Digital Technology in Somatic and Gene Therapy Trials of Pediatric Patients With Ocular Diseases: Protocol for a Scoping Review (Preprint)
Author(s)
Type
Working Paper
Abstract
Background:
Pharmacogenomics suggests that diseases with similar symptomatic presentations often have varying genetic causes, affecting an individual patient’s response to a specific therapeutic strategy. Gene therapies and somatic cell therapies offer unique therapeutic pathways for ocular diseases and often depend on increased understanding of the genotype-phenotype relationship in disease presentation and progression. While demand for personalised medicine is increasing and the required molecular tools are available, its adoption within paediatric ophthalmology remains to be maximised in the post-genomic era. To address the individual hurdles encountered in the field of genomic-related clinical trials and facilitate the uptake of personalised medicine, we propose to conduct a review that will examine and identify the digital technologies used to facilitate data analysis in somatic and gene therapy trials in paediatric patients with ocular diseases.
Objective:
To present an outline of HIT/ICT resources used in somatic and gene therapy clinical trials in children with ocular diseases. This review will enable authors to identify challenges and provide recommendations facilitating the uptake of genetic and somatic therapies as therapeutic tools in paediatric ophthalmology. The review will also determine whether conducting a systematic review will be beneficial.
Methods:
The review will be guided by Arksey/O’Malley’s and Levac’s methodological frameworks. Following the development of Medical Subject Headings (MeSH)/subject headings and keywords, a systematic search of MEDLINE/PubMed, Embase and Scopus will be conducted. Two suitably qualified independent reviewers will determine study eligibility. Following identification of studies, data will be extracted and analysed.
Results:
Database searches will be initiated in September 2018. We expect to complete the review in December 2018.
Conclusions:
Based on review findings, the authors will summarise methods used for facilitating IT integration in personalised medicine. Additionally, it will identify further research gaps and determine whether conduction of further reviews will be beneficial. Clinical Trial: Because this is a scoping review protocol, it is ineligible for registration with PROSPERO.
Pharmacogenomics suggests that diseases with similar symptomatic presentations often have varying genetic causes, affecting an individual patient’s response to a specific therapeutic strategy. Gene therapies and somatic cell therapies offer unique therapeutic pathways for ocular diseases and often depend on increased understanding of the genotype-phenotype relationship in disease presentation and progression. While demand for personalised medicine is increasing and the required molecular tools are available, its adoption within paediatric ophthalmology remains to be maximised in the post-genomic era. To address the individual hurdles encountered in the field of genomic-related clinical trials and facilitate the uptake of personalised medicine, we propose to conduct a review that will examine and identify the digital technologies used to facilitate data analysis in somatic and gene therapy trials in paediatric patients with ocular diseases.
Objective:
To present an outline of HIT/ICT resources used in somatic and gene therapy clinical trials in children with ocular diseases. This review will enable authors to identify challenges and provide recommendations facilitating the uptake of genetic and somatic therapies as therapeutic tools in paediatric ophthalmology. The review will also determine whether conducting a systematic review will be beneficial.
Methods:
The review will be guided by Arksey/O’Malley’s and Levac’s methodological frameworks. Following the development of Medical Subject Headings (MeSH)/subject headings and keywords, a systematic search of MEDLINE/PubMed, Embase and Scopus will be conducted. Two suitably qualified independent reviewers will determine study eligibility. Following identification of studies, data will be extracted and analysed.
Results:
Database searches will be initiated in September 2018. We expect to complete the review in December 2018.
Conclusions:
Based on review findings, the authors will summarise methods used for facilitating IT integration in personalised medicine. Additionally, it will identify further research gaps and determine whether conduction of further reviews will be beneficial. Clinical Trial: Because this is a scoping review protocol, it is ineligible for registration with PROSPERO.
Date Issued
2018-04-05
Citation
2018
Publisher
JMIR Publications Inc.
Copyright Statement
© The authors. All rights reserved. This is a privileged document currently under peer-review/community review (or an accepted/rejected manuscript). Authors have provided JMIR Publications with an exclusive license to publish this preprint on it's website for review and ahead-of-print citation purposes only. While the final peer-reviewed paper may be licensed under a cc-by license on publication, at this stage authors and publisher expressively prohibit redistribution of this draft paper other than for review purposes.