Cyclophosphamide
| Evidence Level: L5 | Predicted Indications: 2 |
Table of Contents
- Cyclophosphamide
- Pharmacist Assessment Report
- Cyclophosphamide: Vertebral anomalies and variable endocrine and T-cell dysfunction
- One-Sentence Summary
- Quick Overview
- Why is This Prediction Reasonable?
- Clinical Trial Evidence
- Literature Evidence
- Australia Market Information
- Cytotoxicity
- Safety Considerations
- Conclusion and Next Steps
- Disclaimer
Cyclophosphamide: Vertebral anomalies and variable endocrine and T-cell dysfunction
One-Sentence Summary
Cyclophosphamide is an alkylating chemotherapy drug used across a range of cancers and lymphomas. The TxGNN model predicts it may be relevant to retroperitoneal neoplasm, but no clinical trials are registered and the supporting literature consists of case reports and small series in which cyclophosphamide was given within combination regimens.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Not recorded in the local data |
| Predicted New Indication | Vertebral anomalies and variable endocrine and T-cell dysfunction |
| TxGNN Prediction Score | 0.9938 |
| Evidence Level | L5 |
| Australia Market Status | Listed on the PBS Schedule (1 of 1 items) – TGA (ARTG) registration status is not included in this data |
| Number of PBS Items | 1 (valid: 1) |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Detailed mechanism of action data is not available in the data compiled for this report. Cyclophosphamide is a well-known alkylating agent that damages the DNA of rapidly dividing cells, which is the general basis for its use in many malignancies.
“Retroperitoneal neoplasm” is an anatomical grouping rather than a single disease. It covers heterogeneous tumours such as paraganglioma, germ cell tumours, rhabdomyosarcoma and desmoplastic small round cell tumour. Cyclophosphamide has been used in some of these as part of multi-drug regimens (for example with vincristine and dacarbazine, or vincristine and doxorubicin). Because the reports are individual cases or small series, any benefit cannot be attributed to cyclophosphamide alone. The prediction is supported mainly by the model score and an indirect mechanistic fit.
Clinical Trial Evidence
Currently no related clinical trials registered.
Literature Evidence
None of the publications below is a controlled trial of cyclophosphamide in retroperitoneal neoplasm as a defined disease. They are case reports, case series and cohort studies of specific tumour types with retroperitoneal involvement, typically using combination chemotherapy.
| PMID | Year | Type | Journal | Key Findings |
|---|---|---|---|---|
| 10656434 | 2000 | Cohort (Phase II) | Clin Cancer Res | Cyclophosphamide plus carboplatin in advanced seminoma: 32 of 46 patients (70%) reached complete remission with chemotherapy alone |
| 9058098 | 1997 | Case series | Intern Med | Cyclophosphamide, vincristine and dacarbazine in malignant paraganglioma with retroperitoneal and hepatic tumours |
| 26813434 | 2015 | Case series | Zhonghua Zhong Liu Za Zhi | Analysis of diagnosis and treatment of desmoplastic small round cell tumour |
| 8874321 | 1996 | Cohort | J Clin Oncol | High-dose ifosfamide, carboplatin and etoposide with stem-cell support in germ cell cancer (cyclophosphamide not part of the regimen) |
| 15739629 | 2004 | Case report and review | Pediatr Hematol Oncol | Malignant retroperitoneal paraganglioma in a child; chemotherapy debulking with cyclophosphamide, vincristine and dacarbazine |
| 34689500 | 2021 | Case report | J Coll Physicians Surg Pak | Testicular primitive neuroectodermal tumour with retroperitoneal nodes treated with IE and VAC chemotherapy |
| 2191575 | 1990 | Case report | Hinyokika Kiyo | Paratesticular rhabdomyosarcoma with retroperitoneal nodal spread; effective VAC therapy |
| 2658509 | 1989 | Case report | Hinyokika Kiyo | Advanced paratesticular rhabdomyosarcoma with retroperitoneal metastasis |
| 4027886 | 1985 | Case report | Cancer | Two infants with unresectable retroperitoneal fibrosarcoma achieved complete responses with doxorubicin, vincristine and cyclophosphamide |
| 14606716 | 2003 | Case report | Intern Med | Retroperitoneal fibrosis with intracranial lesions responded to corticosteroid and cyclophosphamide (a non-malignant condition) |
Australia Market Information
| ARTG Number | Product Name | Dosage Form | Approved Indication |
|---|---|---|---|
| ARTG 1266P | Cyclonex | Tablet 50 mg (anhydrous) | Not recorded in the local data |
One licence is recorded and it is valid (single-ingredient product, oral route).
Cytotoxicity
| Item | Content |
|---|---|
| Cytotoxicity Classification | Conventional cytotoxic (alkylating agent) |
| Myelosuppression Risk | High; neutropenia and other cytopenias are expected |
| Emetogenicity Classification | Moderate (dose dependent) |
| Monitoring Items | Full blood count with differential, liver and renal function, electrolytes, urinalysis for haematuria |
| Handling Protection | Must follow cytotoxic drug handling requirements |
Please refer to the Product Information (PI) warnings and precautions for full details. Bladder toxicity (haemorrhagic cystitis) is a recognised concern with this drug class.
Safety Considerations
- Drug Interactions: The interaction screen returned 100 entries; the first 20 reviewed are mostly Moderate, with some Minor. Main ones include:
- Amphotericin B products (conventional, lipid complex, cholesteryl sulfate, liposomal): Moderate
- Sulfonylureas and related glucose-lowering agents (chlorpropamide, acetohexamide, glimepiride, glipizide, glyburide, tolazamide, tolbutamide, nateglinide, repaglinide, troglitazone): Moderate
- Bupropion, aprepitant, metronidazole, nitisinone: Moderate
- Levofloxacin, ondansetron: Minor
Please refer to the TGA-approved Product Information (PI) for warnings and contraindications.
Conclusion and Next Steps
Decision: Hold
Rationale: There are no registered trials, and the literature consists of case reports and small series in which cyclophosphamide was part of multi-drug regimens for different tumour types. No cited study directly addresses retroperitoneal neoplasm as a single disease, so the evidence level is L5.
To proceed, the following is needed:
- Mechanism of action data for cyclophosphamide
- A defined tumour subtype within retroperitoneal neoplasm (for example sarcoma or germ cell tumour) with a clear clinical question
- Evidence that separates the contribution of cyclophosphamide from the other drugs in combination regimens
- Safety information from the TGA-approved Product Information (warnings and contraindications)
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.