ORIGINAL ARTICLE
Tapering thrombopoietin receptor agonists in primary immune thrombocytopenia: Expert consensus based on the RAND/UCLA modified Delphi panel method

https://doi.org/10.1002/rth2.12457Get rights and content
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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.

Keywords

blood platelets
consensus
idiopathic
platelet count
purpura
receptors
thrombocytopenic
thrombopoietin

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This is an open access article under the terms of the Creative Commons Attribution‐NonCommercial‐NoDerivs License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made.

Handling Editor: Dr Neil Zakai

Funding informationThis study was funded by Novartis.

Name of institution where study took place: Partnership for Health Analytic Research (PHAR), LLC.