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77 Publications visible to you, out of a total of 77
Immunosuppressant and immunomodulatory treatment for dermatomyositis and polymyositis

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BACKGROUND: Idiopathic inflammatory myopathies are chronic diseases with significant mortality and morbidity. Whilst immunosuppressive and immunomodulatory therapies are frequently used, the optimal … therapeutic regimen remains unclear. This is an update of a review first published in 2005. OBJECTIVES: To assess the effects of immunosuppressants and immunomodulatory treatments for dermatomyositis and polymyositis. SEARCH METHODS: We searched the Cochrane Neuromuscular Disease Group Specialized Register (August 2011), the Cochrane Central Register of Controlled Trials (CENTRAL) (Issue 3 2011), MEDLINE (January 1966 to August 2011), EMBASE (January 1980 to August 2011) and clinicaltrials.gov (August 2011). We checked the bibliographies of identified trials and wrote to disease experts. SELECTION CRITERIA: We included all randomised controlled trials (RCTs) or quasi-RCTs involving participants with probable or definite dermatomyositis and polymyositis as defined by the criteria of Bohan and Peter, or definite, probable or mild/early by the criteria of Dalakas. In participants without a classical rash of dermatomyositis, inclusion body myositis should have been excluded by muscle biopsy. We considered any immunosuppressant or immunomodulatory treatment. The two primary outcomes were the change in a function or disability scale measured as the proportion of participants improving one grade, two grades etc, predefined based on the scales used in the studies after at least six months, and a 15% or greater improvement in muscle strength compared with baseline after at least six months. Other outcomes were: the International Myositis Assessment and Clinical Studies Group (IMACS) definition of improvement, number of relapses and time to relapse, remission and time-to-remission, cumulative corticosteroid dose and serious adverse effects. DATA COLLECTION AND ANALYSIS: Two authors independently selected papers, extracted data and assessed risk of bias in included studies. They collected adverse event data from the included studies. MAIN RESULTS: The review authors identified fourteen 14 relevant RCTs. They excluded four trials.The 10 included studies, four of which have been added in this update, included a total of 258 participants. Six studies compared an immunosuppressant or immunomodulator with placebo control, and four studies compared two immunosuppressant regimes with each other. Most of the studies were small (the largest had 62 participants) and many of the reports contained insufficient information to assess risk of bias.Amongst the six studies comparing immunosuppressant with placebo, one study, investigating intravenous immunoglobulin (IVIg), showed statistically significant improvement in scores of muscle strength in the IVIg group over three months. Another study investigating etanercept showed some evidence of a steroid sparing effect, a secondary outcome in this review, but no improvement in other assessed outcomes. The other four randomised placebo-controlled trials assessed either plasma exchange and leukapheresis, eculizumab, infliximab or azathioprine against placebo and all produced negative results.Three of the four studies comparing two immunosuppressant regimes (azathioprine with methotrexate, ciclosporin with methotrexate, and intramuscular methotrexate with oral methotrexate plus azathioprine) showed no statistically significant difference in efficacy between the treatment regimes. The fourth study comparing pulsed oral dexamethasone with daily oral prednisolone and found that the dexamethasone regime had a shorter median time to relapse but fewer side effects.Immunosuppressants were associated with significant side effects. AUTHORS’ CONCLUSIONS: This systematic review highlights the lack of high quality RCTs that assess the efficacy and toxicity of immunosuppressants in inflammatory myositis.

Authors: P. A. Gordon, J. B. Winer, J. E. Hoogendijk, E. H. Choy

Date Published: 2012

Publication Type: Journal

Morbidity and mortality in adult polymyositis and dermatomyositis

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Before the use of corticosteroids, the prognosis for polymyositis/dermatomyositis (PM/DM) was extremely poor. To date, although overall prognosis appears to be better, PM and DM are still considered … to be associated with increased morbidity, primarily related to severe muscle weakness and visceral involvement. Recent series underline that only 20% to 40% of treated patients will achieve PM/DM remission, whereas 60% to 80% will experience a polycyclic or chronic, continuous course of the disease. PM/DM further continues to have a great impact on life in medium- and long-term follow-up, as up to 80% of treated patients are still disabled (using Health Assessment Questionnaire scores). The overall mortality ratio in PM/DM patients also remains threefold higher compared with the general population, with cancer, lung, and cardiac complications and infections being the most common causes of deaths. Predictive factors for a poor prognosis in PM/DM patients are older age, involvement of lung and cardiac systems, dysphagia, cancer, and serum myositis-specific antibodies (including coexistent presence of anti-Ro52 and anti-Jo1 antibodies, anti-signal recognition particle antibody, anti-155/140, and anti-CADM-140 antibodies).

