Prophylactic treatment of bleeding episodes in children &lt;12 years with moderate to severe hereditary factor X deficiency (<scp>FXD</scp>): Efficacy and safety of a high‐purity plasma‐derived factor X (pd<scp>FX</scp>) concentrate

Liesner, Raina; Akanezi, Chioma; Norton, Miranda; Payne, Jeanette

doi:10.1111/hae.13500

Cited by 14 publications

(72 citation statements)

References 20 publications

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“…Larger doses and lower IR were reported in younger patients in the TEN02 trial. The maximum dose in the TEN02 trial was 60 IU/kg every 48 hours with a recommended prophylaxis regimen of 40 IU/kg twice weekly 14 . Younger children (2.6‐5 years of age) demonstrated a lower 30‐minute IR (1.53 IU/dL per IU/kg) compared with older children (6‐12 years of age, 1.91 IU/dL per IU/kg), suggesting pdFX pharmacokinetics may vary with age 14 .…”

Section: Resultsmentioning

confidence: 99%

“…The TEN02 trial reported the outcomes of prophylactic pdFX in patients < 12 years old with moderate or severe FXD. This study included four patients aged 2.6 to 3.6 years old who received at least 6 months (or ≥50 exposure days) of prophylactic pdFX 14 . Two additional pediatric patients were included in the compassionate use trial, TEN05 15 .…”

Section: Introductionmentioning

confidence: 99%

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Use of plasma‐derived factor X concentrate in neonates and infants with congenital factor X deficiency

Zimowski

McGuinn

Abajas

et al. 2020

Journal of Thrombosis and Haemostasis

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Congenital factor X deficiency (FXD) is an autosomal recessive bleeding disorder caused by mutations in the F10 gene with a prevalence of 1:1 000 000. 1 FXD is one of the most severe rare bleeding disorders, and the bleeding phenotype correlates well with plasma FX activity levels (FX:C). Patients with severe FXD often present in the neonatal period with umbilical stump bleeding or intracranial hemorrhage (ICH). Throughout life, these patients can experience hemarthroses, intramuscular hematomas, and gastrointestinal bleeding. Patients with moderate or mild FXD manifest mucocutaneous bleeding and excessive traumatic and postsurgical bleeding. 2-4 Long-term prophylaxis with infusions of FX-containing products is recommended in patients with FXD and a personal or family history of severe bleeding. 1,5-7 Until recently, fresh frozen plasma (FFP) or prothrombin complex concentrates (PCCs) were the primary source of FX infusions. Because of the

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Section: Resultsmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Use of plasma‐derived factor X concentrate in neonates and infants with congenital factor X deficiency

Zimowski

McGuinn

Abajas

et al. 2020

Journal of Thrombosis and Haemostasis

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“…Clinical trials published to date have demonstrated a favourable efficacy profile for pdFX 59,60 . All clinical studies of pdFX, including on‐demand, prophylactic and/or perisurgical treatment, have reported that patients show no evidence of developing inhibitors to pdFX 59–61 . Recently, a case series was published describing 4 neonates and infants with hereditary FX deficiency who exhibited ICH 62 .…”

Section: Discussionmentioning

confidence: 99%

Occurrence and management of severe bleeding episodes in patients with hereditary factor X deficiency

Tarantino

2021

Haemophilia

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Vitamin K–dependent factor X (FX) plays an important role in thrombin formation, and a deficiency in FX can cause impaired coagulation, the severity of which is usually correlated with the degree of deficiency. Due to the critical role that FX plays in the coagulation cascade, FX deficiency is associated with a higher risk of bleeding than deficiencies in other coagulation factors. Patients with the hereditary autosomal‐recessive homozygous form of FX deficiency, which occurs in approximately 1:1,000,000 individuals worldwide, are often diagnosed when they present with spontaneous life‐threatening haemorrhage (most often intracranial haemorrhage) during the first month of life. In addition to central nervous system bleeds, other severe bleeding types experienced by such patients may include umbilical cord bleeding, gastrointestinal or pulmonary haemorrhage, intramuscular haematomas and/or haemarthrosis. Delayed treatment or inadequate replacement of FX may result in developmental delays, musculoskeletal disabilities or death. The high risk of recurrent severe bleeding necessitates prophylactic replacement therapy for many individuals with severe FX deficiency. Available products for replacement therapy include plasma‐derived FX concentrate and prothrombin complex concentrates. Fresh‐frozen plasma may be used when concentrates are not available but is a less efficient means of FX replacement. This article reviews the literature on severe bleeding in individuals with hereditary FX deficiency and discusses current treatment options.

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“…Two Phase III trials, TEN01 and TEN03, demonstrated the safety and efficacy of pdFX for on‐demand treatment of bleeds (TEN01) in subjects with moderate or severe FXD and perioperative bleeding management for planned surgery (TEN03) in subjects with mild‐to‐severe FXD . The recently published TEN02 Phase III study evaluated the prophylactic use of pdFX in children less than 12 years old with moderate or severe FXD . Investigators rated the efficacy of pdFX in reducing or preventing bleeding episodes during routine prophylaxis and treating bleeds as excellent.…”

Section: Discussionmentioning

confidence: 99%

Plasma‐derived Factor X therapy for treatment of intracranial bleeding in a patient with Factor X deficiency: a case report

et al. 2019

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BACKGROUND: Factor X (FX) deficiency (FXD) is an extremely rare autosomal recessive hereditary hematologic disorder, affecting approximately one in 1,000,000 of the general population. CASE REPORT: This case report describes an infantwith hereditary severe FXD who presented with a spontaneous, life-threatening intracranial hemorrhage and was treated with the first licensed plasma-derived FX (pdFX) concentrate. On admission, laboratory assays showed severe coagulopathy of unknown cause; the patient was empirically treated using a multimodal hemostatic approach with prothrombin complex concentrate, fresh-frozen plasma, and tranexamic acid. Subsequent single-factor coagulation and genetic analyses confirmed the hereditary FXD diagnosis, and the therapeutic regimen was changed to a targeted regimen of 250 IU pdFX daily. Based on careful monitoring of the coagulation profile, pdFX administration frequency was increased to twice daily, followed by a reduction to once every 18 hours. The patient was discharged after 7 weeks of hospitalization in good clinical condition and now receives prophylactic pdFX three times weekly.ABBREVIATIONS: aPTT = activated partial thromboplastin time;FX:C = Factor X activity; FXD = Factor X deficiency; ICH = intracranial hemorrhage; INR = international normalized ratio; PCC(s) = prothrombin complex concentrate(s); pdFX = plasmaderived Factor X.From the

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Prophylactic treatment of bleeding episodes in children <12 years with moderate to severe hereditary factor X deficiency (FXD): Efficacy and safety of a high‐purity plasma‐derived factor X (pdFX) concentrate

Abstract: These results demonstrate the efficacy and safety of pdFX for treating children <12 years with moderate/severe hereditary FXD.

Cited by 14 publications

References 20 publications

Use of plasma‐derived factor X concentrate in neonates and infants with congenital factor X deficiency

Use of plasma‐derived factor X concentrate in neonates and infants with congenital factor X deficiency

Occurrence and management of severe bleeding episodes in patients with hereditary factor X deficiency

Plasma‐derived Factor X therapy for treatment of intracranial bleeding in a patient with Factor X deficiency: a case report

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