Pediatric-onset Multiple Sclerosis treatment: a multicentre observational study comparing natalizumab with fingolimod.

Multiple Sclerosis disease modifying treatment fingolimod natalizumab pediatric multiple sclerosis real-world study

Journal

Journal of neurology
ISSN: 1432-1459
Titre abrégé: J Neurol
Pays: Germany
ID NLM: 0423161

Informations de publication

Date de publication:
23 Aug 2024
Historique:
received: 07 06 2024
accepted: 21 07 2024
revised: 17 07 2024
medline: 23 8 2024
pubmed: 23 8 2024
entrez: 23 8 2024
Statut: aheadofprint

Résumé

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. to compare the efficacy of natalizumab versus fingolimod in POMS. 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). 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 T 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.

Sections du résumé

BACKGROUND 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 OBJECTIVE
to compare the efficacy of natalizumab versus fingolimod in POMS.
METHODS 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 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 T
CONCLUSION CONCLUSIONS
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.

Identifiants

DOI: 10.1007/s00415-024-12610-y PMID: 39177751
pubmed: 39177751
doi: 10.1007/s00415-024-12610-y
pii: 10.1007/s00415-024-12610-y
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Informations de copyright

© 2024. The Author(s).

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Auteurs

Antonio Carotenuto (A)

Department of Neuroscience, Reproductive Science and Odontostomatology, Multiple Sclerosis Clinical Care and Research Centre, Federico II University, Naples, Italy. carotenuto.antonio87@gmail.com.

Cristina Di Monaco (C)

Department of Neuroscience, Reproductive Science and Odontostomatology, Multiple Sclerosis Clinical Care and Research Centre, Federico II University, Naples, Italy.

Laura Papetti (L)

Developmental Neurology Unit, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.

Giovanna Borriello (G)

Department of Public Health, University "Federico II" of Naples, Naples, Italy.

Elisabetta Signoriello (E)

Second Division of Neurology, Department of Advanced Medical and Surgical Sciences, University of Campania, Naples, Italy.

Camilla Masciulli (C)

Department of NEUROFARBA, University of Florence, Florence, Italy.

Valentina Tomassini (V)

Multiple Sclerosis Centre, Clinical Neurology, SS. Annunziata University Hospital, Chieti, Italy.
Institute for Advanced Biomedical Technologies (ITAB), Department of Neurosciences, Imaging and Clinical Sciences, University G. d'Annunzio of Chieti-Pescara, Chieti, Italy.

Giovanna De Luca (G)

Multiple Sclerosis Centre, Clinical Neurology, SS. Annunziata University Hospital, Chieti, Italy.

Antonio Ianniello (A)

Department of Human Neurosciences, Sapienza University, Rome, Italy.

Giacomo Lus (G)

Second Division of Neurology, Department of Advanced Medical and Surgical Sciences, University of Campania, Naples, Italy.

Federica Novarella (F)

Department of Neuroscience, Reproductive Science and Odontostomatology, Multiple Sclerosis Clinical Care and Research Centre, Federico II University, Naples, Italy.

Antonio Luca Spiezia (AL)

Department of Neuroscience, Reproductive Science and Odontostomatology, Multiple Sclerosis Clinical Care and Research Centre, Federico II University, Naples, Italy.

Dario Di Somma (D)

Department of Neuroscience, Reproductive Science and Odontostomatology, Multiple Sclerosis Clinical Care and Research Centre, Federico II University, Naples, Italy.

Marcello Moccia (M)

Department of Molecular Medicine and Medical Biotechnology, Federico II University of Naples, Naples, Italy.

Maria Petracca (M)

Department of Human Neurosciences, Sapienza University, Rome, Italy.

Carmine Iacovazzo (C)

Department of Neuroscience, Reproductive Science and Odontostomatology, Multiple Sclerosis Clinical Care and Research Centre, Federico II University, Naples, Italy.

Giuseppe Servillo (G)

Department of Neuroscience, Reproductive Science and Odontostomatology, Multiple Sclerosis Clinical Care and Research Centre, Federico II University, Naples, Italy.

Emilio Portaccio (E)

Department of NEUROFARBA, University of Florence, Florence, Italy.

Maria Triassi (M)

Department of Public Health, University "Federico II" of Naples, Naples, Italy.

Maria Pia Amato (MP)

Department of NEUROFARBA, University of Florence, Florence, Italy.

Carlo Pozzilli (C)

Department of Human Neurosciences, Sapienza University, Rome, Italy.

Massimiliano Valeriani (M)

Developmental Neurology Unit, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
System Medicine Department, Tor Vergata University of Rome, Rome, Italy.
Center for Sensory-Motor Interaction, Aalborg University, Aalborg, Denmark.

Vincenzo Brescia Morra (V)

Department of Neuroscience, Reproductive Science and Odontostomatology, Multiple Sclerosis Clinical Care and Research Centre, Federico II University, Naples, Italy.

Roberta Lanzillo (R)

Department of Neuroscience, Reproductive Science and Odontostomatology, Multiple Sclerosis Clinical Care and Research Centre, Federico II University, Naples, Italy.

Classifications MeSH