Article Text

other Versions

Download PDFPDF
Extended report
Abatacept in the treatment of adult dermatomyositis and polymyositis: a randomised, phase IIb treatment delayed-start trial
  1. Anna Tjärnlund1,
  2. Quan Tang1,
  3. Cecilia Wick1,
  4. Maryam Dastmalchi1,
  5. Herman Mann2,
  6. Jana Tomasová Studýnková2,
  7. Radka Chura3,
  8. Nicola J Gullick3,
  9. Rosaria Salerno3,
  10. Johan Rönnelid4,
  11. Helene Alexanderson5,
  12. Eva Lindroos1,
  13. Rohit Aggarwal6,
  14. Patrick Gordon3,
  15. Jiri Vencovsky2,
  16. Ingrid E Lundberg1
  1. 1Rheumatology Unit, Department of Medicine, Karolinska University Hospital Solna, Karolinska Institutet, Stockholm, Sweden
  2. 2Department of Rheumatology, Institute of Rheumatology, Prague, Czech Republic
  3. 3Department of Rheumatology, King’s College Hospital NHS Foundation Trust, London, UK
  4. 4Department of Immunology, Genetics and Pathology, Uppsala University, Uppsala, Sweden
  5. 5Division of Physiotherapy, Department of Neurobiology, Karolinska Institutet and Physical Therapy Clinic, Karolinska University Hospital, Stockholm, Sweden
  6. 6Division of Rheumatology and Clinical Rheumatology, University of Pittsburgh School of Medicine, Pittsburgh, USA
  1. Correspondence to Anna Tjärnlund; anna.tjarnlund{at}


Objectives To study the effects of abatacept on disease activity and on muscle biopsy features of adult patients with dermatomyositis (DM) or polymyositis (PM).

Methods Twenty patients with DM (n=9) or PM (n=11) with refractory disease were enrolled in a randomised treatment delayed-start trial to receive either immediate active treatment with intravenous abatacept or a 3 month delayed-start. The primary endpoint was number of responders, defined by the International Myositis Assessment and Clinical Studies Group definition of improvement (DOI), after 6 months of treatment. Secondary endpoints included number of responders in the early treatment arm compared with the delayed treatment arm at 3 months. Repeated muscle biopsies were investigated for cellular markers and cytokines.

Results 8/19 patients included in the analyses achieved the DOI at 6 months. At 3 months of study, five (50%) patients were responders after active treatment but only one (11%) patient in the delayed treatment arm. Eight adverse events (AEs) were regarded as related to the drug, four mild and four moderate, and three serious AEs, none related to the drug. There was a significant increase in regulatory T cells (Tregs), whereas other markers were unchanged in repeated muscle biopsies.

Conclusions In this pilot study, treatment of patients with DM and PM with abatacept resulted in lower disease activity in nearly half of the patients. In patients with repeat muscle biopsies, an increased frequency of Foxp3+ Tregs suggests a positive effect of treatment in muscle tissue.

  • Dermatomyositis
  • polymyositis
  • treatment

Statistics from

Request Permissions

If you wish to reuse any or all of this article please use the link below which will take you to the Copyright Clearance Center’s RightsLink service. You will be able to get a quick price and instant permission to reuse the content in many different ways.


  • Handling editor Tore K Kvien

  • Contributors All authors were involved in drafting the article or revising it critically for important intellectual content. All authors approved the final version to be published and had full access to all of the data in the study and take responsibility for the integrity of the data and the accuracy of the data analysis. Study conception and design: AT, QT, CW, MD, HM, JTS, RC, NJG, RS, JR, HA, EL, RA, PG, JV, IEL. Acquisition of data: AT, QT, CW, MD, HM, JTS, RC, NJG, RS, JR, HA, EL, RA, PG, JV, IEL. Analysis and interpretation of data: AT, QT, CW, MD, HM, JTS, RC, NJG, RS, JR, HA, EL, RA, PG, JV, IEL. AT and QT contributed equally to this paper.

  • Funding This study was carried out as an investigator-initiated study funded by a grant from Bristol-Myers Squibb (BMS) and Börje Dahlin Foundation, Swedish Research Council GRANTK2014-52X-14045-14-3, Swedish Rheumatism Association, King Gustaf V 80-Year Foundation, and the regional agreement on medical training and clinical research (ALF) between Stockholm County Council and Karolinska Institutet. BMS provided the study drug. The presented work was initiated, conducted and performed independently of BMS.

  • Competing interests IEL: research grant and advisory board consultant for BMS, research grant from AstraZeneca, consultant for MedImmune, aTyr and IDERA. RA: research grant from BMS.

  • Patient consent Obtained.

  • Ethics approval The institutional review board at each study site.

  • Provenance and peer review Not commissioned; externally peer reviewed.