Childhood Onset Chronic Inflammatory Demyelinating Polyneuropathy: Treatment and Outcome
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Purpose: To investigate the clinical features of childhood chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) as well as the treatment responses and outcomes. Methods: We retrospectively analyzed clinical features and treatment outcomes of 10 cases of childhood CIDP followed up for more than a year. Intravenous immunoglobulin (IVIG), IV steroid and plasmapheresis was considered as the first line therapy and used according to patient’s response. Immunosuppresant agent was added when patients did not respond to the first line therapy. Results: 10 CIDP cases (4 male and 6 female) were enrolled, and the median age of onset was 8 years (range 3-15 years). The mean duration of follow up was 46.2 months (range 12-82 months). Our patients manifested more frequently with subacute onset (n=8, 80%) and polyphasic course (n=6, 60%). All ten patients had lower extremity weakness at presentation (100%), six patients complained of upper extremity weakness (60%), and three patients reported sensory changes (30%). In the monophasic course (n=4), plasmapheresis showed a better treatment response (good 50%, partial 50%) compared to IVIG (good 0%, partial 75%) and steroids (good 0%, partial 0%), especially in progressive phases. In the polyphasic course (n=6), IVIG (good 50%, partial 50%) and plasmapheresis (good 40%, partial 60%) showed similar treatment responses. Five patients were refractory to first line treatment and received immunosuppressants; four patients received cyclosporine and achieved successful disease control without significant side effects. The overall long-term outcomes were favorable, with eight patients (80%) showing minimal symptoms and no relapse within 6 months. Conclusion: Our results suggest that early administration of plasmapheresis in progressive monophasic course and cyclosporine in refractory polyphasic course may be effective in childhood CIDP.
Abstract
Purpose: To investigate the clinical features of childhood chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) as well as the treatment responses and outcomes. Methods: We retrospectively analyzed clinical features and treatment outcomes of 10 cases of childhood CIDP followed up for more than a year. Intravenous immunoglobulin (IVIG), IV steroid and plasmapheresis was considered as the first line therapy and used according to patient’s response. Immunosuppresant agent was added when patients did not respond to the first line therapy. Results: 10 CIDP cases (4 male and 6 female) were enrolled, and the median age of onset was 8 years (range 3-15 years). The mean duration of follow up was 46.2 months (range 12-82 months). Our patients manifested more frequently with subacute onset (n=8, 80%) and polyphasic course (n=6, 60%). All ten patients had lower extremity weakness at presentation (100%), six patients complained of upper extremity weakness (60%), and three patients reported sensory changes (30%). In the monophasic course (n=4), plasmapheresis showed a better treatment response (good 50%, partial 50%) compared to IVIG (good 0%, partial 75%) and steroids (good 0%, partial 0%), especially in progressive phases. In the polyphasic course (n=6), IVIG (good 50%, partial 50%) and plasmapheresis (good 40%, partial 60%) showed similar treatment responses. Five patients were refractory to first line treatment and received immunosuppressants; four patients received cyclosporine and achieved successful disease control without significant side effects. The overall long-term outcomes were favorable, with eight patients (80%) showing minimal symptoms and no relapse within 6 months. Conclusion: Our results suggest that early administration of plasmapheresis in progressive monophasic course and cyclosporine in refractory polyphasic course may be effective in childhood CIDP.
참고문헌 (36)
- Vallat JM, "Chronic inflammatory demyelinating polyradiculoneuropathy: diagnostic and therapeutic challenges for a treatable condition", Lancet Neurol, 9, pp.402~412 (2010)
- Connolly AM, "Chronic inflammatory demyelinating polyneuropathy in childhood", Pediatr Neurol, 24, pp.177~182 (2001)
- Riekhoff AG, "Childhood chronic inflammatory demyelinating polyneuroradiculopathy-- three cases and a review of the literature", Eur J Paediatr Neurol, 16, pp.315~331 (2012)
- Korinthenberg R., "Chronic inflammatory demyelinating polyradiculoneuropathy in children and their response to treatment", Neuropediatrics, 30, pp.190~196 (1999)
- Nevo Y, "Childhood chronic inflammatory demyelinating neuropathies : clinical course and long-term follow-up", Neurology, 47, pp.98~102 (1996)
- Hattori N, "Clinicopathological features of chronic inflammatory demyelinating polyradiculoneuropathy in childhood", J Neurol Sci, 154, pp.66~71 (1998)
- Rossignol E, "Evolution and treatment of childhood chronic inflammatory polyneuropathy", Pediatr Neurol, 36, pp.88~94 (2007)
