Pediatric-onset Multiple Sclerosis treatment: a multicentre observational study comparing natalizumab with fingolimod
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Published on: September 12, 2016
Antonio Carotenuto1, Cristina Di Monaco2, Laura Papetti3
1Department of Neuroscience, Reproductive Science and Odontostomatology, Multiple Sclerosis Clinical Care and Research Centre, Federico II University, Naples, Italy. carotenuto.antonio87@gmail.com.
Insights
Both natalizumab and fingolimod effectively manage relapses in pediatric-onset multiple sclerosis (POMS). Natalizumab also demonstrated a decrease in disability, potentially linked to higher baseline inflammation in these POMS patients.
Area of Science:
- Pediatric Neurology
- Neuroimmunology
- Clinical Therapeutics
Background:
- Pediatric-onset Multiple Sclerosis (POMS) exhibits greater inflammatory activity than adult-onset MS.
- Treatment options for POMS are restricted, with limited approved therapies and off-label use of others.
Purpose of the Study:
- To compare the efficacy of natalizumab versus fingolimod in managing POMS.
- To evaluate treatment effects on clinical and MRI outcomes in POMS patients.
Main Methods:
- An observational, longitudinal, multicenter study involving POMS patients treated with natalizumab (N-POMS) or fingolimod (F-POMS).
- Data collection included Annual Relapse Rate (ARR), Expanded Disability Status Scale (EDSS), Symbol Digit Modality Test (SDMT), and MRI activity at baseline, 12-18 months, and last observation.
Main Results:
- N-POMS patients presented with higher baseline ARR, EDSS, and lower SDMT compared to F-POMS.
- Both treatments improved ARR between baseline and 12-18 months; EDSS and SDMT improved only in the N-POMS group.
- Long-term follow-up (66.1 months) showed sustained ARR control in N-POMS patients.
Conclusions:
- Both natalizumab and fingolimod demonstrate high and sustained efficacy in controlling relapses in POMS.
- Natalizumab treatment is associated with a decrease in disability in POMS, possibly due to baseline inflammatory activity.
Background:
Pediatric-onset Multiple Sclerosis (POMS) patients show more inflammatory disease compared with adult-onset MS. However, highly effective treatments are limited with only fingolimod being approved in Italy and natalizumab prescribed as off-label treatment.
Objectives:
to compare the efficacy of natalizumab versus fingolimod in POMS.
Methods:
This is an observational longitudinal multicentre study including natalizumab- and fingolimod-treated POMS patients (N-POMS and F-POMS, respectively). We collected Annual Relapse Rate (ARR), Expanded Disability Status Scale (EDSS), Symbol Digit Modality Test (SDMT), and MRI activity at baseline (T0), 12-18 months (T1), and last available observation (T2).
Results:
We enrolled 57 N-POMS and 27 F-POMS patients from six Italian MS Centres. At T0, N-POMS patients showed higher ARR (p = 0.03), higher EDSS (p = 0.003) and lower SDMT (p = 0.04) at baseline compared with F-POMS. Between T0 and T1 ARR improved for both N-POMS and F-POMS (p < 0.001), while EDSS (p < 0.001) and SDMT (p = 0.03) improved only for N-POMS. At T2 (66.1 ± 55.4 months) we collected data from 42 out of 57 N-POMS patients showing no further ARR decrease.
Conclusion:
Both natalizumab and fingolimod showed high and sustained efficacy in controlling relapses and natalizumab also associated to a disability decrease in POMS. This latter effect might be partly mediated by the high inflammatory activity at baseline in N-POMS.
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