Abstract
<h3>Objective:</h3> To explore safety and effectiveness of anti-emetic strategies in Alexander disease (AxD). <h3>Background:</h3> 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. <h3>Design/Methods:</h3> 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. <h3>Results:</h3> 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). <h3>Conclusions:</h3> 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. <b>Disclosure:</b> Ms. Sergio has nothing to disclose. Miss Gallison has nothing to disclose. Dr. Porcari has nothing to disclose. The institution of Mr. Joung has received research support from Ionis Pharmaceuticals. Mr. Faig has nothing to disclose. An immediate family member of Geraldine Liu has received publishing royalties from a publication relating to health care. Dr. Waldman has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for SwanBio. An immediate family member of Dr. Waldman has stock in Pfizer. The institution of Dr. Waldman has received research support from Ionis Pharmaceuticals. Dr. Waldman has received publishing royalties from a publication relating to health care.
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