Contributions
Abstract: P666
Type: Poster Sessions
Abstract Category: Clinical aspects of MS - Paediatric MS
Background: Serum neurofilament light chain (sNfL) is a promising biomarker of neuro-axonal injury in adult multiple sclerosis (MS). Data for paediatric MS are still missing.
Objective: To investigate sNfL as a potential biomarker for disease activity and the drug response in paediatric MS patients.
Methods: sNfL was measured in a cohort of 28 paediatric MS patients treated with fingolimod (median age at disease onset was 13.3 years, range 7.2-16.8 years; follow-up was from 12 to 105 months) using a high-sensitivity single molecule array (Simoa) NF-light® Advantage Kit (Quanterix). Twenty-one patients had switched from interferon β, glatiramer acetate or dimethyl fumarate to fingolimod (Group 1) and 7 patients switched from natalizumab to fingolimod (Group 2). sNfL levels were compared with those in a healthy control group (188 controls, aged 3 months to 17.9 years).
Results: Median sNfL level in healthy paediatric controls was 5.3 pg/mL, >=97.5th percentile of 13.9 pg/mL. The majority of pediatric MS patients showed elevated sNfL levels at disease onset (median 37 pg/mL). Under disease modifying therapy, sNfL levels were decreased in 85.7% of patients below 97.5th percentile when compared to age-matched controls. In Group 1, after starting fingolimod, sNfL levels decreased from a median of 15.1 pg/mL (time point of therapy change) to a median of 9.4 pg/mL within 5-10 months. In Group 2, the median sNfL at the end of natalizumab therapy was 7.1 pg/mL. Six to ten months after a switch to fingolimod, sNfL levels remained stable (median 7.6 pg/mL). A comprehensive comparative analysis of NfL and magnetic resonance imaging (MRI) data is in progress.
Conclusion: In general, healthy children do have significantly lower sNfL levels than adults. In paediatric MS, disease modifying therapies significantly reduced sNfL levels to a different extent. Our data provide evidence for sNfL being a useful biomarker for detecting subclinical disease activity and therapy response in paediatric MS.
Disclosure: *Jens Kuhle and Jutta Gärtner equally contributed for this study. This study was funded by Novartis Pharma AG, Basel, Switzerland.
Marie-Christine Reinert: nothing to disclose.
Christian Barro received travel support by Teva and Novartis not related to this work. Zusanna Michalak: nothing to disclose.
Wolfgang Brück has received honoraria for lectures by Bayer Vital, Biogen, Merck Serono, Teva, Genzyme, Roche and Novartis. He is a member of scientific advisory boards for Teva, Biogen, Novartis, Celgene, MedDay and Genzyme. He receives research support from Teva, Biogen, Genzyme, MedDay and Novartis.
Peter Huppke has received honoraria for lectures and consultancy fees from Bayer, Merck, Biogen and Novartis.
Jens Kuhle received speaker fees, research support, travel support, and/or served on advisory boards by ECTRIMS, Swiss MS Society, Swiss National Research Foundation, (320030_160221), University of Basel, Bayer, Biogen, Genzyme, Merck, Novartis, Protagen AG, Roche, Teva.
Jutta Gärtner has received honoraria for lectures and consultancy fees from Bayer, Teva and Novartis.
Harald Kropshofer, Davorka Tomic and David Leppert are employees of Novartis Pharma AG.
Abstract: P666
Type: Poster Sessions
Abstract Category: Clinical aspects of MS - Paediatric MS
Background: Serum neurofilament light chain (sNfL) is a promising biomarker of neuro-axonal injury in adult multiple sclerosis (MS). Data for paediatric MS are still missing.
Objective: To investigate sNfL as a potential biomarker for disease activity and the drug response in paediatric MS patients.
Methods: sNfL was measured in a cohort of 28 paediatric MS patients treated with fingolimod (median age at disease onset was 13.3 years, range 7.2-16.8 years; follow-up was from 12 to 105 months) using a high-sensitivity single molecule array (Simoa) NF-light® Advantage Kit (Quanterix). Twenty-one patients had switched from interferon β, glatiramer acetate or dimethyl fumarate to fingolimod (Group 1) and 7 patients switched from natalizumab to fingolimod (Group 2). sNfL levels were compared with those in a healthy control group (188 controls, aged 3 months to 17.9 years).
Results: Median sNfL level in healthy paediatric controls was 5.3 pg/mL, >=97.5th percentile of 13.9 pg/mL. The majority of pediatric MS patients showed elevated sNfL levels at disease onset (median 37 pg/mL). Under disease modifying therapy, sNfL levels were decreased in 85.7% of patients below 97.5th percentile when compared to age-matched controls. In Group 1, after starting fingolimod, sNfL levels decreased from a median of 15.1 pg/mL (time point of therapy change) to a median of 9.4 pg/mL within 5-10 months. In Group 2, the median sNfL at the end of natalizumab therapy was 7.1 pg/mL. Six to ten months after a switch to fingolimod, sNfL levels remained stable (median 7.6 pg/mL). A comprehensive comparative analysis of NfL and magnetic resonance imaging (MRI) data is in progress.
Conclusion: In general, healthy children do have significantly lower sNfL levels than adults. In paediatric MS, disease modifying therapies significantly reduced sNfL levels to a different extent. Our data provide evidence for sNfL being a useful biomarker for detecting subclinical disease activity and therapy response in paediatric MS.
Disclosure: *Jens Kuhle and Jutta Gärtner equally contributed for this study. This study was funded by Novartis Pharma AG, Basel, Switzerland.
Marie-Christine Reinert: nothing to disclose.
Christian Barro received travel support by Teva and Novartis not related to this work. Zusanna Michalak: nothing to disclose.
Wolfgang Brück has received honoraria for lectures by Bayer Vital, Biogen, Merck Serono, Teva, Genzyme, Roche and Novartis. He is a member of scientific advisory boards for Teva, Biogen, Novartis, Celgene, MedDay and Genzyme. He receives research support from Teva, Biogen, Genzyme, MedDay and Novartis.
Peter Huppke has received honoraria for lectures and consultancy fees from Bayer, Merck, Biogen and Novartis.
Jens Kuhle received speaker fees, research support, travel support, and/or served on advisory boards by ECTRIMS, Swiss MS Society, Swiss National Research Foundation, (320030_160221), University of Basel, Bayer, Biogen, Genzyme, Merck, Novartis, Protagen AG, Roche, Teva.
Jutta Gärtner has received honoraria for lectures and consultancy fees from Bayer, Teva and Novartis.
Harald Kropshofer, Davorka Tomic and David Leppert are employees of Novartis Pharma AG.