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Recombinant factor XIII: a safe and novel treatment for congenital factor XIII deficiency.

Abstract
Congenital factor XIII (FXIII) deficiency is a rare, autosomal-recessive disorder, with most patients having an A-subunit (FXIII-A) deficiency. Patients experience life-threatening bleeds, impaired wound healing, and spontaneous abortions. In many countries, only plasma or cryoprecipitate treatments are available, but these carry a risk for allergic reactions and infection with blood-borne pathogens. The present study was a multinational, open-label, single-arm, phase 3 prophylaxis trial evaluating the efficacy and safety of a novel recombinant FXIII (rFXIII) in congenital FXIII-A subunit deficiency. Forty-one patients ≥ 6 years of age (mean, 26.4; range, 7-60) with congenital FXIII-A subunit deficiency were enrolled. Throughout the rFXIII prophylaxis, only 5 bleeding episodes (all trauma induced) in 4 patients were treated with FXIII-containing products. The crude mean bleeding rate was significantly lower than the historic bleeding rate (0.138 vs 2.91 bleeds/patient/year, respectively) for on-demand treatment. Transient, non-neutralizing, low-titer anti-rFXIII Abs developed in 4 patients, none of whom experienced allergic reactions, any bleeds requiring treatment, or changes in FXIII pharmacokinetics during the trial or follow-up. These non-neutralizing Abs declined below detection limits in all 4 patients despite further exposure to rFXIII or other FXIII-containing products. We conclude that rFXIII is safe and effective in preventing bleeding episodes in patients with congenital FXIII-A subunit deficiency. This study is registered at http://www..clinicaltrials.gov as number NCT00713648.
AuthorsAida Inbal, Johannes Oldenburg, Manuel Carcao, Anders Rosholm, Ramin Tehranchi, Diane Nugent
JournalBlood (Blood) Vol. 119 Issue 22 Pg. 5111-7 (May 31 2012) ISSN: 1528-0020 [Electronic] United States
PMID22451421 (Publication Type: Clinical Trial, Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't)
Chemical References
  • Antibodies
  • Recombinant Proteins
  • F8 protein, human
  • Factor VIII
Topics
  • Adolescent
  • Adult
  • Antibodies (blood)
  • Child
  • Drug Hypersensitivity (etiology)
  • Factor VIII (administration & dosage, adverse effects)
  • Female
  • Hemophilia A (blood, drug therapy)
  • Hemorrhage (blood, drug therapy)
  • Humans
  • Male
  • Middle Aged
  • Recombinant Proteins (administration & dosage, adverse effects)
  • Risk Factors
  • Safety

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