TY - JOUR
T1 - Monthly ranibizumab for choroidal neovascularizations secondary to angioid streaks in pseudoxanthoma elasticum
T2 - A one-year prospective study
AU - Finger, Robert P.
AU - Charbel Issa, Peter
AU - Hendig, Doris
AU - Scholl, Hendrik P.N.
AU - Holz, Frank G.
N1 - Funding Information:
Publication of this article was supported by a University of Bonn BONFOR (Germany) research grant to R.P.F.; the European Commission (EU) FP7, Marie Curie Intra-European Fellowship (237238) to P.C.I.; the Wynn-Gund Translational Research Acceleration Program Enhanced Research and Clinical Training Award, National Neurovision Research Institute (NNRI) – Foundation Fighting Blindness (FFB; NNCD-CL-0310.0049-JHU-WG) to H.P.N.S.; the Macular Degeneration Research Award, American Health Assistance Foundation (AHAF; M2010042) to H.P.N.S., and the European Commission (EU) FP6, Integrated Project “EVI-GENORET” (LSHG-CT-2005-512036) to H.P.N.S. Heidelberg Engineering provided the Spectralis HRA+OCT. Novartis Pharma GmbH provided the test drug and funding for the trial. The sponsors or funding organizations had no role in the design or conduct of this research. The contents of this publication reflect only the author’s views and not the views of the funding organizations. The department receives research support from Novartis Pharma and Heidelberg Engineering. P.C.I. and F.G.H. received lecture fees from Heidelberg Engineering. R.P.F., P.C.I., and F.G.H. received lecture fees from Novartis Pharma. F.G.H. is consultant for Heidelberg Engineering, Optos, Zeiss, Alcon, Pfizer, and Novartis Pharma. Involved in conception and design of the study (P.C.I., R.P.F., H.P.N.S., F.G.H.); data collection (P.C.I., R.P.F., H.P.N.S.); analysis and interpretation (R.P.F.); writing the article (R.P.F.); critical revision of the article (P.C.I., D.H., H.P.N.S., F.G.H.); final approval of the article (all authors); provision of resources (D.H., F.G.H.); statistical expertise (P.C.I., R.P.F.); obtaining funding (D.H., P.C.I., R.P.F., H.P.N.S., F.G.H.); literature search (R.P.F.); and administrative support (D.H., P.C.I., R.P.F., H.P.N.S., F.G.H.). Approval was obtained from the local Institutional Review Board, the European Medicines Agency (EMEA, UK), and the German competent health authority (Paul-Ehrlich Institut, Germany). The principles of the declaration of Helsinki and good clinical practice guidelines were followed. All patients provided written informed consent for study participation. The clinical trial was registered in the ClinicalTrials database ( www.clinicaltrials.gov ; Identifier: NCT00510965 ).
PY - 2011/10
Y1 - 2011/10
N2 - Purpose: To evaluate the efficacy and safety of monthly intravitreal ranibizumab for the treatment of choroidal neovascularizations (CNV) secondary to angioid streaks (AS) in pseudoxanthoma elasticum (PXE). Design: Twelve-month prospective, open-label, uncontrolled, nonrandomized interventional clinical trial. Methods: In 7 patients, 1 eye with an active CNV was injected with 0.5 mg ranibizumab monthly over 1 year. Distance and reading visual acuity, reading speed, angiographic findings, and central retinal thickness (CRT) on optical coherence tomography were assessed at each visit. Central retinal light increment sensitivity (LIS) was assessed by microperimetry at baseline, at 6 months, and 3 to 4 months after the last injection. Results: Best-corrected visual acuity increased significantly from baseline to month 12 (20/63 or 61 ETDRS letters to 20/32 or 73 ETDRS letters; P =.012). The effect was maintained 3 months later (61 ETDRS letters to 72 ETDRS letters; P =.055). Reading acuity and speed could be maintained throughout the study. Central LIS improved (6.6 dB, SD ± 5.9 at baseline to 7.4 dB, SD ± 6.2 at last follow-up; P <.001). Leakage from active CNVs subsided. Mean change in CRT from baseline to month 12 and 15 was -86 μm (P =.074) and -65 μm (P =.182), respectively. No serious adverse events occurred. Conclusions: Efficacy outcomes indicate a beneficial therapeutic effect of intravitreal ranibizumab on central visual function including retinal LIS. Both the functional and morphologic response based on angiographic and OCT findings to ranibizumab treatment implicate an important pathophysiological role of vascular endothelial growth factor in CNVs secondary to AS in PXE. Intravitreal ranibizumab appears to be a safe and efficacious treatment in these patients.
AB - Purpose: To evaluate the efficacy and safety of monthly intravitreal ranibizumab for the treatment of choroidal neovascularizations (CNV) secondary to angioid streaks (AS) in pseudoxanthoma elasticum (PXE). Design: Twelve-month prospective, open-label, uncontrolled, nonrandomized interventional clinical trial. Methods: In 7 patients, 1 eye with an active CNV was injected with 0.5 mg ranibizumab monthly over 1 year. Distance and reading visual acuity, reading speed, angiographic findings, and central retinal thickness (CRT) on optical coherence tomography were assessed at each visit. Central retinal light increment sensitivity (LIS) was assessed by microperimetry at baseline, at 6 months, and 3 to 4 months after the last injection. Results: Best-corrected visual acuity increased significantly from baseline to month 12 (20/63 or 61 ETDRS letters to 20/32 or 73 ETDRS letters; P =.012). The effect was maintained 3 months later (61 ETDRS letters to 72 ETDRS letters; P =.055). Reading acuity and speed could be maintained throughout the study. Central LIS improved (6.6 dB, SD ± 5.9 at baseline to 7.4 dB, SD ± 6.2 at last follow-up; P <.001). Leakage from active CNVs subsided. Mean change in CRT from baseline to month 12 and 15 was -86 μm (P =.074) and -65 μm (P =.182), respectively. No serious adverse events occurred. Conclusions: Efficacy outcomes indicate a beneficial therapeutic effect of intravitreal ranibizumab on central visual function including retinal LIS. Both the functional and morphologic response based on angiographic and OCT findings to ranibizumab treatment implicate an important pathophysiological role of vascular endothelial growth factor in CNVs secondary to AS in PXE. Intravitreal ranibizumab appears to be a safe and efficacious treatment in these patients.
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U2 - 10.1016/j.ajo.2011.03.022
DO - 10.1016/j.ajo.2011.03.022
M3 - Article
C2 - 21704964
AN - SCOPUS:80053313612
SN - 0002-9394
VL - 152
SP - 695
EP - 703
JO - American journal of ophthalmology
JF - American journal of ophthalmology
IS - 4
ER -