Rituximab in paediatric onset multiple sclerosis: a case series

被引:48
作者
Salzer, Jonatan [1 ]
Lycke, Jan [2 ]
Wickstrom, Ronny [3 ]
Naver, Hans [4 ]
Piehl, Fredrik [5 ]
Svenningsson, Anders [1 ]
机构
[1] Umea Univ, Neurol Sect, Dept Pharmacol & Clin Neurosci, S-90187 Umea, Sweden
[2] Sahlgrens Univ Hosp, Dept Clin Neurosci & Rehabil, Gothenburg, Sweden
[3] Karolinska Inst, Dept Womens & Childrens Hlth, Neuropaediat Unit, Stockholm, Sweden
[4] Uppsala Univ, Dept Neurol, Inst Neurosci, Uppsala, Sweden
[5] Karolinska Inst, Dept Clin Neurosci, Stockholm, Sweden
关键词
Paediatric onset multiple sclerosis; Observational study; Immunomodulatory treatment; Rituximab; NATALIZUMAB; THERAPEUTICS; THERAPY; COHORT;
D O I
10.1007/s00415-015-7979-x
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
Paediatric onset multiple sclerosis (POMS) is characterized by high inflammatory activity. No disease modifying treatment has been approved for POMS. The objective of this report was to report the use of rituximab, a B cell depleting monoclonal anti-CD20-antibody, in POMS. This is a retrospective case series at four specialized MS centres in Sweden. Participants were identified through the Swedish MS-registry and our own patient stocks. Data were collected through medical charts review. We identified 14 POMS patients treated with i.v. rituximab 500-1000 mg every 6th to 12th months. Median age at disease onset was 14.7 years, median age at rituximab treatment initiation was 16.5 years, and median treatment duration was 23.6 months. No relapses were reported, and the EDSS scores remained stable or decreased in 13 of 14 cases during rituximab treatment. Beyond 6 months from initiating rituximab treatment, only one new lesion was detected on MRI. No serious AEs were reported. The drug survival was 86 %. Our data indicate that rituximab treatment is safe, effective and well tolerated in children with MS. Nine POMS cases treated with rituximab have previously been published. They had higher disease activity pre-rituximab, but similar safety and efficacy outcomes after treatment. An RCT of rituximab in POMS is warranted.
引用
收藏
页码:322 / 326
页数:5
相关论文
共 21 条
[1]   Rituximab in relapsing-remitting multiple sclerosis: A 72-week, open-label, phase I trial [J].
Bar-Or, Amit ;
Calabresi, Peter A. J. ;
Arnlod, Douglas ;
Markowitz, Clyde ;
Shafer, Stuart ;
Kasper, Lloyd H. ;
Waubant, Ernmanuelle ;
Gazda, Suzanne ;
Fox, Robert J. ;
Panzara, Michael ;
Sarkar, Neena ;
Agarwal, Sunil ;
Smith, Craig H. .
ANNALS OF NEUROLOGY, 2008, 63 (03) :395-400
[2]   Rituximab Use in Pediatric Central Demyelinating Disease [J].
Beres, Shannon J. ;
Graves, Jennifer ;
Waubant, Emmanuelle .
PEDIATRIC NEUROLOGY, 2014, 51 (01) :114-118
[3]   Risk of Natalizumab-Associated Progressive Multifocal Leukoencephalopathy [J].
Bloomgren, Gary ;
Richman, Sandra ;
Hotermans, Christophe ;
Subramanyam, Meena ;
Goelz, Susan ;
Natarajan, Amy ;
Lee, Sophia ;
Plavina, Tatiana ;
Scanlon, James V. ;
Sandrock, Alfred ;
Bozic, Carmen .
NEW ENGLAND JOURNAL OF MEDICINE, 2012, 366 (20) :1870-1880
[4]   Consensus statement: evaluation of new and existing therapeutics for pediatric multiple sclerosis [J].
Chitnis, T. ;
Tenembaum, S. ;
Banwell, B. ;
Krupp, L. ;
Pohl, D. ;
Rostasy, K. ;
Yeh, E. A. ;
Bykova, O. ;
Wassmer, E. ;
Tardieu, M. ;
Kornberg, A. ;
Ghezzi, A. .
MULTIPLE SCLEROSIS JOURNAL, 2012, 18 (01) :116-127
[5]   Utility and safety of rituximab in pediatric autoimmune and inflammatory CNS disease [J].
Dale, Russell C. ;
Brilot, Fabienne ;
Duffy, Lisa V. ;
Twilt, Marinka ;
Waldman, Amy T. ;
Narula, Sona ;
Muscal, Eyal ;
Deiva, Kumaran ;
Andersen, Erik ;
Eyre, Michael R. ;
Eleftheriou, Despina ;
Brogan, Paul A. ;
Kneen, Rachel ;
Alper, Gulay ;
Anlar, Banu ;
Wassmer, Evangeline ;
Heineman, Kirsten ;
Hemingway, Cheryl ;
Riney, Catherine J. ;
Kornberg, Andrew ;
Tardieu, Marc ;
Stocco, Amber ;
Banwell, Brenda ;
Gorman, Mark P. ;
Benseler, Susanne M. ;
Lim, Ming .
NEUROLOGY, 2014, 83 (02) :142-150
[6]   Natalizumab in pediatric multiple sclerosis: results of a cohort of 55 cases [J].
Ghezzi, A. ;
Pozzilli, C. ;
Grimaldi, L. M. E. ;
Moiola, L. ;
Brescia-Morra, V. ;
Lugaresi, A. ;
Lus, G. ;
Rinaldi, F. ;
Rocca, M. A. ;
Trojano, M. ;
Bianchi, A. ;
Comi, G. ;
Filippi, M. .
MULTIPLE SCLEROSIS JOURNAL, 2013, 19 (08) :1106-1112
[7]   Daclizumab Use in Patients With Pediatric Multiple Sclerosis [J].
Gorman, Mark P. ;
Tillema, Jan-Mendelt ;
Ciliax, Annika M. ;
Guttmann, Charles R. G. ;
Chitnis, Tanuja .
ARCHIVES OF NEUROLOGY, 2012, 69 (01) :78-81
[8]  
Gorman MP, 2009, ARCH NEUROL-CHICAGO, V66, P54, DOI 10.1001/archneurol.2008.505
[9]   B-cell depletion with Rituximab in relapsing-remitting multiple sclerosis [J].
Hauser, Stephen L. ;
Waubant, Emmanuelle ;
Arnold, Douglas L. ;
Vollmer, Timothy ;
Antel, Jack ;
Fox, Robert J. ;
Bar-Or, Amit ;
Panzara, Michael ;
Sarkar, Neena ;
Agarwal, Sunil ;
Langer-Gould, Annette ;
Smith, Craig H. .
NEW ENGLAND JOURNAL OF MEDICINE, 2008, 358 (07) :676-688
[10]   Rituximab in Patients with Primary Progressive Multiple Sclerosis Results of a Randomized Double-Blind Placebo-Controlled Multicenter Trial [J].
Hawker, Kathleen ;
O'Connor, Paul ;
Freedman, Mark S. ;
Calabresi, Peter A. ;
Antel, Jack ;
Simon, Jack ;
Hauser, Stephen ;
Waubant, Emmanuelle ;
Vollmer, Timothy ;
Panitch, Hillel ;
Zhang, Jiameng ;
Chin, Peter ;
Smith, Craig H. .
ANNALS OF NEUROLOGY, 2009, 66 (04) :460-471