June 2021
Volume 62, Issue 8
Open Access
ARVO Annual Meeting Abstract  |   June 2021
Corneal cystine crystal deposition: Online medical education can significantly improve knowledge, competence and confidence diagnosing and treating ocular manifestations of nephrotic cystinosis
Author Affiliations & Notes
  • Siggi Trier
    Education, Medscape LLC, New York, New York, United States
  • Pakinam Aboulsaoud
    Education, Medscape LLC, New York, New York, United States
  • John Maeglin
    Education, Medscape LLC, New York, New York, United States
  • Stephanie Cherqui
    Division of Genetics, University of California San Diego, La Jolla, California, United States
  • Paul C Grimm
    Department of Pediatric Nephrology, Lucile Packard Children's Hospital, Stanford University School of Medicine, Stanford, California, United States
  • Rachel J Bishop
    (contractor), National Eye Institute, Bethesda, Maryland, United States
  • Footnotes
    Commercial Relationships   Siggi Trier, Medscape LLC (C); Pakinam Aboulsaoud, Medscape LLC (E); John Maeglin, Medscape LLC (E); Stephanie Cherqui, AVROBIO Inc (C), AVROBIO Inc (I), California Institute of Regenerative Medicine (F), Stelios Therapeutics (C), Stelios Therapeutics (I), The Cystinosis Research Foundation and National Institute of Health (F); Paul Grimm, Alexion (F), Cystinosis Research Foundation (C), Horizon Pharmaceuticals (C); Rachel Bishop, Cystinosis Research Network (C), Leadiant (C)
  • Footnotes
    Support  Developed through an independent educational grant from Recordati
Investigative Ophthalmology & Visual Science June 2021, Vol.62, 135. doi:
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      Siggi Trier, Pakinam Aboulsaoud, John Maeglin, Stephanie Cherqui, Paul C Grimm, Rachel J Bishop; Corneal cystine crystal deposition: Online medical education can significantly improve knowledge, competence and confidence diagnosing and treating ocular manifestations of nephrotic cystinosis. Invest. Ophthalmol. Vis. Sci. 2021;62(8):135.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : To determine if online medical education for ophthalmologists (Opht) could improve their knowledge, competence and confidence to diagnose and treat children with ocular manifestations of nephrotic cystinosis

Methods : Participants completed a 3-item questionnaire plus a confidence assessment before and after watching a 30-minute multispeciality expert roundtable discussion with accompanying slides. A matched pair design was used pre-/post-assessment, with scores compared to assess changes in the proportion of correct responses. A chi-squared test assessed statistical significance at the P <.05 level. Launch Jun 18, 2020; data through Aug 8, 2020. Assessment completers: 239

Results : An average of 44% of Opht responses on pre-assessment were correct, increasing to 66% on post-assessment (p<.001). The activity significantly increased knowledge of ocular manifestations of nephrotic cystinosis in children, including molecular genetics aspects (pre: 28%, post: 47%, p<.001). Diagnostic competence improved, measured in choosing the best diagnostic approach to examine cystine crystal deposition in an applied patient case (pre: 70%, post: 84% p<.001). Treatment competence increased for therapy and application choice for the same case, choosing topical cysteamine eye drops over a systemic dose increase, anti-inflammatory or antibiotic topical treatment (pre: 32%, post: 68%, p<.001). 54% of Opht had a measurable increase in confidence in their ability to treat ocular manifestations of cystinosis.

Conclusions : Nephrotic cystinosis is a rare inherited disease, diagnosis and specific treatment are frequently delayed with a significant impact on overall prognosis.Corneal cystine deposition is visible by slit lamp biomicroscopy and can lead to photophobia, blepharospasm, superficial punctate keratopathy and recurrent corneal erosions.To deplete deposition additional topical cysteamine treatment is required to reach the avascular cornea. Online medical education in the form of an multispeciality expert roundtable discussion with accompanying slides can significantly improve rare disease knowledge and diagnostic, and therapeutic competence and confidence. Additional education is warranted to shorten time to diagnosis for ocular manifestations of nephrotic cystinosis and share data for emerging therapies.

This is a 2021 ARVO Annual Meeting abstract.

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