Abstract Details

Title Efficacy of Eculizumab in Acute Refractory Pediatric Neuromyelitis Optica: A Case Report

Topic Autoimmune Neurology

Presentation(s) P11 - Poster Session 11 (5:30 PM-6:30 PM)

Poster/Presentation
Number 14-012

Objective To present the successful therapeutic use of intravenous (IV) eculizumab in a pediatric patient with acute aquaporin-4 (AQP4) IgG positive neuromyelitis optica spectrum disorder (NMOSD).

Background NMOSD is an immune-mediated disorder of the central nervous system in which pathogenic IgG autoantibodies against the AQP4 water transport channel produce astrocyte, myelin, and neuronal injury through complement-mediated cytotoxicity. First-line treatment of an acute flare of NMOSD includes high dose IV steroids often combined with plasma exchange (PLEX). Eculizumab is a monoclonal antibody that inhibits complement-mediated cytotoxicity and was reported to mildly improve an adult with acute AQP4-IgG seropositive NMOSD failing IV methylprednisolone (IVMP) and PLEX. To our knowledge, there are no reports of eculizumab used in children with an acute NMOSD relapse.

Design/Methods Case report and literature review

Results A 12-year-old male presented with emesis, bulbar weakness, paresthesias, limb weakness, gait instability, urinary retention, and respiratory failure requiring intubation. His extensive workup was negative except for cerebrospinal fluid analysis showing mild pleocytosis and an MRI brain showing a non-enhancing T2 hyperintense lesion in the dorsal medulla. AQP4 IgG titers were positive (serum >1:100,000 [reference <1:5], CSF >1:2,048 [reference <1:2]). He received a 5-day course of IVMP 30 milligrams (mg)/kilogram/day and 5 sessions of PLEX. The patient had minimal improvement, instead developing new numbness and tremor. With worsening symptoms, he was initiated on eculizumab. Eculizumab was administered per the pediatric dosage recommended for atypical hemolytic uremic syndrome: two weekly doses of 600 mg as an induction course, followed by initiation maintenance dosage of 900 mg at week 3 and every 2 weeks thereafter. Our patient demonstrated rapid stabilization and improvement, returning near baseline within months except for mild right-sided residual weakness and fatigue which is gradually improving.

Conclusions This case demonstrates the potential utility of eculizumab for the acute treatment of pediatric AQP4-IgG seropositive NMOSD relapses.

Authors/Disclosures
Ryan Kammeyer, MD (Childrens Hospital Colorado) PRESENTER	The institution of Dr. Kammeyer has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Amgen. The institution of Dr. Kammeyer has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Novartis. Dr. Kammeyer has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Ogborn-Mihm Trial Lawyers. The institution of Dr. Kammeyer has received research support from Rocky Mountain Multiple Sclerosis Center.
Michael Enriquez, MD (University of Colorado)	Dr. Enriquez has nothing to disclose.
Scott Rosenthal, MD (University of Colorado)	Dr. Rosenthal has a non-compensated relationship as a Sub-Investigator with Biohaven and Teva that is relevant to AAN interests or activities.
Amanda L. Piquet, MD, FAAN (University of Colorado)	The institution of Dr. Piquet has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Genentech/Roche. The institution of Dr. Piquet has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. The institution of Dr. Piquet has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Kyverna . The institution of Dr. Piquet has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Genentech/Roche. The institution of Dr. Piquet has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Kyverna. The institution of Dr. Piquet has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Piquet has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Sands Anderson PC. Dr. Piquet has received personal compensation in the range of $5,000-$9,999 for serving as an Expert Witness for Joe Jones Law Firm. Dr. Piquet has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Cortez & Associates. Dr. Piquet has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Falk Waas. The institution of Dr. Piquet has received research support from Rocky Mountain MS Center. The institution of Dr. Piquet has received research support from Roche/Genentech. The institution of Dr. Piquet has received research support from NYU. The institution of Dr. Piquet has received research support from Anokion. The institution of Dr. Piquet has received research support from UCB . The institution of Dr. Piquet has received research support from Foundation for Sarcoidosis. The institution of Dr. Piquet has received research support from Kyverna . Dr. Piquet has received publishing royalties from a publication relating to health care. Dr. Piquet has received publishing royalties from a publication relating to health care. Dr. Piquet has received personal compensation in the range of $10,000-$49,999 for serving as a Litigative Consultant with US-Dept HHS/DICP. Dr. Piquet has a non-compensated relationship as a Medical Advisory Board Member with Autoimmune Encephalitis Alliance (AEA) that is relevant to AAN interests or activities. Dr. Piquet has a non-compensated relationship as a Medical Advisory Board Member with Stiff Person Syndrome Research Foundation (SPSRF) that is relevant to AAN interests or activities.
Jeffrey L. Bennett, MD, PhD, FAAN (University of Colorado School of Medicine)	Dr. Bennett has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Amgen. Dr. Bennett has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Bennett has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Genentech. Dr. Bennett has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Roche. Dr. Bennett has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Mitsubishi Tanabe. Dr. Bennett has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Novartis. Dr. Bennett has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immpact Bio. Dr. Bennett has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Chugai. Dr. Bennett has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Beigene. Dr. Bennett has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Imcyse. Dr. Bennett has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Bennett has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for MIAC. Dr. Bennett has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for CorEvitas. Dr. Bennett has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for Alexion. Dr. Bennett has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Vindico. Dr. Bennett has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Touch IME. Dr. Bennett has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Efficient LLC. Dr. Bennett has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Pavich. Dr. Bennett has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Marie Bush. Dr. Bennett has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Marks Gray. Dr. Bennett has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Knight, Nicastro, MacKay. The institution of Dr. Bennett has received research support from Alexion. The institution of Dr. Bennett has received research support from Genentech. Dr. Bennett has received intellectual property interests from a discovery or technology relating to health care. Dr. Bennett has received publishing royalties from a publication relating to health care.
Loren A. McLendon, MD (Nemours Pediatric Neurology)	The institution of Dr. McLendon has received research support from LaRoche .

��ɫ��

��ɫ��