Decitabine
| Evidence Level: L5 | Predicted Indications: 1 |
Table of Contents
- Decitabine
- Pharmacist Assessment Report
- Decitabine: hypomethylating agent (DNA methyltransferase inhibitor) → refractory cytopenia of childhood
- 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
Decitabine: hypomethylating agent (DNA methyltransferase inhibitor) → refractory cytopenia of childhood
One-Sentence Summary
Decitabine is a hypomethylating agent (DNA methyltransferase inhibitor) used in myeloid malignancies. The TxGNN model predicts it may be effective for refractory cytopenia of childhood, a paediatric myelodysplastic syndrome subtype. Currently 0 clinical trials and 2 publications (one cohort study and one case report, both in paediatric myelodysplastic syndrome) are available, and neither directly studies refractory cytopenia of childhood as a defined cohort.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Not recorded in the local data |
| Predicted New Indication | refractory cytopenia of childhood |
| TxGNN Prediction Score | 0.9903 |
| 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 currently available. Based on the drug class, decitabine is a hypomethylating agent that inhibits DNA methyltransferase. This class is used in myeloid malignancies, including acute myeloid leukaemia and myelodysplastic syndromes.
Refractory cytopenia of childhood is the most common paediatric myelodysplastic syndrome subtype. Aberrant DNA methylation and clonal myeloid dysregulation are plausible shared biology. This link is inferred from the drug class and related myeloid disease, not from supplied mechanism data. The TxGNN score is a computational output and is not clinical evidence.
Clinical Trial Evidence
Currently no related clinical trials registered.
Literature Evidence
| PMID | Year | Type | Journal | Key Findings |
|---|---|---|---|---|
| 35624441 | 2022 | Cohort | BMC Pediatrics | Single-centre, 10-year experience of decitabine plus a minimally myelosuppressive regimen as a bridge to allogeneic stem cell transplantation in children with myelodysplastic syndrome |
| 42125547 | 2026 | Case report | Experimental and Therapeutic Medicine | Decitabine monotherapy in an 8-year-old with paediatric myelodysplastic syndrome, most commonly presenting as refractory cytopenia of childhood, in a patient ineligible for transplantation or immunosuppressive therapy |
The abstracts supplied are truncated, so outcomes cannot be summarised. The evidence level is set to L5 because the cohort study addresses paediatric myelodysplastic syndrome broadly, and the case report abstract excerpt does not confirm the diagnosis.
Australia Market Information
| ARTG Number | Product Name | Dosage Form | Approved Indication |
|---|---|---|---|
| ARTG 13081L | Inqovi 35/100 | Tablet containing decitabine 35 mg with cedazuridine 100 mg | Not recorded in the local data |
Note: this product is a decitabine and cedazuridine combination, so it does not describe how single-ingredient decitabine is approved.
Cytotoxicity
| Item | Content |
|---|---|
| Cytotoxicity Classification | Conventional cytotoxic (hypomethylating nucleoside analogue) |
| Myelosuppression Risk | Please refer to the Product Information (PI) warnings and precautions |
| Emetogenicity Classification | Please refer to the Product Information (PI) warnings and precautions |
| Monitoring Items | Full blood count with differential, liver and renal function |
| Handling Protection | Follow local cytotoxic drug handling requirements |
Safety Considerations
- Drug Interactions: 120 interactions were identified. Major-level examples include deferiprone, samarium (153Sm) lexidronam, adalimumab and baricitinib. Moderate-level examples include mercaptopurine, zidovudine, ganciclovir, gemcitabine, azathioprine and alemtuzumab.
Please refer to the TGA-approved Product Information (PI) for warnings and contraindications.
Conclusion and Next Steps
Decision: Hold
Rationale: The prediction rests on a high model score and a plausible class-based rationale. No registered trials exist, and the two publications are one cohort study and one case report in paediatric myelodysplastic syndrome. Because no study directly addresses refractory cytopenia of childhood, the evidence level is L5 and this remains a research question.
To proceed, the following is needed:
- Studies reporting outcomes specifically in refractory cytopenia of childhood
- Mechanism of action data
- Warnings and contraindications from the TGA Product Information
- Paediatric dosing and safety data, including for the oral combination product
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.