Prophylactic Therapy with Fibrogammin Is Associated with a Decreased Incidence of Bleeding Episodes: A Retrospective Case Control Study.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 1014-1014
Author(s):  
Jeanne M. Lusher ◽  
Steven W. Pipe ◽  
Sarah Alexander ◽  
Diane J. Nugent
Keyword(s):  
Factor Xiii ◽  
Age At Onset ◽  
Retrospective Chart Review ◽  
Phase Iii ◽  
Prophylactic Therapy ◽  
Retrospective Case ◽  
Factor Xiii Deficiency ◽  
Spontaneous Bleeding ◽  
Bleeding Episodes ◽  
Congenital Factor

Abstract In an ongoing Phase III study we are evaluating the prophylactic efficacy and long-term safety of Fibrogammin® P, a purified, pasteurized concentrate of Factor XIII, in patients (pts) with congenital Factor XIII deficiency (an extremely rare, hereditary, life-threatening bleeding disorder). The study is being conducted at 37 sites and has enrolled 61 pts; these 61 represent approximately 2/3 of US patients with FXIII deficiency. For this analysis, a retrospective chart review was undertaken to compare spontaneous bleed-event rates 24 months prior to (when possible) and after the initiation of therapy in a subset of 7 study subjects who did not receive any prophylactic therapy prior to entering the study. The table below presents data for each of these pts on dosing, bleeding episodes and treatment (Rx) during both observation periods (Obs Pd). An additional 4 pts had been treated prophylactically with regular infusions of FFP or cryoprecipitate and transitioned successfully to FXIII therapy; there were no seroconversions to HIV, Hepatitis B/C in any study pts. Pre-Fibrogammin P Therapy On Fibrogammin P Therapy Pt # Age ♦ Obs Pd (mos) / # Bleeds; Bleed Type Rx Obs Pd mos; (# inf) / # bleeds Bleed Type Dose/Kg† ♦ Age at onset of Fibrogammin Prophylactic therapy; † Pts were dosed on a unit basis (1250 unit vials), at 5-wk intervals, variability in weight and vial size lead to a range of per Kg doses; ⋄ p = .01 vs pre-therapy 504 5 yr 24 / 2 Hematomas none 12 (12) / 0 27 2703 10 mo 9 / 2 Umbilical cord; extensive bruising FFP 25 (26) / 1 Head trauma and bruising 29.7 – 44.9 2704 20 mo 6 / 4 Forehead × 2; frenulum × 2; other FFP × 5 26 (26) / 2 Traumatic foot; traumatic head (extracranial) 18.3 – 43.1 2705 9 yr 24 / 3 Lip; ankle; forehead FFP 13 (12) / 0 none 19.9 – 29.7 3301 19 yr 24 / 5 Large hematomas none 9.5 (11) / 0 none 18.9 3302 17 yr 24 / 0 none none 9.5 (12) / 0 none 24.3 3303 14 yr 24 / 8 Intracranial; others × 7 FFP, Cryoppt 10 (11) / 0 none 15.6 Mean 2.5 bleeds/yr 0.2 bleeds/yr⋄ As shown in the table, these children had 2.5 bleeds per year in the period before Fibrogammin prophylaxis, and only 0.2 per year while on Fibrogammin. These results demonstrate a consistent and clinically significant reduction in spontaneous bleeding with prophylactic use of Fibrogammin® P in pts with symptomatic congenital Factor XIII deficiency.

Acquired Factor XIII Deficiency in the Inpatient Setting: Clinical Suspicion, Impact and Treatment: Retrospective Case Series

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 1415-1415
Author(s):  
Fernando Chuliber ◽  
Natalia Paola Schutz ◽  
Victoria Otero ◽  
Luis Barrera ◽  
Diana Altuna ◽  
...  
Keyword(s):  
Factor Xiii ◽  
Case Series ◽  
Screening Tests ◽  
Coagulation Cascade ◽  
Inpatient Setting ◽  
Retrospective Case ◽  
Factor Xiii Deficiency ◽  
Spontaneous Bleeding ◽  
Retrospective Case Series ◽  
Fxiii Deficiency

Abstract Introduction: Activated factor XIII (FXIII) stabilizes fibrin clots at the end of the coagulation cascade by bridging fibrin molecules. Disproportionate surgery related bleeding has been reported in association with FXIII deficiency, in patients with normal coagulopathies screening tests, and without a history of previous bleedings. Methods: Retrospective case series. We performed immunological factor XIIIA (FXIIIA) studies in the inpatient setting of the Hospital Italiano de Buenos Aires, between January 2014 and March 2016. FXIIIA below 50% were considered deficient. Patients with suspicion of congenital Factor XIII deficiency or 2 years old or less were excluded. Descriptive statistics were used. Populations were compared with chi-square, Fisher and T test, or Mann Whitney tests with Stata13 software. Results: FXIII was studied in 52 patients; 37 of these (26 females and 11 males) met inclusion and exclusion criteria. Median age was 43 years (range 9-81). Twenty two patients were studied because of disproportionate surgery related bleeding, 11 because of spontaneous bleeding; and 4 not specified. All patients presented normal coagulopathies screening tests, normal Von Willebrand factor and ristocetine cofactor activity and normal platelet function tests. Eleven patients (30%) had FXIIIA less than 50%. Statistically significant differences between groups were found for median drop in hematocrit points [3.5 (IQR 3-10) vs 1.5 (IQR 0-2, p=0.02)] and median number of red cell units transfused [4 (IQR 0-6) vs 0 (0-2), p=0.01]. Differences in rates of minor and major bleedings were not statistically significant. Only one patient of eleven presented FXIII deficiency associated spontaneous bleeding vs 8 out of 20 patients in the postsurgical setting. FXIII concentrate was used in two patients to treat persistent bleeding, resolving in less than 24 hours. Three patients died, none because of bleeding. Five of the 11 patients with FXIII deficiency returned to normal values when FXIII assay was repeated away from acute setting. Discussion: FXIII acquired deficiency is associated with disproportionate bleeding in postsurgical setting in patients without apparent coagulation defects. FXIII deficiency could be underdiagnosed. Patients with FXIII deficiency in our series had more hematocrit drop and more transfusion requirements. We found no association with spontaneous bleeding. Alltough deficiency could be transient, treatment with FXIII concentrate could be useful to manage serious, persistent or life threatening bleedings. Disclosures No relevant conflicts of interest to declare.

