Efficacy of Anakinra in Refractory Adult-Onset Still's Disease: Multicenter Study of 41 Patients and Literature Review

Adult-onset Still's disease (AOSD) is often refractory to standard therapy. Anakinra (ANK), an interleukin-1 receptor antagonist, has demonstrated efficacy in single cases and small series of AOSD. We assessed the efficacy of ANK in a series of AOSD patients. Multicenter retrospective open-label study. ANK was used due to lack of efficacy to standard synthetic immunosuppressive drugs and in some cases also to at least 1 biologic agent. Forty-one patients (26 women/15 men) were recruited. They had a mean age of 34.4 ± 14 years and a median [interquartile range (IQR)] AOSD duration of 3.5 [2-6] years before ANK onset. At that time the most common clinical features were joint manifestations 87.8%, fever 78%, and cutaneous rash 58.5%. ANK yielded rapid and maintained clinical and laboratory improvement. After 1 year of therapy, the frequency of joint and cutaneous manifestations had decreased to 41.5% and to 7.3% respectively, fever from 78% to 14.6%, anemia from 56.1% to 9.8%, and lymphadenopathy from 26.8% to 4.9%. A dramatic improvement of laboratory parameters was also achieved. The median [IQR] prednisone dose was also reduced from 20 [11.3-47.5] mg/day at ANK onset to 5 [0-10] at 12 months. After a median [IQR] follow-up of 16 [5-50] months, the most important side effects were cutaneous manifestations (n = 8), mild leukopenia (n = 3), myopathy (n = 1), and infections (n = 5). ANK is associated with rapid and maintained clinical and laboratory improvement, even in nonresponders to other biologic agents. However, joint manifestations are more refractory than the systemic manifestations

Biologic therapy in refractory non-multiple sclerosis optic neuritis isolated or associated to immune-mediated inflammatory diseases. A multicenter study

We aimed to assess the efficacy of biologic therapy in refractory non-Multiple Sclerosis (MS) Optic Neuritis (ON), a condition more infrequent, chronic and severe than MS ON. This was an open-label multicenter study of patients with non-MS ON refractory to systemic corticosteroids and at least one conventional immunosuppressive drug. The main outcomes were Best Corrected Visual Acuity (BCVA) and both Macular Thickness (MT) and Retinal Nerve Fiber Layer (RNFL) using Optical Coherence Tomography (OCT). These outcome variables were assessed at baseline, 1 week, and 1, 3, 6 and 12 months after biologic therapy initiation. Remission was defined as the absence of ON symptoms and signs that lasted longer than 24 h, with or without an associated new lesion on magnetic resonance imaging with gadolinium contrast agents for at least 3 months. We studied 19 patients (11 women/8 men; mean age, 34.8 +/- 13.9 years). The underlying diseases were Bechet's disease (n = 5), neuromyelitis optica (n = 3), systemic lupus erythematosus (n = 2), sarcoidosis (n = 1), relapsing polychondritis (n = 1) and anti-neutrophil cytoplasmic antibody -associated vasculitis (n = 1). It was idiopathic in 6 patients. The first biologic agent used in each patient was: adalimumab (n = 6), rituximab (n = 6), infliximab (n = 5) and tocilizumab (n = 2). A second immunosuppressive drug was simultaneously used in 11 patients: methotrexate (n = 11), azathioprine (n = 2), mycophenolate mofetil (n = 1) and hydroxychloroquine (n = 1). Improvement of the main outcomes was observed after 1 year of therapy when compared with baseline data: mean +/- SD BCVA (0.8 +/- 0.3 LogMAR vs. 0.6 +/- 0.3 LogMAR; p = 0.03), mean +/- SD RNFL (190.5 +/- 175.4 mum vs. 183.4 +/- 139.5 mum; p = 0.02), mean +/- SD MT (270.7 +/- 23.2 mum vs. 369.6 +/- 137.4 mum; p = 0.03). Besides, the median (IQR) prednisone-dose was also reduced from 40 (10-61.5) mg/day at baseline to. 2.5 (0-5) mg/day after one year of follow-up; p = 0.001. After a mean +/- SD follow-up of 35 months, 15 patients (78.9%) achieved ocular remission, and 2 (10.5%) experienced severe adverse events. Biologic therapy is effective in patients with refractory non-MS ON

ABATACEPT in rheumatoid arthritis with interstitial lung disease. A multicenter study of 181 patients

Background Interstitial Lung Disease (ILD) is a severe extraarticular manifestation of rheumatoid arthritis (RA). Objectives Our aim was to assess the efficacy of abatacept (ABA) in RA patients with ILD. Methods Retrospective multicenter study of RA patients with ILD treated with ABA. ILD was diagnosed by HRCT. We have analyzed the following variables: a) 1-point change the Modified Medical Research Council (MMRC); b) FVC improvement ≥10%; and improvement ≥10% in DLCO; c) radiological improvement in HRCT scan, d) changes in DAS28 score. Values were compared with baseline e) prednisone doses Results We studied 181 patients (94women/87 men) with ILD associated to RA. The follow-up was 12.1[6.2-24.1] months. The mean age was 64.54 ± 9.7 years. The median to progression of ILD was 12 [3-43.75] months. 81 patients were treated in monotherapy, 100 patients in combination therapy. The most frequent pattern was UIP 45,3%. The most of patients who did not have dyspnea remained asymptomatic. See results in Figure1. DAS28 also improved. We appreciate a decrease in the dose of prednisone compared to the initial dose. Conclusion ABA seems to be effective. However, should be verified in prospective and randomized studies