Abstract
Background: Recent evidence suggests that in patients with immune thrombocytopenia (ITP) with a stable response on thrombopoietin receptor agonists (TPO-RAs), treatment may be tapered and/or discontinued.
Objectives: To provide a guide for tapering and discontinuation of TPO-RA therapy in patients with ITP, based on hematologist survey results, existing evidence and expert consensus.
Patients/methods: UK hematologists completed a survey to characterize self-reported practice patterns related to TPO-RA tapering and discontinuation in patients with ITP. Using a modified Delphi panel approach, ITP experts developed consensus statements regarding the use of TPO-RA tapering and discontinuation.
Results: Survey respondents estimated that 30–34% of their patients were suitable for tapering or discontinuation, and that 29–35% of these patients required treatment re-initiation after an average treatment-free interval of 86–106 days. No clear predictors of patient suitability or response to tapering or discontinuation were identified. The ITP expert consensus was that approximately 30% of patients are eligible for tapering and discontinuation, which may be considered after 6–12 months for patients demonstrating an adequate treatment response (platelet count >50,000/µL at ≥75% of assessments in the preceding 6 months). Treatment re-initiation may be considered if the platelet count decreases or if the patient becomes symptomatic. Individual differences need to be taken into account when considering TPO-RA tapering or discontinuation.
Conclusions: Tapering and discontinuation of TPO-RA therapy may be considered for certain patients with ITP. Further study is needed to better predict patients likely to achieve sustained off-treatment responses after tapering and discontinuation
Objectives: To provide a guide for tapering and discontinuation of TPO-RA therapy in patients with ITP, based on hematologist survey results, existing evidence and expert consensus.
Patients/methods: UK hematologists completed a survey to characterize self-reported practice patterns related to TPO-RA tapering and discontinuation in patients with ITP. Using a modified Delphi panel approach, ITP experts developed consensus statements regarding the use of TPO-RA tapering and discontinuation.
Results: Survey respondents estimated that 30–34% of their patients were suitable for tapering or discontinuation, and that 29–35% of these patients required treatment re-initiation after an average treatment-free interval of 86–106 days. No clear predictors of patient suitability or response to tapering or discontinuation were identified. The ITP expert consensus was that approximately 30% of patients are eligible for tapering and discontinuation, which may be considered after 6–12 months for patients demonstrating an adequate treatment response (platelet count >50,000/µL at ≥75% of assessments in the preceding 6 months). Treatment re-initiation may be considered if the platelet count decreases or if the patient becomes symptomatic. Individual differences need to be taken into account when considering TPO-RA tapering or discontinuation.
Conclusions: Tapering and discontinuation of TPO-RA therapy may be considered for certain patients with ITP. Further study is needed to better predict patients likely to achieve sustained off-treatment responses after tapering and discontinuation
| Original language | English |
|---|---|
| Pages (from-to) | 418-426 |
| Number of pages | 9 |
| Journal | Acta Haematologica |
| Volume | 144 |
| Issue number | 4 |
| Early online date | 31 Mar 2021 |
| DOIs | |
| Publication status | Published - 1 Jul 2021 |
Bibliographical note
Funding Information:The authors would like to thank Elizabeth Kehler and Mark Silvey of the Adelphi Group for their support in the development and analysis of the surveys and consensus meeting. This study was funded by Novartis Pharmaceuticals. Writing and editorial support in the preparation of this manuscript was provided by Nicky Dekker, MD, PhD, of Excerpta Medica, funded by Novartis Pharmaceuticals.
Funding Information:
N. Cooper received speaking and ad board honoraria from Amgen and Novartis. Q.A. Hill received honoraria from Novartis for advisory work and speaking at educational events. J. Grainger received consultancy honoraria from Novartis, Alexion, Ono Pharma, Amgen, and GSK and travel grants from Novartis. J.P. Westwood received honoraria from Novartis for advisory work and speaking at educational events. C. Bradbury received speaker honoraria from BMS, Novartis, and Pfizer; honoraria for advisory work from Ablynx and Novartis; and support for conference attendance from Amgen, Bayer, and Novartis. D. Provan received honoraria and research support from Amgen and Novartis and consultancy honoraria from Amgen and Ono Pharma. J. Thachil received honoraria from Amgen and Novartis. N. Ramscar is a Novartis employee. A. Roy is a Novartis employee.
Publisher Copyright:
© 2021 The Author(s). Published by S. Karger AG, Basel.
Keywords
- eltrombopag
- immune thrombocytopenia
- romiplostim
- thrombopoietin receptor agonists
- treatment discontinuation