Abstract Details

Title Valproic Acid is an Effective Anti-Emetic Treatment in Alexander Disease

Topic Child Neurology and Developmental Neurology

Presentation(s) P10 - Poster Session 10 (8:00 AM-9:00 AM)

Poster/Presentation
Number 4-010

Objective To explore safety and effectiveness of anti-emetic strategies in Alexander disease (AxD).

Background Emesis is a frequent symptom in AxD and leads to inadequate intake and food avoidance behaviors. As a result, weight loss and failure to thrive are common in AxD. We explored emesis treatment strategies to determine their relative benefits and side effects in clinical practice.

Design/Methods Medical records of patients with AxD (<21 years) participating in a natural history cohort study were reviewed for history of emesis, difficulty with weight gain, and medications. Patients were retrospectively evaluated for response to medication, which was defined as a clinically meaningful decrease in emesis. Treatment failure was defined as no improvement, worsened emesis, clinically significant weight loss, or presence of an intolerable side effect. Deceased patients, patients with a Nissen fundoplication, and patients with insufficient longitudinal data were excluded. Fisher’s exact tests were used to compare response rates of valproic acid to those of anti-reflux medications (proton pump inhibitors, H2 antagonists analyzed together) or cyproheptadine.

Results Among 59 subjects with vomiting, 34 subjects met inclusion criteria. Emesis was treated with multiple anti-reflux medications (N=24), cyproheptadine (N=19), and valproic acid (VPA, N=22). Overall, 48% of patients responded to multiple anti-reflux medications, 32% responded to cyproheptadine, and 95% responded to VPA. VPA had a higher success rate in reducing emesis compared to multiple reflux medications (p=0.0001) or cyproheptadine (p<0.0001). Adverse effects from VPA included bone fractures (N=1) in a non-ambulatory patient, hyperammonemia (N=2) which resolved with dose lowering, and mild hair loss (N=2).

Conclusions Recognizing the limitations of retrospective review, 95% of AxD patients on VPA showed improvements in emesis and weight gain. Response rates to VPA were greater compared to anti-reflux medications or cyproheptadine. VPA is well tolerated overall, although use should be monitored for known adverse effects.

Authors/Disclosures
Emily Sergio, Other (Children's Hospital of Philadelphia) PRESENTER	Ms. Sergio has nothing to disclose.
Kathryn Gallison, Other (The Children's Hospital of Philadelphia)	The institution of Miss Gallison has received research support from Ionis Pharmaceuticals.
Giulia S. Porcari, MD (Children's Hospital of Philadelphia)	The institution of Dr. Porcari has received research support from NIH/NINDS (T32 - NS-061779).
Joshua Joung, Other (Children's Hospital of Philadelphia)	Mr. Joung has nothing to disclose.
Walter Faig	Walter Faig has received personal compensation for serving as an employee of Westat.
	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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