Deferiprone
| Evidence Level: L4 | Predicted Indications: 8 |
Table of Contents
- Deferiprone
- Pharmacist Assessment Report
- Deferiprone: From Transfusional Iron Overload in Thalassemia to Hepatic Porphyria
- One-Sentence Summary
- Quick Overview
- Why is This Prediction Reasonable?
- Clinical Trial Evidence
- Literature Evidence
- India Market Information
- Safety Considerations
- Conclusion and Next Steps
- Disclaimer
Deferiprone: From Transfusional Iron Overload in Thalassemia to Hepatic Porphyria
One-Sentence Summary
Deferiprone is an oral iron chelator, originally used for iron overload in thalassemia and related blood disorders. The TxGNN model predicts it may be effective for hepatic porphyria, but the supporting evidence is limited to 2 preclinical publications and no registered clinical trials.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Hemosiderosis and beta-thalassemia-related conditions (including hemoglobinopathy and hereditary persistence of fetal hemoglobin) |
| Predicted New Indication | Hepatic porphyria |
| TxGNN Prediction Score | 0.9920 |
| Evidence Level | L4 |
| India Market Status | Listed in a compiled Indian medicine price dataset (2 of 2) – CDSCO approval status is not included in this data |
| Number of Listed Products | 2 (valid: 2) |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Currently, detailed mechanism of action data is not available. Based on known information, deferiprone is an oral iron chelator, its use in iron overload from thalassemia is established, and mechanistically it may be applicable to hepatic porphyria.
Iron overload and iron-driven oxidative stress worsen uroporphyria in porphyria cutanea tarda and related hepatic porphyrias, partly through inhibition of the enzyme uroporphyrinogen decarboxylase. Lowering hepatic iron could plausibly reduce porphyrin accumulation. Phlebotomy, which removes iron, is already an effective treatment for porphyria cutanea tarda, which supports the idea.
The available studies are preclinical and indirect. A mouse model of porphyria cutanea tarda compared deferiprone with iron-deficient diets. A 2020 study in congenital erythropoietic porphyria, a related but distinct erythropoietic porphyria, found that iron chelation improved hemolytic anemia and skin photosensitivity. No human studies in hepatic porphyria were found.
Clinical Trial Evidence
Currently no related clinical trials registered.
Literature Evidence
| PMID | Year | Type | Journal | Key Findings |
|---|---|---|---|---|
| 32678895 | 2020 | Preclinical (animal model) | Blood | Iron chelation rescued hemolytic anemia and skin photosensitivity in a model of congenital erythropoietic porphyria |
| 17854053 | 2007 | Preclinical (mouse model) | Hepatology | In a mouse model of porphyria cutanea tarda, deferiprone was compared with iron-deficient diets for reducing hepatic uroporphyrin accumulation |
India Market Information
| Authorization Number | Product Name | Dosage Form | Approved Indication |
|---|---|---|---|
| 116019 | Kelfer 250 Capsule (Cipla Ltd) | Bottle of 50 capsules | Not stated in the data compiled |
| 115792 | Kelfer 500 Capsule (Cipla Ltd) | Bottle of 50 capsules | Not stated in the data compiled |
Safety Considerations
- Drug Interactions: 246 interactions were identified in total. The ones listed here are all rated Moderate.
- Polyvalent-cation products, which may bind deferiprone: calcium salts (phosphate, acetate, carbonate, citrate, gluconate, lactate, glubionate), magnesium salts (oxide, carbonate, chloride, citrate, gluconate, hydroxide, sulfate), aluminum hydroxide, magaldrate, kaolin, attapulgite, sucralfate
- Iron
Please refer to the package insert for warnings and contraindications.
Conclusion and Next Steps
Decision: Hold
Rationale: The mechanistic idea is plausible, but the evidence is only preclinical and indirect (one mouse model of porphyria cutanea tarda and one model of a different porphyria). There are no human trials in hepatic porphyria, so the prediction remains a research question.
To proceed, the following is needed:
- Mechanism of action data for deferiprone
- Package insert warnings and contraindications, which are currently missing and block safety screening
- Human data (case series or clinical trials) in porphyria cutanea tarda or other hepatic porphyrias
- Approved indication text for the two Indian registrations
- A plan for managing the interactions with polyvalent-cation products, such as antacids, and with iron
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.