Tocilizumab therapy in juvenile systemic sclerosis: a retrospective single centre pilot study

Rheumatol Int. 2021 Jan;41(1):121-128. doi: 10.1007/s00296-020-04732-z. Epub 2020 Oct 27.


To evaluate the efficacy and safety of anti-interleukin (IL)-6 receptor antibody tocilizumab (TCZ) as a treatment option of juvenile systemic sclerosis (JSS). Nine JSS patients were assigned to a TCZ, additionally to conventional treatment (steroids, methotrexate, mycophenolate-mofetil). The modified Rodnan skin score (mRSS), carbon-monoxide diffusion capacity (DLCO), thorax high-resolution tomography (HRCT), patient global assessment (PGA) and Juvenile Systemic Sclerosis Severity (J4S) score were used to explore the efficacy of treatment. Nine JSS patients were treated with TCZ with a median treatment duration of 10 (1-21) months. Nine patients (77.8%) had radiologically confirmed improvement on thorax HRCT, 7 (77.8%) had decreased PGA (mean pre-treatment PGA 3.7 vs. 2.3 post-treatment PGA 2), 6 (66.7%) had increased DLCO (mean pre-treatment DLCO 69.14% vs. post-treatment DLCO 79.50%) after the TCZ treatment. In all patients mRSS and the J4S decreased: 26.1 vs. 19.7 and 8.2 vs. 4.7, respectively. Changes in mRSS, DLCO, PGA and J4S were statistically significant: p = 0.012, 0.04, 0.026 and 0.007, respectively. All patients tolerated well TCZ treatment. JSS is a rare condition characterized with skin fibrosis and internal organ involvement. Tocilizumab represents a potential treatment option for patients unresponsive to conventional treatment. Long-term prospective studies with higher number of patients are needed to provide more relevant data.

Keywords: Juvenile scleroderma; Juvenile systemic sclerosis; Modified rodnan skin score; Pulmonary fibrosis; Tocilizumab.

MeSH terms

  • Adolescent
  • Adult
  • Antibodies, Monoclonal, Humanized / therapeutic use*
  • Child
  • Female
  • Humans
  • Interleukin-6 / antagonists & inhibitors
  • Male
  • Pilot Projects
  • Retrospective Studies
  • Scleroderma, Systemic / drug therapy*
  • Thorax / drug effects
  • Treatment Outcome
  • Young Adult


  • Antibodies, Monoclonal, Humanized
  • Interleukin-6
  • tocilizumab

Supplementary concepts

  • Juvenile systemic scleroderma