Clinical utility and patient perspectives on the use of extended half-life rFIXFc in the management of hemophilia B

Maricel G. Miguelino, Jerry S Powell

Research output: Contribution to journalArticle

Abstract

Hemophilia B is an X-linked genetic disease caused by mutation of the gene for coagulation protein factor IX (FIX), with an incidence of approximately once every 30,000 male births in all populations and ethnic groups. When severe, the disease leads to spontaneous life threatening bleeding episodes. When untreated, most patients die from bleeding complications before 25 years of age. Current therapy requires frequent intravenous infusions of therapeutic recombinant or plasma-derived protein concentrates containing FIX. Most patients administer the infusions at home every few days, and must limit their physical activities to avoid abnormal bleeding when the FIX activity levels are below normal. After completing the pivotal Phase III clinical trial, a new therapeutic FIX preparation that has been engineered for an extended half-life in circulation, received regulatory approval in March 2014 in Canada and the US. This new FIX represents a major therapeutic advance for patients with hemophilia B. The half-life is prolonged due to fusion of the native FIX molecule with the normal constant region of immunoglobulin G. This fusion molecule then follows the normal immunoglobulin recirculation pathways through endothelial cells, resulting in prolonged times in circulation. In the clinical trials, over 150 patients successfully used eftrenonacog alfa regularly for more than 1 year to prevent spontaneous bleeding, to successfully treat any bleeding episodes, and to provide effective coagulation for major surgery. All infusions were well tolerated and effective, with no inhibitors detected and no safety concerns. This promising therapy should allow patients to use fewer infusions to maintain appropriate FIX activity levels in all clinical settings.

Original languageEnglish (US)
Pages (from-to)389-395
Number of pages7
JournalPatient Preference and Adherence
Volume8
DOIs
StatePublished - Oct 14 2010

Fingerprint

Hemophilia B
Factor IX
Half-Life
Hemorrhage
management
Disease
population group
X-Linked Genetic Diseases
surgery
ethnic group
incidence
Canada
Therapeutics
Phase III Clinical Trials
Population Groups
Ethnic Groups
Intravenous Infusions
Immunoglobulins
Blood Proteins
Endothelial Cells

Keywords

  • Extended half-life
  • Factor IX
  • Genetic coagulation defects
  • Hemophilia B
  • Prophylaxis

ASJC Scopus subject areas

  • Social Sciences (miscellaneous)
  • Medicine (miscellaneous)
  • Health Policy
  • Pharmacology, Toxicology and Pharmaceutics (miscellaneous)

Cite this

Clinical utility and patient perspectives on the use of extended half-life rFIXFc in the management of hemophilia B. / Miguelino, Maricel G.; Powell, Jerry S.

In: Patient Preference and Adherence, Vol. 8, 14.10.2010, p. 389-395.

Research output: Contribution to journalArticle

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