Approach to Janus kinase inhibition for juvenile dermatomyositis among CARRA and PReS providers

Matthew A Sherman¹, Rebecca Nicolai², Emily K Datyner³, Silvia Rosina⁴, Angela Hamilton⁵, Kaveh Ardalan⁶, Brigitte Bader-Meunier⁷, Amanda G Brown⁸, Marc H A Jansen^{9

10}, Susan Kim¹¹, Bianca Lang¹², Raquel Campanilho-Marques^{13

14}, Liza J McCann¹⁵, Helga Sanner^{16

17}, Saskia R Veldkamp¹⁸, Meredyth G Ll Wilkinson^{19

20

21}, Belina Y Yi²², Hanna Kim²³, Stacey E Tarvin⁵, Charalampia Papadopoulou^{21

24}; CARRA JDM Therapeutics Workgroup and the PReS JDM Working Party

Collaborators, Affiliations

Collaborators

CARRA JDM Therapeutics Workgroup and the PReS JDM Working Party:
Matthew A Sherman, Rebecca Nicolai, Emily K Datyner, Silvia Rosina, Angela Hamilton, Kaveh Ardalan, Brigitte Bader-Meunier, Amanda G Brown, Marc H A Jansen, Susan Kim, Bianca Lang, Raquel Campanilho-Marques, Liza J McCann, Helga Sanner, Saskia R Veldkamp, Meredyth G Ll Wilkinson, Belina Y Yi, Hanna Kim, Stacey E Tarvin, Charalampia Papadopoulou

Affiliations

¹ Division of Allergy, Immunology, and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
² Division of Rheumatology, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
³ Department of Pediatric Rheumatology, Vanderbilt University Medical Center, Nashville, TN, USA.
⁴ UOC Reumatologia E Malattie Autoinfiammatorie, IRCCS Istituto Giannina Gaslini, Genoa, Italy.
⁵ Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA.
⁶ Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
⁷ Department of Pediatric Immunology and Rheumatology, Necker Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.
⁸ Department of Rheumatology, Children's Hospital of Arkansas, University of Arkansas for Medical Sciences, Little Rock, AR, USA.
⁹ Department of Paediatric Immunology & Rheumatology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht, The Netherlands.
¹⁰ RITA, European Reference Networks, Brussels, Belgium.
¹¹ Department of Pediatrics, University of California, San Francisco, CA, USA.
¹² Department of Pediatrics, Dalhousie University and IWK Health, Halifax, NS, Canada.
¹³ Department of Rheumatology, Unidade Local de Saúde de Santa Maria, Lisbon, Portugal.
¹⁴ Instituto de Medicina Molecular João Lobo Antunes, Lisbon Academic Medical Centre, Lisbon, Portugal.
¹⁵ Department of Paediatric Rheumatology, Alder Hey Children's NHS Foundation Trust, Liverpool, UK.
¹⁶ Department of Rheumatology, Oslo University Hospital, Oslo, Norway.
¹⁷ Department of Health Sciences, Oslo New University College, Oslo, Norway.
¹⁸ Center for Translational Immunology, University Medical Center Utrecht, Utrecht, The Netherlands.
¹⁹ Infection, Immunity and Inflammation Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, London, UK.
²⁰ Centre for Adolescent Rheumatology Versus Arthritis at UCL UCLH and GOSH, London, UK.
²¹ Rare Diseases Theme NIHR Biomedical Research Centre, Great Ormond Street Hospital, London, UK.
²² Department of Pediatrics, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
²³ Juvenile Myositis Pathogenesis and Therapeutics Unit, National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Institutes of Health, Bethesda, MD, USA.
²⁴ Department Paediatric Rheumatology, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.

PMID: 39928372
PMCID: PMC12316366
DOI: 10.1093/rheumatology/keaf086

Approach to Janus kinase inhibition for juvenile dermatomyositis among CARRA and PReS providers

Matthew A Sherman et al. Rheumatology (Oxford). 2025.

. 2025 Aug 1;64(8):4732-4737.

doi: 10.1093/rheumatology/keaf086.

Authors

Collaborators

CARRA JDM Therapeutics Workgroup and the PReS JDM Working Party:
Matthew A Sherman, Rebecca Nicolai, Emily K Datyner, Silvia Rosina, Angela Hamilton, Kaveh Ardalan, Brigitte Bader-Meunier, Amanda G Brown, Marc H A Jansen, Susan Kim, Bianca Lang, Raquel Campanilho-Marques, Liza J McCann, Helga Sanner, Saskia R Veldkamp, Meredyth G Ll Wilkinson, Belina Y Yi, Hanna Kim, Stacey E Tarvin, Charalampia Papadopoulou

