Rate of Prolonged Response After Stopping Thrombopoietin-receptor Agonists Treatment in ITP

Summary

Thrombopoietin-receptor agonists (Tpo-RAs) have profoundly changed the management of ITP. However, today, there are no international recommendations concerning the long-term use of these costly, potentially pro-thrombotic agents, and that could induce bone marrow fibrosis in case of prolonged treatment. Tpo-RAs have been thought to play only a supporting role in ITP management. But our center along with many other research centers, have reported unexpected cases of durable remission after Tpo-RAs discontinuation in adult chronic ITP. In these retrospective studies, more than 20 % of patients were able to achieve prolonged remission. The purpose of this study is to demonstrate that a substantial proportion of ITP patients may achieve a prolonged response after Tpo-RA discontinuation. The investigators developed, in this study, a standardized procedure to discontinue Eltrombopag and Romiplostim, wherein the dose will be slowly tapered to limit the risk of bleeding. In case of relapse after Tpo-RA discontinuation, the decision to start a new therapy will be based on the clinician's judgment.

Timeline

Start

2017-09-26

Primary completion

2020-08-26

Completion

2021-02-16

Publications

• Cottu A, Guillet S, Viallard JF, Riviere E, Cheze S, Gobert D, Neel A, Graveleau J, Marolleau JP, Lefrere F, Moulis G, Lega JC, Moignet-Autrel A, Robbins A, Crickx E, Boutin E, Noel N, Malphettes M, Galicier L, Audia S, Bonnotte B, Lambotte O, Fain O, Gerfaud-Valentin M, Terriou L, Martis N, Morin AS, Perlat A, Le Gallou T, Roy-Peaud F, Puyade M, Comont T, Limal N, Languille L, Michel M, Godeau B, Mahevas M. Long-term follow-up of the STOPAGO study. Blood. 2025 Jan 9;145(2):244-247. doi: 10.1182/blood.2024025707.
• Guillet S, Crickx E, Azzaoui I, Chappert P, Boutin E, Viallard JF, Riviere E, Gobert D, Galicier L, Malphettes M, Cheze S, Lefrere F, Audia S, Bonnotte B, Lambotte O, Noel N, Fain O, Moulis G, Hamidou M, Gerfaud-Valentin M, Marolleau JP, Terriou L, Martis N, Morin AS, Perlat A, Le Gallou T, Roy-Peaud F, Robbins A, Lega JC, Puyade M, Comont T, Limal N, Languille L, Zarrour A, Luka M, Menager M, Belmondo T, Hue S, Canoui-Poitrine F, Michel M, Godeau B, Mahevas M. Prolonged response after TPO-RA discontinuation in primary ITP: results of a prospective multicenter study. Blood. 2023 Jun 8;141(23):2867-2877. doi: 10.1182/blood.2022018665.

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Indications