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B cell depletion for autoimmune diseases in paediatric patients
Annette F Jansson1, Claudia Sengler, Jasmin Kuemmerle-Deschner
1Ludwig-Maximilians University, Munich, Germany.
Insights
Rituximab (RTX) therapy shows efficacy in pediatric autoimmune diseases, reducing disease activity and steroid use. However, long-term B cell depletion can cause immunological side effects, necessitating standardized treatment guidelines.
Area of Science:
- Pediatric Rheumatology and Nephrology
- Immunology
- Pharmacology
Background:
- Limited data exists on B cell depletion therapy for severe autoimmune diseases in children.
- Rituximab (RTX) is a B cell depleting agent used off-label in pediatric autoimmune conditions.
Purpose of the Study:
- To describe the spectrum of autoimmune disorders treated with RTX in pediatric patients.
- To evaluate RTX therapeutic regimens, efficacy, and immunological side effects.
- To discuss the need for standardized treatment guidelines and future research.
Main Methods:
- Retrospective cohort study of 65 pediatric patients treated with RTX.
- Follow-up of at least 6 months.
- Data collected through German pediatric rheumatology and nephrology societies.
Main Results:
- RTX was used for systemic lupus erythematosus, vasculitis, hematological autoimmune diseases, and others.
- 18% experienced infusion-related adverse events.
- 22% achieved complete remission, 52% partial remission after 6 months.
- Steroid dose reduced by over half in 46% of patients.
- 9-48% showed decreased immunoglobulin levels (IgG, IgM, IgA).
- 8 patients developed immunoglobulin deficiency or prolonged CD20 depletion after >12 months.
Conclusions:
- Rituximab therapy effectively reduces disease activity in pediatric autoimmune diseases.
- Long-term immunological alterations, including immunoglobulin deficiency, can occur in over 10% of patients.
- Controlled prospective studies and standardized protocols are needed for RTX use in children.
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.
