Hereditary Factor X Deficiency Is a Rare Bleeding Disorder that Affects About 1 in a Million People1,2

  • Characterized by insufficient levels of factor X
  • Affects both women and men
  • People with factor X deficiency have a high risk of excess bleeding
  • Often hereditary, as an autosomal recessive disorder
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Hereditary Factor X Deficiency in Neonates
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View 10 Fast Facts About Hereditary Factor X Deficiency

Diagnostic Indicators of Factor X Deficiency Include Bleeding Symptoms and Prolonged PT and aPTT1,2

Diagram showing the steps to Factor X deficiency diagnosis including: 1. past history of symptoms, 2. ruling out other bleeding disorders, 3. testing and 4. diagnosis.
aPTT, activated partial thromboplastin time; PT, prothrombin time.
Early diagnosis of factor X deficiency may allow for earlier initiation of appropriate care and treatment

Factor X Deficiency May Present with Ongoing Symptoms

Photo representing Fernando
Fernando coped with more than 25 years of joint swelling, pain, and bruising, until he was tested and diagnosed with factor X deficiency after being hospitalized for an intracranial hemorrhage. (Actual patient experience; name/photo changed for privacy)

Symptom occurrence with factor X deficiency (N=42)3*

Red spot on wrist
Easy bruising
55%
Red spot on wrist
Epistaxis
36%
Red spot on swollen elbow
Hemarthrosis
33%
Red spot on wrist
Intracranial
hemorrhage
21%

Important Note

Up to 1 in 5 symptomatic patients with factor X deficiency have had an intracranial hemorrhage.
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  • In this analysis, additional symptoms of factor X deficiency included hematomas (43%), gum bleeding (31%), gastrointestinal hemorrhage (12%), and hematuria (7%)

*Refers to data collected from one series of patients from three Central European and two Latin American countries in a 2006 study; numbers reflect characterization of the 42 symptomatic patients out of 102 total subjects with confirmed reduced FX activity and identified F10 gene causative mutations (<50% of the sample were symptomatic).

Patients with factor X deficiency may have a high risk of spontaneous, severe, or life-threatening bleeds3

The majority of women with factor X deficiency have a history of heavy menstrual bleeding3

Photo representing Isabelle
Isabelle struggled for years with heavy menstrual bleeding but was not tested or diagnosed with factor X deficiency until age 21, when she required a blood transfusion following dental surgery. (Actual patient experience; name/photo changed for privacy)
Woman icon
70%

In women of reproductive age with hereditary factor X deficiency:

  • Up to 70% (12/17) have heavy menstrual bleeding3†
  • Risks with pregnancy include miscarriage, uterine bleeding, postpartum hemorrhage, and preterm labor
†Refers to data collected from one series of patients from three Central European and two Latin American countries in a 2006 study; numbers reflect characterization of the 42 symptomatic patients (n=17 females of reproductive age) out of 102 total subjects with confirmed reduced FX activity and identified F10 gene causative mutations (<50% of the sample were symptomatic).
Patients with factor X deficiency may present with bleeds related to women’s health

Undiagnosed and untreated neonates can be at high risk for severe bleeds

Affected neonates are at risk of experiencing devastating outcomes if hereditary factor X deficiency is not promptly recognized and treated

Newborn baby icon

Symptoms in neonates3,4:

  • Intracranial hemorrhage (ICH)
  • Gastrointestinal (GI) bleeding
  • Bleeding that won’t stop normally from the umbilical stump or post-circumcision
  • Abnormal bruising or bleeding
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With Hereditary Factor X Deficiency, ICH and GI Bleeds are Likely to Occur Early in Life3,4

Calendar highlighting the 10th day with text: 9.7 days Median Age at ICH or GI Bleed
  • In a study of 102 adult and pediatric patients with hereditary factor X deficiency, 42 of whom were symptomatic—
    • In neonates, ICH and GI bleeds presented at a median age of 9.7 days
“Patients with FXD often present in infancy with unprovoked umbilical stump, intracranial, or gastrointestinal bleeding…” 5
Zimowski KL, et al. 2020.
Use of plasma-derived factor X concentrate in neonates and infants with congenital factor X deficiency.
ICH due to factor X deficiency can result in permanent impairment or mortality in neonates4

Severity Classification is Unique to Factor X4,6–8

SEVERE

<10 IU/dL
factor X activity
icon showing a blood sample with almost no factor X

High risk of major spontaneous bleeding

MODERATE

10–40 IU/dL
factor X activity
icon showing a blood sample with very little factor X

Minor spontaneous or triggered bleeding risk

MILD

>40–65 IU/dL
factor X activity
icon showing a blood sample with less than a normal amount of factor X

