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Intra-arterial and oral digoxin therapy for retinoblastoma
Mrinali Patel1, Yannis M Paulus, Y Pierre Gobin
1Ophthalmic Oncology Service, Memorial Sloan-Kettering Cancer Center, New York, USA.
Purpose:
Preclinical studies demonstrate that cardiac glycosides such as ouabain and digoxin have antitumor effects on retinoblastoma cells in vitro and in a xenograft murine model of retinoblastoma.
Methods:
Based on these findings, we report a case of intra-arterial followed by systemic oral digoxin therapy in a patient with unilateral retinoblastoma that had failed prior intra-arterial chemotherapy.
Results:
Oral administration of digoxin produced no effect, while intra-arterial digoxin therapy produced a modest but measurable response that was likely limited by the inability to achieve sustained drug concentration in the eye.
Conclusions:
This case highlights both the potential promise and limitations of cardiac glycoside therapy in retinoblastoma.
Insights
Cardiac glycosides show promise for retinoblastoma treatment. Intra-arterial digoxin offered a modest response in one patient, but sustained drug delivery to the eye remains a challenge.
Area of Science:
- Oncology
- Ophthalmology
- Pharmacology
Background:
- Preclinical studies suggest cardiac glycosides, including ouabain and digoxin, possess antitumor properties against retinoblastoma.
- Retinoblastoma is a pediatric eye cancer with limited treatment options for advanced or refractory cases.
Observation:
- A case study investigated digoxin therapy in a patient with unilateral retinoblastoma that was refractory to intra-arterial chemotherapy.
- The treatment involved an initial intra-arterial administration of digoxin followed by systemic oral therapy.
Findings:
- Systemic oral digoxin administration demonstrated no discernible effect on the retinoblastoma.
- Intra-arterial digoxin therapy resulted in a modest, measurable tumor response.
- The efficacy of intra-arterial digoxin was likely constrained by challenges in maintaining sustained therapeutic drug concentrations within the ocular environment.
Implications:
- This case underscores the potential therapeutic role of cardiac glycosides in retinoblastoma management.
- Limitations in achieving adequate intraocular drug levels highlight the need for innovative delivery strategies for cardiac glycoside therapy in retinoblastoma.
