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How I approach: Previously untreated patients with severe congenital hemophilia A

Courtney D Thornburg1,2

  • 1Rady Children's Hospital San Diego, San Diego, California.

Pediatric Blood & Cancer
|September 26, 2018
PubMed

Previously untreated patients with severe hemophilia A are a vulnerable population at risk for severe bleeding which is currently managed with exogenous clotting factor replacement. The primary burden of current treatment is high-titer inhibitor development. Evolving data on current treatment products as well as emerging therapeutics may inform treatment decisions to prevent bleeding and inhibitor formation. Considerations for diagnosis, education, and shared decision-making related to product choice and treatment regimen are discussed.

Keywords:
factor replacementgenotypinghemophiliainhibitorprophylaxis

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