Cyclosporine

Evidence Level: L5

Predicted Indications: 7

Cyclosporine

Pharmacist Assessment Report

Cyclosporine: calcineurin inhibitor immunosuppressant → granulomatous disease, chronic, autosomal recessive

One-Sentence Summary

Cyclosporine is a calcineurin inhibitor immunosuppressant. The TxGNN model predicts it may be relevant to granulomatous disease, chronic, autosomal recessive, but the data compiled for this report contain no clinical trials and 1 publication. That publication describes stem cell transplantation outcomes and does not test cyclosporine as a treatment for this disease.

Quick Overview

Item	Content
Original Indication	Not recorded in the local data
Predicted New Indication	granulomatous disease, chronic, autosomal recessive
TxGNN Prediction Score	0.9968
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 are not available in the data compiled for this report. Cyclosporine is generally known as a calcineurin inhibitor that suppresses T-cell activation. It is widely used as part of graft-versus-host disease (GVHD) prophylaxis in haematopoietic stem cell transplantation (HSCT).

The single publication linked to this prediction reports survival after sibling or unrelated donor HSCT for chronic granulomatous disease (CGD). Cyclosporine’s role there would be supportive immunosuppression around the transplant. It would not correct the underlying defect in the NADPH oxidase system that causes CGD. The very high TxGNN score is therefore not backed by direct clinical evidence, and the mechanistic link is indirect.

Literature Evidence

PMID	Year	Type	Journal	Key Findings
22078471	2012	Cohort	The Journal of Allergy and Clinical Immunology	Reports survival after sibling or unrelated donor HSCT in CGD. Cyclosporine is not the studied intervention.

Australia Market Information

ARTG Number	Product Name	Dosage Form	Approved Indication
ARTG 12663L	Ikervis	Eye drops 1 mg per mL, single dose units 0.3 mL, 30	Not recorded in the local data

The only licensed product listed is an ophthalmic preparation. Its route differs from the systemic use that transplant-related immunosuppression would require.

Safety Considerations

Label-based warnings and contraindications are not available in the data compiled for this report. Please refer to the TGA-approved Product Information (PI) for safety information.

Drug interactions (100 recorded; examples from the interaction database):

Major: loperamide
Moderate: rabeprazole, hydrocortisone, amphotericin B, bupropion, aprepitant, mesalazine, triamcinolone, acetylsalicylic acid, dexamethasone, betamethasone, metronidazole, budesonide
Minor: ranitidine, doxycycline

Conclusion and Next Steps

Decision: Hold

Rationale: No clinical trial or publication tests cyclosporine in chronic granulomatous disease itself. The only cited study concerns stem cell transplantation, so this prediction rests on the model score alone (L5).

To proceed, the following is needed:

Studies that directly evaluate cyclosporine in chronic granulomatous disease, rather than as transplant support
Mechanism of action data to support a specific biological link
TGA Product Information safety data, including warnings and contraindications
Clarification of whether a systemic formulation would be needed, since the only licensed product here is an eye drop
Disclaimer

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