scholarly journals Detecting meaningful change using the North Star Ambulatory Assessment in Duchenne muscular dystrophy

2013 ◽  
Vol 55 (11) ◽  
pp. 1046-1052 ◽  
Author(s):  
Anna G Mayhew ◽  
Stefan J Cano ◽  
Elaine Scott ◽  
Michelle Eagle ◽  
Kate Bushby ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Ambulatory Assessment ◽  
Meaningful Change ◽  
The North

scholarly journals Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy

PLoS ONE ◽  
2019 ◽  
Vol 14 (9) ◽  
pp. e0221097 ◽  
Author(s):  
Francesco Muntoni ◽  
Joana Domingos ◽  
Adnan Y. Manzur ◽  
Anna Mayhew ◽  
Michela Guglieri ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Ambulatory Assessment ◽  
Individual Item ◽  
The North

Overview of morbidity, mortality and survival in Duchenne muscular dystrophy in the North East of England

2015 ◽  
Vol 25 ◽  
pp. S301-S302
Author(s):  
C. Marini Bettolo ◽  
H. van Ruiten ◽  
M. Guglieri ◽  
M. Eagle ◽  
V. Straub ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
North East ◽  
The North

scholarly journals Development of a Clinical Global Impression of Change (CGI-C) and a Caregiver Global Impression of Change (CaGI-C) measure for ambulant individuals with Duchenne muscular dystrophy

2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Hannah Staunton ◽  
Claire Trennery ◽  
Rob Arbuckle ◽  
Maitea Guridi ◽  
Elena Zhuravleva ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Clinical Global Impression ◽  
Global Changes ◽  
Meaningful Change ◽  
Treatment Benefit ◽  
Specific Patient ◽  
Concept Elicitation ◽  
Global Impression Of Change

Abstract Background In clinical trials for rare diseases, such as Duchenne muscular dystrophy, clinical outcome assessments (COA) used to assess treatment benefit are often generic and may not be sensitive enough to detect change in specific patient populations. Thus, there is a need for disease specific COAs that track meaningful change among individuals. When developing such measures, input from clinicians, caregivers and patients is critical for assessing clinically relevant concepts and ensuring validity of the measure. Method The aim of this study was to develop two Duchenne-specific global impression items for use in clinical trials. The development of the Duchenne Clinical Global Impression of Change (CGI-C) and Caregiver Global Impression of Change (CaGI-C) was informed by findings from concept elicitation (CE) interviews with clinicians, caregivers and individuals with Duchenne. Through cognitive debriefing (CD) interviews, clinicians and caregivers evaluated draft CGI-C and CaGI-C items to ensure relevance and understanding of the items and instructions. Suggestions made during the CD interviews were incorporated into the finalized CGI-C and CaGI-C measures. Results The symptoms most frequently reported by clinicians, caregivers and individuals with Duchenne were muscle weakness, fatigue, cardiac difficulties and pain. Regarding physical functioning, all three populations noted that small changes in functional ability were meaningful, particularly when independence was impacted. Caregivers and clinicians reported that changes in speed, endurance and quality of movement were important, as was improvement in the ability of individuals to keep up with their peers. A change in the ability to complete everyday activities was also significant to families. These results were used to create two global impression of change items and instruction documents for use by clinicians (CGI-C) and caregivers (CaGI-C). Overall, both items were well understood by participants. The descriptions and examples developed from the CE interviews were reported to be relevant and appropriate for illustrating different levels of meaningful change in patients with Duchenne. Modifications were made based on caregiver and clinician CD feedback . Conclusions As part of a holistic measurement strategy, such COA can be incorporated into the clinical trial setting to assess global changes in relevant symptoms and functional impacts associated with Duchenne.

Suitability of North Star Ambulatory Assessment in young boys with Duchenne muscular dystrophy

2015 ◽  
Vol 25 (1) ◽  
pp. 14-18 ◽  
Author(s):  
Roberto De Sanctis ◽  
Marika Pane ◽  
Serena Sivo ◽  
Valeria Ricotti ◽  
Giovanni Baranello ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Ambulatory Assessment
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