Change in Drusen Volume as a Novel Clinical Trial Endpoint for the Study of Complement Inhibition in Age-related Macular Degeneration

Change in Drusen Volume as a Novel Clinical Trial Endpoint for the Study of Complement Inhibition in Age-related Macular Degeneration

Author Garcia Filho, Carlos Alexandre de Amorim Autor UNIFESP Google Scholar
Yehoshua, Zohar Google Scholar
Gregori, Giovanni Google Scholar
Nunes, Renata Portella Autor UNIFESP Google Scholar
Penha, Fernando Marcondes Autor UNIFESP Google Scholar
Moshfeghi, Andrew A. Google Scholar
Zhang, Kang Google Scholar
Feuer, William Google Scholar
Rosenfeld, Philip J. Google Scholar
Institution Univ Miami
Universidade Federal de São Paulo (UNIFESP)
Univ Calif San Diego
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.
Language English
Sponsor Alexion Pharmaceuticals
Macula Vision Research Foundation
Carl Zeiss Meditec
Research to Prevent Blindness
NEI core center grant
Department of Defense (DOD)
Jerome A. Yavitz Charitable Foundation
Emma Clyde Hodge Memorial Foundation
Florman Family Foundation
Gemcon Family Foundation
Grant number NEI core center grant: P30 EY014801
Department of Defense (DOD): W81XWH-09-1-0675
Date 2014-01-01
Published in Ophthalmic Surgery Lasers & Imaging. Thorofare: Slack Inc, v. 45, n. 1, p. 18-31, 2014.
ISSN 1542-8877 (Sherpa/Romeo, impact factor)
Publisher Slack Inc
Extent 18-31
Origin http://dx.doi.org/10.3928/23258160-20131217-01
Access rights Open access Open Access
Type Article
Web of Science ID WOS:000333812200003
URI http://repositorio.unifesp.br/handle/11600/37187

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