Tocilizumab in the treatment of mixed connective tissue disease and overlap syndrome in children

Cabrera, Natalia; Duquesne, Agnès; Desjonquères, Marine; Larbre, Jean‐Paul; Lega, Jean‐Christophe; Fabien, Nicole; Belot, Alexandre

doi:10.1136/rmdopen-2016-000271

Cited by 13 publications

(4 citation statements)

References 14 publications

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“…A case report described an adult patient with concomitant DM, SSc, and RA whose disease activity improved with tocilizumab therapy [31]. In one case, a 12-year-old patient with juvenile DM, treated with glucocorticoids and methotrexate (MTX), relapsed with Raynaud’s phenomenon, sclerodactyly, and polyarthritis [32]. After several drugs failed, tocilizumab was initiated, but improved joint manifestations only.…”

Section: Resultsmentioning

confidence: 99%

Off-label use of tocilizumab to treat non-juvenile idiopathic arthritis in pediatric rheumatic patients: a literature review

et al. 2018

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Tocilizumab, an anti-interleukin-6 (IL-6) agent, is indicated as a treatment for several autoimmune or inflammatory diseases, including rheumatoid arthritis and juvenile idiopathic arthritis (JIA). IL-6 plays roles in both immune system dysregulation and inflammation, and thus efforts to extend the utility of tocilizumab in patients with autoinflammatory conditions are ongoing. Here, we survey the literature on the off-label use of tocilizumab in patients with juvenile-onset rheumatic diseases including juvenile systemic lupus erythematosus (SLE), juvenile dermatomyositis (DM), vasculitis, juvenile scleroderma, and other autoinflammatory diseases. There is no real evidence that tocilizumab is useful for patients with SLE and juvenile DM, but several cases of childhood Takayasu arteritis have experienced promising outcomes. In juvenile-onset scleroderma, for which no therapy that can halt disease progression is available, tocilizumab may stop progression and the associated functional impairment. Tocilizumab prevents systemic inflammation in patients with Kawasaki’s disease, but may develop coronary aneurysms. Tocilizumab has been used to treat several pediatric autoinflammatory diseases, including JIA-associated uveitis and Castleman’s disease. Further work in larger populations is necessary to confirm the effects of tocilizumab in patients with pediatric rheumatic diseases.

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Section: Resultsmentioning

confidence: 99%

Off-label use of tocilizumab to treat non-juvenile idiopathic arthritis in pediatric rheumatic patients: a literature review

et al. 2018

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“…A recently published review of current pharmacological treatment of idiopathic inflammatory myopathies [69] described three cases of refractory myositis treated with tocilizumab with promising results [118][119][120] and three case reports showing beneficial effects of co-stimulation blockade using Abatacept in myositis [121][122][123]. A randomized clinical trial is underway to test this hypothesis (ClinicalTrials.gov Identifier: NCT02594735).…”

Section: Other Biologic Agentsmentioning

confidence: 99%

Treatment of Juvenile Dermatomyositis: An Update

Papadopoulou

Wedderburn

2017

Pediatr Drugs

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Abstract:The Idiopathic Inflammatory Myopathies of childhood consist a heterogeneous group of autoimmune diseases characterised by proximal muscle weakness and pathognomonic skin rashes. The overall prognosis of juvenile myositis has improved significantly over recent years, but the long-term outcome differs substantially from patient to patient suggestive of distinct clinical phenotypes with variable responses to treatment. High doses of corticosteroids remain the cornerstone of therapy along with other immunosuppressant therapies depending on disease severity and response. The advent of biological drugs has revolutionised the management of various paediatric rheumatologic diseases, including inflammatory myopathies. There are few data from randomised controlled trials to guide management decisions, thus several algorithms for the treatment of juvenile myositis have been developed using international expert opinion. The general treatment goals now include elimination of active disease and normalization of physical function, so as to preserve normal growth and development, and to prevent long-term damage and deformities. This review summarizes the newer and possible future therapies of juvenile inflammatory myopathies, including evidence supporting their efficacy and safety.

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“…Considering the mechanism of IL-6 and the effects of tocilizumab, the drug can be used in several disorders in which autoinflammatory reactions are the main pathogenesis [ 25 ]. A little evidence of promising responses to tocilizumab in children is also reported in systemic lupus erythematosus [ 26 ] and juvenile dermatomyositis [ 27 ], as well as Takayasu arteritis [ 25 ]. Globally, the literature review supports the use of tocilizumab therapy as one option in the treatment of several pediatric rheumatic diseases, not limited to JIA [ 25 ].…”

Section: Discussionmentioning

confidence: 99%

Tocilizumab as a Useful Tool for Thyroid Eye Disease in Pediatric Population: A Case Report

Moleiro

Vilares-Morgado

Alves

et al. 2022

Case Rep Ophthalmol

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Thyroid eye disease (TED) characterizes by inflammation and remodeling of orbital tissues. Although the majority of pediatric TED is mild, some children present progressive and severe disease. The approach to severe disease in this group of patients, especially when noncortico-responsive, is challenging. The purpose of this work was to describe the effective use of tocilizumab as second-line therapy in steroid-resistant pediatric TED. A 13-year-old female with a history of Graves’ disease presented with right eye proptosis for at least 8 months associated with mild pain on eye movements and ocular surface complaints. The ophthalmologic evaluation revealed Hertel exophthalmometry readings of 22 mm on the right eye (OD) and 19 mm on the left (OS). The remaining ophthalmic examination was unremarkable. Intravenous methylprednisolone pulses of 500 mg were initiated without any improvement after 4 treatments. Following multidisciplinary team discussion, therapy was switched to monthly tocilizumab injections at 4 mg/kg. Significant reduction of proptosis and resolution of pain and ocular surface complaints were noted immediately after the treatment switch. Exophthalmometry readings after the end of treatment that included 4 tocilizumab injections were 20 mm on OD and 19 mm on OS. No side effects were reported during the entire follow-up. Six months after treatment cessation, the patient remains stable, without any signs of orbitopathy relapse and no ophthalmologic complaints. This is the second case report showing the effectiveness of tocilizumab in pediatric TED and the first one showing its efficacy when steroids fail. Our results support the potential safety and efficacy of this immunosuppressor in children with TED.

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Tocilizumab in the treatment of mixed connective tissue disease and overlap syndrome in children

Cited by 13 publications

References 14 publications

Off-label use of tocilizumab to treat non-juvenile idiopathic arthritis in pediatric rheumatic patients: a literature review

Off-label use of tocilizumab to treat non-juvenile idiopathic arthritis in pediatric rheumatic patients: a literature review

Treatment of Juvenile Dermatomyositis: An Update

Tocilizumab as a Useful Tool for Thyroid Eye Disease in Pediatric Population: A Case Report

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