Background To reduce treatment burden and optimise patient outcomes in diabetic macular oedema, w... more Background To reduce treatment burden and optimise patient outcomes in diabetic macular oedema, we present 1-year results from two phase 3 trials of faricimab, a novel angiopoietin-2 and vascular endothelial growth factor-A bispecific antibody. Methods YOSEMITE and RHINE were randomised, double-masked, non-inferiority trials across 353 sites worldwide. Adults with vision loss due to centre-involving diabetic macular oedema were randomly assigned (1:1:1) to intravitreal faricimab 6•0 mg every 8 weeks, faricimab 6•0 mg per personalised treatment interval (PTI), or aflibercept 2•0 mg every 8 weeks up to week 100. PTI dosing intervals were extended, maintained, or reduced (every 4 weeks up to every 16 weeks) based on disease activity at active dosing visits. The primary endpoint was mean change in best-corrected visual acuity at 1 year, averaged over weeks 48, 52, and 56. Efficacy analyses included the intention-to-treat population (non-inferiority margin 4 Early Treatment Diabetic Retinopathy Study [ETDRS] letters); safety analyses included patients with at least one dose of study treatment. These trials are registered with ClinicalTrials.gov (YOSEMITE NCT03622580 and RHINE NCT03622593). Findings 3247 patients were screened for eligibility in YOSEMITE (n=1532) and RHINE (n=1715). After exclusions, 940 patients were enrolled into YOSEMITE between
NEURO-ONCOLOGY • JUNE 2018 mutation and recapitulation of her germline APC frameshift variant. Wh... more NEURO-ONCOLOGY • JUNE 2018 mutation and recapitulation of her germline APC frameshift variant. Whole transcriptome analysis of tumor RNA revealed overexpression of multiple Wnt/β-catenin and SHH pathway genes. This case represents the first FAPassociated craniopharyngioma in childhood and the first characterized with somatic and germline exome sequencing, expanding our understanding of the molecular underpinnings driving tumorigenesis in this unique patient.
JD designed the study and clinically assessed the patients. EL, ML, XP, EH, and MG did the labora... more JD designed the study and clinically assessed the patients. EL, ML, XP, EH, and MG did the laboratory studies and prepared the figures. EL, SJM, RB-G, and JD were involved in study design, data analysis, and writing of the report. Conflicts of interest RC has received honoraria from Boehringer-Ingelheim and Orion Pharma for projects unrelated to the current study. SJM has received reimbursement for travel and accommodation expenses as well as funding support from Pfizer. JD has received royalties from a patent related to Ma2 autoantibody test and has filed patent applications for NMDA and GABA B receptor autoantibody tests. JD has received funding from Euroimmun for projects unrelated to the current study. All other authors have no conflicts of interest.
Background To reduce treatment burden and optimise patient outcomes in diabetic macular oedema, w... more Background To reduce treatment burden and optimise patient outcomes in diabetic macular oedema, we present 1-year results from two phase 3 trials of faricimab, a novel angiopoietin-2 and vascular endothelial growth factor-A bispecific antibody. Methods YOSEMITE and RHINE were randomised, double-masked, non-inferiority trials across 353 sites worldwide. Adults with vision loss due to centre-involving diabetic macular oedema were randomly assigned (1:1:1) to intravitreal faricimab 6•0 mg every 8 weeks, faricimab 6•0 mg per personalised treatment interval (PTI), or aflibercept 2•0 mg every 8 weeks up to week 100. PTI dosing intervals were extended, maintained, or reduced (every 4 weeks up to every 16 weeks) based on disease activity at active dosing visits. The primary endpoint was mean change in best-corrected visual acuity at 1 year, averaged over weeks 48, 52, and 56. Efficacy analyses included the intention-to-treat population (non-inferiority margin 4 Early Treatment Diabetic Retinopathy Study [ETDRS] letters); safety analyses included patients with at least one dose of study treatment. These trials are registered with ClinicalTrials.gov (YOSEMITE NCT03622580 and RHINE NCT03622593). Findings 3247 patients were screened for eligibility in YOSEMITE (n=1532) and RHINE (n=1715). After exclusions, 940 patients were enrolled into YOSEMITE between
NEURO-ONCOLOGY • JUNE 2018 mutation and recapitulation of her germline APC frameshift variant. Wh... more NEURO-ONCOLOGY • JUNE 2018 mutation and recapitulation of her germline APC frameshift variant. Whole transcriptome analysis of tumor RNA revealed overexpression of multiple Wnt/β-catenin and SHH pathway genes. This case represents the first FAPassociated craniopharyngioma in childhood and the first characterized with somatic and germline exome sequencing, expanding our understanding of the molecular underpinnings driving tumorigenesis in this unique patient.
JD designed the study and clinically assessed the patients. EL, ML, XP, EH, and MG did the labora... more JD designed the study and clinically assessed the patients. EL, ML, XP, EH, and MG did the laboratory studies and prepared the figures. EL, SJM, RB-G, and JD were involved in study design, data analysis, and writing of the report. Conflicts of interest RC has received honoraria from Boehringer-Ingelheim and Orion Pharma for projects unrelated to the current study. SJM has received reimbursement for travel and accommodation expenses as well as funding support from Pfizer. JD has received royalties from a patent related to Ma2 autoantibody test and has filed patent applications for NMDA and GABA B receptor autoantibody tests. JD has received funding from Euroimmun for projects unrelated to the current study. All other authors have no conflicts of interest.
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Papers by Miguel Guzman