Patient reported outcomes in GNE myopathy: incorporating a valid assessment of physical function in a rare disease.
Christina Slota, Margaret Bevans, Li Yang and 3 others
PMID 28637129WHAT IT FOUND
Three patient-reported function measures were usable and stable in 35 people with GNE myopathy, but they did not separate low from moderate impairment and may not fit advanced disease.
Key findings
01The HAP adjusted activity score and the IBMFRS showed moderate to large positive relationships with clinician assessments of physical function.
02The IBMFRS and ABC scale showed consistent item responses and stable scores between baseline and 6 months.
03At baseline, 91.4% of participants reported a history of falls and 74.3% used an assistive device.
STILL TO COME
How it was doneWhat they foundWhat it means for PTsWhat it means for OTs
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What it does not show
The sample was small: 35 at baseline and 16 at 6 months, because GNE myopathy is rare. Only participants who could travel were eligible, so severely impaired or wheelchair-dependent people may be under-represented. The study could not confirm validity in advanced disease; 31.4% could not complete the six-minute walk test, and missing data occurred for tests that advanced patients could not perform. The PROs did not separate low-functioning from moderate-functioning groups. The ABC scale correlated less strongly with physical assessments than the HAP adjusted score and IBMFRS.
Declared interests
The authors report no declarations of interest. The paper is listed as supported by NIH extramural research.
The easy way to misread this
Do not assume these questionnaires are appropriate for wheelchair-dependent or severely limited patients. The study could not confirm validity there, and 31.4% of subjects could not complete the six-minute walk test.