Inhibitor Development in Severe Congenital Factor V Deficiency.

Area of Science:

• Hematology
• Immunology
• Pediatrics

Background:

• Severe congenital Factor V (FV) deficiency is a rare bleeding disorder.
• Acquired inhibitors against coagulation factors, including FV, can complicate management.
• Development of FV inhibitors can lead to refractory bleeding episodes.

Purpose of the Study:

• To describe a case of acquired anti-FV inhibitor in a pediatric patient with congenital FV deficiency.
• To report the management and successful eradication of the anti-FV inhibitor.
• To evaluate the efficacy of immune tolerance induction and immunosuppressive therapy.

Main Methods:

• Diagnosis of severe congenital FV deficiency.
• Development of anti-FV inhibitor post-fresh frozen plasma (FFP) transfusion.
• Confirmation of inhibitor using ELISA.
• Bleeding control with recombinant factor VIIa (rFVIIa).
• Initiation of immune tolerance induction with FFP and immunosuppressive therapy.

Main Results:

• The patient developed a high-titer anti-FV inhibitor.
• Bleeding was effectively managed with rFVIIa.
• FV inhibitor levels became undetectable after one year of treatment.
• The patient achieved a favorable clinical outcome with no further bleeding.

Conclusions:

• Acquired FV inhibitors can develop in patients with congenital FV deficiency.
• A combination of FFP, rFVIIa, and immunosuppressive therapy can successfully eradicate FV inhibitors.
• Immune tolerance induction protocols are effective in managing acquired factor inhibitors.