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Crizanlizumab for retinal vasculopathy with cerebral leukoencephalopathy in a phase II clinical study.
J Clin Invest
December 2024
John F. Hardesty, MD Department of Ophthalmology and Visual Sciences, Washington University School of Medicine, St. Louis, Missouri, USA.
Crizanlizumab for retinal vasculopathy with cerebral leukoencephalopathy in a phase II clinical study.
J Clin Invest
May 2024
John F. Hardesty, MD Department of Ophthalmology and Visual Sciences, and.
BackgroundRetinal vasculopathy with cerebral leukoencephalopathy and systemic manifestations (RVCL-S) is a rare, autosomal dominant, universally fatal disease without effective treatment options. This study explores the safety and preliminary efficacy of crizanlizumab, a humanized monoclonal antibody against P-selectin approved for the prevention of sickle cell crises, in slowing retinal nonperfusion and preserving vision in patients with RVCL-S.METHODSEleven patients with RVCL-S with confirmed exonuclease 3 prime repair exonuclease 1 (TREX1) mutations received monthly crizanlizumab infusions over 2 years.
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