Change in Drusen volume as a novel clinical trial endpoint for the study of complement inhibition in age-related macular degeneration

Carlos Alexandre De Amorim Garcia Filho, Zohar Yehoshua, Giovanni Gregori, Renata Portella Nunes, Fernando M. Penha, Andrew A. Moshfeghi, Kang Zhang, William Feuer, Philip J. Rosenfeld

Research output: Contribution to journalArticle

43 Scopus citations

Abstract

BACKGROUND AND OBJECTIVE: To evaluate the change in drusen volume following treatment with eculizumab, a systemic inhibitor of complement component 5. PATIENTS AND METHODS: Single-center, prospective, randomized, double-masked clinical trial. Patients were randomized 2:1 to receive intravenous eculizumab or placebo over 26 weeks. Main outcome measure: decrease in drusen volume of at least 50% at 26-week follow-up. RESULTS: Mean drusen cube root volumes were 0.49 mm and 0.47 mm (P = .64) at baseline and 0.51 mm and 0.42 mm (P = .17) at 26 weeks in the eculizumab and placebo groups, respectively. In the placebo group, one eye had a decrease in drusen volume of at least 50% and two eyes developed neovascularization through 26 weeks. CONCLUSION: Systemic complement inhibition with eculizumab did not significantly reduce drusen volume. Drusen growth was dependent on the number of complement at-risk alleles. Future trials should consider the use of a composite clinical trial endpoint in which efficacy is defined by the treatment's ability to prevent drusen growth, neovascularization, and the formation of geographic atrophy over 1 year.

Original languageEnglish (US)
Pages (from-to)18-31
Number of pages14
JournalOphthalmic Surgery Lasers and Imaging Retina
Volume45
Issue number1
DOIs
StatePublished - Jan 1 2014

ASJC Scopus subject areas

  • Surgery
  • Ophthalmology

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