Background Natalizumab (NAT) has a strong impact on disease activity of aggressive pediatric multiple sclerosis (MS), with no difference in safety profile compared to adult MS. However, available data are limited by short follow-up. Our aim was to report long-term follow-up data (up to 11 years) of a large Italian pediatric MS cohort treated with NAT. Materials and methods We retrospectively collected data of pediatric MS patients treated with NAT included in a previous study and prospectively followed in Italian MS centers. We compared disease activity pre, during, and post-NAT and we performed survival analyses of time to evidence of disease activity (EDA) during NAT, time to reach EDA post-NAT, and time to NAT discontinuation. Results Ninety-two patients were included from 19 MS centers in Italy. At NAT initiation, cohort's characteristics were as follows: 55 females; 14.7 +/- 2.4 (mean +/- SD) years of age; 34 naive to disease modifying therapies; 1-year pre-NAT annualized relapse rate (ARR): 2.2 +/- 1.2; EDSS (median [IQR]): 2.5 [2.0-3.0]; gadolinium-enhancing lesions: 2 [1-5]; 41 JCV positives. During NAT treatment (61.9 +/- 35.2 mean infusions), ARR lowered to 0.08 +/- 0.23 (p < 0.001), EDSS score to 1.5 [1.0-2.5] at last infusion (p < 0.001), and 51% patients had EDA (21% after 6 months of rebaseline). No serious adverse events were reported. Forty-nine patients discontinued NAT, mainly due to PML concern; the majority (29/49) had disease reactivation in the subsequent 12 months, of which three with a clinical rebound. Conclusion NAT treatment maintains its high efficacy for a long time in pediatric MS patients, with no new safety issues.

Long-term follow-up (up to 11 years) of an Italian pediatric MS cohort treated with natalizumab: a multicenter, observational study / Baroncini, Damiano; Ghezzi, Angelo; Guaschino, Clara; Moiola, Lucia; Filippi, Massimo; Ianniello, Antonio; Pozzilli, Carlo; Lanzillo, Roberta; Brescia-Morra, Vincenzo; Margoni, Monica; Gallo, Paolo; Callari, Graziella; Grimaldi, Luigi; Lus, Giacomo; Calabrese, Massimiliano; Simone, Marta; Marfia, Girolama Alessandra; Rasia, Sarah; Cargnelutti, Daniela; Comi, Giancarlo; Zaffaroni, Mauro. - In: NEUROLOGICAL SCIENCES. - ISSN 1590-1874. - 43:11(2022), pp. 6415-6423. [10.1007/s10072-022-06211-8]

Long-term follow-up (up to 11 years) of an Italian pediatric MS cohort treated with natalizumab: a multicenter, observational study

Ianniello, Antonio;Pozzilli, Carlo;
2022

Abstract

Background Natalizumab (NAT) has a strong impact on disease activity of aggressive pediatric multiple sclerosis (MS), with no difference in safety profile compared to adult MS. However, available data are limited by short follow-up. Our aim was to report long-term follow-up data (up to 11 years) of a large Italian pediatric MS cohort treated with NAT. Materials and methods We retrospectively collected data of pediatric MS patients treated with NAT included in a previous study and prospectively followed in Italian MS centers. We compared disease activity pre, during, and post-NAT and we performed survival analyses of time to evidence of disease activity (EDA) during NAT, time to reach EDA post-NAT, and time to NAT discontinuation. Results Ninety-two patients were included from 19 MS centers in Italy. At NAT initiation, cohort's characteristics were as follows: 55 females; 14.7 +/- 2.4 (mean +/- SD) years of age; 34 naive to disease modifying therapies; 1-year pre-NAT annualized relapse rate (ARR): 2.2 +/- 1.2; EDSS (median [IQR]): 2.5 [2.0-3.0]; gadolinium-enhancing lesions: 2 [1-5]; 41 JCV positives. During NAT treatment (61.9 +/- 35.2 mean infusions), ARR lowered to 0.08 +/- 0.23 (p < 0.001), EDSS score to 1.5 [1.0-2.5] at last infusion (p < 0.001), and 51% patients had EDA (21% after 6 months of rebaseline). No serious adverse events were reported. Forty-nine patients discontinued NAT, mainly due to PML concern; the majority (29/49) had disease reactivation in the subsequent 12 months, of which three with a clinical rebound. Conclusion NAT treatment maintains its high efficacy for a long time in pediatric MS patients, with no new safety issues.
2022
multiple sclerosis; natalizumab; pediatric; treatment
01 Pubblicazione su rivista::01a Articolo in rivista
Long-term follow-up (up to 11 years) of an Italian pediatric MS cohort treated with natalizumab: a multicenter, observational study / Baroncini, Damiano; Ghezzi, Angelo; Guaschino, Clara; Moiola, Lucia; Filippi, Massimo; Ianniello, Antonio; Pozzilli, Carlo; Lanzillo, Roberta; Brescia-Morra, Vincenzo; Margoni, Monica; Gallo, Paolo; Callari, Graziella; Grimaldi, Luigi; Lus, Giacomo; Calabrese, Massimiliano; Simone, Marta; Marfia, Girolama Alessandra; Rasia, Sarah; Cargnelutti, Daniela; Comi, Giancarlo; Zaffaroni, Mauro. - In: NEUROLOGICAL SCIENCES. - ISSN 1590-1874. - 43:11(2022), pp. 6415-6423. [10.1007/s10072-022-06211-8]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11573/1669848
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