New Families with Hereditary Hemorrhagic Trait due to Deficiency of Fibrin Stabilizing Factor (F. XIII)

1968 ◽  
Vol 20 (03/04) ◽  
pp. 534-541 ◽  
Author(s):  
O Egeberg
Keyword(s):  
Factor Xiii ◽  
Family Members ◽  
Recessive Inheritance ◽  
Factor Xiii Deficiency ◽  
History Of ◽  
Congenital Factor

SummarySevere hemorrhagic disorder due to congenital factor XIII deficiency is described in two unrelated Norwegian girls.Plasma cephalin time was for both patients extraordinarily short during episodes of bleeding and hematomas. No such hyperactivity reaction was demonstrable in unaffected condition some months later.Estimations of blood factor XIII levels revealed a partial defect in the parents of both children, and also in some other family members, consistent with an autosomal incompletely recessive inheritance of the defect. Some of the presumptive heterozygotes had a history of light bleeding phenomenons; whether this was related to their partial lack of factor XIII is so far uncertain.

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

Blood ◽  
2012 ◽  
Vol 119 (22) ◽  
pp. 5111-5117 ◽  
Author(s):  
Aida Inbal ◽  
Johannes Oldenburg ◽  
Manuel Carcao ◽  
Anders Rosholm ◽  
Ramin Tehranchi ◽  
...  
Keyword(s):  
Factor Xiii ◽  
Autosomal Recessive Disorder ◽  
Allergic Reactions ◽  
Open Label ◽  
Impaired Wound Healing ◽  
Bleeding Rate ◽  
Life Threatening ◽  
A Subunit ◽  
Bleeding Episodes ◽  
Congenital Factor

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.

Successful pregnancy in a woman with congenital factor XIII deficiency treated with substitutive therapy

1987 ◽  
Vol 55 (1) ◽  
pp. 45-48 ◽  
Author(s):  
F. Rodeghiero ◽  
G. C. Castaman ◽  
E. Bona ◽  
M. Ruggeri ◽  
E. Dini
Keyword(s):  
Factor Xiii ◽  
Factor Xiii Deficiency ◽  
Successful Pregnancy ◽  
Congenital Factor

scholarly journals A Novel Molecular Mechanism of Severe Congenital Factor XIII Deficiency

2020 ◽  
Author(s):  
Jingjing Han ◽  
Miao Jiang ◽  
Jian Su ◽  
Ziqiang Yu ◽  
Xia Bai ◽  
...  
Keyword(s):  
Molecular Mechanism ◽  
Factor Xiii ◽  
Factor Xiii Deficiency ◽  
Congenital Factor

Noninvasive prenatal diagnosis of congenital factor XIII deficiency in Iran

2022 ◽  
Vol Publish Ahead of Print ◽  
Author(s):  
Hoda Motlagh ◽  
Akbar Dorgalaleh ◽  
Shadi Tabibian ◽  
Majid Naderi ◽  
Farhad Zaker
Keyword(s):  
Prenatal Diagnosis ◽  
Factor Xiii ◽  
Factor Xiii Deficiency ◽  
Noninvasive Prenatal Diagnosis ◽  
Congenital Factor

scholarly journals Use of Catridecacog in a patient with severe Factor XIII deficiency undergoing surgery

2019 ◽  
Vol 11 (1) ◽  
Author(s):  
Gianluca Sottilotta ◽  
Francesca Luise ◽  
Vincenzo Oriana ◽  
Angela Piromalli ◽  
Rosa Santacroce ◽  
...  
Keyword(s):  
Inguinal Hernia ◽  
Surgical Procedures ◽  
Factor Xiii ◽  
Bleeding Disorder ◽  
Factor Xiii Deficiency ◽  
Fxiii Deficiency ◽  
Congenital Factor

Despite many articles regarding the antihemorrhagic treatment and prophylaxis, there is a lack of experience about how to best conduct major surgical procedures in patients with congenital factor XIII (FXIII) deficiency. Here we report a case of surgery (right inguinal hernia, complicated by heaviness and pain) performed in a patient with FXIII deficiency, receiving recombinant FXIII prophylaxis (Catridecacog 35 UI/kg every 28±2 days). Our experience shows that Catridecacog can be used safely and effectively not only for continued prophylaxis but also in surgery and adds to the very limited body of evidence currently available on surgery in this bleeding disorder.

Sign in / Sign up

Export Citation Format

Share Document