Efficacy, safety and pharmacokinetics of a new high‐purity factor X concentrate in women and girls with hereditary factor X deficiency

Kulkarni, Roshni; James, Andra H.; Norton, Miranda; Shapiro, Amy D.

doi:10.1111/jth.13983

Cited by 13 publications

(36 citation statements)

References 11 publications

(24 reference statements)

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Section: Antenatal Complicationsmentioning

confidence: 66%

“…In nine case series, menstrual or gynaecological problems were not mentioned . 46/72 (64%) women required blood products [35 Fresh frozen plasma (FFP), 2 Prothrombin complex concentrate (PCCs) and 9 FX concentrate] and 7/72 (10%) cases required blood transfusion . Tranexamic acid was mentioned as therapy in five cases .…”

Section: Resultsmentioning

confidence: 99%

“…46/72 (64%) women required blood products [35 Fresh frozen plasma (FFP), 2 Prothrombin complex concentrate (PCCs) and 9 FX concentrate] and 7/72 (10%) cases required blood transfusion . Tranexamic acid was mentioned as therapy in five cases . Combined oral contraception (COC) was used in seven cases .…”

Section: Resultsmentioning

confidence: 99%

“…As mentioned earlier, four pregnancies resulted in a miscarriage (13%), three in the first trimester and one was a late miscarriage at 21 weeks . Only one case had prophylactic treatment (twice‐weekly pdFX) and miscarried at 6 weeks. Two cases did not receive prophylaxis; including one miscarriage at 21 weeks and one first trimester miscarriage (gestational age not specified).…”

Section: Antenatal Complicationsmentioning

confidence: 99%

“…Prophylaxis was given during delivery, and postpartum period in 14 pregnancies and 16 pregnancies did not receive prophylaxis during this period (FX levels were < 0.01 IU/mL in one, 0.06 IU/mL in another one, 0.4 IU/mL in a third case and in another levels were not reported), and in one case, there was no record of prophylaxis administered …”

Section: Postnatal Complicationsmentioning

confidence: 99%

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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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Section: Antenatal Complicationsmentioning

confidence: 66%

Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

Section: Antenatal Complicationsmentioning

confidence: 99%

Section: Postnatal Complicationsmentioning

confidence: 99%

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

Spiliopoulos

Kadir

2019

Haemophilia

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Plasma‐derived factor X concentrate compassionate use for hereditary factor X deficiency: Long‐term safety and efficacy in a retrospective data‐collection study

Huang

Liesner

Austin

et al. 2021

Research and Practice in Thrombosis and Haemostasis

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Background Coagadex is a high‐purity plasma‐derived factor X concentrate (pdFX) developed to treat hereditary factor X deficiency (FXD). Objective Evaluate the efficacy and safety of pdFX administered to patients with hereditary FXD. Methods This was an open‐label, multicenter, retrospective analysis of patients receiving pdFX for compassionate use. Efficacy end points included treatments administered, the number and treatment of bleeds, and investigator assessments. Adverse drug reactions (ADRs) were monitored. Results Fifteen patients were included: seven received routine prophylaxis, seven received on‐demand treatment, and one alternated. Most were aged ≥12 years (n = 13) and had severe hereditary FXD (n = 12). The median follow‐up time was 19.2 months (range, 3.5‐48.8). The number of infusions per patient per month was higher for the routine prophylaxis group (median [range], 5.4 [1.4‐10.1]) than for the on‐demand group (0.8 [0.1‐2.3]), as was the dose per infusion (27.9 [21.9‐53.6] IU/kg vs 20.0 [13.6‐27.7] IU/kg). Patients experienced 88 bleeds (34 minor, 7 major, 47 unclassified). The monthly bleed rate per patient was 0.04 in the routine prophylaxis group (based on 17 bleeds in four patients) and 0.8 in the on‐demand group (based on 71 bleeds in eight patients). pdFX was used to treat 79 bleeds and was rated effective in all instances. In an overall assessment, investigators rated pdFX as excellent for 14 patients (93.3%) and good for 1 patient (6.3%). No ADRs or safety concerns were reported. Conclusions This analysis supports the use of pdFX as a safe, effective treatment for hereditary FXD. Routine prophylaxis with pdFX may reduce bleed frequency.

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