Author: I. Marie

Date Published: 2012

Publication Type: Journal

Predictors of survival in a cohort of patients with polymyositis and dermatomyositis: effect of corticosteroids, methotrexate and azathioprine

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INTRODUCTION: The idiopathic inflammatory myopathies are rare diseases for which data regarding the natural history, response to therapies and factors affecting mortality are needed. We performed this … study to examine the effects of treatment and clinical features on survival in polymyositis and dermatomyositis patients. METHODS: A total of 160 consecutive patients (77 with polymyositis and 83 with dermatomyositis) seen at the University of Michigan from 1997 to 2003 were included. Medical records were abstracted for clinical, laboratory and therapeutic data, including initial steroid regimen and immunosuppressive use. State vital records were utilized to derive mortality and cause of death data. Survival was modeled by left-truncated Kaplan-Meier estimation and Cox regression. RESULTS: The 5- and 10-year survival estimates were 77% (95% CI = 66 to 85), and 62% (95% CI = 48 to 73), respectively, and the rates were similar for polymyositis and dermatomyositis. Survival between the sexes was similar through 5 years and significantly lower thereafter for males (10-year survival: 18% male, 73% female; P = 0.002 for 5- to 10-year interval). The sex disparity was restricted to the polymyositis group. Increased age at diagnosis and non-Caucasian race were associated with lower survival. Intravenous versus oral corticosteroid use was associated with a higher risk of death among Caucasians (HR = 10.6, 95% CI = 2.1 to 52.8). Early survival between patients treated with methotrexate versus azathioprine was similar, but survival at 10 years was higher for the methotrexate-treated group (76% vs 52%, P = 0.046 for 5- to 10-year interval). CONCLUSIONS: Patients treated initially with intravenous corticosteroids had higher mortality, which was likely related to disease severity. Both methotrexate and azathioprine showed similar early survival benefits as first-line immunosuppressive drugs. Survival was higher between 5 and 10 years in the methotrexate-treated group, but could not be confirmed in multivariable modeling for the full follow-up period. Other important predictors of long-term survival included younger age, female sex and Caucasian race.

Authors: E. Schiopu, K. Phillips, P. M. MacDonald, L. J. Crofford, E. C. Somers

Date Published: 2012

Publication Type: Journal

Finalisation and validation of the rheumatoid arthritis impact of disease score, a patient-derived composite measure of impact of rheumatoid arthritis: a EULAR initiative

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OBJECTIVE: A patient-derived composite measure of the impact of rheumatoid arthritis (RA), the rheumatoid arthritis impact of disease (RAID) score, takes into account pain, functional capacity, fatigue, … physical and emotional wellbeing, quality of sleep and coping. The objectives were to finalise the RAID and examine its psychometric properties. METHODS: An international multicentre cross-sectional and longitudinal study of consecutive RA patients from 12 European countries was conducted to examine the psychometric properties of the different combinations of instruments that might be included within the RAID combinations scale (numeric rating scales (NRS) or various questionnaires). Construct validity was assessed cross-sectionally by Spearman correlation, reliability by intraclass correlation coefficient (ICC) in 50 stable patients, and sensitivity to change by standardised response means (SRM) in 88 patients whose treatment was intensified. RESULTS: 570 patients (79% women, mean +/- SD age 56 +/- 13 years, disease duration 12.5 +/- 10.3 years, disease activity score (DAS28) 4.1 +/- 1.6) participated in the validation study. NRS questions performed as well as longer combinations of questionnaires: the final RAID score is composed of seven NRS questions. The final RAID correlated strongly with patient global (R=0.76) and significantly also with other outcomes (DAS28 R=0.69, short form 36 physical -0.59 and mental -0.55, p<0.0001 for all). Reliability was high (ICC 0.90; 95% CI 0.84 to 0.94) and sensitivity to change was good (SRM 0.98 (0.96 to 1.00) compared with DAS28 SRM 1.06 (1.01 to 1.11)). CONCLUSION: The RAID score is a patient-derived composite score assessing the seven most important domains of impact of RA. This score is now validated; sensitivity to change should be further examined in larger studies.

Authors: L. Gossec, S. Paternotte, G. J. Aanerud, A. Balanescu, D. T. Boumpas, L. Carmona, M. de Wit, B. A. Dijkmans, M. Dougados, M. Englbrecht, F. Gogus, T. Heiberg, C. Hernandez, J. R. Kirwan, E. M. Mola, M. M. Cerinic, K. Otsa, G. Schett, M. Scholte-Voshaar, T. Sokka, G. von Krause, G. A. Wells, T. K. Kvien

Date Published: 2011

Publication Type: Journal

Patients with polymyositis or dermatomyositis have reduced grip force and health-related quality of life in comparison with reference values: an observational study

