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Assessment of a Clinical Trial Metric for Rett Syndrome: Critical Analysis of the Rett Syndrome Behavioural Questionnaire.

التفاصيل البيبلوغرافية
العنوان: Assessment of a Clinical Trial Metric for Rett Syndrome: Critical Analysis of the Rett Syndrome Behavioural Questionnaire.
المؤلفون: Hou W; Stony Brook University Medical Center, Stony Brook, New York., Bhattacharya U; University of Georgia, Athens, Georgia., Pradana WA; University of Georgia, Athens, Georgia., Tarquinio DC; Center for Rare Neurological Diseases, Norcross, Georgia. Electronic address: daniel@rareneuro.com.
المصدر: Pediatric neurology [Pediatr Neurol] 2020 Jun; Vol. 107, pp. 48-56. Date of Electronic Publication: 2020 Feb 04.
نوع المنشور: Journal Article; Research Support, Non-U.S. Gov't
اللغة: English
بيانات الدورية: Publisher: Elsevier Science Publishing Country of Publication: United States NLM ID: 8508183 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1873-5150 (Electronic) Linking ISSN: 08878994 NLM ISO Abbreviation: Pediatr Neurol Subsets: MEDLINE
أسماء مطبوعة: Publication: New York Ny : Elsevier Science Publishing
Original Publication: Chippewa Falls, Wis. : Professional Publications, [c1985-
مواضيع طبية MeSH: Behavioral Symptoms/*diagnosis , Clinical Trials as Topic/*standards , Outcome Assessment, Health Care/*standards , Psychiatric Status Rating Scales/*standards , Psychometrics/*standards , Rett Syndrome/*diagnosis, Adolescent ; Adult ; Behavioral Symptoms/etiology ; Biomarkers ; Child ; Child, Preschool ; Cohort Studies ; Female ; Humans ; Rett Syndrome/complications ; Surveys and Questionnaires ; Young Adult
مستخلص: Background: Rett syndrome is a neurodevelopmental disorder with potential for improvement through novel targeted therapeutics. Reliable outcome measures are critical to the development of treatments. We examined the merits and flaws of the Rett Syndrome Behavioural Questionnaire, an outcome measure for clinical trials.
Methods: The Rett Syndrome Behavioural Questionnaire was administered alongside other clinical scales in three cohorts, an online survey, a clinic-based study, and the screening period for a clinical trial. Data were collected from individuals with Rett syndrome and related disorders at three time points, separated by a minimum of one week and a maximum of two months. We hypothesized that for clinical trial use, little change should occur among visits. Distribution statistics, internal consistency, intraclass correlation coefficient, percent agreement, and Cohen's kappa were examined.
Results: Among 149 with classic Rett syndrome, the Rett Syndrome Behavioural Questionnaire was completed 377 times. Median total score was 33, ranging from 3 to 73. Of the 51 items tested in the original Rett Syndrome Behavioural Questionnaire study, 24 exhibited either floor or ceiling effects. Friedman's analysis of variance revealed significant difference among visits (P = 0.024), and graphical analysis using Bland-Altman plots demonstrated systematic positive bias with a 95% confidence interval including up to 12.9 points higher to 15.7 points lower at retest. Median agreement measured by kappa was 0.53 for retest at visit 2 and 0.49 for retest at visit 3.
Conclusions: The Rett Syndrome Behavioural Questionnaire did not achieve acceptable standards as an outcome assessment for clinical trials in Rett syndrome.
(Copyright © 2020 Elsevier Inc. All rights reserved.)
التعليقات: Comment in: Pediatr Neurol. 2020 Oct;111:88. (PMID: 32921537)
Comment in: Pediatr Neurol. 2020 Oct;111:4. (PMID: 32951657)
فهرسة مساهمة: Keywords: Biomarker; Outcome measure; Response to treatment; Rett syndrome
سلسلة جزيئية: ClinicalTrials.gov NCT02696044
المشرفين على المادة: 0 (Biomarkers)
تواريخ الأحداث: Date Created: 20200314 Date Completed: 20210423 Latest Revision: 20210423
رمز التحديث: 20240829
DOI: 10.1016/j.pediatrneurol.2020.01.009
PMID: 32165033
قاعدة البيانات: MEDLINE
الوصف
تدمد:1873-5150
DOI:10.1016/j.pediatrneurol.2020.01.009