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September 28, 2021Frontiers in OncologyOpen Access

Off-Label Use of Thrombopoietin Receptor Agonists: Case Series and Review of the Literature

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Why the study?

Data on off-label thrombopoietin receptor agonist use in refractory thrombocytopenia beyond current indications remain scarce, with potential risks such as thrombosis.

Does off-label use of TPO-RAs improve response rates in patients with refractory thrombocytopenia?

Comparison

Off-label thrombopoietin receptor agonist use across three etiology groups

Design

Single-center case series

Authors

MCMarco CapecchiFSFabio SerpentiJGJuri Alessandro Giannotta

Discussion

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Overview

Off-label TPO-RA use in refractory thrombocytopenia warrants caution; leaves open questions on efficacy and thrombosis risk pending controlled data.

Structured PICO

Does off-label use of TPO-RAs improve response rates in patients with refractory thrombocytopenia?

P
Population
14 patients with refractory thrombocytopenia due to myelodysplastic syndromes, post-transplantation, or lymphoproliferative diseases
I
Intervention
Off-label thrombopoietin receptor agonists (TPO-RAs)
O
Outcome
Overall response rate (ORR) and safety (adverse events, particularly thrombosis)surrogate

Off-label use of TPO-RAs appears effective and safe for managing refractory thrombocytopenia in myelodysplastic syndromes, post-transplantation, and lymphoproliferative diseases.

Cite This Study

Capecchi et al. (2021) studied this question.

synapsesocial.com/papers/6a729cde35aa2c282ce357b0https://doi.org/10.3389/fonc.2021.680411
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Evolving Patterns of TPO-RA Use in Children: A Decade of Single-Centre Experience and Narrative Review2026
  2. 2Novel Perspectives on Thrombopoietin Receptor Agonists Applications2024 · 4 citations
  3. 3Role of Thrombopoietin Receptor Agonists in Inherited Thrombocytopenia2021 · 29 citations
  4. 4Bridging the gaps between randomized controlled trials and real-world use of thrombopoietin receptor agonists for adult primary immune thrombocytopenia: a systematic review and meta-analysis2025
  5. 5Thrombopoietin Receptor Agonists (TPO-RAs): Drug Class Considerations for Pharmacists2021 · 116 citations