Tapering Thrombopoietin Receptor Agonists in Primary Immune Thrombocytopenia: Expert Consensus Based on the RAND/UCLA Modified Delphi Panel Method

Overview

Journal Res Pract Thromb Haemost

Publisher Elsevier

Date 2021 Feb 4

PMID 33537531

Citations 8

Authors

Adam Cuker

Jenny M Despotovic

Rachael F Grace

Caroline Kruse

Michele P Lambert

Howard A Liebman

Roger M Lyons

Keith R McCrae

Vinod Pullarkat

Jeffrey S Wasser

David Beenhouwer

Sarah N Gibbs

Irina Yermilov

Michael S Broder

Affiliations

Soon will be listed here.

Abstract

Background: Thrombopoietin receptor agonists (TPO-RAs) are used to treat primary immune thrombocytopenia (ITP). Some patients have discontinued treatment while maintaining a hemostatic platelet count.

Objectives: To develop expert consensus on when it is appropriate to consider tapering TPO-RAs in ITP, how to taper patients off therapy, how to monitor patients after discontinuation, and how to restart therapy.

Methods: We used a RAND/UCLA modified Delphi panel method. Ratings were completed independently by each expert before and after a meeting. Second-round ratings were used to develop the panel's guidance. The panel was double-blinded: The sponsor and nonchair experts did not know each other's identities.

Results: Guidance on when it is appropriate to taper TPO-RAs in children and adults was developed based on patient platelet count, history of bleeding, intensification of treatment, trauma risk, and use of anticoagulants/platelet inhibitors. For example, it is appropriate to taper TPO-RAs in patients who have normal/above-normal platelet counts, have no history of major bleeding, and have not required an intensification of treatment in the past 6 months; it is inappropriate to taper TPO-RAs in patients with low platelet counts. Duration of ITP, months on TPO-RA, or timing of platelet response to TPO-RA did not have an impact on the panel's guidance on appropriateness to taper. Guidance on how to taper patients off therapy, how to monitor patients after discontinuation, and how to restart therapy is also provided.

Conclusion: This guidance could support clinical decision making and the development of clinical trials that prospectively test the safety of tapering TPO-RAs.

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