ArticleNeurology2019
Clinical trials of disease-modifying agents in pediatric MS: Opportunities, challenges, and recommendations from the IPMSSG.
Erratum issuedOpen access · hybridAbstract readConsensus Statement
In one paragraph
Article in Neurology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 30 papers, 3 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
30citing papers in PubMed, 3 pooled it
7.1field-weighted citation impact, top 2% of its field
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
30 citing papers in PubMed, 3 syntheses or guidelines pooled it, 73 citations in OpenAlex.
- Placebo in Paediatric Clinical Trials: Systematic Literature Review and Framework-Based Synthesis.Journal of paediatrics and child health · 2026Pooled it
- Natalizumab for multiple sclerosis.The Cochrane database of systematic reviews · 2025Pooled it
- Adult and pediatric relapsing multiple sclerosis phase II and phase III trial design and their primary end points: A systematic review.Clinical and translational science · 2024Pooled it
- A multicentre, prospective, randomized, open-label pragmatic trial to compare the effectiveness and safety of interferon beta-1a and glatiramer-acetate in paediatric patients affected by Multiple Sclerosis.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2025Trial
- Transitioning from pediatric to adult multiple sclerosis care: Challenges, strategies and therapy.Journal of pediatric rehabilitation medicine · 2025Review
- Optimizing Drug Selection in Children with Multiple Sclerosis: What Do We Know and What Remains Unanswered?Paediatric drugs · 2025Review
- Therapeutic Advances in Pediatric Multiple Sclerosis.Children (Basel, Switzerland) · 2025Review
- Comparative Effectiveness of Natalizumab, Fingolimod, and Injectable Therapies in Pediatric-Onset Multiple Sclerosis: A Registry-Based Study.Neurology · 2024Article
- A cross-sectional study of MRI features and the gut microbiome in pediatric-onset multiple sclerosis.Annals of clinical and translational neurology · 2024Article
- Multiple sclerosis in a 4-year-old boy: a case report and literature review.Frontiers in neurology · 2024Article
- Pediatric Multiple Sclerosis: Changing the Trajectory of Progression.Current neurology and neuroscience reports · 2023Review
- Current knowledge, challenges and innovations in developmental pharmacology: A combined conect4children Expert Group and European Society for Developmental, Perinatal and Paediatric Pharmacology White Paper.British journal of clinical pharmacology · 2022Review
- The Impact of Highly Effective Treatment in Pediatric-Onset Multiple Sclerosis: A Case Series.Children (Basel, Switzerland) · 2022Article
- Evolution of clinically isolated syndrome to pediatric-onset multiple sclerosis and a review of the literature.Turkish journal of medical sciences · 2022Review
- Multiple Sclerosis in Children: Differential Diagnosis, Prognosis, and Disease-Modifying Treatment.CNS drugs · 2022Review
- Improving pediatric multiple sclerosis interventional phase III study design: a meta-analysis.Therapeutic advances in neurological disorders · 2022Article
- Therapeutic Advances in Multiple Sclerosis.Frontiers in neurology · 2022Review
- Update on the management of multiple sclerosis during the COVID-19 pandemic and post pandemic: An international consensus statement.Journal of neuroimmunology · 2021Review
- Enhancing Mood, Cognition, and Quality of Life in Pediatric Multiple Sclerosis.Paediatric drugs · 2021Review
- COVID-19 and Treatment and Immunization of Children-The Time to Redefine Pediatric Age Groups is Here.Rambam Maimonides medical journal · 2021Review
Corrections and comments
- Erratum issued
Authors and funding
11 authors at 2 institutions in 7 countries.