Affiliations

¹ Division of Allergy, Immunology, and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
² Division of Rheumatology, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
³ Department of Pediatric Rheumatology, Vanderbilt University Medical Center, Nashville, TN, USA.
⁴ UOC Reumatologia E Malattie Autoinfiammatorie, IRCCS Istituto Giannina Gaslini, Genoa, Italy.
⁵ Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA.
⁶ Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
⁷ Department of Pediatric Immunology and Rheumatology, Necker Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.
⁸ Department of Rheumatology, Children's Hospital of Arkansas, University of Arkansas for Medical Sciences, Little Rock, AR, USA.
⁹ Department of Paediatric Immunology & Rheumatology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht, The Netherlands.
¹⁰ RITA, European Reference Networks, Brussels, Belgium.
¹¹ Department of Pediatrics, University of California, San Francisco, CA, USA.
¹² Department of Pediatrics, Dalhousie University and IWK Health, Halifax, NS, Canada.
¹³ Department of Rheumatology, Unidade Local de Saúde de Santa Maria, Lisbon, Portugal.
¹⁴ Instituto de Medicina Molecular João Lobo Antunes, Lisbon Academic Medical Centre, Lisbon, Portugal.
¹⁵ Department of Paediatric Rheumatology, Alder Hey Children's NHS Foundation Trust, Liverpool, UK.
¹⁶ Department of Rheumatology, Oslo University Hospital, Oslo, Norway.
¹⁷ Department of Health Sciences, Oslo New University College, Oslo, Norway.
¹⁸ Center for Translational Immunology, University Medical Center Utrecht, Utrecht, The Netherlands.
¹⁹ Infection, Immunity and Inflammation Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, London, UK.
²⁰ Centre for Adolescent Rheumatology Versus Arthritis at UCL UCLH and GOSH, London, UK.
²¹ Rare Diseases Theme NIHR Biomedical Research Centre, Great Ormond Street Hospital, London, UK.
²² Department of Pediatrics, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
²³ Juvenile Myositis Pathogenesis and Therapeutics Unit, National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Institutes of Health, Bethesda, MD, USA.
²⁴ Department Paediatric Rheumatology, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.

PMID: 39928372
PMCID: PMC12316366
DOI: 10.1093/rheumatology/keaf086

Abstract

Objectives: Janus kinase inhibition (JAKi) has been proposed as a treatment for idiopathic inflammatory myopathies to target increased interferon signalling. Predominantly retrospective reports have demonstrated effectiveness of JAKi in refractory JDM. However, JAKi remains an off-label treatment for JDM and there may be variation in use worldwide. An international survey was conducted to investigate approaches to JAKi for JDM.

Methods: The Childhood Arthritis and Rheumatology Research Alliance (CARRA) JDM Therapeutics workgroup and core members of the Paediatric Rheumatology European Society (PReS) JDM working party devised an electronic survey to assess the use of JAKi in JDM. CARRA and PReS members were invited by e-mail to complete the survey.

Results: There were 229 respondents (18%), with 50% from the USA and 29% from Europe. One hundred and fifty had used JAKi for over 450 patients with JDM; among them, 77% noted clinical improvement in most or all patients and 17% reported side effects. The highest ranked perceived barriers to JAKi use were lack of clinical data and inability to obtain insurance approval. The highest ranked clinical indications for starting JAKi were refractory skin disease, refractory muscle disease, inability to wean steroids and intolerance to other steroid-sparing agents.

Conclusion: Paediatric rheumatologists use JAKi off-label for refractory JDM. Most providers noted clinical improvement in their patients. Barriers to JAKi use include lack of clinical data and insurance coverage. Clinical trials are needed to provide better data on the efficacy and safety of JAKi.

Keywords: Janus kinase inhibitors; juvenile dermatomyositis; pediatric rheumatology.

Published by Oxford University Press on behalf of the British Society for Rheumatology 2025.

PubMed Disclaimer

Figures

**Figure 1.**
Location of respondents and respondent preferences for systemic JAKi indications and medications. (A) Map indicating location of respondents and number of responses per country. (B) Respondents ranked the most important clinical indications for initiating systemic JAKi. (C) Systemic JAKi that respondents would consider using. (D) Concomitant medications that respondents would consider using with systemic JAKi. For (B), respondents selected and ranked their top five indications. For (C) and (D), respondents could select all applicable. GI: gastrointestinal; JAKi: Janus kinase inhibitor

See this image and copyright information in PMC

References

1. Papadopoulou C, Chew C, Wilkinson MGL, McCann L, Wedderburn LR. Juvenile idiopathic inflammatory myositis: an update on pathophysiology and clinical care. Nat Rev Rheumatol 2023;19:343–62. - PMC - PubMed
1. Bellutti Enders F, Bader-Meunier B, Baildam E et al. Consensus-based recommendations for the management of juvenile dermatomyositis. Ann Rheum Dis 2017;76:329–40. - PMC - PubMed
1. Hinze CH, Oommen PT, Dressler F et al. Development of practice and consensus-based strategies including a treat-to-target approach for the management of moderate and severe juvenile dermatomyositis in Germany and Austria. Pediatr Rheumatol Online J 2018;16:40. - PMC - PubMed
1. Kobayashi I, Akioka S, Kobayashi N et al. Clinical practice guidance for juvenile dermatomyositis (JDM) 2018-Update. Mod Rheumatol 2020;30:411–23. - PubMed
1. Oldroyd AGS, Lilleker JB, Amin T et al. ; British Society for Rheumatology Standards, Audit and Guidelines Working Group. British Society for Rheumatology guideline on management of paediatric, adolescent and adult patients with idiopathic inflammatory myopathy. Rheumatology (Oxford) 2022;61:1760–8. - PMC - PubMed

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Approach to Janus kinase inhibition for juvenile dermatomyositis among CARRA and PReS providers

Collaborators

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Approach to Janus kinase inhibition for juvenile dermatomyositis among CARRA and PReS providers

Authors

Collaborators

Affiliations

Abstract

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References

MeSH terms

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