Inhibitor development after liver transplantation in congenital factor VII deficiency

Abstract

Congenital factor VII (FVII) deficiency is the commonest type of the rare bleeding disorders. Very few cases of congenital FVII deficiency developed inhibitor and liver transplant is considered as definitive treatment. In the literature, twelve patients with congenital FVII deficiency developed inhibitors. Two had spontaneous resolution of inhibitors and one did not respond to high dose recombinant factor VIIa (rFVIIa) and died. Regarding liver transplant in congenital FVII patients, seven patients underwent liver transplant with good prognosis. We report a 5-year-old girl with confirmed severe congenital FVII deficiency since neonatal period. She suffered from recurrent intracranial bleeding despite rFVIIa replacement. After auxiliary liver transplant at the age of 4, she continued to show persistent deranged clotting profile and was found to have inhibitor towards FVII. Interestingly, she was still responsive to rFVIIa replacement.

Inhibitors in Patients with Congenital Bleeding Disorders Other Than Hemophilia

“…Interestingly, she was responsive to rFVIIa despite the presence of the inhibitor. 72 Also, successful prophylactic rFVIIa in a patient with inherited FVII deficiency with inhibitors to FVII was reported by Tokgoz and colleagues, 68 suggesting a possible weak affinity of this alloantibody to rFVIIa. By contrast, Borhany and colleagues 71 reported a FVII inhibitor case who did not respond to rFVIIa therapy despite extensive immunosuppressive (methylprednisolone and cyclophosphamide) treatment.…”

“…30 Thirteen inhibitor cases have been reported so far in eight studies, following use of either plasma-derived or rFVII products. [65][66][67][68][69][70][71][72] In a prospective study of 101 spontaneous or traumatic bleeds occurring in 75 patients with congenital FVII deficiency, two inhibitors were detected in two repeatedly treated patients (one post-plasma-derived FVII, one post-rFVIIa). 67 See and colleagues 72 reported the case of FVII inhibitor development following liver transplantation in a 5-yearold girl with severe congenital FVII deficiency.…”

Sinh Chất Ức Chế Ở Bệnh Nhân Thiếu Yếu Tố Vii Báo Cáo Trường Hợp Bệnh

“…Bệnh thiếu yếu tố VII bẩm sinh hay bệnh thiếu proconvertin là bệnh hiếm, ước tính khoảng 1/500.000 người, không phụ thuộc vào dân tộc hay địa lý [6]. So với tần suất các bệnh thiếu yếu tố đông máu hiếm khác như thiếu fi brinogen, prothrombin (FII), yếu tố V, thiếu phối hợp yếu tố V và VIII, X, XI, XIII, có tần suất thay đổi từ 1/5000.000 đến 1/2.000.000 [3] -Điều trị thiếu FVII bẩm sinh có sinh chất ức chế bằng rFVIIa liều cao có hiệu quả ngừng chảy máu, cứu sống bệnh nhân.…”