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Published on: September 12, 2016
Prognostic indicators of improvement with therapeutic plasma exchange in pediatric demyelination
Andrea Savransky1, Adrian Rubstein1, Marina Huaman Rios1
1From the Department of Neurology (A.S., M.H.R., S.L.V., M.C.V., S.P.S., S.T.) and Transfusion Medicine (G.M., R.A., A.M.P.), National Pediatric Hospital Dr. J.P. Garrahan; and Private Institute of Statistics (A.R.), Buenos Aires, Argentina.
Insights
Therapeutic plasma exchange (TPE) effectively improved neurological function in children with acute inflammatory demyelinating central nervous system (CNS) syndromes. This rescue therapy demonstrated a low rate of adverse events, making it a beneficial treatment option.
Area of Science:
- Pediatric Neurology
- Neuroimmunology
- Demyelinating Diseases
Background:
- Acute inflammatory demyelinating central nervous system (CNS) syndromes in children can lead to significant disability.
- Steroid-refractory cases require alternative rescue therapies to prevent long-term neurological deficits.
Purpose of the Study:
- To evaluate the safety and clinical efficacy of therapeutic plasma exchange (TPE) as a rescue therapy for pediatric patients with acute CNS inflammatory demyelinating events.
- To identify baseline prognostic factors associated with treatment response to TPE.
Main Methods:
- Retrospective analysis of a pediatric cohort (2003-2017) receiving TPE for steroid-refractory acute CNS inflammatory events.
- Functional outcomes assessed using Expanded Disability Status Scale (EDSS), visual, bladder, and gait scales, pre- and post-TPE.
Main Results:
- 72% of children showed moderate to marked neurological improvement by the end of TPE, with 88.5% improvement at 6-month follow-up.
- Lower baseline EDSS, visual, and gait scores independently predicted better treatment outcomes.
- Adverse events occurred in 5.9% of procedures, with severe events in 4 cases.
Conclusions:
- Therapeutic plasma exchange (TPE) is an effective rescue therapy for children with acute inflammatory demyelinating CNS syndromes, leading to functional recovery.
- No specific therapeutic window for TPE initiation was identified; however, vigilance for serious adverse events is crucial.
Objectives:
To determine the safety and clinical benefit of therapeutic plasma exchange (TPE) as rescue therapy in children with acute inflammatory demyelinating CNS syndromes and to identify baseline prognostic indicators of treatment improvement.
Methods:
This single-center retrospective pediatric cohort included all consecutive patients admitted to our hospital over the period from 2003 to 2017 because of a steroid-refractory acute CNS event presumed to be inflammatory who required TPE. Functional status assessment to identify improvement included the following performance category scales: visual outcome, bladder control, gait, and Expanded Disability Status Scale (EDSS). These assessments were performed before and after TPE in every patient.
Results:
Sixty-five children requiring TPE to treat 78 CNS attacks were included for analysis. Median age at TPE was 10.5 years (1.9-18 years); 45% were girls. Seropositivity (aquaporin-4 water channel-immunoglobulin G [IgG] or myelin oligodendrocyte glycoprotein-IgG) was found in 20 of 42 (48%) patients. Attack phenotypes leading to TPE were optic neuritis (ON) in 42%, longitudinally extensive transverse myelitis (LETM) in 31%, ON + LETM in 15%, and other combined syndromes in 11%. Overall, moderate to marked neurologic improvement was observed in 72% of children at the end of TPE and in 88.5% at 6 months of follow-up. Lower baseline scores on the EDSS, visual outcome, and gait scales were found to be independent prognostic indicators of treatment benefit. Sex, age at onset and at TPE, attack phenotype, disease duration, and time from attack onset to TPE initiation were not significantly associated with treatment outcome. Adverse events were observed in 31 of 524 (5.9%) procedures, being severe in 4.
Conclusions:
TPE was an effective rescue therapy associated with functional improvement. No therapeutic window for TPE initiation was identified in this pediatric cohort. Overall frequency of adverse events was low; however, serious events should be anticipated and avoided.
Classification Of Evidence:
This study provides Class IV evidence that for children with acute inflammatory demyelinating CNS syndromes, TPE leads to functional improvement.
