Development of a Retinopathy of Prematurity Activity Scale and Clinical Outcome Measures for Use in Clinical Trials

Lois E.H. Smith, Ann Hellström, Andreas Stahl, Alistair Fielder, Wiley Chambers, Jane Moseley, Cynthia Toth, David K. Wallace, Brian A. Darlow, Jacob V. Aranda, Boubou Hallberg, Jonathan M. Davis

Research output: Contribution to journalArticle

1 Citation (Scopus)

Abstract

Importance: To facilitate drug and device development for neonates, the International Neonatal Consortium brings together key stakeholders, including pharmaceutical companies, practitioners, regulators, funding agencies, scientists, and families, to address the need for objective, standardized clinical trial outcome measurements to fulfill regulatory requirements. Retinopathy of prematurity (ROP) is a disease that affects preterm neonates. The current International Classification of Retinopathy of Prematurity does not take into account all of the characteristics of ROP and does not adequately discriminate small changes in disease after treatment. These factors are critical for evaluating outcomes in clinical trials. Observations: There is need for an updated ROP acute disease activity and structure scale as well as end-stage structure and ophthalmologic outcome measures designed for use at different ages. The scale and measures, based on current diagnostic methods and treatments, could be used as a guideline for clinical intervention trials. The scale is intended to be validated against retrospective data and revised for use in future trials. An iterative revision process can be accomplished if new measures are added to clinical trials and evaluated at the end of each trial for prognostic value. The new measures would then be incorporated into a new version of the activity scale and the outcome measures revised. Conclusions and Relevance: An ROP activity scale and outcome measures to obtain the most robust and discriminatory data for clinical trials are needed. The scales should be dynamic and modified as knowledge and imaging modalities improve and then validated using data from well-documented clinical trials. This approach is relevant to improving clinical trial data quality.

Original languageEnglish (US)
JournalJAMA Ophthalmology
DOIs
StateAccepted/In press - Jan 1 2018

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Retinopathy of Prematurity
Outcome Assessment (Health Care)
Clinical Trials
Acute Disease
Pharmaceutical Preparations
Guidelines
Equipment and Supplies

ASJC Scopus subject areas

  • Ophthalmology

Cite this

Smith, L. E. H., Hellström, A., Stahl, A., Fielder, A., Chambers, W., Moseley, J., ... Davis, J. M. (Accepted/In press). Development of a Retinopathy of Prematurity Activity Scale and Clinical Outcome Measures for Use in Clinical Trials. JAMA Ophthalmology. https://doi.org/10.1001/jamaophthalmol.2018.5984

Development of a Retinopathy of Prematurity Activity Scale and Clinical Outcome Measures for Use in Clinical Trials. / Smith, Lois E.H.; Hellström, Ann; Stahl, Andreas; Fielder, Alistair; Chambers, Wiley; Moseley, Jane; Toth, Cynthia; Wallace, David K.; Darlow, Brian A.; Aranda, Jacob V.; Hallberg, Boubou; Davis, Jonathan M.

In: JAMA Ophthalmology, 01.01.2018.

Research output: Contribution to journalArticle

Smith, LEH, Hellström, A, Stahl, A, Fielder, A, Chambers, W, Moseley, J, Toth, C, Wallace, DK, Darlow, BA, Aranda, JV, Hallberg, B & Davis, JM 2018, 'Development of a Retinopathy of Prematurity Activity Scale and Clinical Outcome Measures for Use in Clinical Trials', JAMA Ophthalmology. https://doi.org/10.1001/jamaophthalmol.2018.5984
Smith, Lois E.H. ; Hellström, Ann ; Stahl, Andreas ; Fielder, Alistair ; Chambers, Wiley ; Moseley, Jane ; Toth, Cynthia ; Wallace, David K. ; Darlow, Brian A. ; Aranda, Jacob V. ; Hallberg, Boubou ; Davis, Jonathan M. / Development of a Retinopathy of Prematurity Activity Scale and Clinical Outcome Measures for Use in Clinical Trials. In: JAMA Ophthalmology. 2018.
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