Congenital factor X deficiency in Japan.

Mori, Kazuo; Sakai, Hideaki; Nakano, Nobuaki; Suzuki, Seikichi; Sugai, Kuniaki; Hisa, Shizue; Gotō, Yoshio

doi:10.1620/tjem.133.1

Cited by 17 publications

(4 citation statements)

References 24 publications

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“…The mean t 1/2 of FVII:C in the MC710-treated ranged from 2.1 to 3.4 h (Table 2a) and was similar to the previous reported value (mean t 1/2 of FVII:C in haemophilia patients without haemorrhage: 2.82 ± 0.53 h [11]). On the other hand, the mean t 1/2 of FX:C ranged from 20.2 to 23.2 h (Table 2c) and was shorter than reported values (t 1/2 of FX:C 24-56 h [12,13]); however, this difference may have been due to the effect of the concurrent presence of FVIIa at a high concentration, different test conditions (agents, blood sampling point) and analytical method. The results of the PD analysis also showed that MC710 had a strong ability to improve prolonged APTT and shorten PT in haemophilia patients with inhibitors.…”

Section: Discussionmentioning

confidence: 61%

Clinical pharmacological study of a plasma‐derived factor VIIa and factor X mixture (MC710) in haemophilia patients with inhibitors – Phase I trial

et al. 2011

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MC710, a combined product of plasma-derived activated factor VII (FVIIa) and factor X (FX) at a protein weight ratio of 1:10, is a novel bypassing agent for haemostasis in haemophilia patients with inhibitors. In this study, pharmacokinetic (PK), pharmacodynamic (PD) parameters and safety of single doses of MC710 were investigated in 11 male haemophilia patients with inhibitors in a non-bleeding state. This was a multi-centre, open-labelled, non-randomized, active controlled crossover, dose-escalation study of five doses (20-120 μg kg(-1) of FVIIa) with re-administration of different MC710 dosages to the same subjects. The active controls were NovoSeven (120 μg kg(-1)) and/or FEIBA (50 and 75 U kg(-1)) which were used to compare PD parameters. The area under the curve (AUC) and maximum plasma concentration (C(max)) of MC710 active ingredients increased dose-dependently within the range of 20 and 120 μg kg(-1). After administration of MC710, activated partial thromboplastin time (APTT) was dose-dependently improved and prothrombin time (PT) was shortened to approximately 6 s at 10 min, and APTT improvement and PT shortening effects were maintained until 12 h after administration of MC710 at all doses. No serious or severe adverse event was observed after administration of MC710; furthermore, several diagnostic marker values and those changes did not indicate any signs of disseminated intravascular coagulation (DIC). These results suggest that MC710 would have haemostatic potential equal to or greater than NovoSeven and FEIBA and was be tolerable when given at doses up to 120 μg kg(-1).

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Section: Discussionmentioning

confidence: 61%

Clinical pharmacological study of a plasma‐derived factor VIIa and factor X mixture (MC710) in haemophilia patients with inhibitors – Phase I trial

et al. 2011

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“…Pedigree analysis has also suggested a two allele genetic model to explain the normal bimodal distribution of factor X activity (Siervogel et al,19 79). In addition to reduced levels of factor X activity, there may be deficiencies in factor X antigen (Denson et al, 1970), although this does not appear to be invariably the case (Denson et al, 1970;Girolami et al, 1972;Parkin et al, 1974;Porter et al, 1979;Moroi et al, 1981). Transient deficiencies of plasma factor X activity have been observed in association with anticoagulant therapy (Hemker et al, 1968;Fair et al, 1979), exposure to fungicide (Graham et al, 1959), infection with Mycoplasma pneumoniae (Peuscher et al, 1979) and a case of combined adenocarcinoma of the kidney and adrenal cortical carcinoma (Stefanini & Wiggishoff, 1976).…”

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confidence: 99%

Heterogeneity of hereditary and acquired factor X deficiencies by combined immunochemical and functional analyses

