Development of an adapted Clinical Global Impression scale for use in Angelman syndrome.
Journal
Journal of neurodevelopmental disorders
ISSN: 1866-1955
Titre abrégé: J Neurodev Disord
Pays: England
ID NLM: 101483832
Informations de publication
Date de publication:
04 01 2021
04 01 2021
Historique:
received:
07
07
2020
accepted:
01
12
2020
entrez:
5
1
2021
pubmed:
6
1
2021
medline:
27
8
2021
Statut:
epublish
Résumé
The Clinical Global Impression-Severity (CGI-S) and CGI-Improvement (CGI-I) scales are widely accepted tools that measure overall disease severity and change, synthesizing the clinician's impression of the global state of an individual. Frequently employed in clinical trials for neuropsychiatric disorders, the CGI scales are typically used in conjunction with disease-specific rating scales. When no disease-specific rating scale is available, the CGI scales can be adapted to reflect the specific symptom domains that are relevant to the disorder. Angelman syndrome (AS) is a rare, clinically heterogeneous condition for which there is no disease-specific rating scale. This paper describes efforts to develop standardized, adapted CGI scales specific to AS for use in clinical trials. In order to develop adapted CGI scales specific to AS, we (1) reviewed literature and interviewed caregivers and clinicians to determine the most impactful symptoms, (2) engaged expert panels to define and operationalize the symptom domains identified, (3) developed detailed rating anchors for each domain and for global severity and improvement ratings, (4) reviewed the anchors with expert clinicians and established minimally clinically meaningful change for each symptom domain, and (5) generated mock patient vignettes to test the reliability of the resulting scales and to standardize rater training. This systematic approach to developing, validating, and training raters on a standardized, adapted CGI scale specifically for AS is described herein. The resulting CGI-S/I-AS scales capture six critical domains (behavior, gross and fine motor function, expressive and receptive communication, and sleep) defined by caregivers and expert clinicians as the most challenging for patients with AS and their families. Rigorous training and careful calibration for clinicians will allow the CGI-S/-I-AS scales to be reliable in the context of randomized controlled trials. The CGI-S/-I-AS scales are being utilized in a Phase 3 trial of gaboxadol for the treatment of AS.
Sections du résumé
BACKGROUND
The Clinical Global Impression-Severity (CGI-S) and CGI-Improvement (CGI-I) scales are widely accepted tools that measure overall disease severity and change, synthesizing the clinician's impression of the global state of an individual. Frequently employed in clinical trials for neuropsychiatric disorders, the CGI scales are typically used in conjunction with disease-specific rating scales. When no disease-specific rating scale is available, the CGI scales can be adapted to reflect the specific symptom domains that are relevant to the disorder. Angelman syndrome (AS) is a rare, clinically heterogeneous condition for which there is no disease-specific rating scale. This paper describes efforts to develop standardized, adapted CGI scales specific to AS for use in clinical trials.
METHODS
In order to develop adapted CGI scales specific to AS, we (1) reviewed literature and interviewed caregivers and clinicians to determine the most impactful symptoms, (2) engaged expert panels to define and operationalize the symptom domains identified, (3) developed detailed rating anchors for each domain and for global severity and improvement ratings, (4) reviewed the anchors with expert clinicians and established minimally clinically meaningful change for each symptom domain, and (5) generated mock patient vignettes to test the reliability of the resulting scales and to standardize rater training. This systematic approach to developing, validating, and training raters on a standardized, adapted CGI scale specifically for AS is described herein.
RESULTS
The resulting CGI-S/I-AS scales capture six critical domains (behavior, gross and fine motor function, expressive and receptive communication, and sleep) defined by caregivers and expert clinicians as the most challenging for patients with AS and their families.
CONCLUSIONS
Rigorous training and careful calibration for clinicians will allow the CGI-S/-I-AS scales to be reliable in the context of randomized controlled trials. The CGI-S/-I-AS scales are being utilized in a Phase 3 trial of gaboxadol for the treatment of AS.
Identifiants
pubmed: 33397286
doi: 10.1186/s11689-020-09349-8
pii: 10.1186/s11689-020-09349-8
pmc: PMC7784030
doi:
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
3Subventions
Organisme : NICHD NIH HHS
ID : P50 HD103537
Pays : United States
Organisme : NCATS NIH HHS
ID : UL1 TR001863
Pays : United States
Références
Int Clin Psychopharmacol. 1997 Nov;12(6):333-8
pubmed: 9547135
J Chiropr Med. 2017 Dec;16(4):346
pubmed: 29276468
Child Psychiatry Hum Dev. 2020 Sep 2;:
pubmed: 32880036
Acta Psychiatr Scand. 2000 Dec;102(6):461-5
pubmed: 11142437
Psychiatry (Edgmont). 2007 Jul;4(7):28-37
pubmed: 20526405
J Nerv Ment Dis. 2006 Nov;194(11):845-52
pubmed: 17102709
Int Psychogeriatr. 1996 Summer;8(2):277-88; discussion 288-90
pubmed: 8994897
J Clin Epidemiol. 2008 Feb;61(2):102-9
pubmed: 18177782
Pediatrics. 2017 Feb;139(2):
pubmed: 28100688
Mol Psychiatry. 2020 Aug 13;:
pubmed: 32792659
Behav Sci (Basel). 2017 Jun 27;7(3):
pubmed: 28653978
Psychol Bull. 1979 Mar;86(2):420-8
pubmed: 18839484
Proc Natl Acad Sci U S A. 2014 Oct 28;111(43):15550-5
pubmed: 25313065
Patient. 2019 Feb;12(1):97-112
pubmed: 29987743
Orphanet J Rare Dis. 2017 Oct 16;12(1):164
pubmed: 29037196
Acta Psychiatr Scand Suppl. 2003;(416):16-23
pubmed: 12755850
Neuropsychopharmacology. 2006 Feb;31(2):406-12
pubmed: 16123745
Am J Med Genet A. 2006 Mar 1;140(5):413-8
pubmed: 16470747
BMC Psychiatry. 2011 May 14;11:83
pubmed: 21569566
Psychiatry Res. 1997 Dec 5;73(3):159-71
pubmed: 9481807
J Child Neurol. 2015 Nov;30(13):1743-8
pubmed: 25895911
J Clin Psychiatry. 2012 Apr;73(4):526-32
pubmed: 22579152
Am J Med Genet A. 2011 Jan;155A(1):81-90
pubmed: 21204213