Emmanuelle WaubantFrom the UCSF MS Center (E.W.), San Francisco, CA; The Children's Hospital of Philadelphia (B.B.), Perelman School of Medicine, University of Pennsylvania; Birmingham Children's Hospital (E.W.), UK; Department of Health Sciences (M.-P. S.), University of Genova and Ospedale Policlinico San Martino IRCCS; Department NEUROFARBA (M.-P.A.), University of Florence, Italy; IRCCS Fondazione Don Carlo Gnocchi (M.-P.A.), Florence, Italy; Department of Neurology (R.H.), Erasmus MC, Rotterdam, the Netherlands; MS Comprehensive Care Center at NYU Langone (L.K.), New York, NY; Division of Paediatric Neurology (K.R.), Children's Hospital Datteln, University Witten/Herdecke, Datteln, Germany; Pediatric MS Center (S.T.), Department of Neurology, National Pediatric Hospital Dr. Garrahan, Buenos Aires, Argentina; and Partners Pediatric MS Center (T.C.), Massachusetts General Hospital, Boston. emmanuelle.waubant@ucsf.edu.
Brenda BanwellFrom the UCSF MS Center (E.W.), San Francisco, CA; The Children's Hospital of Philadelphia (B.B.), Perelman School of Medicine, University of Pennsylvania; Birmingham Children's Hospital (E.W.), UK; Department of Health Sciences (M.-P. S.), University of Genova and Ospedale Policlinico San Martino IRCCS; Department NEUROFARBA (M.-P.A.), University of Florence, Italy; IRCCS Fondazione Don Carlo Gnocchi (M.-P.A.), Florence, Italy; Department of Neurology (R.H.), Erasmus MC, Rotterdam, the Netherlands; MS Comprehensive Care Center at NYU Langone (L.K.), New York, NY; Division of Paediatric Neurology (K.R.), Children's Hospital Datteln, University Witten/Herdecke, Datteln, Germany; Pediatric MS Center (S.T.), Department of Neurology, National Pediatric Hospital Dr. Garrahan, Buenos Aires, Argentina; and Partners Pediatric MS Center (T.C.), Massachusetts General Hospital, Boston.
Evangeline WassmerFrom the UCSF MS Center (E.W.), San Francisco, CA; The Children's Hospital of Philadelphia (B.B.), Perelman School of Medicine, University of Pennsylvania; Birmingham Children's Hospital (E.W.), UK; Department of Health Sciences (M.-P. S.), University of Genova and Ospedale Policlinico San Martino IRCCS; Department NEUROFARBA (M.-P.A.), University of Florence, Italy; IRCCS Fondazione Don Carlo Gnocchi (M.-P.A.), Florence, Italy; Department of Neurology (R.H.), Erasmus MC, Rotterdam, the Netherlands; MS Comprehensive Care Center at NYU Langone (L.K.), New York, NY; Division of Paediatric Neurology (K.R.), Children's Hospital Datteln, University Witten/Herdecke, Datteln, Germany; Pediatric MS Center (S.T.), Department of Neurology, National Pediatric Hospital Dr. Garrahan, Buenos Aires, Argentina; and Partners Pediatric MS Center (T.C.), Massachusetts General Hospital, Boston.
Maria-Pia SormaniFrom the UCSF MS Center (E.W.), San Francisco, CA; The Children's Hospital of Philadelphia (B.B.), Perelman School of Medicine, University of Pennsylvania; Birmingham Children's Hospital (E.W.), UK; Department of Health Sciences (M.-P. S.), University of Genova and Ospedale Policlinico San Martino IRCCS; Department NEUROFARBA (M.-P.A.), University of Florence, Italy; IRCCS Fondazione Don Carlo Gnocchi (M.-P.A.), Florence, Italy; Department of Neurology (R.H.), Erasmus MC, Rotterdam, the Netherlands; MS Comprehensive Care Center at NYU Langone (L.K.), New York, NY; Division of Paediatric Neurology (K.R.), Children's Hospital Datteln, University Witten/Herdecke, Datteln, Germany; Pediatric MS Center (S.T.), Department of Neurology, National Pediatric Hospital Dr. Garrahan, Buenos Aires, Argentina; and Partners Pediatric MS Center (T.C.), Massachusetts General Hospital, Boston.
