobin level of ≥1 g/dL (correct)

    • Which of the following is NOT an approach to treating anemia in NTDT?

  • Stimulating hepcidin production
  • Transfusing red blood cells (correct)
  • Inhibiting ferroportin
  • Activating pyruvate kinase

    • What was the response rate in the luspatercept group in the BEYOND trial?

    <p>77%</p> Signup and view all the answers

    What is the name of the phase 2 randomized placebo-controlled trial evaluating the efficacy and safety of luspatercept?

    <p>BEYOND trial</p> Signup and view all the answers

    What is the name of the ferroportin inhibitor mentioned in the text?

    <p>VIT-276322</p> Signup and view all the answers

    What is the name of the pyruvate kinase activators mentioned in the text?

    <p>Mitapivat and Etavopivat</p> Signup and view all the answers

    In which year was the application for regulatory approval of luspatercept withdrawn in the United States?

    <p>2022</p> Signup and view all the answers

    What is the name of the antisense oligonucleotide mentioned in the text?

    <p>TMPRSS6LRx</p> Signup and view all the answers

    What is the name of the small interfering ribonucleic acid mentioned in the text?

    <p>SLN124</p> Signup and view all the answers

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