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B cell depletion for autoimmune diseases in paediatric patients

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Abstract

Data on B cell depletion therapy in severe autoimmune diseases in paediatric patients are very limited. We conducted a retrospective cohort study and recruited patients who were treated with rituximab (RTX) and followed up for at least 6 months through the German societies of paediatric rheumatology and nephrology. The aim was to describe the spectrum of autoimmune disorders for which RTX was used and to describe the applied therapeutic regimens, the observed efficacy, as well as potential immunological side effects. The need to develop standard treatment guidelines for future trials should be discussed. Sixty-five patients were included. Nineteen patients suffered from systemic lupus erythematosus, 13 from vasculitic disorders, 12 from hematological autoimmune diseases, 5 from mixed connective tissue disorders, 4 from juvenile idiopathic arthritis, and 9 had other autoimmune diseases. Adverse, infusion-related events were reported in 12/65 (18%) patients. Considering laboratory and clinical parameters, 13 patients (22%) were in complete remission, 31 (52%) were in partial remission, 6 (10%) were unchanged and 10 (17%) had progressed after 6 months. In 46% of the patients, the steroid dose could be more than halved. IgG, IgM and IgA decreased from normal levels prior to RTX therapy to below normal levels at 6 months in 2/22 (9%), 10/21 (48%), and 4/22 (18%) patients, respectively. Immunoglobulin deficiency or prolonged CD20 depletion was reported in eight patients after an observation period longer than 12 months. RTX therapy led to a perceivable reduction in disease activity. However, long-term immunological alterations may occur in more than 10% of the patients. Guidelines and protocols for off-label therapy are desirable to document reasonable follow-up data. Controlled prospective studies for RTX therapies in children with standardised therapeutic and diagnostic protocols are urgently needed.

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Disclosures

The study was supported by Roche Pharma AG, Grenzach-Wyhlen and by “Kindness for Kids—Foundation for Children with Rare Diseases”. The sponsors of the study had no decisive role in study design, data collection, data analysis, data interpretation, and writing of the manuscript.

A.F. Jansson: received an unrestricted grant by Roche Pharma AG, Grenzach-Wyhlen, manufacturer of MabThera®.

C. Sengler: none

J. Kuemmerle-Deschner: none

B. Gruhn: none

A.B. Kranz: none

H. Lehmann: none

D. Kleinert: none

L. Pape: none

D. Moebius: none

J. Peitz: none

D. Foell: none

H.J. Girschick: none

I. Foeldvari: none

J.P. Haas: none

D Haffner: none

V. Grote: none

Author information

Authors and Affiliations

  1. Dr. von Hauner Children’s Hospital, Ludwig-Maximilians University, Munich, Germany

    Annette F. Jansson & Veit Grote

  2. Universitätskinderklinik Charité, Berlin, Germany

    Claudia Sengler

  3. Universitätsklinik f. Kinderheilkunde u. Jugendmedizin, Tübingen, Germany

    Jasmin Kuemmerle-Deschner

  4. Klinik für Kinder- und Jugendmedizin, Friedrich-Schiller-Universität, Jena, Germany

    Bernd Gruhn

  5. Universitäts-Kinderklinik, Essen, Germany

    A. Birgitta Kranz

  6. Universitätskinderklinik, Gießen, Germany

    Hartwig Lehmann

  7. Kinderklinik, Memmingen, Germany

    Daniela Kleinert

  8. Kinderklinik der Medizinischen Hochschule, Hannover, Germany

    Lars Pape

  9. Carl-Thiem Klinikum, Cottbus, Germany

    Dagmar Moebius

  10. Unikinderklinik, Köln, Germany

    Joachim Peitz

  11. Universitätskinderklinik, Münster, Germany

    Dirk Foell

  12. Vivantes Hospital Friedrichhain, Berlin, Germany

    Hermann J. Girschick

  13. Zentrum für Kinder-und Jugendrheumatologie am Klinikum Eilbek, Hamburg, Germany

    Ivan Foeldvari

  14. Deutsches Zentrum für Kinder- und Jugendrheumatologie, Garmisch-Partenkirchen, Germany

    Johannes P. Haas

  15. Universitäts-Kinder- und Jugendklinik, Rostock, Germany

    Dieter Haffner

  16. Dr. von Hauner Children’s Hospital, Ludwig-Maximilians University, Lindwurmstr. 4, 80337, Munich, Germany

    Veit Grote

Authors
  1. Annette F. Jansson
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  2. Claudia Sengler
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  10. Ivan Foeldvari
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  11. Dieter Haffner
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  12. Johannes P. Haas
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  13. Dagmar Moebius
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  14. Dirk Foell
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  15. Joachim Peitz
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  16. Veit Grote
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Correspondence to Veit Grote.

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Jansson, A.F., Sengler, C., Kuemmerle-Deschner, J. et al. B cell depletion for autoimmune diseases in paediatric patients. Clin Rheumatol 30, 87–97 (2011). https://doi.org/10.1007/s10067-010-1630-0

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  • DOI: https://doi.org/10.1007/s10067-010-1630-0

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