Maria-Pia AmatoFrom the UCSF MS Center (E.W.), San Francisco, CA; The Children's Hospital of Philadelphia (B.B.), Perelman School of Medicine, University of Pennsylvania; Birmingham Children's Hospital (E.W.), UK; Department of Health Sciences (M.-P. S.), University of Genova and Ospedale Policlinico San Martino IRCCS; Department NEUROFARBA (M.-P.A.), University of Florence, Italy; IRCCS Fondazione Don Carlo Gnocchi (M.-P.A.), Florence, Italy; Department of Neurology (R.H.), Erasmus MC, Rotterdam, the Netherlands; MS Comprehensive Care Center at NYU Langone (L.K.), New York, NY; Division of Paediatric Neurology (K.R.), Children's Hospital Datteln, University Witten/Herdecke, Datteln, Germany; Pediatric MS Center (S.T.), Department of Neurology, National Pediatric Hospital Dr. Garrahan, Buenos Aires, Argentina; and Partners Pediatric MS Center (T.C.), Massachusetts General Hospital, Boston.
Rogier HintzenFrom the UCSF MS Center (E.W.), San Francisco, CA; The Children's Hospital of Philadelphia (B.B.), Perelman School of Medicine, University of Pennsylvania; Birmingham Children's Hospital (E.W.), UK; Department of Health Sciences (M.-P. S.), University of Genova and Ospedale Policlinico San Martino IRCCS; Department NEUROFARBA (M.-P.A.), University of Florence, Italy; IRCCS Fondazione Don Carlo Gnocchi (M.-P.A.), Florence, Italy; Department of Neurology (R.H.), Erasmus MC, Rotterdam, the Netherlands; MS Comprehensive Care Center at NYU Langone (L.K.), New York, NY; Division of Paediatric Neurology (K.R.), Children's Hospital Datteln, University Witten/Herdecke, Datteln, Germany; Pediatric MS Center (S.T.), Department of Neurology, National Pediatric Hospital Dr. Garrahan, Buenos Aires, Argentina; and Partners Pediatric MS Center (T.C.), Massachusetts General Hospital, Boston.
Lauren KruppFrom the UCSF MS Center (E.W.), San Francisco, CA; The Children's Hospital of Philadelphia (B.B.), Perelman School of Medicine, University of Pennsylvania; Birmingham Children's Hospital (E.W.), UK; Department of Health Sciences (M.-P. S.), University of Genova and Ospedale Policlinico San Martino IRCCS; Department NEUROFARBA (M.-P.A.), University of Florence, Italy; IRCCS Fondazione Don Carlo Gnocchi (M.-P.A.), Florence, Italy; Department of Neurology (R.H.), Erasmus MC, Rotterdam, the Netherlands; MS Comprehensive Care Center at NYU Langone (L.K.), New York, NY; Division of Paediatric Neurology (K.R.), Children's Hospital Datteln, University Witten/Herdecke, Datteln, Germany; Pediatric MS Center (S.T.), Department of Neurology, National Pediatric Hospital Dr. Garrahan, Buenos Aires, Argentina; and Partners Pediatric MS Center (T.C.), Massachusetts General Hospital, Boston.
Kevin RostásyFrom the UCSF MS Center (E.W.), San Francisco, CA; The Children's Hospital of Philadelphia (B.B.), Perelman School of Medicine, University of Pennsylvania; Birmingham Children's Hospital (E.W.), UK; Department of Health Sciences (M.-P. S.), University of Genova and Ospedale Policlinico San Martino IRCCS; Department NEUROFARBA (M.-P.A.), University of Florence, Italy; IRCCS Fondazione Don Carlo Gnocchi (M.-P.A.), Florence, Italy; Department of Neurology (R.H.), Erasmus MC, Rotterdam, the Netherlands; MS Comprehensive Care Center at NYU Langone (L.K.), New York, NY; Division of Paediatric Neurology (K.R.), Children's Hospital Datteln, University Witten/Herdecke, Datteln, Germany; Pediatric MS Center (S.T.), Department of Neurology, National Pediatric Hospital Dr. Garrahan, Buenos Aires, Argentina; and Partners Pediatric MS Center (T.C.), Massachusetts General Hospital, Boston.