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OBJECTIVES: The aims of this study were to investigate hand function in PM and DM patients and compare this with reference values in healthy individuals and also to investigate if hand function correlated … with activity performance and health-related quality of life. METHODS: An observational cross-sectional study was performed in 18 women and 13 men with PM or DM with established disease. Grip force and hand mobility were assessed by Grippit and Escola Paulista de Medicina-Range of Motion scale. Activity performance was measured with myositis activities profile and health-related quality of life by short form-36 (SF-36). RESULTS: Women and men with PM and DM with mean disease duration of 6.8 (5.5) years had a significantly lower grip force than gender- and age-matched healthy individuals (women 71% and men 60%). They also had significantly lower mean values in all dimensions of the health-related quality of life instrument SF-36 compared with the Swedish population. In patients with PM and DM, the grip force correlated significantly with the ability to perform domestic activities. In women with PM and DM, the grip force correlated significantly with the health-related quality of life dimensions vitality and mental health. There were no significant differences between patients with PM and DM regarding grip force, hand mobility, activity performance or health-related quality of life. CONCLUSIONS: Patients with PM or DM have reduced grip force that could influence activity performance and health-related quality of life.

Authors: M. Regardt, E. Welin Henriksson, H. Alexanderson, I. E. Lundberg

Date Published: 2011

Publication Type: Journal

Measures of adult and juvenile dermatomyositis, polymyositis, and inclusion body myositis: Physician and Patient/Parent Global Activity, Manual Muscle Testing (MMT), Health Assessment Questionnaire (HAQ)/Childhood Health Assessment Questionnaire (C-HAQ), Childhood Myositis Assessment Scale (CMAS), Myositis Disease Activity Assessment Tool (MDAAT), Disease Activity Score (DAS), Short Form 36 (SF-36), Child Health Questionnaire (CHQ), physician global damage, Myositis Damage Index (MDI), Quantitative Muscle Testing (QMT), Myositis Functional Index-2 (FI-2), Myositis Activities Profile (MAP), Inclusion Body Myositis Functional Rating Scale (IBMFRS), Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI), Cutaneous Assessment Tool (CAT), Dermatomyositis Skin Severity Index (DSSI), Skindex, and Dermatology Life Quality Index (DLQI)

Abstract

Not specified

Authors: L. G. Rider, V. P. Werth, A. M. Huber, H. Alexanderson, A. P. Rao, N. Ruperto, L. Herbelin, R. Barohn, D. Isenberg, F. W. Miller

Date Published: 2011

Publication Type: Journal

Long-term outcome of patients with polymyositis/ dermatomyositis and anti-PM-Scl antibody

Abstract (Expand)

BACKGROUND: To date, no series has analysed long-term outcome in patients with polymyositis/dermatomyositis (PM/DM) with anti-PM-Scl antibody. OBJECTIVES: The aims of the present study were: (i) to … assess clinical features and long-term outcome, including organ complications, functional course and mortality rate, in patients with isolated PM/DM with anti-PM-Scl antibody; and (ii) to evaluate prevalence, characteristics and long-term outcome of interstitial lung disease (ILD) in patients with isolated PM/DM with anti-PM-Scl antibody. METHODS: The medical records of 20 consecutive patients with isolated PM/DM with anti-PM-Scl antibody were reviewed. RESULTS: Two patients (10%) achieved remission of PM/DM, whereas 14 (70%) improved and four (20%) had a worsened clinical status. Short-term recurrences (during tapering of therapy) occurred in nine patients and long-term recurrences (after discontinuation of therapy) in three patients. Moreover, patients with PM/DM with anti-PM-Scl antibody exhibited severe complications, as follows: oesophageal involvement (n = 4) requiring enteral feeding in three cases, ventilatory insufficiency (n = 3) requiring mechanical ventilation in two cases; three other patients had cancer. Interestingly, patients with PM/DM with anti-PM-Scl antibody often presented symptoms that are usually found in antisynthetase syndrome, i.e. hyperkeratotic rhagadiform hand symptoms (n = 2; 10%), Raynaud’s phenomenon (n = 8; 40%), arthralgia/arthritis (n = 7; 35%) and ILD (n = 12; 60%). In our cohort, the associated ILD often required combined therapy of steroids and immunosuppressive agents. CONCLUSIONS: Our series suggests that the presence of anti-PM-Scl antibody is not a good prognostic factor in patients with PM/DM, as there appears to be an association with lung and oesophageal involvement; in addition, anti-PM-Scl antibody may coexist with malignancy in patients with PM/DM. Furthermore, anti-PM-Scl antibody-positive patients with PM/DM often exhibit ’mechanic’s hands’, Raynaud’s phenomenon and joint involvement. Our latter findings raise the possibility that the immunogenetic background influences the autoantibody status of these patients; HLA-DR3 has, in fact, been found in association with antisynthetase syndrome antibodies and with anti-PM-Scl antibodies.

Authors: I. Marie, L. Lahaxe, O. Benveniste, K. Delavigne, D. Adoue, L. Mouthon, E. Hachulla, J. Constans, K. Tiev, E. Diot, H. Levesque, O. Boyer, F. Jouen

Date Published: 2010

Publication Type: Journal

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