Hereditary Factor X Deficiency Is Rare—But Not for the Patient Who Has It

Person standing in the middle of the letter x
In the general population—
hereditary factor X deficiency affects about 1 in 1 million1
Group of people standing around the letter x
Among people who have been diagnosed with a rare bleeding disorder—
hereditary factor X deficiency affects about 1 in 12*2
Person standing in the middle of the letter x
To the patient in your office with undetected hereditary factor X deficiency—
you are the one who can make a difference

*Based on worldwide distribution of rare bleeding disorders for factor deficiencies of I, II, V, VI, VII, X, XIII, or V+VIII.

Many people with hereditary factor X deficiency may not know they have it and may endure years of suffering before receiving an accurate diagnosis.

About Factor X Deficiency

Factor X deficiency is a rare bleeding disorder caused by dysfunctional factor X protein or insufficient levels of factor X in the blood2

  • People with factor X deficiency have a higher risk of excess bleeding that includes menorrhagia, bleeding in the joints, and a high risk of intracranial hemorrhage

Factor X deficiency is often hereditary, as an autosomal recessive disorder2

  • The condition occurs equally in males and females
  • Prevalence of a factor X deficiency heterozygous carrier state is estimated to be as high as 1 in 500 people

Factor X Deficiency and the Pathway to Diagnosis

As a critical component of the blood coagulation cascade, factor X plays a role in the intrinsic, extrinsic, and common pathways:

  • Intrinsic pathway – Measured clinically as PTT
  • Extrinsic pathway – Measured clinically as PT
  • Common pathway – Factor X initiates the final common pathway for fibrin formation
Hereditary factor X deficiency is suggested when both the PT and PTT are prolonged. Diagnosis is confirmed by testing specific FX activity levels3

PTT, partial thromboplastin time; PT, prothrombin time.

Symptoms of Factor X Deficiency5

Factor X deficiency can present at any age and varies in severity. Patients may experience many different types of bleeds, including potentially life-threatening intracranial bleeds.

In females:

In this analysis, menorrhagia occurred in ~70% of women of reproductive age who had hereditary factor X deficiency

Person silhouette icon
Menorrhagia
70%
Did you know?
About 1 in 5 patients with factor X deficiency have had an intracranial hemorrhage.

Research Shows Bleed Types Vary in Subjects with Factor X Deficiency (N=42)

  • Red spot on wrist
    Easy bruising
    55%
  • Spot on ankle
    Hematomas
    43%
  • Nose bleed
    Epistaxis
    36%
  • Bleeding in the joints
    Hemarthrosis
    33%
  • Gums bleeding
    Gum bleeding
    31%
  • Intracranial hemorrhage
    Intracranial hemorrhage
    21%
  • Gastrointestinal hemorrhage
    Gastrointestinal hemorrhage
    12%
  • Blood in urine
    Hematuria
    7%
Baby icon

In neonates:

Abnormal bleeding may occur with circumcision or from umbilical stump (often when the stump falls off at 1-2 weeks)

Severity Classification of Hereditary Factor X Deficiency6,7

Factor X severity classifications are based on factor X coagulant activity level. In the general population, factor X coagulant activity level is 65-120 IU/dL (reference ranges may vary)6

  • A 1998 publication classified “severe” factor X deficiency as <1 IU/dL6
Classification of Hereditary Factor X Deficiency6
SevereModerateMild
Factor X functional activity<1% (<1 IU/dL)1–5% (1–5 IU/dL)>5% (>5 IU/dL)

More recently, a 2012 proposed classification by the Rare Bleeding Disorders Working Group defined “severe” deficiency more broadly—as <10 IU/dL7

The analysis indicated:

  • Patients with factor X activity levels <10% (10 IU/dL) had a high risk of major spontaneous bleeding (eg, central nervous system and gastrointestinal bleeding)
  • A strong association existed between factor X activity level and clinical bleeding severity
  • Patients with levels between 10 and 40 IU/dL suffered minor spontaneous or triggered bleeding
Proposed Classification by the Rare Bleeding Disorders Working Group7
SevereModerateMild
Factor X functional activity<10% (<10 IU/dL)10–40% (10–40 IU/dL)>40% (>40 IU/dL)

