Disclosures: Eric M. Westerbeck, MD: No financial relationships or conflicts of interest

Case Diagnosis: 7-year-old female with weakness and urinary incontinence

Case Description: The patient presented to the emergency department with weakness and urinary incontinence with imaging and positive aquaporin-4 antibody consistent with Neuromyelitis Optica Spectrum Disorder (NMOSD). She was treated with IV steroids followed by IVIG and was transferred to inpatient rehabilitation. She had recurrence of symptoms at rehabilitation, with functional decline, and again treated with oral and IV steroids. Following continued decline she was transferred to acute care and treated with 11 plasmapheresis sessions with limited benefit. She developed severe retching and intractable vomiting. She started eculizumab treatment and demonstrated significant clinical and functional improvement. She was transferred back to rehabilitation for ongoing therapies and infusions with improvement in function prior to discharge home.

Setting: Pediatric Inpatient Rehabilitation HospitalAssessment/

Results: Within hours of eculizumab administration, the patient demonstrated improvements in alertness, head control, and retching/vomiting improved. She ultimately transferred back to rehabilitation and tolerated additional administrations of eculizumab, first on a weekly basis and eventually a biweekly basis. Complications during the remainder of her rehabilitation course included fever and anemia, but could not be definitively attributed to eculizumab. She had significant functional gains by discharge, able to ambulate community distances and negotiate stairs with minimal assistance.

Discussion: Eculizumab, a humanized monoclonal antibody against complement component C5, has been used in pediatric patients for treatment of atypical hemolytic uremic syndrome and adult patients for treatment of NMOSD. While a trial in pediatric patients with NMOSD is ongoing, this is the first report of eculizumab for treatment of NMOSD in a pediatric patient, and the first report of this treatment being safely delivered in a pediatric inpatient rehabilitation unit.

Conclusion: Following administration of eculizumab for NMOSD, our pediatric patient showed reversal of functional decline and achieved functional gains. Administration of eculizumab was well-tolerated and feasible during comprehensive inpatient rehabilitation admission.

Level of Evidence: Level V

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PM&R Meeting Abstracts - https://pmrjabstracts.org/abstract/eculizumab-for-treatment-of-pediatric-neuromyelitis-optica-spectrum-disorder-implications-for-rehabilitation/