PMID- 28993346 OWN - NLM STAT- MEDLINE DCOM- 20171213 LR - 20220331 IS - 1468-2060 (Electronic) IS - 0003-4967 (Linking) VI - 77 IP - 1 DP - 2018 Jan TI - Abatacept in the treatment of adult dermatomyositis and polymyositis: a randomised, phase IIb treatment delayed-start trial. PG - 55-62 LID - 10.1136/annrheumdis-2017-211751 [doi] AB - 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. CI - (c) Article author(s) (or their employer(s) unless otherwise stated in the text of the article) 2018. All rights reserved. No commercial use is permitted unless otherwise expressly granted. FAU - Tjarnlund, Anna AU - Tjarnlund A AD - Rheumatology Unit, Department of Medicine, Karolinska University Hospital Solna, Karolinska Institutet, Stockholm, Sweden. FAU - Tang, Quan AU - Tang Q AD - Rheumatology Unit, Department of Medicine, Karolinska University Hospital Solna, Karolinska Institutet, Stockholm, Sweden. FAU - Wick, Cecilia AU - Wick C AD - Rheumatology Unit, Department of Medicine, Karolinska University Hospital Solna, Karolinska Institutet, Stockholm, Sweden. FAU - Dastmalchi, Maryam AU - Dastmalchi M AD - Rheumatology Unit, Department of Medicine, Karolinska University Hospital Solna, Karolinska Institutet, Stockholm, Sweden. FAU - Mann, Herman AU - Mann H AD - Department of Rheumatology, Institute of Rheumatology, Prague, Czech Republic. FAU - Tomasova Studynkova, Jana AU - Tomasova Studynkova J AD - Department of Rheumatology, Institute of Rheumatology, Prague, Czech Republic. FAU - Chura, Radka AU - Chura R AD - Department of Rheumatology, King's College Hospital NHS Foundation Trust, London, UK. FAU - Gullick, Nicola J AU - Gullick NJ AD - Department of Rheumatology, King's College Hospital NHS Foundation Trust, London, UK. FAU - Salerno, Rosaria AU - Salerno R AD - Department of Rheumatology, King's College Hospital NHS Foundation Trust, London, UK. FAU - Ronnelid, Johan AU - Ronnelid J AD - Department of Immunology, Genetics and Pathology, Uppsala University, Uppsala, Sweden. FAU - Alexanderson, Helene AU - Alexanderson H AD - Division of Physiotherapy, Department of Neurobiology, Karolinska Institutet and Physical Therapy Clinic, Karolinska University Hospital, Stockholm, Sweden. FAU - Lindroos, Eva AU - Lindroos E AD - Rheumatology Unit, Department of Medicine, Karolinska University Hospital Solna, Karolinska Institutet, Stockholm, Sweden. FAU - Aggarwal, Rohit AU - Aggarwal R AD - Division of Rheumatology and Clinical Rheumatology, University of Pittsburgh School of Medicine, Pittsburgh, USA. FAU - Gordon, Patrick AU - Gordon P AD - Department of Rheumatology, King's College Hospital NHS Foundation Trust, London, UK. FAU - Vencovsky, Jiri AU - Vencovsky J AUID- ORCID: 0000-0002-0851-0713 AD - Department of Rheumatology, Institute of Rheumatology, Prague, Czech Republic. FAU - Lundberg, Ingrid E AU - Lundberg IE AD - Rheumatology Unit, Department of Medicine, Karolinska University Hospital Solna, Karolinska Institutet, Stockholm, Sweden. LA - eng PT - Clinical Trial, Phase II PT - Journal Article PT - Randomized Controlled Trial DEP - 20171009 PL - England TA - Ann Rheum Dis JT - Annals of the rheumatic diseases JID - 0372355 RN - 0 (Immunosuppressive Agents) RN - 7D0YB67S97 (Abatacept) SB - IM MH - Abatacept/*administration & dosage MH - Adult MH - Dermatomyositis/*drug therapy MH - Drug Administration Schedule MH - Female MH - Humans MH - Immunosuppressive Agents/*administration & dosage MH - Male MH - Middle Aged MH - Pilot Projects MH - Polymyositis/*drug therapy MH - Treatment Outcome OTO - NOTNLM OT - Dermatomyositis OT - polymyositis OT - treatment COIS- 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. EDAT- 2017/10/11 06:00 MHDA- 2017/12/14 06:00 CRDT- 2017/10/11 06:00 PHST- 2017/05/07 00:00 [received] PHST- 2017/07/14 00:00 [revised] PHST- 2017/08/05 00:00 [accepted] PHST- 2017/10/11 06:00 [pubmed] PHST- 2017/12/14 06:00 [medline] PHST- 2017/10/11 06:00 [entrez] AID - annrheumdis-2017-211751 [pii] AID - 10.1136/annrheumdis-2017-211751 [doi] PST - ppublish SO - Ann Rheum Dis. 2018 Jan;77(1):55-62. doi: 10.1136/annrheumdis-2017-211751. Epub 2017 Oct 9.