Fair

Edgington

1985

Br J Haematol

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Limited information is available regarding molecular abnormalities associated with derived factor X deficiencies. In order to assess the types of molecular aberrations that may occur in this group of haemostatic diseases we have analysed plasmas of 33 individuals from 28 kindred exhibiting factor X deficiency. These included those of hereditary type, transiently acquired deficiency as well as factor X deficiency associated with amyloidosis. Plasmas were analysed by one-stage assays for factor X activation by the extrinsic coagulation pathway, intrinsic coagulation pathway and Russell's viper venom. Selected plasmas were analysed in two-stage assays. Normal factor X concentration by specific radioimmunoassay was 6.38 +/- 1.29 micrograms/ml. Factor X associated with factor X deficiency were grouped by their specific activities measured for the three activation pathways and antigen concentration. The results suggest that a broad spectrum of molecular aberrations exist in the factor X deficiency states. The most common group of factor X deficiency was associated with abnormal activation of factor X by all three pathways. Variants of factor X associated with primary amyloidosis and transient acquired deficiency appeared to be abnormal molecules and not just reduced factor X concentration. Hereditary abnormal factor X molecules include the spectrum of potential defective molecules. The relationship of factor X structure to function and the heterogeneity of these defective molecules is discussed.

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“…In total, 332 women were identified; 30 women in 30 case reports (Table ) and 302 women in 19 case series. In the case reports, the FX coagulant activity was <0.01 IU/mL in 14 women, 0.01‐0.05 IU/mL in 8 women and >0.05 IU/mL in four women, and in four women, the severity of FX deficiency was not mentioned . In the case series, 63 patients had FX coagulant activity ≤0.01 IU/mL, 95 patients >0.01 IU/mL (range 0.01‐0.26) and in the rest FX level was not reported.…”

Section: Resultsmentioning

confidence: 93%

Congenital Factor X deficiency in women: A systematic review of the literature

Spiliopoulos

Kadir

2019

Haemophilia

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Factor X deficiency (FXD) is a rare autosomal recessive bleeding disorder with a variable phenotypic severity. In women, heavy menstrual bleeding (HMB), recurrent ovulation bleeding with haemoperitoneum and bleeding complications in pregnancy such as retroplacental haematoma and postpartum haemorrhage have been reported. The aim of this review was to examine gynaecological problems and obstetric complications in women with congenital FXD. A total number of 49 relevant articles were identified, including 332 women, dating from 1960 to 2018. Heavy menstrual bleeding was reported in 72/284 (25%) women in total, 14/30 (47%) in case reports and 58/254 (23%) in 11 case series, 64% and 10% required blood products and blood transfusion, respectively. Haemoperitoneum from ovulation bleeding or ruptured haemorrhagic ovarian cyst requiring blood transfusion occurred in 8/322 (2.4%) women, six required surgical intervention, including oophorectomy in two. 31 pregnancies were reported in 19 women. There were four miscarriages (including a late miscarriage at 21 weeks). There was a high rate of preterm birth and neonatal death occurring in eight (30%) and three (11%) of pregnancies reaching viability stage. Postpartum haemorrhage (PPH) occurred in six (22%) of deliveries, one requiring hysterectomy. In conclusion, women with FXD are at an increased risk of heavy bleeding during menstruation and ovulation as well as adverse pregnancy outcome and postpartum haemorrhage. Collaboration in a multidisciplinary team including an obstetrician/gynaecologist, a perinatologist and a haematologist is necessary for the prevention and management of these complications.

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Congenital factor X deficiency in Japan.

Cited by 17 publications

References 24 publications

Clinical pharmacological study of a plasma‐derived factor VIIa and factor X mixture (MC710) in haemophilia patients with inhibitors – Phase I trial

Clinical pharmacological study of a plasma‐derived factor VIIa and factor X mixture (MC710) in haemophilia patients with inhibitors – Phase I trial

Heterogeneity of hereditary and acquired factor X deficiencies by combined immunochemical and functional analyses

Congenital Factor X deficiency in women: A systematic review of the literature

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