Successful long-time treatment with mycophenolate-mofetil in a child with acquired factor VIII inhibitor

Summary

Here, we report about a boy (age: 18 years) who developed an acquired factor VIII inhibitor at the age of 9 years. He presented with bleeding in his right ankle, multiple haematomas and a high-titer factor VIII type II inhibitor (400 BU). Therapy: He received treatment with MMF (CellCept→), dexamethasoneimmunoglobulin pulses, and rituximab together with high dose FVIII (Hannover protocol). His inhibitor titer decreased rapidly, and half-life and recovery normalized. Inhibitor titres increased after reduction of the factor VIII dose, and increased further after MMF was stopped. A second treatment course with MMF again resulted in reduction of the titre, improvement in half life and recovery, and no more bleedings. Inhibitor reappeared with MMF dose reduction, again accompanied by severe bleeding. Additional rituximab stopped the bleedings, and treatment with MMF has been continued since. Conclusion: Although the laboratory parameters showed no complete remission, severe bleedings and expensive factor replacement could be avoided by long-term treatment with MMF.

Zusammenfassung

Ein 18-jähriger Patient entwickelte im Alter von 9 Jahren einen erworbenen hochtitrigen Faktor-VIII-Inhibitor (400 BE). Klinisch imponierten eine Blutung ins rechte Sprunggelenk und multiple Hämatome. Der Inhibitor zeigte die typische nicht-lineare Typ-II-Kinetik. Therapie: Unter einer immunsuppressiven Therapie mit MMF (Cellcept→), Dexamethason-Immunglobulin-Blöcken und Rituximab sowie einer Hochdosis-Faktor-VIII-Therapie (Hannover-Protokoll) zeigte sich zunächst ein gutes Ansprechen. Der Inhibitor-Titer sank rasch, Halbwertszeit und Recovery normalisierten sich. Nach Reduktion der Faktordosis und Absetzen des MMF stieg der Hemmkörpertiter wieder an und es traten erneut Blutungen auf. Eine zweite Behandlung mit MMF zeigte wiederum ein Therapieansprechen sowie Blutungsfreiheit. Bei dem Versuch, im Verlauf die MMF-Dosis zu reduzieren, kam es erneut zu schweren Blutungen, die durch die zusätzliche Gabe von Rituximab beherrscht werden konnten. Schlussfolgerung: Durch die fortgesetzte Behandlung mit MMF als Monotherapie erreichte der Patient laboranalytisch zwar keine komplette Remission, schwere Blutungen und eine kostenintensive Faktortherapie konnten jedoch vermieden werden.

Keywords

Acquired haemophilia - factor VIII inhibitor - mycophenolate-mofetil - immuno-suppression

Schlüsselwörter

Erworbene Hämophilie - Faktor-VIII-Inhibitor - Mycophenolat-mofetil - Immunsuppression

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