- Ryan MM, "Childhood chronic inflammatory demyelinating polyneuropathy: clinical course and long-term outcome", Neuromuscul Disord, 10, pp.398~406 (2000)
- Simmons Z, "Chronic inflammatory demyelinating polyradiculoneuropathy in children: I. Presentation, electrodiagnostic studies, and initial clinical course, with comparison to adults", Muscle Nerve, 20, pp.1008~1015 (1997)
- Sladky JT., "What is the best initial treatment for childhood chronic inflammatory demyelinating polyneuropathy: corticosteroids or intravenous immunoglobulin?", Muscle Nerve, 38, pp.1638~1643 (2008)
- Magy L, "Evidence-based treatment of chronic immunemediated neuropathies", Expert Opin Pharmacother, 10, pp.1741~1754 (2009)
- Mahdi-Rogers M, "Immunomodulatory treatment other than corticosteroids, immunoglobulin and plasma exchange for chronic inflammatory demyelinating polyradiculoneuropathy", Cochrane Database Syst Rev, pp.CD003280~ (2013)
- Van den Bergh PY, "European Federation of Neurological Societies", Peripheral Nerve Society guideline on management of chronic inflammatory demyelinating polyradiculoneuropathy: report of a joint task force of the European Federation of Neurological Societies and the Peripheral Nerve Society - first revision, 2010, pp.17, 356~363
- van Swieten JC, "Interobserver agreement for the assessment of handicap in stroke patients", Stroke, 19, pp.604~607 (1988)
- McMillan HJ, "Childhood chronic inflammatory demyelinating polyradiculoneuropathy: combined analysis of a large cohort and eleven published series", Neuromuscul Disord, 23, pp.103~111 (2013)
- Mygland A, "Onset and course of chronic inflammatory demyelinating polyneuropathy", Muscle Nerve, 31, pp.589~593 (2005)
- Kuwabara S, "Long term prognosis of chronic inflammatory demyelinating polyneuropathy: a five year follow up of 38 cases", J Neurol Neurosurg Psychiatry, 77, pp.66~70 (2006)
- Nevo Y, "88th ENMC international workshop: childhood chronic inflammatory demyelinating polyneuropathy (including revised diagnostic criteria), Naarden, The Netherlands, December 8-10, 2000", Neuromuscul Disord, 12, pp.195~200 (2002)
- Köller H, "Chronic inflammatory demyelinating polyneuropathy", N Engl J Med, 352, pp.1343~1356 (2005)
- Odaka M, "Intractable chronic inflammatory demyelinating polyneuropathy treated successfully with ciclosporin", J Neurol Neurosurg Psychiatry, 76, pp.1115~1120 (2005)
- Cocito D, "A nationwide retrospective analysis on the effect of immune therapies in patients with chronic inflammatory demyelinating polyradiculoneuropathy", Eur J Neurol, 17, pp.289~294 (2010)
- Viala K, "A current view of the diagnosis, clinical variants, response to treatment and prognosis of chronic inflammatory demyelinating polyradiculoneuropathy", J Peripher Nerv Syst, 15, pp.50~56 (2010)
- Matsuda M, "Cyclosporin A in treatment of refractory patients with chronic inflammatory demyelinating polyradiculoneuropathy", J Neurol Sci, 224, pp.29~35 (2004)
- Mahattanakul W, "Treatment of chronic inflammatory demyelinating polyneuropathy with cyclosporin-A", J Neurol Neurosurg Psychiatry, 60, pp.185~187 (1996)
- Barnett MH, "Cyclosporin A in resistant chronic inflammatory demyelinating polyradiculoneuropathy", Muscle Nerve, 21, pp.454~460 (1998)
- Gorson KC., "An update on the management of chronic inflammatory demyelinating polyneuropathy", Ther Adv Neurol Disord, 5, pp.359~373 (2012)
- Visudtibhan A, "Cyclosporine in chronic inflammatory demyelinating polyradiculoneuropathy", Pediatr Neurol, 33, pp.368~372 (2005)
- Sabatelli M, "Pure motor chronic inflammatory demyelinating polyneuropathy", J Neurol, 248, pp.772~777 (2001)
- Cocito D, "Immunosuppressive treatment in refractory chronic inflammatory demyelinating polyradiculoneuropathy. A nationwide retrospective analysis", Eur J Neurol, 18, pp.1417~1421 (2011)
- Bosboom WM, "The diagnostic value of sural nerve T cells in chronic inflammatory demyelinating polyneuropathy", Neurology, 53, pp.837~845 (1999)
- Sanvito L, "Circulating subsets and CD4(+)CD25(+) regulatory T cell function in chronic inflammatory demyelinating polyradiculoneuropathy", Autoimmunity, 42, pp.667~677 (2009)
- Chi LJ, "Distribution of Th17 cells and Th1 cells in peripheral blood and cerebrospinal fluid in chronic inflammatory demyelinating polyradiculoneuropathy", J Peripher Nerv Syst, 15, pp.345~356 (2010)
- Dyck PJ, "Combined azathioprine and prednisone in chronic inflammatory-demyelinating polyneuropathy", Neurology, 35, pp.1173~1176 (1985)
- RMC Trial Group, "Randomised controlled trial of methotrexate for chronic inflammatory demyelinating polyradiculoneuropathy (RMC trial): a pilot, multicentre study", Lancet Neurol, 8, pp.158~164 (2009)
- Hadden RD, "Randomized trial of interferon beta-1a in chronic inflammatory demyelinating polyradiculoneuropathy", Neurology, 53, pp.57~61 (1999)
- Hughes RA, "Intramuscular interferon beta-1a in chronic inflammatory demyelinating polyradiculoneuropathy", Neurology, 74, pp.651~657 (2010)