Tocilizumab is a promising treatment option for therapy resistant juvenile localized scleroderma patients

Introduction Juvenile localized scleroderma (jlSc) usually responds well to treatment with methotrexate or mycophenolate. In case of nonresponse or partial response, tocilizumab (TOC) appears to be a promising option. Methods Participants of the Pediatric Rheumatology Email Board were asked to report patients with jlSc treated with TOC. Results Six centers responded and reported 11 patients. The mean age at disease onset was 5.5 years. Disease duration at time of the initiation of TOC was 4.5 years. A total of 5 patients had linear subtype, 2 Parry Romberg syndrome, and 1 morphea en coup de sabre. Three had a generalized subtype, 2 a mixed subtype, and 1 a limited subtype/morphea. Before starting TOC, 10 of 11 patients received methotrexate, 7/11 combination methotrexate and mycophenolate, 1 abatacept, and 1 antitumor necrosis factor therapy. The indications for starting TOC were: (i) an increase in the score of the Localized Scleroderma Activity Index (mLoSSI) in 9 patients; and (ii) evidence of increased extra-cutaneous activity in 2 patients. The mean duration of TOC therapy was 14.75 months. Three of 11 patients received TOC as monotherapy, and 8/11 as combination therapy. Therapy success was reflected by a decreased mLoSSI in 9/11 patients, no new lesion occurrence, and – with Parry Romberg syndrome subtype – no increase in facial atrophy. In 8/8 patients, both the physicians’ and patients’ global assessment of disease activity decreased. In 3/3 patients, the number of active joints decreased. The mean modified Rodnan skin score decreased from 8.7 to 5.6. Conclusions In this small cohort of patients, TOC seems to be a promising rescue medication.