ITI with high-dose FIX and combined immunosuppressive therapy in a patient with severe haemophilia B and inhibitor.
Standard
ITI with high-dose FIX and combined immunosuppressive therapy in a patient with severe haemophilia B and inhibitor. / Beutel, Karin; Hauch, H; Rischewski, J; Kordes, Uwe; Schneppenheim, J; Schneppenheim, Reinhard.
in: HAMOSTASEOLOGIE, Jahrgang 29, Nr. 2, 2, 2009, S. 155-157.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
Harvard
APA
Vancouver
Bibtex
}
RIS
TY - JOUR
T1 - ITI with high-dose FIX and combined immunosuppressive therapy in a patient with severe haemophilia B and inhibitor.
AU - Beutel, Karin
AU - Hauch, H
AU - Rischewski, J
AU - Kordes, Uwe
AU - Schneppenheim, J
AU - Schneppenheim, Reinhard
PY - 2009
Y1 - 2009
N2 - Inhibitor development is a rare but serious event in hemophilia B patients. Management is hampered by the frequent occurrence of allergic reactions to factor IX, low success rates of current inhibitor elimination protocols and the risk of development of nephrotic syndrome. Single cases of immune tolerance induction (ITI) including immunosuppressive agents like mycophenolat mofetil (MMF) or rituximab have been reported. We present a case of successful inhibitor elimination with a combined immune-modulating therapy and high-dose factor IX (FIX). This boy had developed a FIX inhibitor at the age of 5 years and had a history of allergic reactions to FIX and to FEIBAO. Under on-demand treatment with recombinant activated FVII the inhibitor became undetectable but the boy suffered from multiple joint and muscle bleeds. At the age of 11.5 years ITI was attempted with a combination of rituximab, MMF, dexamethasone, intravenous immunoglobulins and high-dose FIX. The inhibitor did not reappear and FIX half-life normalized. No allergic reaction, no signs of nephrotic syndrome and no serious infections were observed.
AB - Inhibitor development is a rare but serious event in hemophilia B patients. Management is hampered by the frequent occurrence of allergic reactions to factor IX, low success rates of current inhibitor elimination protocols and the risk of development of nephrotic syndrome. Single cases of immune tolerance induction (ITI) including immunosuppressive agents like mycophenolat mofetil (MMF) or rituximab have been reported. We present a case of successful inhibitor elimination with a combined immune-modulating therapy and high-dose factor IX (FIX). This boy had developed a FIX inhibitor at the age of 5 years and had a history of allergic reactions to FIX and to FEIBAO. Under on-demand treatment with recombinant activated FVII the inhibitor became undetectable but the boy suffered from multiple joint and muscle bleeds. At the age of 11.5 years ITI was attempted with a combination of rituximab, MMF, dexamethasone, intravenous immunoglobulins and high-dose FIX. The inhibitor did not reappear and FIX half-life normalized. No allergic reaction, no signs of nephrotic syndrome and no serious infections were observed.
M3 - SCORING: Zeitschriftenaufsatz
VL - 29
SP - 155
EP - 157
JO - HAMOSTASEOLOGIE
JF - HAMOSTASEOLOGIE
SN - 0720-9355
IS - 2
M1 - 2
ER -