gms | German Medical Science

45. Kongress der Deutschen Gesellschaft für Rheumatologie, 31. Jahrestagung der Deutschen Gesellschaft für Orthopädische Rheumatologie, 27. Jahrestagung der Gesellschaft für Kinder- und Jugendrheumatologie

06.09. - 09.09.2017, Stuttgart

Tocilizumab is a promising treatment option for therapy resistent juvenile localised scleroderma patients

Meeting Abstract

  • Ivan Foeldvari - Schön Klinik Hamburg-Eilbek, Hamburger Zentrum für Kinder- und Jugendrheumatologie, Hamburg
  • Jordi Anton - University Children´s Hospital, Pediatric Rheumatology, Barcelona, Spain
  • Mark Friswell - Great North Children’s Hospital, Newcastle, UK
  • Blanca Bica - Universidade Federal do Rio de Janeiro, Rio de Janeiro, Brazil
  • Jaime de Inocencio - Pediatric Rheumatology Unit Hospital Universitario, Madrid, Spain
  • Angela Aquilani - Ospedale Bambino Gesù, Rome, Italy
  • Nicola Helmus - Schön Klinik Hamburg-Eilbek, Hamburger Zentrum für Kinder- und Jugendrheumatologie, Hamburg

Deutsche Gesellschaft für Rheumatologie. Deutsche Gesellschaft für Orthopädische Rheumatologie. Gesellschaft für Kinder- und Jugendrheumatologie. 45. Kongress der Deutschen Gesellschaft für Rheumatologie (DGRh), 31. Jahrestagung der Deutschen Gesellschaft für Orthopädische Rheumatologie (DGORh), 27. Jahrestagung der Gesellschaft für Kinder- und Jugendrheumatologie (GKJR). Stuttgart, 06.-09.09.2017. Düsseldorf: German Medical Science GMS Publishing House; 2017. DocKR.28

doi: 10.3205/17dgrh146, urn:nbn:de:0183-17dgrh1461

Veröffentlicht: 4. September 2017

© 2017 Foeldvari et al.
Dieser Artikel ist ein Open-Access-Artikel und steht unter den Lizenzbedingungen der Creative Commons Attribution 4.0 License (Namensnennung). Lizenz-Angaben siehe



Background: Juvenile localised scleroderma (jlSc) respond to treatment of methotrexate or mycophenolate. In case of nonresponse or partial response, Tocilizumab (TOC) seems to be a promising option.

Methods: Participants of the paediatric rheumatology email board were asked, to report patients with jlSc treated with TOC.

Results: Six centers responded to the survey from the email board, with around 800 participatns, and reported 11 patients. The mean age of the patients at disease onset was 5.5 years. Disease duration at time of the intiation of TOC was 53.5. months (range 9 to 109). 5 patients had linear subtype, 3 of them with facial involvement, 2 of them Parry Romberg and one of them coup de sabre. Three had generalized subtype , 2 mixed subtype and 1 limited subtype/morphea. Before starting TOC patients received 10/11 Methotrexate, 7/11 Mycophenolate, 1 abatacept and 1 anti-TNF therapy. Reason to start TOC was in 9 patients increase in Localised Scleroderma Activity Index (mLoSSI). In two patients increased extracutaneous activity was the indication, in one increased activity of arthritis and in the other increased activity of the central nervous system involvement.

The mean duration of tocilizumab therapy was 14.75 months. 2 patients received s.c. accoding the poly JIA dosing and all other i.v.. There were diferent i.v doses applied, 5 of them 8mg/kg every 4 weeks, one of them 8 mg /kg every three weeks, 1 every two weeks and 1 pateints received 10 mg /kg every 3 weeks. 3/11 received TOC as monotherapy. 8/11 as combination therapy, 6 of them with Methotrexate and one of each with Mycophenolate or Tacrolimus. Therapy success was refelected by a decreased mLoSSI in 8/11 patients and in 6 patients by a decrease in the Localosed Scleroderma Skin Damage Index[1] (LoSDI). No new lesion occured during the treatment and in the patients with Parry Romerg subtype(n=2) no increase in the facial atrophy occured. In 8/8 patients physician global (VAS 0-100) decreased and in 8/8 the patients global disease activity (VAS 0-100) decreased. In 3/3 patients, were it was applicable, the number of acitve joints decreased, in one patients the limb discrepancy decreased. The mean modified Rodnan skin score assessed in 8 patients decreased from the mean value of 9.6 to 5.5.

Conclusion: In this small cohort of patients TOC seems to be a promising rescue medication in methotrexate/mycophenolate nonresponsive patients. A prospective controlled study would be important to prove the seen effect in a controlled way.