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Br J Haematol. 2014 Jun;165(6):865-9. doi: 10.1111/bjh.12888. Epub 2014 Apr 12.

The temporary use of thrombopoietin-receptor agonists may induce a prolonged remission in adult chronic immune thrombocytopenia. Results of a French observational study.

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  • 1Service de Médecine Interne, Centre de référence des cytopénies auto-immunes de l'adulte, Hôpital Henri Mondor, Assistance Publique-Hôpitaux de Paris, Université Paris Est Créteil, Créteil, France; Etablissement Français du Sang Ile de France, Inserm U955, Créteil, Hôpital Henri Mondor, Paris, France.

Abstract

Thrombopoietin-receptor agonists (Tpo-RAs) are highly effective in immune thrombocytopenia (ITP). Recently, cases of durable remission after Tpo-RA discontinuation in adult ITP have been reported. We aimed to describe the subset of patients in whom transient Tpo-RA therapy may induce a durable response. We studied all adults with primary ITP treated with at least one Tpo-RA over a 5-year period (n = 54) and seen at one of three participating referral centres in France. Tpo-RAs were discontinued in 20 of 28 patients who achieved a complete response. We excluded six patients because a previous treatment at the start of Tpo-RA treatment may have interfered with the response. Overall, eight patients with chronic ITP showed a sustained response [median follow-up: 13·5 months (range 5-27 months)]. We could not identify a predictive factor of sustained response. In conclusion, a substantial proportion of ITP patients receiving Tpo-RAs can maintain a durable response after treatment discontinuation.

© 2014 John Wiley & Sons Ltd.

KEYWORDS:

durable response; immune thrombocytopenia; prolonged remission; thrombopoietin-receptor agonists

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