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Evolving Complexity in Hemophilia Management

Stacy E Croteau1

  • 1Boston Children's Hospital, 300 Longwood Avenue, Boston, MA 02115, USA.

Pediatric Clinics of North America
|May 28, 2018
PubMed

Rapid expansion of therapeutic options have increased the complexity of hemophilia care. Previously, on-demand therapy aimed to reduce morbidity and early mortality; however, now aggressive prophylaxis, particularly in children, encourages an active lifestyle. Accurate diagnosis, recognition of early threats to musculoskeletal health, and optimization of therapy are critical for both males and females affected by hemophilia. The diversity of emerging hemophilia therapies, from modified factor protein concentrates, to gene therapy, to nonfactor hemostatic strategies, provide an exciting opportunity to target unmet needs in the bleeding disorder community.

Keywords:
Bispecific antibodyBleedingFactor IXFactor VIIIFactor concentrateGene therapyHemarthrosisInhibitors

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