Disease Directory Factor XIII Deficiency
Blood

Factor XIII Deficiency

Also known as: fibrin stabilizing factor deficiency, FXIII deficiency, Laki-Lorand factor deficiency

Prevalence

1 in 1,000,000 to 1 in 5,000,000

Onset

Neonatal period; umbilical stump bleeding is often the first sign

Type

Autosomal recessive

Gene

F13A1, F13B

About Factor XIII Deficiency

Factor XIII deficiency is an ultra-rare congenital bleeding disorder caused by biallelic mutations in F13A1 (encoding the FXIII-A catalytic subunit) or F13B (encoding the carrier FXIII-B subunit), resulting in deficiency of the transglutaminase that cross-links fibrin polymers to form a mechanically stable clot. Because standard coagulation tests (PT, aPTT) are normal in FXIII deficiency, diagnosis is often delayed, and the condition is characterized by a distinctive pattern of delayed bleeding occurring hours to days after injury once primary hemostasis is established but clot stability is compromised. Severe FXIII deficiency carries high risks of intracranial hemorrhage, recurrent miscarriage, and impaired wound healing.

Common Clinical Features

Umbilical cord stump bleeding in the neonatal period (pathognomonic sign) Delayed bleeding after injury, surgery, or dental procedures Recurrent intracranial hemorrhage Habitual miscarriage in affected women due to impaired placental implantation Poor wound healing and abnormal scar formation Deep muscle hematomas and hemarthroses Normal PT, aPTT, platelet count, and bleeding time despite severe bleeding Positive urea clot solubility test in severe deficiency

Clinical Trial Eligibility Tips

What to know before applying to Factor XIII Deficiency trials.

Factor XIII activity level (chromogenic or functional assay) is essential for diagnosis confirmation and trial eligibility; severe deficiency is defined as FXIII activity below 1-5%, and activity levels correlate with bleeding phenotype.

Genetic mutation identification in F13A1 or F13B helps classify type (A subunit vs B subunit deficiency), with type A being more common and clinically severe; bring genetic test results to screening appointments.

Prophylactic FXIII replacement therapy history, including product used, dose, frequency, and any breakthrough bleeding events, is required documentation for trials evaluating recombinant FXIII or novel long-acting replacement products.

Patient Resources

Patient Organization

National Hemophilia Foundation

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Orphanet

European reference resource for rare diseases (ORPHA:331)

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NORD

National Organization for Rare Disorders

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