Treating juvenile dermatomyositis to target: Paediatric Rheumatology European Society/Childhood Arthritis and Rheumatology Research Alliance-endorsed recommendations from an international task force

  • Angelo Ravelli*
  • , Silvia Rosina
  • , Jayne M MacMahon
  • , Talia Baird
  • , Ana Isabel Rebollo-Giménez
  • , Claas Hinze
  • , Liza J McCann
  • , Ann M Reed
  • , Lisa G Rider
  • , Matilde Arvigo
  • , Brigitte Bader-Meunier
  • , Claudio Bruno
  • , Li Caifeng
  • , Raquel Campanilho-Marques
  • , Sara Cuccato
  • , Chiara Fiorillo
  • , Nikki A Hahn
  • , Adam M Huber
  • , Marc Jansen
  • , Ozgur Kasapcopur
  • Maria Martha Katsikas, Susan Kim, Polly Livermore, Sue Maillard, Clara Malattia, Angela Nyangore Migowa, Takako Miyamae, Ruth Murphy, Rebecca Nicolai, Charalampia Papadopoulou, Clarissa Pilkington, Helga Sanner, Sujata Sawhney, Elzbieta Smolewska, Stacey E Tarvin, Georgina Tiller, Natasa Toplak, Lucy R Wedderburn, Francesca Bovis, Alessandro Consolaro, Brian M Feldman
*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

Abstract

OBJECTIVES: Despite the recent prognostic improvement, a sizeable proportion of patients with juvenile dermatomyositis (JDM) respond suboptimally to contemporary therapies. This study aimed to develop recommendations for treating JDM to target.

METHODS: A Steering Committee formulated a set of provisional recommendations based on evidence derived from a systematic literature review and a retrospective chart review of patients. These were discussed, amended, and voted on by an international Task Force, including 28 paediatric rheumatologists, 2 specialists in neuromuscular diseases, 1 dermatologist, 1 physical therapist, 1 research nurse, 2 patients with JDM, and 1 parent of a patient with JDM. Items that achieved at least an 80% majority vote were accepted as final recommendations.

RESULTS: Although the literature review did not reveal trials that compared a treat-to-target strategy with a nonsteered approach, it provided indirect evidence about specific end points that could serve as targets that facilitated development of recommendations. The group reached consensus on 7 overarching principles and 12 recommendations. It was agreed that both patients/parents and treaters should share decisions in setting treatment targets and therapeutic strategies, with inactive disease as the preferred target and minimal disease activity an alternative one. Inactive disease is targeted to be achieved within 12 months after treatment start. Interim targets include minimal and moderate clinical improvement within 6 weeks and 3 months, respectively, and normalisation of muscle strength within 6 months. High-dose glucocorticoids remain fundamental in the initial management, but progressive tapering and discontinuation within 12 months through optimisation of concomitant immunomodulatory therapy was advised. A research agenda was formulated.

CONCLUSIONS: The Task Force developed recommendations for treating JDM to target, being aware that the evidence is not strong and needs to be expanded by future research. Implementation of the recommendations in clinical practice will help to reach optimal outcomes for JDM.

Original languageEnglish
Pages (from-to)1055-1067
Number of pages13
JournalAnnals of the rheumatic diseases
Volume84
Issue number7
Early online date22 May 2025
DOIs
Publication statusPublished - Jul 2025

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