Original research: Evaluation of the Retinopathy of Prematurity Activity Scale (ROP-ActS) in a randomised controlled trial aiming for prevention of severe ROP: a substudy of the Mega Donna Mega trial

BMJ Open Ophthalmology

article

Aldina Pivodic¹  Helena Johansson²  Lois EH Smith⁴  Chatarina Löfqvist¹  Kerstin Albertsson-Wikland⁶  Staffan Nilsson⁷  Ann Hellström¹ 

[1] Department of Clinical Neuroscience , Institute of Neuroscience and Physiology, Sahlgrenska Academy, University of Gothenburg;Mary MacKillop Institute for Health Research, Australian Catholic University;Sahlgrenska Osteoporosis Centre, Institute of Medicine, University of Gothenburg;Department of Ophthalmology , Boston Children's Hospital, Harvard Medical School;Institute of Health Care Science, Sahlgrenska Academy, University of Gothenburg;Department of Physiology/Endocrinology , Institute of Neuroscience and Physiology, Sahlgrenska Academy, University of Gothenburg;Department of Mathematical Sciences , Chalmers University of Technology;Department of Laboratory Medicine , Institute of Biomedicine, Sahlgrenska Academy, University of Gothenburg

关键词: Treatment other;    Vision;    Retina;    Diagnostic tests/Investigation;   

DOI  :  10.1136/bmjophth-2021-000923

学科分类：工业工程学

来源: BMJ Publishing Group

【 摘 要 】

Objective The current grading of retinopathy of prematurity (ROP) does not sufficiently discriminate disease severity for evaluation of trial interventions. The published ROP Activity Scales (original: ROP-ActS and modified: mROP-ActS), describing increasing severity of ROP, versus the categorical variables severe ROP, stage, zone and plus disease were evaluated as discriminators of the effect of an ROP preventive treatment.Methods and analysis The Mega Donna Mega trial investigated ROP in infants born <28-week gestational age (GA), randomised to arachidonic acid (AA) and docosahexaenoic acid (DHA) supplementation or no supplementation. Of 207 infants, 86% with finalised ROP screening were included in this substudy. ROP-ActS versus standard variables were evaluated using Fisher’s non-parametric permutation test, multivariable logistic and linear regression and marginal fractional response models.Results The AA:DHA group (n=84) and the control group (n=93) were well balanced. The maximum ROP-ActS measurement was numerically but not significantly lower in the AA:DHA group (mean: 4.0 (95% CI 2.9 to 5.0)) versus the control group (mean: 5.3 (95% CI 4.1 to 6.4)), p=0.11. In infants with any ROP, the corresponding scale measurements were 6.8 (95% CI 5.4 to 8.2) and 8.7 (95% CI 7.5 to 10.0), p=0.039. Longitudinal profiles of the scale were visually distinguished for the categories of sex and GA for the intervention versus control.Conclusions The preventive effect of AA:DHA supplementation versus no supplementation was better discriminated by the trial’s primary outcome, severe ROP, than by ROP-ActS. The sensitivity and the linear qualities of ROP-ActS require further validations on large data sets and perhaps modifications.Trial registration number NCT03201588.

【 授权许可】

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