Decitabine
| Evidence Level: L3 | Predicted Indications: 1 |
Table of Contents
Decitabine: From Myelodysplastic Syndromes to Refractory Cytopenia of Childhood
One-Sentence Summary
Decitabine is a hypomethylating agent marketed in the US for myelodysplastic syndromes (MDS). The TxGNN model predicts it may be useful for refractory cytopenia of childhood (RCC), the most common pediatric MDS subtype. Support is thin: 0 registered clinical trials and 1 publication (a single-center retrospective cohort in pediatric MDS).
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Myelodysplastic syndromes (from general drug knowledge; the approved-indication text was empty in the input data) |
| Predicted New Indication | Refractory cytopenia of childhood |
| TxGNN Prediction Score | 99.03% (a model output, not clinical evidence) |
| Evidence Level | L3 |
| US Market Status | ✓ Marketed |
| Number of Licenses | 20 (generic ANDAs) |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Detailed mechanism-of-action data is not currently available in the input. Decitabine is a DNA methyltransferase inhibitor (hypomethylating agent). It is thought to reverse abnormal DNA hypermethylation in dysplastic blood-forming clones. This explanation comes from general knowledge of the drug class, not from the Evidence Pack.
RCC is the most common pediatric MDS subtype, so its biology overlaps with adult MDS, the setting where decitabine is already used. That makes the prediction biologically plausible.
The one available paper does not test decitabine alone. It studies decitabine as part of a minimally myelosuppressive regimen used to bridge children to allogeneic stem cell transplantation. The evidence therefore supports decitabine as a bridging or conditioning component, not as standalone therapy for RCC. The paper covers pediatric MDS in general, and no RCC-specific results are confirmed.
Clinical Trial Evidence
Currently no related clinical trials registered.
Literature Evidence
| PMID | Year | Type | Journal | Key Findings |
|---|---|---|---|---|
| 35624441 | 2022 | Cohort (inferred as retrospective, single center) | BMC Pediatrics | 10-year single-center experience with decitabine plus a minimally myelosuppressive regimen bridging to allo-HSCT in children with MDS. Only the study objective was available; outcome data and RCC-specific results were not confirmed. |
US Market Information
| Authorization Number | Product Name | Dosage Form | Manufacturer |
|---|---|---|---|
| ANDA210756 | Decitabine | Lyophilized powder for injection | Fresenius Kabi USA, LLC |
| ANDA204607 | Decitabine | Lyophilized powder for injection | Mylan Institutional LLC |
| ANDA203131 | Decitabine | Lyophilized powder for injection | Dr. Reddy’s Laboratories Inc. |
| ANDA215355 | Decitabine | Lyophilized powder for injection | Camber Pharmaceuticals, Inc. |
| ANDA203131 | Decitabine | Lyophilized powder for injection | Dr. Reddy’s Laboratories Limited |
The approved-indication text was not provided for these authorizations. All listed products are injectables.
Cytotoxicity
This section is based on general knowledge of the drug class, not on the Evidence Pack. Please also refer to the package insert warnings and precautions.
| Item | Content |
|---|---|
| Cytotoxicity Classification | Conventional cytotoxic (hypomethylating nucleoside analog) |
| Myelosuppression Risk | High (neutropenia and thrombocytopenia are common) |
| Emetogenicity Classification | Low |
| Monitoring Items | CBC with differential and platelets, liver and renal function |
| Handling Protection | Follow hazardous (cytotoxic) drug handling regulations |
Safety Considerations
Please refer to the package insert for safety information.
Conclusion and Next Steps
Decision: Hold
Rationale: The only evidence is one retrospective single-center cohort. It studies decitabine in a combination regimen for pediatric MDS in general, not as monotherapy or specifically in RCC. The package insert safety data is also missing, which blocks the safety screening step. The pediatric setting and the drug’s high myelosuppression risk make that gap especially important.
To proceed, the following is needed:
- The FDA package insert (warnings, contraindications, approved indications), parsed into the safety data
- Mechanism of action data from DrugBank
- The full text of PMID 35624441, to confirm study design, population (RCC subgroup) and outcomes
- A search of trial registries (including ICTRP) and the literature for RCC-specific decitabine studies
- Pediatric dosing and toxicity data, particularly for combination regimens before transplant
This report is for research reference only and does not constitute medical advice. Drug repurposing candidates require clinical validation before any application.
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.