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PReS-FINAL-2038: Clinical efficacy and tolerability of tocilizumab in patients with persistant systemic JIA
© Mihailova et al.; licensee BioMed Central Ltd. 2013
Published: 5 December 2013
Tocilizumab was recommended for the treatment of systemic juvenile idiopathic arthritis (sJIA) in children whose disease has responded inadequately to non-steroidal anti-inflammatory drugs, systemic corticosteroids and methotrexate.
To study the efficacy and tolerability of Tocilizumab (TCZ) in patients with longstanding refractory to the conventional treatment and TNF-inhibitors sJIA.
Seven children in the age range 2-12 years (mean 6.3 years) with disease duration 1.2 - 9 years (mean 4.6 years) with active persistent sJIA have been enrolled in one-year prospective study. All 7 patients had oligoarticular joint involvement and received previous treatment with NSAIDS, Methotrexate and corticosteroids. 4/7 had concomitant therapy with Etanercept and 2/7 second DMARD. In all patients TCZ was administered immediately after discontinuation of the biological treatment or the second DMARD in a dose of 8 mg/kg for patients ≥ 30 kg and 12 mg/kg for patients < 30 kg every two weeks. At baseline all patients had active disease. Response to the treatment was measured according to the ACR Pedi criteria at the end of the 1st, 3rd, 6th, 9th and 12th month.
Clinical examination, laboratory investigations, growth parameters' assessment and screening for adverse events have been performed every 2 weeks.
At the end of the 1st month we found complete resolution of the systemic symptoms and acute phase response in all patients. At the end of the 3rd month, ACR Pedi 30, 50, 70 and 90 responses were achieved by 7 (100%), 5 (71%), 1(14%) and 0 (0%) patients, respectively. By 6th month the observed responses we as follows: ACR Pedi 30 - 7 pts (100%), ACR Pedi 50 - 7 pts (100%), ACR Pedi 70 - 5 pts (71%), ACR 90 - 1 patient (14%). At the end of 9th month ACR Pedi 30, 50, 70 and 90 responses were achieved by 7 (100%), 7 (100%), 6 (86%) and 4 (57%) patients. All patients maintained these parameters by 12th month.
After achieving control of the disease activity, we started to reduce the dose of steroids and/or Methotrexate leading to their discontinuation and monotherapy with TCZ in 4 patients. In all of these patients remission on medication was documented by 12th month. At the end of the observational period the linear growth velocity of all treated patients reached the average normal values per year of their age matched peers.
Some patients experienced mild and reversible adverse reactions such as: mild neutropenia or thrombocytopenia, slight increase of liver enzymes and increased cholesterol levels. All side effects resolved with the decrease of the dosage and did not lead to the discontinuation of the treatment.
Tocilizumab is an effective and well tolerated drug in children with systemic longstanding persistent JIA, refractory to the conventional treatment and TNF-inhibition. Monotherapy with TCZ is an appropriate option for controlling the disease activity, improving the physical growth and minimizing the overall toxicity of the treatment of this disease.
Disclosure of interest
This article is published under license to BioMed Central Ltd. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.