PURPOSE. To investigate bilateral symmetry of visual impairment in cone–rod dystrophy (CRD) patients and understand the feasibility of clinical trial designs treating one eye and using the untreated eye as an internal control. METHODS. This was a retrospective study of visual function loss measures in 436 CRD patients followed at the Ophthalmology Department of the Catholic University in Rome. Clinical measures considered were best-corrected visual acuity, focal macular cone electroretinogram (fERG), and Ganzfeld cone-mediated and rod-mediated electroretinograms. Interocular agreement in each of these clinical indexes was assessed by t- and Wilcoxon tests for paired samples, structural (Deming) regression analysis, and intraclass correlation. Baseline and follow-up measures were analyzed. A separate analysis was performed on the subset of 61 CRD patients carrying likely disease-causing mutations in the ABCA4 gene. RESULTS. Statistical tests show a very high degree of bilateral symmetry in the extent and progression of visual impairment in the fellow eyes of CRD patients. CONCLUSIONS. These data contribute to a better understanding of CRDs and support the feasibility of clinical trial designs involving unilateral eye treatment with the use of fellow eye as internal control.

Galli Resta, L., Falsini, B., De Rossi, G., Piccardi, M., Ziccardi, L., Fadda, A., Minnella, A. M., Marangoni, D., Placidi, G., Campagna, F., Abed, E., Bertelli, M., Zuntini, M., Resta, G., Bilateral symmetry of visual function loss in cone–rod dystrophies, <<INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE>>, 2016; 57 (8): 3759-3768. [doi:10.1167/iovs.15-18313] [http://hdl.handle.net/10807/91672]

Bilateral symmetry of visual function loss in cone–rod dystrophies

Falsini, Benedetto
;
De Rossi, Giuseppe;Piccardi, Marco;Ziccardi, Lucia;Minnella, Angelo Maria;Marangoni, Dario;Placidi, Giorgio;Campagna, Francesca;Abed, Edoardo;
2016

Abstract

PURPOSE. To investigate bilateral symmetry of visual impairment in cone–rod dystrophy (CRD) patients and understand the feasibility of clinical trial designs treating one eye and using the untreated eye as an internal control. METHODS. This was a retrospective study of visual function loss measures in 436 CRD patients followed at the Ophthalmology Department of the Catholic University in Rome. Clinical measures considered were best-corrected visual acuity, focal macular cone electroretinogram (fERG), and Ganzfeld cone-mediated and rod-mediated electroretinograms. Interocular agreement in each of these clinical indexes was assessed by t- and Wilcoxon tests for paired samples, structural (Deming) regression analysis, and intraclass correlation. Baseline and follow-up measures were analyzed. A separate analysis was performed on the subset of 61 CRD patients carrying likely disease-causing mutations in the ABCA4 gene. RESULTS. Statistical tests show a very high degree of bilateral symmetry in the extent and progression of visual impairment in the fellow eyes of CRD patients. CONCLUSIONS. These data contribute to a better understanding of CRDs and support the feasibility of clinical trial designs involving unilateral eye treatment with the use of fellow eye as internal control.
Inglese
Galli Resta, L., Falsini, B., De Rossi, G., Piccardi, M., Ziccardi, L., Fadda, A., Minnella, A. M., Marangoni, D., Placidi, G., Campagna, F., Abed, E., Bertelli, M., Zuntini, M., Resta, G., Bilateral symmetry of visual function loss in cone–rod dystrophies, <<INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE>>, 2016; 57 (8): 3759-3768. [doi:10.1167/iovs.15-18313] [http://hdl.handle.net/10807/91672]
File in questo prodotto:
Non ci sono file associati a questo prodotto.

I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificativo per citare o creare un link a questo documento: http://hdl.handle.net/10807/91672
Citazioni
  • ???jsp.display-item.citation.pmc??? 5
  • Scopus 8
  • ???jsp.display-item.citation.isi??? 8
social impact