Silvia TenembaumFrom the UCSF MS Center (E.W.), San Francisco, CA; The Children's Hospital of Philadelphia (B.B.), Perelman School of Medicine, University of Pennsylvania; Birmingham Children's Hospital (E.W.), UK; Department of Health Sciences (M.-P. S.), University of Genova and Ospedale Policlinico San Martino IRCCS; Department NEUROFARBA (M.-P.A.), University of Florence, Italy; IRCCS Fondazione Don Carlo Gnocchi (M.-P.A.), Florence, Italy; Department of Neurology (R.H.), Erasmus MC, Rotterdam, the Netherlands; MS Comprehensive Care Center at NYU Langone (L.K.), New York, NY; Division of Paediatric Neurology (K.R.), Children's Hospital Datteln, University Witten/Herdecke, Datteln, Germany; Pediatric MS Center (S.T.), Department of Neurology, National Pediatric Hospital Dr. Garrahan, Buenos Aires, Argentina; and Partners Pediatric MS Center (T.C.), Massachusetts General Hospital, Boston.
Tanuja ChitnisFrom the UCSF MS Center (E.W.), San Francisco, CA; The Children's Hospital of Philadelphia (B.B.), Perelman School of Medicine, University of Pennsylvania; Birmingham Children's Hospital (E.W.), UK; Department of Health Sciences (M.-P. S.), University of Genova and Ospedale Policlinico San Martino IRCCS; Department NEUROFARBA (M.-P.A.), University of Florence, Italy; IRCCS Fondazione Don Carlo Gnocchi (M.-P.A.), Florence, Italy; Department of Neurology (R.H.), Erasmus MC, Rotterdam, the Netherlands; MS Comprehensive Care Center at NYU Langone (L.K.), New York, NY; Division of Paediatric Neurology (K.R.), Children's Hospital Datteln, University Witten/Herdecke, Datteln, Germany; Pediatric MS Center (S.T.), Department of Neurology, National Pediatric Hospital Dr. Garrahan, Buenos Aires, Argentina; and Partners Pediatric MS Center (T.C.), Massachusetts General Hospital, Boston.
IPMSSG
Australian National University · AUOspedale Policlinico San Martino · IT
Funding
The Radiologically Isolated Syndrome in ChildrenK23NS101099 · NINDS · YALE UNIVERSITY · PI MAKHANI, NAILA · 2017 to 2022
$792kNINDS NIH HHS K23 NS101099
Abstract
objectiveThe impetus for this consensus discussion was to recommend clinical trial designs that can deliver high-quality data for effective therapies for pediatric patients, in a reasonable timeframe, with a key focus on short- and long-term safety.
methodsThe International Pediatric Multiple Sclerosis Study Group convened a meeting of experts to review the advances in the understanding of pediatric-onset multiple sclerosis (MS) and the advent of clinical trials for this population.
resultsIn the last few years, convincing evidence has emerged that the biological processes involved in MS are largely shared across the age span. As such, treatments proven efficacious for the care of adults with MS have a biological rationale for use in pediatric MS given the relapsing-remitting course at onset and high relapse frequency. There are also ethical considerations on conducting clinical trials in this age group including the use of placebo owing to highly active disease. It is imperative to reconsider study design and implementation based on what information is needed. Are studies needed for efficacy or should safety be the primary goal? Further, there have been major recruitment challenges in recently completed and ongoing pediatric MS trials. Phase 3 trials for every newly approved therapy for adult MS in the pediatric MS population are simply not feasible.
conclusionsA primary goal is to ensure high-quality evidence-based treatment for children and adolescents with MS, which will improve our understanding of the safety of these agents and remove regulatory or insurance-based limitations in access to treatment.
Indexed as
Clinical Trials as TopicAdolescentChildEvidence-Based MedicineHumansMultiple Sclerosis, Relapsing-Remitting
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