Testing and Diagnosis of Factor X Deficiency

Factor X deficiency is diagnosed using a “Factor X Activity” blood test. This can confirm suspicions based on symptom assessments, prolonged PT and PTT, or other findings.1

Delayed diagnosis is common with rare bleeding disorders.2 These real-life cases underscore the importance of early diagnosis—

Photo of Maritza
Maritza continued 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.
Photo of Adrian
Adrian coped with more than 25 years of joint swelling, pain, and bruising, until he was finally tested and diagnosed with factor X deficiency as an adult after being hospitalized for a brain bleed.
Laboratory Test Codes to Determine Factor X Deficiency7
LaboratoryTest CodeTest Name
Labcorp086306Factor X Activity
Quest Diagnostics™359Factor X Activity, Clotting
BioReference Laboratories5735-6Factor X Activity (FACT.X ACT.)
TriCore Reference LaboratoriesFACT10Factor X Activity
CPT Code for Factor X Activity: 85260

Early Diagnosis of Factor X Deficiency May Allow for Appropriate Care and Treatment Sooner1,2

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.
Without attention to prompt and accurate diagnosis, patients with hereditary factor X deficiency may suffer from symptoms and go undiagnosed for years.2

Treatment Considerations

Expert consensus recommends use of single-factor concentrates for treatment of rare bleeding disorders (including hereditary factor X deficiency)3,8

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“Expert consensus now considers that optimal therapy for rare coagulation disorders is the use of single-factor concentrates for specific replacement of the deficient factor wherever possible to reduce the risk of adverse reactions and accumulation of other non-deficient coagulation factors”3
Dr. Amy Shapiro,
Indiana Hemophilia & Thrombosis Center
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“Single-factor concentrates should be used as therapy whenever possible in patients with rare bleeding disorders”8
European consensus proposals for treatment of
haemophilia with coagulation factor concentrates
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Some treatment options contain factor X but are not FDA-approved to treat factor X deficiency and are not single-factor concentrates. They are lower purity for factor X, containing multiple clotting factors not needed for treatment of hereditary factor X deficiency:

Prothrombin Complex Concentrates (PCCs)9

  • Contain estimated factor X content based on historical measurements—a factor X assay level is not listed on each vial
  • Contain multiple other clotting factors in addition to factor X
  • Are manufactured based on a factor IX assay, and the amounts of each factor in a vial can vary considerably between products and between lots of the same product

Elevated levels of factors II, VII, and IX may be associated with an increased risk of thromboembolic complications.

References: 1. Brown DL, Kouides PA. Haemophilia. 2008;14(6):1176-1182. 2. Palla R, et al. Blood. 2015;125(13):2052-2061. 3. Shapiro A. Expert Opin Drug Metab Toxicol. 2017;13(1):97-104. doi: 10.1080/17425255.2016.1237504. Epub 2016 Nov 2. PMID: 27797267. 4. Hayward CPM. Int J Lab Hem. 2018;40(Suppl. 1):6–14. 5. Hermann FH, Auerswald G, Ruiz-Saez A, et al. Haemophilia. 2006;12:479-489. 6. Peyvandi F, Mannucci PM, Lak M, et al. Brit J Haematol. 1998;102:626-628. 7. Peyvandi F, Di Michele D, Bolton-Maggs PHB, et al. J Thromb Haemost. 2012;10:1938-1943. 8. Giangrande P, et al. Haemophilia. 2014;20:322-325. 9. Menegatti M, Peyvandi F. Semin Thromb Hemost. 2009;35:407-415.

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 and moderate hereditary factor X deficiency

Limitation of Use

Perioperative management of bleeding in major surgery in patients with severe hereditary factor X deficiency has not been studied.

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.