A qualitative study of perceptions of meaningful change in spinal muscular atrophy

Autor: Kenneth Hobby, Jill Jarecki, Sarah McGraw, Jeff Henne, Wei-Shi Yeh, Ying Qian
Jazyk: angličtina
Rok vydání: 2017
Předmět:
0301 basic medicine
Adult
Male
Parents
medicine.medical_specialty
Activities of daily living
Adolescent
Health Personnel
Clinical Neurology
030105 genetics & heredity
lcsh:RC346-429
Muscular Atrophy
Spinal
03 medical and health sciences
Young Adult
0302 clinical medicine
Physical medicine and rehabilitation
Quality of life
Activities of Daily Living
medicine
Humans
Respiratory function
Functional ability
Patient Reported Outcome Measures
Child
lcsh:Neurology. Diseases of the nervous system
Qualitative Research
Patient perspectives
business.industry
Meaningful change
Infant
General Medicine
Spinal muscular atrophy
medicine.disease
SMA
Focus group
Child
Preschool
Physical therapy
Quality of Life
Female
Neurology (clinical)
business
Qualitative
030217 neurology & neurosurgery
Qualitative research
Research Article
Zdroj: BMC Neurology
BMC Neurology, Vol 17, Iss 1, Pp 1-9 (2017)
ISSN: 1471-2377
Popis: Background This qualitative study examined how individuals with Spinal Muscular Atrophy (SMA), their caregivers, and clinicians defined meaningful change, primarily in the Type II and non-ambulant type III patient populations, associated with treatment of this condition. In addition, we explored participants’ views about two measures of motor function routinely used in clinical trials for these SMA subtypes, namely the expanded version of the Hammersmith Functional Motor Scale (HFMSE) and the Upper Limb Module (ULM). Methods The 123 participants (21 with SMA, 64 parents, and 11 clinicians), recruited through SMA advocacy organizations, participated in one of 16 focus groups or 37 interviews. The sessions were audio-recorded, and verbatim transcripts were analyzed using a grounded theory approach. Results For the participants, meaningful change was relative to functional ability, and small changes in motor function could have an important impact on quality of life. Because patients and families feared progressive loss of functional ability, the participants saw maintenance of abilities as a meaningful outcome. They believed that measures of motor function covered important items, but worried that the HFMSE and ULM might not be sensitive enough to capture small changes. In addition, they felt that outcome measures should assess other important features of life with SMA, including the ability to perform daily activities, respiratory function, swallowing, fatigue, and endurance. Conclusions Given the heterogeneity of SMA, it is important to expand the assessment of treatment effects to a broader range of outcomes using measures sensitive enough to detect small changes.
Databáze: OpenAIRE
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