To create a tool, The Children’s Hospital of Philadelphia Pediatric Dysphagia Instrument (CHOP PDI), to assess swallow function and safety in neurodegenerative disease.

Bulbar dysfunction is prominent in Alexander Disease (AxD), yet there are no published measures that qualitatively and quantitatively capture swallow function in medically fragile patients. We created the CHOP PDI as a performance outcome measure for an AxD Natural History Study. 

The CHOP PDI evaluates the oral and pharyngeal phases of the swallow using clinical observations of liquids, purees, and solids. A numeric score is given based on oral skills across textures; a 0 is given in the presence of signs concerning for aspiration or adaptations are needed to consume the food (eg. chopping, thickening, adapted cup). A maximum score of 27 (indicating normal swallow function) is possible in subjects older than 3 years. The CHOP PDI will be presented in its entirety. Descriptive statistics and Cohen's kappa were calculated, and t tests were performed.

The CHOP PDI was administered to 33 AxD patients >3 years at evaluation (mean age 14.7 years, range 3.7–53.4). The mean score was 20.8 (SD 5.3, range 6-27). Subjects with G-tubes (N=8) had lower scores (mean 18.8, range 6-25.5) compared to those without enteral feeds (N=25, mean 21.5, range 7-27, p=0.2097). Interrater reliability was established in 11 patients, simultaneously scored by two clinicians with expertise in pediatric dysphagia (Cohen’s kappa 0.75). Longitudinal analysis (>1 year apart) of 16 subjects showed improved scores in 6 (37.5%), worsening in 8 (50%), and stability in 2 (12.5%).

The CHOP PDI shows promise for comprehensively evaluating longitudinal swallow function while avoiding radiation and other pitfalls associated with the video fluoroscopic swallow study. This tool successfully captured deficits in both children and adults with AxD and can be adapted for other neurodegenerative diseases with bulbar dysfunction.