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
Diagnostic Indicators of Factor X Deficiency Include Bleeding Symptoms and Prolonged PT and aPTT1,2
Factor X Deficiency May Present with Ongoing Symptoms
Symptom occurrence with factor X deficiency (N=42)3
- In this analysis, additional symptoms of factor X deficiency included hematomas (43%), gum bleeding (31%), gastrointestinal hemorrhage (12%), and hematuria (7%)
The majority of women with factor X deficiency have a history of heavy menstrual bleeding3
- Heavy menstrual bleeding occurs in up to 70% of women of reproductive age who have hereditary factor X deficiency3
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
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
Prolonged PT and aPTT is a diagnostic indicator that a test for factor X deficiency may be appropriate
Diagnosis is made via a plasma coagulation factor X activity assay
ICH and GI Bleeds are Likely to Occur Early in Life3,4
In a study of 42 adult and pediatric patients with symptomatic hereditary factor X deficiency, 21% (9 of 42) experienced ICH and 12% (5 of 42) experienced GI hemorrhage—
Use of plasma-derived factor X concentrate in neonates and infants with congenital factor X deficiency.
Severity Classification is Unique to Factor X 4,6
Severity Classification by Factor X Activity*
Low bleeding risk
Minor spontaneous bleeding
High risk of major spontaneous bleeding
- Decreasing factor X activity has been shown to correlate with increasing bleeding severity7
- 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/dL6,7 †
- In the general population, factor X activity ranges from 65–120 IU/dL (reference ranges may vary)8
*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/dL.8
†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 levels.
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 (RB; 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).
Clinical guidelines and expert consensus recommend use of plasma-derived factor X concentrate for the treatment of hereditary factor X deficiency4,7,9
Recommendations concerning products licensed for the treatment of hemophilia and other bleeding disorders, 2022.
Diagnosis, therapeutic advances, and key recommendations for the management of factor X deficiency.
Blood Reviews. 2021.
Occurrence and management of severe bleeding episodes in patients with hereditary factor X deficiency.
Treatment of rare factor deficiencies other than hemophilia.
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. 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. 10. Menegatti M, Peyvandi F. Blood. 2019;133:415-424.
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.