Childhood chronic inflammatory demyelinating polyradiculoneuropathy: Combined analysis of a large cohort and eleven published series |
| |
| Authors: | Hugh J. McMillan Peter B. Kang H. Royden Jones Basil T. Darras |
| |
| Affiliation: | 1. Institute of Neurogenetics, University of Lübeck, Lübeck, Germany;2. Department of Pediatric and Adult Movement Disorders and Neuropsychiatry, Institute of Neurogenetics, University of Lübeck, Lübeck, Germany;1. Departments of Neurology and Pediatrics, Columbia University Irving Medical Center, New York, NY 10032, USA;2. Unit of Neuromuscular and Neurodegenerative Disorders, Post-Graduate Bambino Gesù Children''s Research Hospital, IRCCS, Rome, Italy;3. Department of Neurology, Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA;4. Departments of Medical Genetics and Pediatrics, National Taiwan University Hospital, Taipei, Taiwan;5. Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, USA;6. Division of Neurology, Department of Pediatrics, Nemours Children''s Hospital, Orlando, FL, USA;7. Department of Neuropediatrics and Muscle Disorders, Faculty of Medicine, University of Freiburg, Freiburg, Germany;8. Department of Neuropediatrics, University Medical Hospital, Bonn, Germany;9. Ann & Robert H. Lurie Children''s Hospital of Chicago, Chicago, IL, USA;10. Children''s Hospital of Colorado, University of Colorado School of Medicine, Aurora, CO, USA;11. Royal Children''s Hospital, University of Melbourne, Murdoch Children''s Research Institute, Melbourne, Australia;12. Department of Pediatrics and Neurology, University of Utah, Salt Lake City, UT, USA;13. Department of Pediatric Neurology, Hacettepe University, Ankara, Turkey;14. Sidra Medicine, Department of Pediatrics, Qatar Foundation, Doha, Qatar;15. Division of Clinical and Metabolic Genetics, Department of Pediatrics, Hamad Medical Corporation, Doha, Qatar;p. NEMO Clinical Center – NEuroMuscular Omniservice, Milan, Italy;q. Department of Biomedical Sciences for Health, Università degli Studi di Milano, Milan, Italy;r. Graduate Institute of Clinical Medicine, College of Medicine, Kaohsiung Medical University; Departments of Pediatrics and Laboratory Medicine, Kaohsiung Medical University Hospital, Kaohsiung Medical University, Kaohsiung, Taiwan;s. Department of Neurology, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA;t. Biogen, Cambridge, MA, USA;u. Biogen, Maidenhead, Berkshire, UK;2. Randall Division for Cell and Molecular Biophysics, Muscle Signalling Section, London, United Kingdom;3. Department of Basic and Clinical Neuroscience, IoPPN, King''s College, London, United Kingdom;4. The Dubowitz Neuromuscular Centre, Developmental Neurosciences Programme, UCL Great Ormond Street Institute of Child Health & Great Ormond Street Hospital for Children, London, United Kingdom |
| |
| Abstract: | ![]()
The clinical presentation, disease course, response to treatment, and long-term outcome of thirty childhood chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) patients are presented representing the largest cohort reported to date. Most children (60%) presented with chronic (>8-weeks) symptom-onset while a smaller proportion showed sub-acute (4–8 weeks) or acute (‘‘GBS-like’’; <4 weeks) onset of disease. No gender predilection was observed. The majority of patients had a relapsing (70%) versus a monophasic (30%) temporal profile. Most received initial IVIG monotherapy; 80% showing a good response. Long-term follow-up (mean = 3.8 years) was available for 23 patients; 45% were off all immunomodulatory medications, demonstrating no detectable (55%) or minimal (43%) clinical deficits. Our data were compared with 11 previously published childhood CIDP series providing a comprehensive review of 143 childhood CIDP cases. The combined initial or first-line treatment response across all studies was favourable for IVIG (79% patients) and corticosteroids (84% patients). Response to first-line plasma exchange was poor (only 14% patients improved) although it may offer some transient or partial benefit as an adjuvant or temporary therapy for selected patients. The combined long-term outcome of our cohort and the literature reveals a favourable prognosis for most patients. The combined modified Rankin scale decreased from 3.7 (at presentation) to 0.7 (at last follow-up). This review provides important data pertaining to clinical course, treatment response and long-term outcome of this relatively uncommon paediatric autoimmune disease. |
| |
| Keywords: | |
| 本文献已被 ScienceDirect 等数据库收录！ |
|
