Filgrastim
| Evidence Level: L5 | Predicted Indications: 10 |
Table of Contents
- Filgrastim
- Pharmacist Assessment Report
- Filgrastim: From Severe Congenital Neutropenia to Primary Release Disorder of Platelets
- One-Sentence Summary
- Quick Overview
- Why is This Prediction Reasonable?
- Clinical Trial Evidence
- Literature Evidence
- India Market Information
- Safety Considerations
- Conclusion and Next Steps
- Disclaimer
Filgrastim: From Severe Congenital Neutropenia to Primary Release Disorder of Platelets
One-Sentence Summary
Filgrastim is a G-CSF analog originally used to treat constitutional and severe congenital neutropenia, cyclic hematopoiesis, and chronic familial neutropenia. The TxGNN model predicts it may be effective for primary release disorder of platelets, but no clinical trials and no publications currently support this prediction, so it rests on a computational score alone.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Constitutional neutropenia; severe congenital neutropenia; cyclic hematopoiesis; chronic familial neutropenia |
| Predicted New Indication | Primary release disorder of platelets |
| TxGNN Prediction Score | 1.0000 |
| Evidence Level | L5 |
| India Market Status | Listed in a compiled Indian medicine price dataset (at least 20) – CDSCO approval status is not included in this data |
| Number of Listed Products | at least 20 |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Currently, detailed mechanism of action data is not available. Based on known information, filgrastim is a recombinant G-CSF analog that acts mainly on neutrophil-lineage progenitors through the G-CSF receptor. Its efficacy in neutropenic conditions is established.
Primary release disorder of platelets is a qualitative platelet function defect, involving storage pool or secretion abnormalities. No plausible G-CSF mechanism is documented for it. The high TxGNN score comes from the TxGNN model (deep learning) and may reflect similarities the model learned between related diseases, not biological evidence, so the link between the original and new indications is weak.
The other top-ranked predictions (pseudo-von Willebrand disease, Glanzmann thrombasthenia, Scott syndrome, collagen receptor defects, C1 inhibitor deficiency and others) show the same pattern. Each involves a defect that filgrastim does not target.
Clinical Trial Evidence
Currently no related clinical trials are registered for the top predicted indication.
For reference, the 10th-ranked prediction, platelet-type bleeding disorder (score 0.9969), retrieved seven trials. All are hematopoietic stem cell transplant studies in which filgrastim is only a supportive component. None tests filgrastim in a platelet disorder, and all were graded low relevance.
| Trial Number | Phase | Status | Enrollment | Key Findings |
|---|---|---|---|---|
| NCT00296023 | NA | Completed | 25 | Low-dose allogeneic stem cell transplant for high-risk low-grade hematologic malignancies |
| NCT00004232 | Phase 1 | Completed | Not reported | CD34-enriched stem cell augmentation in allogeneic transplant |
| NCT00054236 | Phase 1 | Completed | 55 | Multiple cord blood unit transplant after non-myeloablative conditioning |
| NCT00301912 | Phase 2 | Withdrawn | 0 | Busulfan/fludarabine preparative therapy; no data |
| NCT00295997 | NA | Unknown | 35 | Non-myeloablative matched unrelated donor transplant |
| NCT00080925 | Phase 1 | Completed | 20 | T-cell depleted reduced-intensity haploidentical transplant |
| NCT00004904 | Phase 1 | Completed | Not reported | T-cell depleted partially matched donor transplant with CD34 “veto” cells |
Literature Evidence
Currently no related literature available.
India Market Information
All 20 registrations are valid single-ingredient injectables. No approved indication text was provided for any of them. The five main ones are listed below.
| Authorization Number | Product Name | Dosage Form | Approved Indication |
|---|---|---|---|
| 84930 | Fillif 300mcg Injection (Torrent) | Prefilled syringe, 1 ml | Not stated |
| 89966 | Ferograstim 300mcg Injection (Feron Healthcare) | Vial | Not stated |
| 95110 | Grastim Injection (Dr Reddy’s) | Vial, 1 ml | Not stated |
| 243969 | Xgrast 300mcg Injection (Emcure) | Vial, 1 ml | Not stated |
| 150319 | Neukine 300mcg Injection (Intas) | Prefilled syringe, 1 ml | Not stated |
Safety Considerations
- Drug Interactions: 145 interactions were identified in total. The ones listed below are all rated Moderate. They are mostly cytotoxic chemotherapy, targeted anticancer agents, and radiopharmaceuticals:
- Paclitaxel
- Mercaptopurine
- Azacitidine
- Bendamustine
- Arsenic trioxide
- Alemtuzumab
- Aldesleukin
- Abemaciclib
- Acalabrutinib
- Ibritumomab tiuxetan
- Tositumomab (including I-131)
- Iobenguane (I-131)
- Fludeoxyglucose (18F)
- Levamisole
Please refer to the package insert for warnings and contraindications, as no label-based data were available.
Conclusion and Next Steps
Decision: Hold
Rationale: The prediction is supported only by a model score (Evidence Level L5). No trial or publication tests filgrastim in primary release disorder of platelets, and its mechanism, which acts on the neutrophil lineage, does not plausibly address a platelet secretion defect. The same is true of the other top-ranked predictions.
To proceed, the following is needed:
- Mechanism of action data from DrugBank, to allow a proper mechanistic-link analysis
- Package insert warnings and contraindications from Indian manufacturers, which are required before any safety screening
- A literature search for any direct evidence of G-CSF in platelet function disorders
- Preclinical or mechanistic studies showing a plausible G-CSF effect on platelet release function
- Route compatibility and similarity-to-original assessments, which are still pending
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.