@entity19434 is a rare, @entity74 , with most @entity1 having an @entity1129 . @entity1 experience life-threatening @entity548 , @entity73 , and @entity2208 . In many countries, only plasma or cryoprecipitate treatments are available, but these carry a risk for @entity56 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 @entity69859 . Forty-one @entity1 >=  6 years of age (mean, 26.4; range, 7-60) with @entity69859 were enrolled. Throughout the rFXIII prophylaxis, only 5 @entity548 episodes (all @entity130 induced) in 4 @entity1 were treated with FXIII-containing products. The crude mean @entity548 rate was significantly lower than the historic @entity548 rate (0.138 vs 2.91 @entity548 / @entity1 /year, respectively) for on-demand treatment. Transient, non-neutralizing, low-titer anti-rFXIII Abs developed in 4 @entity1 , none of whom experienced @entity56 , any @entity548 requiring treatment, or changes in FXIII pharmacokinetics during the trial or follow-up. These non-neutralizing Abs declined below detection limits in all 4 @entity1 despite further exposure to rFXIII or other FXIII-containing products. We conclude that rFXIII is safe and effective in preventing @entity548 episodes in @entity1 with @entity69859 . This study is registered at http://www..clinicaltrials.gov as number NCT00713648.

Recombinant factor XIII: a safe and novel treatment for XXXX .

@entity1 :: ('9606', 'Species') :: ['Patients', 'patients', 'patient'] @entity2208 :: ('MESH:D000022', 'Disease') :: ['spontaneous abortions'] @entity69859 :: ('MESH:C567688', 'Disease') :: ['congenital FXIII-A subunit deficiency'] @entity548 :: ('MESH:D006470', 'Disease') :: ['bleeding', 'bleeds'] @entity73 :: ('MESH:D003072', 'Disease') :: ['impaired wound healing'] @entity56 :: ('MESH:D004342', 'Disease') :: ['allergic reactions'] @entity74 :: ('MESH:D030342', 'Disease') :: ['autosomal-recessive disorder'] @entity1129 :: ('MESH:D001260', 'Disease') :: ['A-subunit (FXIII-A) deficiency'] @entity19434 :: ('MESH:D005177', 'Disease') :: ['Congenital factor XIII (FXIII) deficiency'] @entity130 :: ('MESH:D014947', 'Disease') :: ['trauma']
@entity19434 :: (MESH:D005177,Disease) :: ['congenital factor XIII deficiency']
