Literature DB >> 28196731

Natural History of Conversion of Leber's Hereditary Optic Neuropathy: A Prospective Case Series.

Tiffany Jean Hwang1, Rustum Karanjia2, Milton Nunes Moraes-Filho3, Jesse Gale4, Jeffrey Show Tran1, Edward R Chu1, Solange R Salomao5, Adriana Berezovsky5, Rubens Belfort6, Milton Nunes Moraes3, Federico Sadun7, Anna Maria DeNegri8, Chiara La Morgia9, Piero Barboni10, Carolina do V F Ramos11, Carlos Filipe Chicani1, Peter A Quiros12, Valerio Carelli9, Alfredo A Sadun13.   

Abstract

PURPOSE: To illustrate the natural history of Leber's hereditary optic neuropathy (LHON).
DESIGN: Prospective observational case series. PARTICIPANTS: The Soave-Brazil pedigree of m.11778G>A/ND4 mitochondrial DNA LHON mutation.
METHODS: A prospectively acquired database of the Soave-Brazil pedigree was reviewed. Data from 285 individuals were included in the database over a 15-year period. The pedigree was reviewed for unaffected mutation carriers who converted to affected status, 6 patients with LHON were identified. The medical records were reviewed 1 year preconversion to 1 year postconversion for visual acuity (logarithm of the minimum angle of resolution [logMAR]), Humphrey Visual Field (HVF) mean deviation (MD), and retinal nerve fiber layer (RNFL) thickness, as measured by Cirrus (Carl Zeiss, Oberkochen, Germany) optic coherence tomography (OCT). The RNFL thickness values were normalized for age. Visual acuity, HVF, and processed RNFL data from each of the 12 eyes were then sorted into 2-month time periods relative to the date of conversion, within which they were averaged. MAIN OUTCOME MEASURES: The main outcome measures were visual acuity, HVF MD, and RNFL thickness.
RESULTS: Decreased visual acuity preceded conversion by up to 2 months and then declined up to 8 months postconversion. Decrease in HVF MD occurred at least 4 months preceding conversion, after which values decreased until plateau at 6 to 8 months. Average RNFL thickness was above normal baseline thickness in all 4 quadrants as measured by OCT at the time of conversion. Increase in RNFL thickness preceded conversion as early as 4 to 6 months, peaked at conversion, and decreased until individual plateaus. The temporal quadrant was first to be involved, then the inferior and superior quadrants, and the nasal quadrant showed the latest and least changes.
CONCLUSIONS: Subclinical changes preceded the date of conversion and may reflect the complicated nature of identifying the date of conversion in LHON. Early increases in RNFL preceding conversion suggest that structural changes precede clinically significant vision loss. Asynchronous quadrant involvement supports a previously published mathematical model. The natural history of LHON is not a subacute process, as previously believed, but progresses more slowly, taking up to 8 months to plateau.
Copyright © 2017 American Academy of Ophthalmology. Published by Elsevier Inc. All rights reserved.

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Year:  2017        PMID: 28196731     DOI: 10.1016/j.ophtha.2017.01.002

Source DB:  PubMed          Journal:  Ophthalmology        ISSN: 0161-6420            Impact factor:   12.079


  14 in total

1.  14th EUNOS Congress: PORTO, PORTUGAL, 16-19 JUNE 2019.

Authors: 
Journal:  Neuroophthalmology       Date:  2019-06-07

Review 2.  Pathophysiology of Conversion to Symptomatic Leber Hereditary Optic Neuropathy and Therapeutic Implications: a Review.

Authors:  Alvaro J Mejia-Vergara; Nicolas Seleme; Alfredo A Sadun; Rustum Karanjia
Journal:  Curr Neurol Neurosci Rep       Date:  2020-04-15       Impact factor: 5.081

3.  Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A ND4 Mutation: Systematic Review and Indirect Comparison.

Authors:  Nancy J Newman; Patrick Yu-Wai-Man; Valerio Carelli; Valerie Biousse; Mark L Moster; Catherine Vignal-Clermont; Robert C Sergott; Thomas Klopstock; Alfredo A Sadun; Jean-François Girmens; Chiara La Morgia; Adam A DeBusk; Neringa Jurkute; Claudia Priglinger; Rustum Karanjia; Constant Josse; Julie Salzmann; François Montestruc; Michel Roux; Magali Taiel; José-Alain Sahel
Journal:  Front Neurol       Date:  2021-05-24       Impact factor: 4.003

Review 4.  Leber hereditary optic neuropathy: bridging the translational gap.

Authors:  Neringa Jurkute; Patrick Yu-Wai-Man
Journal:  Curr Opin Ophthalmol       Date:  2017-09       Impact factor: 3.761

5.  Optical coherence tomography angiography changes in radial peripapillary capillaries in Leber hereditary optic neuropathy.

Authors:  Mitsuhiro Matsuzaki; Yasuhiko Hirami; Hirofumi Uyama; Yasuo Kurimoto
Journal:  Am J Ophthalmol Case Rep       Date:  2018-01-04

6.  Ocular Involvement in Friedreich Ataxia Patients and its Relationship with Neurological Disability, a Follow-up Study.

Authors:  Pilar Rojas; Ana I Ramírez; Rosa de Hoz; Manuel Cadena; Antonio Ferreras; Blanca Monsalve; Elena Salobrar-García; José L Muñoz-Blanco; José L Urcelay-Segura; Juan J Salazar; José M Ramírez
Journal:  Diagnostics (Basel)       Date:  2020-01-29

7.  Impaired Ganglion Cell Function Objectively Assessed by the Photopic Negative Response in Affected and Asymptomatic Members From Brazilian Families With Leber's Hereditary Optic Neuropathy.

Authors:  Gabriel Izan Santos Botelho; Solange Rios Salomão; Célia Harumi Tengan; Rustum Karanjia; Felipo Victor Moura; Daniel Martins Rocha; Paula Baptista Eliseo da Silva; Arthur Gustavo Fernandes; Sung Eun Song Watanabe; Paula Yuri Sacai; Rubens Belfort; Valerio Carelli; Alfredo Arrigo Sadun; Adriana Berezovsky
Journal:  Front Neurol       Date:  2021-01-18       Impact factor: 4.003

Review 8.  Principles of OCTA and Applications in Clinical Neurology.

Authors:  Adam Wylęgała
Journal:  Curr Neurol Neurosci Rep       Date:  2018-10-18       Impact factor: 5.081

Review 9.  Therapeutic Options in Hereditary Optic Neuropathies.

Authors:  Giulia Amore; Martina Romagnoli; Michele Carbonelli; Piero Barboni; Valerio Carelli; Chiara La Morgia
Journal:  Drugs       Date:  2021-01       Impact factor: 9.546

10.  Cross-Sectional Analysis of Baseline Visual Parameters in Subjects Recruited Into the RESCUE and REVERSE ND4-LHON Gene Therapy Studies.

Authors:  Mark L Moster; Robert C Sergott; Nancy J Newman; Patrick Yu-Wai-Man; Valerio Carelli; Molly Scannell Bryan; Gerard Smits; Valérie Biousse; Catherine Vignal-Clermont; Thomas Klopstock; Alfredo A Sadun; Adam A DeBusk; Michele Carbonelli; Rabih Hage; Siegfried Priglinger; Rustum Karanjia; Laure Blouin; Magali Taiel; Barrett Katz; José Alain Sahel
Journal:  J Neuroophthalmol       Date:  2021-09-01       Impact factor: 4.415

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