Efficacy and Safety of Plasmapheresis in Children With Acute Transverse or Flaccid Myelitis, and Guillain-Barré Syndrome.

BACKGROUND

Acute transverse myelitis (ATM), acute flaccid myelitis with polio-like myeloradiculitis (AFM) and Guillain-Barré syndrome (GBS) are severe neuroimmune disorders associated with high morbidity. First-line treatments include corticosteroids and intravenous immunoglobulins (IVIg), but they may be insufficient. Therapeutic plasma exchange (TPE) and immunoadsorption (IA) are second- or third-line options in pediatrics, despite their recognized efficacy in adults. Data on pediatric use remain limited. This study aimed to evaluate the efficacy and safety of apheresis in children with ATM, GBS or AFM.

METHODS

We conducted a retrospective monocentric study at Necker University Hospital, including pediatric patients diagnosed with ATM, GBS or AFM who underwent TPE/IA between 2014 and 2024. Functional outcomes were assessed using the modified Rankin Scale (mRS) at five time points, from peak disease severity to last follow-up. Safety was evaluated based on severe adverse events.

RESULTS

Among 23 children (6 years [Q1-Q3]), 20 received TPE and 3 IA. 17 of 23 patients (74%) showed significant improvement by the end of treatment. The median mRS was 5 (IQR 4-5) before apheresis, improving to 4 (IQR 2-4) at the end of TPE/IA, and decreasing to 2 at 6 months. Brainstem function fully recovered in all surviving patients. Among 14 children with sphincter dysfunction, 5 (36%) still required intermittent catheterization. No treatment discontinuations occurred, but two patients died, one from venous air embolism, one after life-sustaining treatment limitation.

CONCLUSION

TPE and IA appear effective in pediatric ATM, AFM and GBS. Larger studies are needed to confirm long-term efficacy and refine treatment guidelines.