Abstract Details

Title Language Skills in Children with Alexander Disease

Topic Child Neurology and Developmental Neurology

Presentation(s) P4 - Poster Session 4 (5:30 PM-6:30 PM)

Poster/Presentation
Number 7-050

Objective To understand the language skills of children with Alexander Disease (AxD) and determine if AxD subgroups contribute to the variability in language performance.

Background Despite language deficits being a common feature among children with AxD, relatively little is known about the communication profiles of the AxD subgroups.

Design/Methods

AxD participants in a natural history study completed a comprehensive speech and language test battery. Participants <21 years completed The Preschool Language Scale-Fifth Edition (PLS-5) or the Clinical Evaluation of Language Fundamentals-Fifth Edition (CELF-5), selected according to the participant’s linguistic level (PLS-5 <6 years, CELF-5 >6 years). Scores for the Auditory Comprehension (AC), Expressive Communication (EC), and Total Language Score (TLS) [PLS-5] and The Core Language Score (CLS), Receptive Language Index (RLI), and Expressive Language Index (ELI) [CELF-5] were calculated. Participants were grouped into the statistically defined subtypes of Type I and Type II. Scores in AxD subtypes were compared with 2-sample t tests.

Results

Among 38 enrolled AxD participants, 22 completed the PLS-5; 17 (77%) showed below-average (<85) receptive and expressive language skills for the TLS. Performance on the CELF-5 varied by disease type: mean CLS for Type I (N=9) was 58.8 vs. 87.4 for Type II (N=7) [p=0.02]. Overall, the percentage of participants who scored ≤85 by subtest was as follows: TLS or CLS 71%, AC or RLI 71%, EC or ELI 74%.

Conclusions Subjects with AxD demonstrate significant language deficits. Type II participants had higher receptive and expressive language scores than the Type I subgroup. Children in the Type II subgroup performed within the low-average range on overall language scores, whereas children with Type I presented with scores in the very low/severe range. The results provide baseline language performance information that can be used to evaluate treatment effects in the future.

Authors/Disclosures
Debra Levin, MA, CCC-SLP (Children'S Hospital of Philadelphia) PRESENTER	No disclosure on file
Hannah R. Cooper (Children'S Hospital of Philadelphia)	Ms. Cooper has nothing to disclose.
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
Amy T. Waldman, MD (Children's Hospital of Philadelphia)	Dr. Waldman has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for SwanBio. An immediate family member of Dr. Waldman has or had stock in Pfizer. The institution of Dr. Waldman has received research support from Ionis Pharmaceuticals. The institution of Dr. Waldman has received research support from Roche/Genentech. The institution of Dr. Waldman has received research support from Ionis Pharmaceuticals. The institution of Dr. Waldman has received research support from Calico. Dr. Waldman has received publishing royalties from a publication relating to health care. Dr. Waldman has received publishing royalties from a publication relating to health care.

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