Defibrotide
| Evidence Level: L5 | Predicted Indications: 50 |
Quick Overview
| Item | Value |
|---|---|
| Drug Name | Defibrotide |
| DrugBank ID | DB04932 |
| Brand Names (EU) | Defitelio |
| Evidence Level | L5 |
| Predicted Indications | 50 |
| Top Prediction Score | 99.91% |
Approved Indication (EMA)
Defitelio is indicated for the treatment of severe hepatic veno-occlusive disease (VOD) also known as sinusoidal obstructive syndrome (SOS) in haematopoietic stem-cell transplantation (HSCT) therapy. It is indicated in adults and in adolescents, children and infants over 1 month of age.
Predicted New Indications
TxGNN model predictions for potential drug repurposing:
| Rank | Indication | Score | Source |
|---|---|---|---|
| 1 | pseudo-von Willebrand disease | 99.91% | DL |
| 2 | primary release disorder of platelets | 99.91% | DL |
| 3 | Glanzmann thrombasthenia | 99.88% | DL |
| 4 | thrombotic thrombocytopenic purpura | 99.71% | DL |
| 5 | Scott syndrome | 99.67% | DL |
| 6 | bleeding diathesis due to a collagen receptor defect | 99.43% | DL |
| 7 | hemorrhagic disorder due to a constitutional thrombocytopenia | 99.39% | DL |
| 8 | congenital factor V deficiency | 99.30% | DL |
| 9 | fetal and neonatal alloimmune thrombocytopenia | 99.23% | DL |
| 10 | thrombocytopenic purpura | 99.22% | DL |
| 11 | inherited thrombophilia | 99.16% | DL |
| 12 | hepatic infarction | 99.14% | DL |
| 13 | peliosis hepatis | 98.98% | DL |
| 14 | familial apolipoprotein C-II deficiency | 98.79% | DL |
| 15 | syndrome with combined immunodeficiency | 98.75% | DL |
| 16 | platelet-type bleeding disorder | 98.69% | DL |
| 17 | Ehlers-Danlos syndrome, fibronectinemic type | 98.68% | DL |
| 18 | hepatic veno-occlusive disease | 98.67% | DL |
| 19 | flood factor deficiency | 98.64% | DL |
| 20 | methylcobalamin deficiency type cblG | 98.25% | DL |
Showing top 20 of 50 predictions.
About TxGNN Predictions
Prediction Sources
| Source | Description |
|---|---|
| KG | Knowledge Graph - Network topology-based associations |
| DL | Deep Learning - Neural network score prediction |
Evidence Levels
| Level | Definition |
|---|---|
| L1 | Multiple Phase 3 RCTs / Systematic Reviews |
| L2 | Single RCT or multiple Phase 2 trials |
| L3 | Observational studies / Large case series |
| L4 | Preclinical / Mechanistic / Case reports |
| L5 | AI prediction only (current) |
Clinical Validation Needed
Research Use Only: These predictions are computational hypotheses that require clinical validation. They should NOT be used for clinical decision-making.
Next Steps for Validation
- Literature Review: Search PubMed for existing evidence
- Clinical Trial Search: Check ClinicalTrials.gov for ongoing studies
- Mechanistic Analysis: Evaluate biological plausibility
- Preclinical Studies: Conduct in vitro/in vivo validation
- Clinical Trials: Design and conduct human studies
Data Access
- FHIR API:
/fhir/ClinicalUseDefinition/ - CSV Download: All Predictions
- GitHub: yao-care/EuTxGNN
Citation
If using this data, please cite:
@article{huang2023txgnn,
title={A foundation model for clinician-centered drug repurposing},
author={Huang, Kexin and others},
journal={Nature Medicine},
year={2023},
doi={10.1038/s41591-023-02233-x}
}
Disclaimer: This report is for research purposes only and does not constitute medical advice. Drug repurposing predictions require rigorous clinical validation before any therapeutic application.