Safety and efficacy of rituximab in the treatment of paediatric autoimmune diseases

Česká verze

Authors: V. Lukášová ¹; H. Schneiderová ²; V. Fiamoli ¹; O. Zapletal ¹; S. Köhlerová ¹; J. Blatný ¹
Authors place of work: Oddělení dětské hematologie FN Brno a Masarykova univerzita Brno ¹; Pediatrická klinika FN Brno a Masarykova univerzita Brno ²
Published in the journal: Transfuze Hematol. dnes,24, 2018, No. 4, p. 270-282.
Category: Původní práce

Summary

Rituximab is a monoclonal antibody directed against CD20 molecules expressed on the surface of B lymphocytes. After binding to the cell surface, rituximab induces B cell death and this leads to transient depletion of B cells associated with a reduction/disappearance of pathological (auto) antibodies. This effect is used in the treatment of autoimmune diseases (off-label mode).

Aim of this work is to assess the safety and efficacy of rituximab in a group of 11 paediatric patients with autoimmune diseases. Data were collected retrospectively, thus the results of certain parameters were not available for all enrolled patients. From a safety point of view, attention was focused on both acute drug-related side effects and long-term adverse effects, including long-term B cell depletion, hypogammaglobulinemia and serious infections. Effectivity of the treatment was assessed as achievement of partial and/or complete remission criteria for respective diagnoses.

In the study population, acute adverse reactions were reported in 5 patients (45.5% of all patients). In 2 patients (25% of patients with data available), even after substantial follow-up, B cell recovery did not return to physiological values. Moderate hypogammaglobulinemia after therapy was seen in 2 patients (25% of patients with data available). Serious infections after therapy were reported in 1 patient (9.1% of all patients). We considered rituximab to be effective in 45.5% of our patients. Complete remission was achieved in 9% of patients, partial remission in 36.5% of patients. Long-term remission lasting more than 1 year was reached and maintained in 27.3% of the monitored patients.

Our results correspond to current recommendations, that rituximab should be considered as second-line treatment in patients with autoimmune disease in children.

Key words:

rituximab – autoimmune diseases in children – adverse events – hypogammaglobulinemia – remission

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