OBJECTIVES: The phase 3 ProDERM study demonstrated intravenous immunoglobulin (IVIg) was safe and effective in patients with dermatomyositis (DM). This analysis assessed clinical and serological predictors of IVIg response in DM patients from ProDERM. METHODS: ProDERM was a prospective, randomized, placebo-controlled study of DM patients. For weeks 0-16, patients received 2.0 g/kg IVIg (Octagam, 10%) or placebo every 4 weeks. Eligible patients entered the open-label extension phase, where all received IVIg to week 40. Univariate and multivariate analyses examined associations between baseline variables and total improvement score (TIS), including myositis disease activity assessment tool (MDAAT; assessing different organ involvement), and myositis-specific and myositis-associated autoantibodies. RESULTS: Ninety-five patients were enrolled. Univariate analyses found no significant association between TIS at week 16 or 40 and age; sex; ethnicity; disease duration/activity; cutaneous, skeletal, gastrointestinal or muscle disease activity; or previous failed or concomitant medications. Multivariate analysis found patients with higher MDAAT cutaneous scores had a better chance of at least minimal TIS improvement. Higher MDAAT pulmonary scores were associated with a lower, but still considerable, chance of improvement. Patients with TIF1-γ antibodies had a better TIS response; however, after controlling for cutaneous disease activity, there was no significant association between antibody classification (including anti-TIF1-γ) and efficacy outcome. CONCLUSION: IVIg was effective in treating DM patients regardless of demographic features and autoantibody status (for most autoantibodies). Patients with higher cutaneous disease activity and/or anti-TIF1-γ responded best to IVIg, while pulmonary disease activity predicted a lower, but still effective, IVIg response, warranting further investigation. TRIAL REGISTRATION: ClinicalTrials.gov, http://clinicaltrials.gov, NCT02728752.

Department of Dermatology and Allergology University of Szeged Szeged Hungary

Department of Neurology Friedrich Baur Institute Ludwig Maximilians University of Munich Munich Germany

Department of Neurology University of Kansas Medical Center Kansas City KS USA

Division of Clinical Immunology Faculty of Medicine University of Debrecen Debrecen Hungary

Division of Rheumatology and Clinical Immunology University of Pittsburgh School of Medicine Pittsburgh PA USA

Division of Rheumatology Department of Medicine University of California Los Angeles CA USA

Division of Rheumatology Medical College of Georgia at Augusta University Augusta GA USA

Global Medical and Scientific Affairs Octapharma Pharmazeutika Produktionsges m b H Vienna Austria

Institute of Rheumatology Research Department and Department of Rheumatology 1st Medical Faculty Charles University Prague Czech Republic

Phoenix Neurological Associates Ltd Phoenix AZ USA

Tareev Clinic of Internal Diseases Sechenov 1st Moscow State Medical University Moscow Russia

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NCT02728752