Lenograstim

證據等級: L5

預測適應症: 4 個

Lenograstim: From Neutrophil Support/Stem Cell Mobilization to Primary Release Disorder of Platelets

One-Sentence Summary

Lenograstim is a recombinant human G-CSF (granulocyte colony-stimulating factor) used to stimulate neutrophil precursor proliferation and to mobilize peripheral hematopoietic stem cells. The TxGNN model predicts it may be effective for Primary Release Disorder of Platelets, but currently no clinical trials or literature directly support this specific indication — only 13 tangentially related hematopoietic stem cell transplantation trials were identified, all graded as low relevance (Grade C).

Quick Overview

Item	Content
Original Indication	Not formally recorded in this Evidence Pack; per the drug’s known pharmacology (documented in the repurposing rationale below), lenograstim is used to stimulate neutrophil recovery and for peripheral hematopoietic stem cell (HSC) mobilization
Predicted New Indication	Primary release disorder of platelets
TxGNN Prediction Score	99.91%
Evidence Level	L4 (mechanism/indirect trial evidence only, no direct trials or literature)
Market Status (Taiwan)	Not Marketed
Number of NDAs	0
Recommended Decision	Hold

Why is This Prediction Reasonable?

Detailed structured mechanism-of-action (MOA) data for lenograstim is not available in this Evidence Pack (flagged as a High-severity data gap). However, the repurposing rationale supplied alongside the TxGNN prediction describes lenograstim as a glycosylated recombinant human G-CSF that acts primarily on the G-CSF receptor (CSF3R), stimulating proliferation and differentiation of granulocyte-lineage precursor cells in bone marrow and promoting neutrophil release and peripheral stem cell mobilization.

Primary release disorder of platelets, by contrast, is a platelet function defect typically involving impaired granule storage or release (e.g., δ-granule or α-granule defects) in megakaryocyte/platelet biology — a pathway distinct from the granulocytic lineage that G-CSF/CSF3R signaling targets. The Evidence Pack’s own analysis notes that there is no known direct receptor or signaling overlap between lenograstim’s pharmacology and the platelet granule release pathway.

Given this, the very high TxGNN score (99.91%) most likely reflects an indirect association in the knowledge graph — for example, shared “hematologic/bone marrow disease” nodes or co-occurrence in hematopoietic transplant/supportive-care contexts — rather than a direct, biologically plausible mechanism. This is consistent with the supporting clinical trials found: all 13 are hematopoietic stem cell transplantation studies where G-CSF-class drugs, if used at all, serve as supportive stem-cell mobilization agents rather than as a treatment directed at platelet release disorder itself.

Clinical Trial Evidence

Trial Number	Phase	Status	Enrollment	Key Findings
NCT00281879	Phase 2	Terminated	200	Unrelated donor HSC transplant for hematological malignancies; not focused on platelet release disorder (Grade C — low relevance)
NCT04047628	Phase 3	Recruiting	156	Autologous HSC transplant vs. best available therapy for treatment-resistant MS; G-CSF-class agents used only as supportive mobilization therapy (Grade C)
NCT06859424	Phase 2	Recruiting	358	Platform trial on post-transplant cyclophosphamide GVHD prophylaxis; no direct link to platelet release disorder (Grade C)
NCT00245037	Phase 1/2	Completed	147	Non-myeloablative allogeneic HSC transplant for hematologic malignancies; indirect relevance only (Grade C)
NCT05170828	Phase 1	Withdrawn	0	Cryopreserved mismatched-donor bone marrow transplant study; withdrawn with no enrollment data (Grade C)
NCT01335932	Phase 2	Completed	160	Ganciclovir/valganciclovir for CMV reactivation prevention in respiratory failure; no direct relevance to lenograstim or platelet disorder (Grade C)
NCT00043979	Phase 2	Completed	60	Allogeneic/syngeneic blood stem cell transplant in pediatric sarcomas; not directly relevant (Grade C)
NCT04540120	Phase 2	Terminated	49	Dapansutrile for moderate COVID-19/cytokine release syndrome; relevance to lenograstim unclear (Grade C)
NCT05436418	Phase 1/2	Recruiting	260	Lowest effective dose of post-transplant cyclophosphamide for GVHD prophylaxis; not directly targeting platelet release disorder (Grade C)
NCT00076752	Phase 2	Completed	9	Intensified lymphodepletion + autologous HSC transplant for severe SLE; not directly relevant (Grade C)

Note: All 13 trials identified for this indication are hematopoietic stem cell transplantation studies in which G-CSF-class drugs (if used) serve only as supportive stem-cell mobilization agents, not as a treatment directed at primary platelet release disorder. None constitutes direct clinical evidence for this indication.

Literature Evidence

Currently no related literature available.

US Market Information

Lenograstim is not currently marketed or registered (0 NDAs/licenses) under this Evidence Pack’s regulatory data. No approved product license or indication text is available to summarize.

Safety Considerations

Please refer to the package insert for safety information.

(All safety fields in this Evidence Pack — key warnings, contraindications, and drug interactions — are currently marked as data gaps. Notably, TFDA label warnings/contraindications data is flagged as a Blocking severity gap, meaning this candidate cannot yet pass the S1 safety pre-screening stage.)

Conclusion and Next Steps

Decision: Hold

Rationale: The mechanistic link between lenograstim (a G-CSF/CSF3R agonist acting on the granulocytic lineage) and primary release disorder of platelets (a platelet granule storage/release defect) is weak and not supported by any direct clinical trial or literature evidence — only tangentially related HSC transplant trials exist. In addition, this drug is not marketed in Taiwan, and a Blocking-severity data gap (missing TFDA label warnings/contraindications) prevents any safety pre-screening at this time.

To proceed, the following is needed:

TFDA package insert data (warnings, contraindications) — currently a Blocking data gap (DG001)
Confirmed mechanism-of-action documentation from DrugBank — currently a High-severity data gap (DG002)
Targeted preclinical or mechanistic studies evaluating any plausible role of G-CSF signaling in platelet granule release biology
Note: the three other TxGNN-predicted indications for lenograstim (Glanzmann thrombasthenia, pseudo-von Willebrand disease, severe nonproliferative diabetic retinopathy) are all Evidence Level L5 — model prediction only, with no supporting trials or literature — and are likewise recommended for Hold.
Disclaimer

This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.