Tocilizumab in two children with pansclerotic morphoea: a hopeful therapy for refractory cases?

Clin Exp Rheumatol. 2017 Sep-Oct;35 Suppl 106(4):211-213. Epub 2017 Sep 29.

Abstract

Pansclerotic morphoea (PM) is a subtype of juvenile localised scleroderma characterised by severe course with generalised full-thickness skin involvement and possible growth and functional impairment. PM treatment comprises a combination of immunosuppressive agents such as corticosteroids, methotrexate, mycophenolate mofetil, PUVA and antithymocyte globulin and biological agents used in off-label. A possible role of IL-6 in the regulation of firoblast differentiation and stimulation of collagen synthesis has been suggested and in patients with systemic sclerosis (SSc) the treatment with tocilizumab (TCZ) was associated to improvement of skin thickness and joint motion. We describe the first two cases of children with PM refractory to different immunosuppressive agents in which the use of TCZ reduced disease activity and stopped disease progression. Therefore, we suggest that an earlier use of this agent in such severe cases could be considered before irreversible sclerosis and tissue damage occurs.Juvenile localised scleroderma (JLS) comprises a group of autoimmune fibrosing conditions involving skin and subcutaneous tissues following an initial inflammatory reaction. Pansclerotic morphoea (PM), an extremely rare and severe subtype of JLS, is characterised by generalised full-thickness skin involvement that may extend over deeper tissues and bone with subsequent growth disturbance and disabling outcome. We describe the first two children with PM refractory to immunosuppressive treatments in which the off-label use of tocilizumab (TCZ), fully humanised anti IL-6R antibody, allowed to control the inflammation and stopped the extension of the disease.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Antibodies, Monoclonal, Humanized / therapeutic use*
  • Child, Preschool
  • Female
  • Humans
  • Immunosuppressive Agents / therapeutic use
  • Male
  • Receptors, Interleukin-6 / antagonists & inhibitors
  • Scleroderma, Localized / drug therapy*

Substances

  • Antibodies, Monoclonal, Humanized
  • Immunosuppressive Agents
  • Receptors, Interleukin-6
  • tocilizumab