Lenograstim
| Evidence Level: L4 | Predicted Indications: 4 |
Table of Contents
Lenograstim: From Unspecified Indication to Primary Release Disorder of Platelets
One-Sentence Summary
Lenograstim is a glycosylated recombinant human G-CSF; the evidence pack does not record a documented original indication (no UK marketing authorisation is currently held), but its established clinical role is stimulating neutrophil precursor proliferation for neutropenia and stem-cell mobilisation. The TxGNN model predicts it may be effective for Primary Release Disorder of Platelets, but this is currently supported only by 13 loosely-related clinical trials (mostly haematopoietic stem cell transplant supportive-care studies) and no published literature.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Not documented in this evidence pack (drug currently unlicensed/not marketed in the UK) |
| Predicted New Indication | Primary Release Disorder of Platelets |
| TxGNN Prediction Score | 99.91% |
| Evidence Level | L4 |
| UK Market Status | Not marketed |
| Number of Marketing Authorisations | 0 |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Currently, detailed mechanism of action data is not available in this evidence pack. Based on known information, Lenograstim is a glycosylated analogue of filgrastim (recombinant human G-CSF), which acts primarily on granulocyte precursor cells to promote proliferation and differentiation into neutrophils. Its established clinical roles are the treatment of neutropenia and mobilisation of peripheral blood stem cells for transplantation — not platelet biology.
Platelet production and release are governed principally by the thrombopoietin (TPO)/MPL signalling pathway acting on megakaryocytes, a pathway mechanistically distinct from the granulocyte-focused action of G-CSF. The 13 clinical trials identified in this evidence pack are almost entirely allogeneic/autologous haematopoietic stem cell transplantation (HSCT) studies in which G-CSF-class agents are used as supportive therapy (stem cell mobilisation or post-transplant neutrophil recovery), not as a treatment directed at platelet release disorders.
Given this, the TxGNN association most likely reflects a co-occurrence pattern in the knowledge graph (G-CSF frequently appearing alongside haematological/transplant contexts where platelet parameters are also monitored) rather than a genuine, mechanistically grounded treatment signal. This should be treated as a research hypothesis requiring further mechanistic validation, not as clinical evidence of efficacy.
Clinical Trial Evidence
| Trial Number | Phase | Status | Enrolment | Key Findings |
|---|---|---|---|---|
| NCT04047628 | Phase 3 | Recruiting | 156 | RCT comparing autologous HSCT vs best available therapy in treatment-resistant relapsing MS; G-CSF likely used for stem cell mobilisation, not a platelet-disorder efficacy endpoint |
| NCT05436418 | Phase 1/2 | Recruiting | 260 | Dose-finding study of post-transplant cyclophosphamide GVHD prophylaxis after PBSC transplant; G-CSF used for stem cell mobilisation only |
| NCT05170828 | Phase 1 | Withdrawn (enrolment 0) | 0 | Cryopreserved unrelated donor bone marrow transplant study; withdrawn, no evidentiary value |
| NCT01503918 | Phase 2 | Completed | 124 | Antiviral prophylaxis for CMV reactivation in critical care; unrelated to platelet biology |
| NCT04540120 | Phase 2 | Terminated | 49 | Oral NLRP3 inhibitor trial for moderate COVID-19; no relevance to G-CSF or platelet disorders |
| NCT01335932 | Phase 2 | Completed | 160 | Ganciclovir/valganciclovir for CMV reactivation prevention in lung injury; not a platelet-disorder trial |
| NCT00043979 | Phase 2 | Completed | 60 | Pilot allogeneic/syngeneic stem cell transplant in paediatric sarcoma; G-CSF as supportive care only |
| NCT00245037 | Phase 1/2 | Completed | 147 | Non-myeloablative allogeneic HSCT for haematologic malignancies; supportive use of G-CSF, no platelet-disorder endpoint |
| NCT00923364 | Phase 2 | Completed | 19 | Reduced-intensity HSCT pilot in GATA2 mutation patients; not directed at platelet release disorder |
| NCT00354172 | Phase 2 | Terminated | 16 | Umbilical cord blood transplant for myeloid leukaemia; terminated, no platelet-disorder relevance |
Note: All trials above are graded B or C relevance by the underlying evidence review — none directly evaluates Lenograstim as a treatment for primary platelet release disorder.
Literature Evidence
Currently no related literature available.
UK Market Information
No UK marketing authorisation is currently held for Lenograstim according to this evidence pack (market status: Not marketed; 0 licences on file).
Safety Considerations
Please refer to the SmPC and BNF for safety information. Report suspected adverse reactions via the Yellow Card Scheme.
Conclusion and Next Steps
Decision: Hold
Rationale: The mechanistic rationale linking G-CSF to platelet release disorders is weak and possibly co-occurrence-driven rather than causal, and none of the 13 identified trials directly tests Lenograstim as a treatment for this indication — they are all HSCT supportive-care studies. There is no literature evidence, and the evidence pack itself flags this as a “Research Question” (decision stage S1), not a candidate ready for clinical development.
To proceed, the following is needed:
- Confirmed mechanism of action data for Lenograstim (currently a data gap; DrugBank query required)
- UK SmPC warnings, contraindications and DDI data (currently a blocking data gap for safety screening)
- Preclinical/mechanistic studies specifically examining G-CSF effects on megakaryocyte function or platelet release pathways
- If mechanistic plausibility is established, a dedicated trial with primary platelet release disorder as a primary (not incidental) endpoint
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.