Low bleeding risk

  • Decreasing factor X activity has been shown to correlate with increasing bleeding severity7
  • Severity classification as shown is currently widely accepted but differs from that existing at the time of COAGADEX clinical trials, which classified “severe” factor X deficiency activity as <1 IU/dL8
  • The COAGADEX prescribing information recommendation is to monitor trough blood levels of factor X targeting ≥5 IU/dL and adjust dosage to clinical response and trough levels9
  • Analysis of registry data from the European Network of Rare Bleeding Disorders (EN-RBD) suggests maintaining a factor X level of ≥10 IU/dL as the appropriate target for prophylaxis, due to the high risk of major spontaneous bleeding (such as GI or CNS) at <10 IU/dL7,10
  • In the general population, factor X activity ranges from 65–120 IU/dL (reference ranges may vary)8
Peyvandi F, et al. J Thromb Haemost. 2012.
A review of data by the Rare Bleeding Disorders Working Group under the Factor VIII and Factor IX Scientific and Standardisation Committee of the International Society on Thrombosis and Haemostasis, exploring the association between residual plasma coagulant factor activity and clinical bleeding profile for each rare bleeding disorder (RBD; N=4,359 patients with RBDs in North American, European, UK, and Indian registries). Factor X severity classification based on analysis of registry data from the European Network of RBDs (EN-RBD; n=592 patients with RBDs).
Unexpected bleeding has been observed over a broad range of factor X deficiency4,6

Treatment Considerations

Clinical guidelines and expert consensus recommend use of plasma-derived factor X concentrate for the treatment of hereditary factor X deficiency4,7,9

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The 2022 National Hemophilia Foundation (NHF) MASAC treatment guidelines recommend only plasma-derived factor X concentrate for the treatment of hereditary factor X deficiency11
NHF Medical and Scientific Advisory Council (MASAC)
Recommendations concerning products licensed for the treatment of hemophilia and other bleeding disorders, 2022.
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“The treatment of factor X deficiency has evolved substantially, from plasma replacement therapy (fresh frozen plasma) to prothrombin complex concentrates (PCCs) to, most recently, the development and licensing approval of a high-purity, plasma-derived, specific single-factor concentrate (pdFX). While patient numbers are small, clinical studies have demonstrated the ability of pdFX therapy to improve hemostasis while limiting the safety concerns associated with earlier treatments such as volume overload (particularly in neonates and young children), anaphylaxis, and thrombosis.”7
Peyvandi F, et al.
Diagnosis, therapeutic advances, and key recommendations for the management of factor X deficiency.
Blood Reviews. 2021.
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“Single-factor FX concentrate is currently considered the standard of care for both on-demand and prophylactic treatment of patients with hereditary FX deficiency…The use of prophylaxis to reduce bleeding risk is essential for patients with FX deficiency given the risk of spontaneous and/or life-threatening bleeds.”4
Tarantino MD.
Occurrence and management of severe bleeding episodes in patients with hereditary factor X deficiency.
Haemophilia. 2021.
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“Patients with severe FX deficiency tend to be the most seriously affected patients with rare coagulation disorders…”12
Menegatti M, et al.
Treatment of rare factor deficiencies other than hemophilia.
Blood. 2019.
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Learn more about the first and only product specifically for children and adults with hereditary factor X deficiency

References: 1. Brown DL, Kouides PA. Haemophilia. 2008;14(6):1176-1182. 2. Palla R, et al. Blood. 2015;125(13):2052-2061. 3. Hermann FH, et al. Haemophilia. 2006;12:479-489. 4. Tarantino MD. Haemophilia. 2021;27:531-543. 5. Zimowski KL, McGuinn YL, Abajas YL, et al. J Thromb Haemost. 2020;18:2551-2556. 6. Peyvandi F, Palla R, et al. J Thromb Haemost. 2012;10:615-621. 7. Peyvandi F, Auerswald G, Austin SK, et al. Blood Reviews. 2021;50:100833. 8. Peyvandi F, et al. Brit J Haematol. 1998;102:626-628. 9. COAGADEX® (Coagulation Factor X, Human) Prescribing Information. Durham, NC: BPL Limited. 2023. 10. Peyvandi F, et al. J Thromb Haemost. 2012;10:1938-1943. 11. Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation. MASAC Document #272. https://www.hemophilia.org/healthcare-professionals/guidelines-on-care/masac-documents/masac-document-272-masac-recommendations-concerning-products-licensed-for-the-treatment-of-hemophilia-and-other-bleeding-disorders. Accessed November 9, 2022. 12. Menegatti M, Peyvandi F. Blood. 2019;133:415-424.

Replace exactly what's missing

Indications and Usage for COAGADEX

COAGADEX, a plasma-derived blood coagulation factor X concentrate, is indicated in adults and children with hereditary factor X deficiency for:

  • Routine prophylaxis to reduce the frequency of bleeding episodes
  • On-demand treatment and control of bleeding episodes
  • Perioperative management of bleeding in patients with mild, moderate and severe hereditary factor X deficiency

Contraindication for COAGADEX

COAGADEX is contraindicated in patients who have had life-threatening hypersensitivity reactions to COAGADEX.

Important Safety Information for COAGADEX

Allergic type hypersensitivity reactions, including anaphylaxis, are possible with COAGADEX. If symptoms occur, patients should discontinue use of the product immediately, contact their physician, and administer appropriate treatment.

The formation of neutralizing antibodies (inhibitors) to factor X is a possible complication in the management of individuals with factor X deficiency. Carefully monitor patients taking COAGADEX for the development of inhibitors by appropriate clinical observations and laboratory tests.

COAGADEX is made from human plasma and may contain infectious agents, e.g. viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent. No cases of transmission of viral diseases, vCJD or CJD, have been associated with the use of COAGADEX.

In clinical studies, the most common adverse reactions (frequency ≥5% of subjects) with COAGADEX were infusion site erythema, infusion site pain, fatigue and back pain.

Please see complete Prescribing Information for COAGADEX.

IMPORTANT SAFETY INFORMATION