Vinblastine
| Evidence Level: L3 | Predicted Indications: 10 |
Table of Contents
Vinblastine: From Hodgkin Lymphoma and Other Lymphomas to Rhabdomyosarcoma
One-Sentence Summary
Vinblastine is a vinca alkaloid chemotherapy drug, originally used for Hodgkin lymphoma, non-Hodgkin lymphoma, mycosis fungoides (cutaneous T-cell lymphoma), mantle cell lymphoma and Langerhans cell histiocytosis. The TxGNN model predicts it may be effective for Rhabdomyosarcoma, but there are currently 0 registered clinical trials and only 7 publications (mostly case reports, small series and preclinical studies), so the evidence remains limited.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Hodgkin lymphoma, non-Hodgkin lymphoma, mycosis fungoides and primary cutaneous T-cell lymphoma, mantle cell lymphoma, Langerhans cell histiocytosis (from the knowledge graph; the US label indication text is not available) |
| Predicted New Indication | Rhabdomyosarcoma |
| TxGNN Prediction Score | 99.86% |
| Evidence Level | L3 |
| US Market Status | Cancelled only (the single ANDA is cancelled) |
| Number of NDAs | 1 (ANDA; cancelled) |
| 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 known pharmacology, vinblastine is a vinca alkaloid that binds tubulin and inhibits microtubule assembly, causing mitotic arrest. Rapidly dividing tumour cells are therefore plausibly sensitive to it.
Its established uses are in lymphomas and related haematological malignancies. Rhabdomyosarcoma is a fast-growing soft tissue sarcoma, mainly of childhood, so an antimitotic agent is mechanistically reasonable. Published reports include vinblastine in combination regimens (for example cisplatin, vinblastine and bleomycin) in children with refractory prostatic rhabdomyosarcoma, and a 2023 adult perianal case with a partial response on a combination containing vinblastine. Preclinical work in rhabdomyosarcoma models also describes synthetic lethality between PLK1 inhibitors and microtubule-destabilizing drugs.
These signals remain anecdotal. In the combination reports, vinblastine’s individual contribution cannot be separated from the other agents.
Other predicted indications in the data are weaker or comparable:
- Monocytic leukemia (L3): historical 1970s clinical series reported remissions in acute monocytic leukemia, but the evidence is uncontrolled and largely superseded by modern therapy.
- Neuroblastoma (L3): preclinical synergy with rapamycin and a phase I study of vinblastine plus sirolimus in paediatric solid tumours.
- Several rhabdomyosarcoma subtypes, liver sarcoma, ganglioneuroblastoma: little or no specific evidence, so these rest mainly on the model score.
Clinical Trial Evidence
Currently no related clinical trials registered.
Literature Evidence
| PMID | Year | Type | Journal | Key Findings |
|---|---|---|---|---|
| 41216926 | 2026 | Cohort (indirect) | Pediatric Blood & Cancer | CWS-96 and CWS-2002P prospective trials in localised non-rhabdomyosarcoma soft tissue sarcoma in children and adolescents; indirect relevance |
| 22156656 | 2011 | Pilot study | Oncotarget | Pilot study of a paediatric metronomic 4-drug regimen, evaluated as an option for resistant cancer |
| 38050209 | 2023 | Case report/Review | Medicine | Adult perianal rhabdomyosarcoma; partial response after nivolumab, dacarbazine, cisplatin and vinblastine, followed by surgery |
| 26024389 | 2015 | Preclinical | Cell Death & Differentiation | PLK1 inhibitors and microtubule-destabilizing drugs show synthetic lethality in rhabdomyosarcoma models |
| 3329524 | 1987 | Preclinical | Anti-Cancer Drug Design | Basis of therapeutic selectivity of vinca alkaloids examined in rhabdomyosarcoma xenografts in mice |
| 2451411 | 1987 | Case report/Review | Hinyokika Kiyo | Refractory prostatic rhabdomyosarcoma in a 3-year-old; bulky pelvic mass reduced rapidly on cisplatin, vinblastine and peplomycin |
| 14328283 | 1965 | Review (historical) | British Journal of Clinical Practice | Historical overview of tumour chemotherapy in children |
US Market Information
| Authorization Number | Product Name | Dosage Form | Approved Indication |
|---|---|---|---|
| ANDA089515 (cancelled) | Vinblastine Sulfate (Fresenius Kabi USA, LLC) | Injection | Not available |
Cytotoxicity
| Item | Content |
|---|---|
| Cytotoxicity Classification | Conventional cytotoxic (vinca alkaloid, antimitotic) |
| Myelosuppression Risk | High. Neutropenia and thrombocytopenia are the main concerns (also flagged in the knowledge-graph contraindication list) |
| Emetogenicity Classification | Low to moderate |
| Monitoring Items | Complete blood count with differential, liver function (including bilirubin), renal function, neurological assessment for peripheral neuropathy |
| Handling Protection | Must follow cytotoxic drug handling regulations. Please refer to the package insert warnings and precautions |
Safety Considerations
- Contraindication signals (from the knowledge graph, not label text): liver failure, thrombocytopenia, neutropenia, agranulocytosis, peripheral neuropathy and other peripheral nervous system disease, hyperbilirubinemia (including Gilbert syndrome), lung disease, myocardial ischemia and coronary artery disease, and infectious disease (including fungal infections and dermatophytosis). Anxiety and dysthymic disorders and hyperuricemia also appear in this list.
- Drug Interactions: 100 interactions were identified. Examples include:
- Major: clarithromycin
- Moderate: aprepitant, dexamethasone, metronidazole, eliglustat, miconazole, rolapitant, rosuvastatin, simvastatin, tinidazole, glycerol phenylbutyrate, troglitazone
- Minor: levofloxacin
- Literature safety signals: severe ileus with cisplatin-vinblastine infusion in neuroblastoma, and neurotoxicity concerns related to tubulin-targeting agents.
Please refer to the package insert for the official warnings and contraindications, which are not available in the data compiled for this report.
Conclusion and Next Steps
Decision: Hold
Rationale: The TxGNN score is high (99.86%) and the antimitotic mechanism is plausible. However, there are no registered trials, the literature is limited to case reports, small series and preclinical work, and vinblastine’s individual contribution in combination regimens cannot be isolated. The only US authorization is cancelled, and the official label safety data are missing.
To proceed, the following is needed:
- FDA package insert warnings and contraindications (a blocking gap for safety screening)
- Detailed mechanism of action data from DrugBank
- Comparison with the current standard rhabdomyosarcoma regimens (vincristine-based), including whether vinblastine adds value
- Prospective or registry data on vinblastine in rhabdomyosarcoma
- A plan to confirm injectable product availability in the US, since the only listed ANDA is cancelled
- A safety monitoring plan covering myelosuppression, neurotoxicity, hepatic impairment and CYP3A4 interactions (e.g. clarithromycin)
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.