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Final Program


Final Program

1116 - Challenges and Unknowns of Biologics In Demyelinating Conditions in Pediatric Patients

Thursday, February 27, 2025
5:00 PM - 6:30 PM MST
Case Diagnosis: Neuromyelitis Optica

Case Description: An 11-year-old female presented with acute ascending bilateral leg numbness, weakness, and gait impairments. MRI revealed extensive hyperintense cord signal abnormality from C2 to the conus medullaris. An infectious and inflammatory workup was unrevealing but aquaporin-4 immunoglobulin G (AQP4-IgG) antibody was positive, establishing a diagnosis of neuromyelitis optica (NMO). She was treated initially with intravenous methylprednisolone and plasma exchange followed by eculizumab every 2 weeks for preventative treatment. After several months, a transition to rituximab was trialed but resulted in a disabling relapse requiring re-hospitalization. Repeat MRI demonstrated extensive new myelitis from C2-T10. Following acute management, she was admitted to inpatient rehabilitation back on eculizumab therapy, where she made great recovery in strength and functionality. The patient is currently ambulating without an assistive device and remains in an intensive outpatient therapy program with close physiatry follow-up.

Discussions:

NMO is a rare autoimmune inflammatory demyelinating disease often associated with AQP4-IgG antibodies that predominantly presents in females aged 30-40. Pediatric cases comprise less than 5%. Clinical manifestations vary but often involve optic neuritis, acute transverse myelitis, and brainstem manifestations. NMO can be disabling, and relapse episodes can cause permanent disability.


Current NMO treatment recommendations are largely based on adult populations, making management in pediatric cases difficult. Rituximab is a common first-line therapy in adults but resulted in treatment failure in this patient and a setback in her functionality, whereas the newer biologic agent eculizumab elicited a better response. There also lacks a functional scale specifically designed for NMO, which underscores a physiatrist’s role in monitoring patients' recovery and rehabilitation.

Conclusions: NMO and many other demyelinating conditions lack clear guidelines for management in the pediatric population. Research on newer biologics is needed as treatment failure can lead to significant relapses in function and quality of life.