Stargardt Disease Treatment with Topical Cholinergic Medication, Low Dose Echothiophate Iodide

Gerard M Nolan, MD September 2021

The purpose of this web presentation is to share with my peers and their patients my 20 year experiences treating patients with Stargardt disease with off label use of echothiophate iodide.   During this time, I have treated more than 300 Stargardt disease patients with this topical cholinergic medication.  Most patients have shown a sustained improvement of visual acuity and color vision.

My hope is that Stargardt disease patients will be able to receive this treatment by their local ophthalmologist and not have to incur the expense of travel.

There are no conflicts of interest in this shared research.  There has been no financial support, contribution or grant funding received from any individual or organization.

This presentation is intended for use by ophthalmologists only and is not intended to suggest or encourage any type of self-diagnosis or self-treatment of any eye disease.  It is hoped this presentation may expand possible treatments for patients under the care of their ophthalmologists.

Abstract

Purpose: This paper presents evidence of improvement of visual acuity and color vision in patients with Stargardt disease achieved by the application of a topical cholinergic medication (TCM), i.e., low dose echothiophate iodide.

Design: Uni-center retrospective study

Participants: Three hundred nine patients with confirmed Stargardt disease

Methods: Three hundred nine participants were followed with 115 participants analyzed over one year.  Thirty-one participants were followed for 5 years and 7 for greater than 10 years.  Best corrected visual-acuity (BCVA) was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol.  Standardized reporting forms were used to collect participants’ clinical observations.  The data was managed using the Excel spreadsheet application.

Main Outcome Measures: Maximum visual improvement (MVI) and improvement at 1 year, 5 years and greater than 10 years; color vision improvement.

Results: Analysis showed a significant MVI.  Significant improvement at 1 year, 5 years and greater than 10 years was also realized.  The MVI depended on the baseline BCVA as follows:

  • Mean BCVA of eyes with baseline BCVA of 20/25 or better improved by 0.11 logMAR (5.5 ETDRS letters)
  • Mean BCVA of eyes with baseline BCVA between 20/25 and 20/70 improved by 0.19 logMAR (9.5 ETDRS letters)
  • Mean BCVA of eyes with a baseline BCVA between 20/70 and 20/200 improved by 0.22 logMAR (11 ETDRS letters)
  • Mean BCVA of eyes with a baseline BCVA between 20/200 and 20/400 improved by 0.27 logMAR (13.5 ETDRS letters)
  • Mean BCVA of eyes with a baseline BCVA less than 20/400 improved by 0.39 logMAR (19.5 ETDRS letters)

Color vision testing by Ishihara Color Test (10 plates) improved by a mean of 2.7 plates.

Conclusions: Gains in visual acuity in Stargardt disease patients appear significant and prolonged over time using a TCM, i.e., low dose echothiophate.  These improvements are especially effective in patients with baseline BCVA 20/200 to 20/400 and baseline BCVA less than 20/400.  There were also improvements in color vision.

Patient Case Histories

For more patient case studies and information please contact gnolan@nolaneye.com .