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

Author(s): Inbal A ; Oldenburg J ; Carcao M ; Rosholm A ; Tehranchi R ; Nugent D

Corporate source(s)

Nugent D, UC Irvine Med Sch, Childrens Hosp Orange County, Dept Hematol, 455 S Maine St, Orange, CA 92868 USA,


Blood 2012, Vol. 119, pg. 5111-5117


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. (Blood. 2012; 119(22): 5111-5117)