NTDT Patient Management

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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?

77% 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?

BEYOND trial Signup and view all the answers

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

VIT-276322 Signup and view all the answers

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

Mitapivat and Etavopivat Signup and view all the answers

In which year was the application for regulatory approval of luspatercept withdrawn in the United States?

2022 Signup and view all the answers

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

TMPRSS6LRx Signup and view all the answers

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

SLN124 Signup and view all the answers

NTDT Patient Management

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Questions and Answers

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

What was the primary endpoint met in the BEYOND trial?

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

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

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

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

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

In which year was the application for regulatory approval of luspatercept withdrawn in the United States?

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

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

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