Vincristine
| Evidence Level: L3 | Predicted Indications: 3 |
Table of Contents
Vincristine: From Lymphoid Cancers to Ganglioneuroblastoma
One-Sentence Summary
Vincristine is a vinca alkaloid chemotherapy drug, originally used for acute lymphoblastic leukemia, Hodgkin and non-Hodgkin lymphomas, and Langerhans cell histiocytosis. The TxGNN model predicts it may be effective for Ganglioneuroblastoma, with 0 registered clinical trials and 6 publications (mostly case reports) currently supporting this direction.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Acute lymphoblastic leukemia, Hodgkin lymphoma, non-Hodgkin lymphoma, mantle cell lymphoma, Langerhans cell histiocytosis (from the knowledge graph; no approved indication text is available in the US license record) |
| Predicted New Indication | Ganglioneuroblastoma |
| TxGNN Prediction Score | 99.31% |
| Evidence Level | L3 |
| US Market Status | Cancelled only (the single ANDA is cancelled) |
| Number of NDAs | 1 (ANDA) |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Detailed mechanism of action data is not available in the data compiled for this report. Based on general pharmacological knowledge, vincristine binds tubulin and blocks microtubule polymerization, causing mitotic arrest in rapidly dividing cells.
Ganglioneuroblastoma is a neuroblastic tumor in the same family as neuroblastoma. Vincristine is a standard component of multi-agent chemotherapy for neuroblastic tumors, typically with cyclophosphamide, doxorubicin, and cisplatin. This biological and therapeutic proximity is consistent with the high TxGNN score.
There is an important caveat. The supplied literature is mostly case reports, plus one prospective trial in a broader neuroblastic tumor population. Vincristine’s individual contribution within combination regimens cannot be isolated, and most supplied titles do not name vincristine. The link therefore rests on knowledge of standard regimens and is not confirmed by the data compiled here.
Clinical Trial Evidence
Currently no related clinical trials registered.
Literature Evidence
| PMID | Year | Type | Journal | Key Findings |
|---|---|---|---|---|
| 31342649 | 2019 | Prospective clinical trial (non-randomized) | Pediatric Blood & Cancer | JN-L-10 trial using image-defined risk factors to guide surgical timing in low-risk neuroblastic tumors; vincristine is not specifically evaluated |
| 8255850 | 1993 | Case report | Postgraduate Medical Journal | Spinal ganglioneuroblastoma in a 21-year-old achieved histologically proven complete remission with chemotherapy alone (doxorubicin, vincristine, cyclophosphamide, etoposide, ifosfamide, cisplatin) |
| 15701990 | 2005 | Case report | J Pediatr Hematol Oncol | Ganglioneuroblastoma presenting with obstructive jaundice; treated with a regimen of cisplatin, pirarubicin/doxorubicin, cyclophosphamide, and vincristine |
| 7421294 | 1980 | Case series | J Thorac Cardiovasc Surg | 31 patients with intrathoracic ganglioneuroblastoma treated with resection, radiation, or chemotherapy; 27 survived, with follow-up up to 25 years |
| 3071124 | 1988 | Case report | Hinyokika Kiyo | Multimodality treatment of adult adrenal ganglioneuroblastoma with giant regional lymph node metastasis |
| 8888754 | 1996 | Case report | J Pediatr Hematol Oncol | Rare gastric involvement in an infant with stage 4 multifocal ganglioneuroblastoma |
US Market Information
| Authorization Number | Product Name | Dosage Form | Approved Indication |
|---|---|---|---|
| ANDA071484 (cancelled) | VinCRIStine Sulfate (Hospira, Inc.) | Injection, solution | Not available |
Cytotoxicity
| Item | Content |
|---|---|
| Cytotoxicity Classification | Conventional cytotoxic (vinca alkaloid, antimicrotubule agent) |
| Myelosuppression Risk | Please refer to the package insert warnings and precautions |
| Emetogenicity Classification | Please refer to the package insert warnings and precautions |
| Monitoring Items | Complete blood count (leukopenia appears in the contraindication data), liver function and bilirubin, neurological assessment for peripheral neuropathy |
| Handling Protection | Must follow cytotoxic drug handling regulations |
Safety Considerations
- Contraindications (knowledge-graph data, not label text): Liver failure, cholestasis and other hyperbilirubinemia conditions (including Gilbert syndrome), leukopenia, peripheral neuropathy and neuromuscular disease (including Charcot-Marie-Tooth disease), acute urate nephropathy, and certain infectious diseases (fungal infection, dermatophytosis).
- Drug Interactions: 100 interactions were identified; the list reviewed here is a partial one. The major interaction is with clarithromycin. Moderate interactions include aprepitant, dexamethasone, metronidazole, eliglustat, naltrexone, miconazole, rolapitant, rosuvastatin, simvastatin, tinidazole, and troglitazone. Many of these involve CYP3A4 or transporter effects, so co-medication needs careful review.
Package insert warnings and contraindications were not available, so please refer to the package insert for the full safety information.
Conclusion and Next Steps
Decision: Hold
Rationale: The evidence is limited to case reports and one non-randomized trial in a broader population, with no registered trials specific to this indication. Vincristine’s own contribution cannot be separated from combination regimens. Its US license is cancelled, and label-level safety data are missing, so the case does not yet support advancing.
To proceed, the following is needed:
- FDA package insert warnings and contraindications (a blocking data gap for safety screening)
- Mechanism of action data from DrugBank
- Systematic review of neuroblastic tumor trials (for example, COG or SIOPEN protocols) to isolate vincristine’s role
- Confirmation of a current marketed vincristine product and its approved indications in the US
- A plan for managing neurotoxicity, hepatic impairment, and drug interaction risks in the target population
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.