Interferon Beta-1B

證據等級: L5 預測適應症: 10

目錄

  1. Interferon Beta-1B
  2. Interferon Beta-1b: From No Approved Danish Indication to Hairy Cell Leukemia
    1. One-Sentence Summary
    2. Quick Overview
    3. Why is This Prediction Reasonable?
    4. Clinical Trial Evidence
    5. Literature Evidence
    6. Denmark Market Information
    7. Safety Considerations
    8. Conclusion and Next Steps
    9. Disclaimer

## 藥師評估報告

Interferon Beta-1b: From No Approved Danish Indication to Hairy Cell Leukemia

One-Sentence Summary

Interferon beta-1b currently holds no marketing authorisation in Denmark, so no approved original indication is recorded for this drug in Danish regulatory data. The TxGNN model predicts it may be effective for Hairy Cell Leukemia, but this direction is currently supported only by 0 registered clinical trials and 4 historical publications (1987–1990), with no active trials or contemporary controlled data.

Quick Overview

Item Content
Original Indication Not available — drug is not marketed in Denmark; no approved indication text on file
Predicted New Indication Hairy Cell Leukemia
TxGNN Prediction Score 99.16%
Evidence Level L3
Denmark Market Status Not Marketed
Number of Marketing Authorisations 0
Recommended Decision Hold

Why is This Prediction Reasonable?

Currently, detailed mechanism of action data for Interferon beta-1b is not available in this evidence pack (flagged as a High-severity data gap). Based on the supporting literature, Interferon beta-1b is a type I interferon with potent antiproliferative and immunomodulatory activity — it induces cell-cycle arrest and enhances NK-cell/monocyte activity. These mechanisms partially overlap with those of interferon alfa, which was historically used as first-line therapy for hairy cell leukemia (HCL) before purine analogues became standard.

Because the drug’s own original indication is not recorded in this dataset (Denmark: not marketed, 0 licenses), the rationale for this prediction rests on drug-class analogy rather than on a documented original-to-new indication relationship. The historical literature (1987–1990) shows that beta-ser interferon produced haematological responses in HCL comparable in direction to alpha-interferon, supporting biological plausibility.

However, the standard of care for HCL has since shifted to purine analogues (cladribine/pentostatin), and all available IFN-beta evidence for HCL consists of small, uncontrolled Phase 1/2 studies or case series from the late 1980s, with no contemporary comparative trials and zero currently registered clinical trials for this indication.

Clinical Trial Evidence

Currently no related clinical trials registered.

Literature Evidence

PMID Year Type Journal Key Findings
2736487 1989 Prospective comparative cohort Cancer 10 HCL patients treated with recombinant beta-serine-interferon; 63% achieved normalization of peripheral blood counts, an additional 25% showed improvement in ≥1 haematologic parameter; persistent hairy cells remained in bone marrow of all patients
2082943 1990 Case series / uncontrolled Phase 2 American Journal of Hematology 12 HCL patients (10 previously treated) given IV beta-ser interferon 90 MU three times weekly; bone marrow involvement 90–100% hairy cells at baseline
2198792 1990 Case series American Journal of Clinical Oncology Patients who failed alpha-2a- or beta-ser-interferon achieved complete response with subsequent pentostatin (2’-deoxycoformycin), illustrating IFN-beta as a bridge/failed first-line option rather than definitive therapy
3312839 1987 Retrospective cohort (single institution) Leukemia UCLA experience across 51 HCL patients on type I interferons; haematological improvement in 96% (alpha-2b), 69% (alpha-N1), and 71% (beta-serine, early follow-up)

Denmark Market Information

Currently not marketed in Denmark — no marketing authorisation is on file for this product.

Safety Considerations

Please refer to the approved Summary of Product Characteristics (SmPC) for safety information.

Conclusion and Next Steps

Decision: Hold

Rationale: The Hairy Cell Leukemia prediction is supported only by L3-level evidence — small, uncontrolled, decades-old (1987–1990) cohort and case-series data with zero currently registered clinical trials. Combined with a Blocking data gap on Danish label warnings/contraindications (DG001) and a High-severity gap on mechanism of action (DG002), plus the drug’s current “not marketed” status in Denmark, the evidence base is insufficient to proceed beyond a research question at this time.

To proceed, the following is needed:

  • SmPC warnings, precautions, and contraindications for Interferon beta-1b (Blocking gap DG001)
  • Confirmed mechanism of action data from DrugBank or equivalent source (DG002)
  • Contemporary comparative evidence for HCL, given that purine analogues (cladribine/pentostatin) are now standard of care
  • Danish/EU regulatory pathway assessment, since the product currently holds no marketing authorisation in Denmark

Note: This evidence pack also contains other TxGNN-predicted indications for Interferon beta-1b with substantially stronger evidence — notably CNS autoimmune/demyelinating disease indications (ranks 3–8), which are supported by dozens of completed trials including multiple completed Phase 3 RCTs and extensive systematic-review literature. These directions likely warrant a separate, dedicated evaluation given their materially higher evidence maturity.

Disclaimer

This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.



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