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


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 as number NCT00713648.

DOI: 10.1182/blood-2011-10-386045

6 Figures and Tables

Citations per Year

78 Citations

Semantic Scholar estimates that this publication has 78 citations based on the available data.

See our FAQ for additional information.

Cite this paper

@article{Inbal2012RecombinantFX, title={Recombinant factor XIII: a safe and novel treatment for congenital factor XIII deficiency.}, author={Aida Inbal and Johannes Oldenburg and Manuel D Carcao and Anders Rosholm and Ramin Tehranchi and Diane Nugent}, journal={Blood}, year={2012}, volume={119 22}, pages={5111-7} }