Abatacept in the treatment of adult dermatomyositis and polymyositis: a randomised, phase IIb treatment delayed-start trial.

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.

Patient involvement in rheumatology outpatient service design and delivery: a case study.

BACKGROUND: Patient involvement is increasingly recognized as important within the UK National Health Service to ensure that services delivered are relevant to users' needs. Organizations are encouraged to work with service users to achieve excellence in care. Patient education can improve health outcomes and reduce health-care costs. Mobile technologies could play a vital role in this. AIM: Patient-centred development of innovative strategies to improve the experience of rheumatology outpatients. CASE STUDY: The Group Rheumatology Initiative Involving Patients (GRIIP) project was set up in 2013 as a joint venture between patients, clinicians, academics and management at a London hospital. The project saw (i) the formation of an independent patient group which provided suggestions for service improvement - outcomes included clearer signs in the outpatient waiting area, extended phlebotomy opening hours and better access to podiatry; (ii) a rolling patient educational evening programme initiated in 2014 with topics chosen by patient experts - feedback has been positive and attendance continues to grow; and (iii) a mobile application (app) co-designed with patients launched in 2015 which provides relevant information for outpatient clinic attendees and data capture for clinicians - downloads have steadily increased as users adopt this new technology. CONCLUSION: Patients can effectively contribute to service improvement provided they are supported, respected as equals, and the organization is willing to undergo a cultural change.