Contributions
Abstract: P863
Type: Poster
Abstract Category: Clinical aspects of MS - 3 Paediatric MS
Background: Available disease modifying therapies (DMTs) appear to be safe and effective in children and adolescents and are included in paediatric multiple sclerosis (MS) treatment guidelines. However, only small observational studies and retrospective case series have been reported to date. This systematic literature review identifies available studies assessing the effectiveness, safety, and tolerability of DMTs for paediatric patients with MS.
Methods: A systematic search was performed in MEDLINE, EMBASE, and the Cochrane Central Register of Controlled Trials on November 7, 2016 to identify randomised controlled trials (RCTs) and observational studies. Manual searches of five conference proceedings published 2014-2016 were also performed. Clinical trial registries were searched to identify ongoing or planned RCTs.
Results: From 2499 records and 236 conference abstracts, 44 publications pertaining to 34 studies were included (32 articles and 12 conference abstracts). Study designs were predominantly retrospective. Across the reported studies, the sizes of the population ranged from 4 to 307 patients. The mean age at treatment initiation spanned 3.3-17.0 years. The proportion of females ranged 25%-100%, and the majority of study populations were ≥50% female. Across the studies, prior to initiation of therapy, the mean Expanded Disability Status Scale (EDSS) ranged 0.6-3.7, and the mean annualised relapse rate (ARR) spanned 1.1-3.8 relapses per year. The treatments that were most to least commonly assessed across the studies included intramuscular (IM) interferon beta (IFNB) 1a, natalizumab, subcutaneous IFNB-1a, IFNB-1b, glatiramer acetate (GA), pooled treatments of IFNBs or GA, fingolimod, rituximab, dimethyl fumarate, mitoxantrone, cyclophosphamide, and daclizumab. Post treatment mean ARR ranged 0-1.6 relapses per year, and post treatment mean EDSS spanned 0.6-2.4. Adverse events from most to least commonly reported included injection reactions, abnormal liver enzyme levels, flu-like symptoms, headaches, fatigue, nausea, myalgia, anaemia, gastrointestinal related events, and fever. Currently, there are three ongoing RCTs, assessing fingolimod vs IM IFNB-1a, teriflunomide vs placebo, and dimethyl fumarate vs IM IFNB-1a.
Conclusions: Most of the evidence on the effectiveness and safety of paediatric MS treatments is based on retrospective studies. Results of ongoing RCTs are expected to strengthen evidence-based treatment decisions.
Disclosure: This study was sponsored by Novartis Pharmaceuticals Corporation.
Lauren B. Krupp has received consulting fees, royalties, or research funding from Abbvie, Biogen, EMD Serono, Genzyme, Johnson & Johnson, Novartis Pharmaceuticals Corporation, Pfizer, RedHill Biopharma, and Sanofi.
Maria Cecilia Vieira, Fernanda C. Boulos, and Haley Toledano are employees of Novartis Pharmaceuticals Corporation.
Desi Peneva, Mir-Masoud Pourrahmat, and Eric Druyts are employees of Precision Health Economics, a health economics consultancy providing services to the life science industry. Novartis Pharmaceuticals Corporation provided financial support for this research to Precision Health Economics.
Abstract: P863
Type: Poster
Abstract Category: Clinical aspects of MS - 3 Paediatric MS
Background: Available disease modifying therapies (DMTs) appear to be safe and effective in children and adolescents and are included in paediatric multiple sclerosis (MS) treatment guidelines. However, only small observational studies and retrospective case series have been reported to date. This systematic literature review identifies available studies assessing the effectiveness, safety, and tolerability of DMTs for paediatric patients with MS.
Methods: A systematic search was performed in MEDLINE, EMBASE, and the Cochrane Central Register of Controlled Trials on November 7, 2016 to identify randomised controlled trials (RCTs) and observational studies. Manual searches of five conference proceedings published 2014-2016 were also performed. Clinical trial registries were searched to identify ongoing or planned RCTs.
Results: From 2499 records and 236 conference abstracts, 44 publications pertaining to 34 studies were included (32 articles and 12 conference abstracts). Study designs were predominantly retrospective. Across the reported studies, the sizes of the population ranged from 4 to 307 patients. The mean age at treatment initiation spanned 3.3-17.0 years. The proportion of females ranged 25%-100%, and the majority of study populations were ≥50% female. Across the studies, prior to initiation of therapy, the mean Expanded Disability Status Scale (EDSS) ranged 0.6-3.7, and the mean annualised relapse rate (ARR) spanned 1.1-3.8 relapses per year. The treatments that were most to least commonly assessed across the studies included intramuscular (IM) interferon beta (IFNB) 1a, natalizumab, subcutaneous IFNB-1a, IFNB-1b, glatiramer acetate (GA), pooled treatments of IFNBs or GA, fingolimod, rituximab, dimethyl fumarate, mitoxantrone, cyclophosphamide, and daclizumab. Post treatment mean ARR ranged 0-1.6 relapses per year, and post treatment mean EDSS spanned 0.6-2.4. Adverse events from most to least commonly reported included injection reactions, abnormal liver enzyme levels, flu-like symptoms, headaches, fatigue, nausea, myalgia, anaemia, gastrointestinal related events, and fever. Currently, there are three ongoing RCTs, assessing fingolimod vs IM IFNB-1a, teriflunomide vs placebo, and dimethyl fumarate vs IM IFNB-1a.
Conclusions: Most of the evidence on the effectiveness and safety of paediatric MS treatments is based on retrospective studies. Results of ongoing RCTs are expected to strengthen evidence-based treatment decisions.
Disclosure: This study was sponsored by Novartis Pharmaceuticals Corporation.
Lauren B. Krupp has received consulting fees, royalties, or research funding from Abbvie, Biogen, EMD Serono, Genzyme, Johnson & Johnson, Novartis Pharmaceuticals Corporation, Pfizer, RedHill Biopharma, and Sanofi.
Maria Cecilia Vieira, Fernanda C. Boulos, and Haley Toledano are employees of Novartis Pharmaceuticals Corporation.
Desi Peneva, Mir-Masoud Pourrahmat, and Eric Druyts are employees of Precision Health Economics, a health economics consultancy providing services to the life science industry. Novartis Pharmaceuticals Corporation provided financial support for this research to Precision Health Economics.