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Mental health
Protocol
Development of the Tailored Rett Intervention and Assessment Longitudinal (TRIAL) database and the Rett Evaluation of Symptoms and Treatments (REST) Questionnaire
  1. Paramala Santosh1,2,3,
  2. Kate Lievesley1,2,
  3. Federico Fiori1,2,3,
  4. Jatinder Singh1
  1. 1 Department of Child and Adolescent Psychiatry, King’s College London, London, UK
  2. 2 HealthTracker Ltd, Gillingham, Kent, UK
  3. 3 Centre for Interventional Paediatric Psychopharmacology and Rare Diseases, South London and Maudsley NHS Foundation Trust, London, UK
  1. Correspondence to Dr Paramala Santosh; paramala.1.santosh{at}kcl.ac.uk

Abstract

Introduction Rett syndrome (RTT) is a pervasive neurodevelopmental disorder that presents with deficits in brain functioning leading to language and learning regression, characteristic hand stereotypies and developmental delay. Different mutations in the gene implicated in RTT—methyl-CpG-binding protein 2 (MECP2) establishes RTT as a disorder with divergent symptomatology ranging from individuals with severe to milder phenotypes. A reliable and single multidimensional questionnaire is needed that can embrace all symptoms, and the relationships between them, and can map clinically meaningful data to symptomatology across the lifespan in patients with RTT. As part of the HealthTracker-based Tailored Rett Intervention and Assessment Longitudinal (TRIAL) database, the Rett Evaluation of Symptoms and Treatments (REST) Questionnaire will be able to marry with the physiological aspects of the disease obtained using wearable sensor technology, along with genetic and psychosocial data to stratify patients. Taken together, the web-based TRIAL database will empower clinicians and researchers with the confidence to delineate between different aspects of disorder symptomatology to streamline care pathways for individuals or for those patients entering clinical trials. This protocol describes the anticipated development of the REST questionnaire and the TRIAL database which links with the outcomes of the wearable sensor technology, and will serve as a barometer for longitudinal patient monitoring in patients with RTT.

Methods and analysis The US Food and Drug Administration Guidance for Patient-Reported Outcome Measures will be used as a template to inform the methodology of the study. It will follow an iterative framework that will include item/concept identification, item/concept elicitation in parent/carer-mediated focus groups, expert clinician feedback, web-based presentation of questionnaires, initial scale development, instrument refinement and instrument validation.

Ethics and dissemination The study has received favourable opinion from the National Health Service (NHS) Research Ethics Committee (REC): NHS Research Ethics Committee (REC)—London, Bromley Research Ethics Committee (reference: 15/LO/1772).

  • biomarkers
  • health trackerTM
  • questionnaire development and validation
  • rett syndrome
  • tailored rett intervention and assessment longitudinal (TRIAL) database
  • wearable sensor technology

This is an Open Access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/

https://doi.org/10.1136/bmjopen-2016-015342

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    Footnotes

    • Contributors JS drafted, wrote and revised the manuscript and wrote the documentation required for ethical approval of the study. KL provided important intellectual review of the manuscript and reviewed the documentation required for ethical approval of the study. FF reviewed the statistical components and reviewed the manuscript. PS secured funding and conceived the study and revised the manuscript critically for important intellectual content.

    • Funding A part of this research study was funded by Reverse Rett UK (Ref No: PCCTABR) and HealthTracker for significantly subsidising the costs for this study.

    • Competing interests PS is the coinventor of the HealthTracker and is the chief executive officer and shareholder in HealthTracker. FF is a data analyst and KL is a project manager employed by HealthTracker. JS is on the professional advisory board for Reverse Rett UK and acts as a scientific advisor.

    • Ethics approval NHS Research Ethics Committee (REC)—London, Bromley Research Ethics Committee (reference: 15/LO/1772).

    • Provenance and peer review Not commissioned; externally peer reviewed.

    • Correction notice This paper has been amended since it was published Online First. Owing to a scripting error, some of the publisher names in the references were replaced with 'BMJ Publishing Group'. This only affected the full text version, not the PDF. We have since corrected theseerrors and the correct publishers have been inserted into the references.