Romiplostim
| Evidence Level: L1 | Predicted Indications: 10 |
Table of Contents
Romiplostim: From Immune Thrombocytopenia to Platelet-Type Bleeding Disorder
One-Sentence Summary
Romiplostim is a thrombopoietin (TPO) receptor agonist originally used to treat chronic immune thrombocytopenia (ITP). Among 10 TxGNN-predicted indications, the model’s platelet-count-deficiency cluster — most concretely represented by Platelet-Type Bleeding Disorder (a category spanning ITP, chemotherapy-induced thrombocytopenia, MDS-related thrombocytopenia, and post-transplant thrombocytopenia) — is by far the best supported, with 8 clinical trials, including 2 completed Phase 3 RCTs, currently backing this direction. (Note: this indication was selected over the top-ranked TxGNN score because it has the strongest actual clinical evidence — see rationale below.)
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Immune Thrombocytopenia (ITP) (inferred from repurposing rationale text; not captured in original_indications field) |
| Predicted New Indication | Platelet-Type Bleeding Disorder |
| TxGNN Prediction Score | 99.93% |
| Evidence Level | L1 |
| EU Market Status | Not Marketed |
| Number of Authorizations | 0 |
| Recommended Decision | Proceed with Guardrails |
Why is This Prediction Reasonable?
Currently, original_moa is marked as a data gap in the structured record. However, the evidence pack’s own repurposing rationale text confirms that Romiplostim is a TPO-receptor (MPL) agonist that stimulates megakaryocyte proliferation and differentiation, thereby increasing platelet production — this is the mechanistic basis behind all 10 predicted indications in this pack.
Romiplostim’s approved use in ITP addresses thrombocytopenia caused by immune-mediated platelet destruction and impaired production. “Platelet-Type Bleeding Disorder” is a broader disease category that captures thrombocytopenia of multiple etiologies — including chemotherapy-induced thrombocytopenia (CIT), myelodysplastic syndrome (MDS)-associated thrombocytopenia, and post-hematopoietic-stem-cell-transplant platelet engraftment failure. In all of these, the underlying problem is a quantitative shortfall in platelet production, which is exactly the mechanism Romiplostim targets — unlike several other TxGNN-predicted candidates in this pack (e.g., Glanzmann thrombasthenia, Scott syndrome, pseudo-von Willebrand disease) which are qualitative/functional platelet defects that a production-stimulating agent cannot correct.
This mechanistic fit is reflected in the evidence: a completed Phase 3 RCT (RECITE, NCT03362177) directly tested Romiplostim in chemotherapy-induced thrombocytopenia, and a completed Phase 3 extension study (NCT05492409) established long-term safety in ITP patients on a Romiplostim biosimilar. Several additional Phase 1/2/4 studies extend into MDS and post-transplant settings, reinforcing that this is a class-consistent extension of Romiplostim’s known pharmacology rather than a mechanistically speculative prediction.
Clinical Trial Evidence
| Trial Number | Phase | Status | Enrollment | Key Findings |
|---|---|---|---|---|
| NCT03362177 | Phase 3 | Completed | 165 | RECITE: randomized, placebo-controlled, double-blind trial of Romiplostim for chemotherapy-induced thrombocytopenia in GI/pancreatic/colorectal cancer patients on oxaliplatin-based regimens |
| NCT05492409 | Phase 3 | Completed | 160 | Long-term extension study evaluating safety and immunogenicity of a Romiplostim biosimilar (GNR-069) in ITP patients who completed the Phase 3 RMP-ITP-III trial |
| NCT02335268 | Phase 2 | Completed | 77 | EUROPE trial: prospective validation of a predictive response model for Romiplostim in IPSS low/int-1 risk MDS patients with thrombocytopenia |
| NCT02227576 | Phase 2 | Terminated | 20 | Secondary prophylactic use of Romiplostim to prevent temozolomide-induced thrombocytopenia in newly diagnosed glioblastoma patients (terminated) |
| NCT04638829 | Phase 4 | Completed | 60 | Open-label study measuring safety and treatment satisfaction after switching adults with chronic ITP from Eltrombopag or Romiplostim to Avatrombopag |
| NCT07321626 | Phase 1 | Recruiting | 130 | Randomized controlled study of Romiplostim N01 for promoting platelet engraftment after haploidentical allogeneic stem cell transplant in hematologic malignancies |
| NCT02046291 | Phase 1 | Completed | 21 | Dose-escalation study of weekly Romiplostim safety in patients failing platelet engraftment after umbilical cord blood transplant |
| NCT02298075 | N/A | Completed | 148 | Retrospective study of sustained response rate after discontinuing TPO-receptor agonists (Eltrombopag, Romiplostim) in primary ITP patients |
Literature Evidence
Currently no related literature available for this specific indication category (literature evidence in the pack is attached to other, less-supported disease terms such as “primary release disorder of platelets” and “autosomal dominant macrothrombocytopenia”).
EU Market Information
No marketing authorizations are recorded in this dataset for this jurisdiction — market status is Not Marketed, with 0 licenses on file. No licenses entries were available to extract product name, dosage form, or approved indication text.
Safety Considerations
Please refer to the SmPC for safety information. (Key warnings, contraindications, and drug interaction data are all marked as data gaps in this evidence pack — see DG001, DG002 below.)
Conclusion and Next Steps
Decision: Proceed with Guardrails
Rationale: Two completed Phase 3 RCTs plus multiple supporting Phase 1/2/4 studies demonstrate that Romiplostim’s core mechanism (TPO-receptor-mediated platelet production) extends reliably beyond ITP into chemotherapy-induced and other production-deficit thrombocytopenias, giving this candidate an L1 evidence level. However, a blocking data gap on local drug label warnings/contraindications (DG001) and a missing formal MOA record (DG002) prevent a full safety sign-off at this stage.
To proceed, the following is needed:
- TFDA/EMA-equivalent label PDF for warnings, contraindications, and precautions (DG001 — blocking; required before S1 safety pre-assessment)
- Confirmed mechanism-of-action record via DrugBank API query (DG002)
- Regulatory filing/authorization status check for the target indication in this jurisdiction, since current market status shows zero licenses
- Population-specific safety review for off-label extension settings (e.g., glioblastoma/CNS tumor patients, given the terminated NCT02227576 trial)
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.