"No evidence of disease activity"-Status in einer "real-world"-Patientenkohorte mit schubförmig remittierender Multipler Sklerose in Deutschland

Summary Background With the approval of innovative and effective therapeutic treatment concepts the therapy of relapsing remitting multiple sclerosis (RRMS) has changed. The concept of “no evidence of disease activity” (NEDA) became a new treatment goal. NEDA-3 concept compromises freedom of clinical disease progression and relapses as well as inflammatory MRI activity. So far, data on NEDA status were mainly collected in post-hoc analyses of drug approval studies. Yet, less is known about the significance in “real-world” clinical settings under new MS therapies. For this reason our study aims at investigation of NEDA in a large heterogeneous German cohort of relapsing MS patients or patients with clinically isolated syndrome. Methods Data of 306 patients with relapsing remitting multiple sclerosis or with clinically isolated syndrome from 2009 to 2016 were included in this retrospective, single-center study at the Department of Neurology of the University Hospital Erlangen. Inclusion required sufficient clinical information and standardized in-house cranial MRI data sets at baseline and at follow-up at least after one year or, if the data sets were complete, up to two or three follow-up years. In this cohort NEDA-3-status, its correlation to clinical features, its association to disease-modifying therapies or NEDA failure (evidence of disease EDA) were analyzed. Results In this heterogeneous cohort of patients with RRMS at early stages of disease (median EDSS 1,5, mean disease duration 30 months) at baseline, 45,1% retained NEDA-3 after one year, 28,9% after two years follow-up and 21,4% after three years. The detection of inflammatory MRI-activity was the reason for loss of NEDA in 63,9%. Clinically isolated syndrome patients sustained more frequently NEDA status. The treatment with monoclonal antibody therapies showed an advantage in sustaining NEDA status as compared to injectables or oral therapies. Discussion Our “real-world” analysis confirmed the potential and adaptability of NEDA for the evaluation and surveillance of MS disease activity, progression and therapy efficacy. In spite of application of high efficient immunomodulatory treatment, NEDA-3 was difficult to sustain. Only a minority preserved NEDA-3. Monoclonal antibody treatment was attended with higher NEDA rates. Further investigations have to be performed beyond of standardized clinical or medication approval studies to finally evaluate the value of NEDA concept in “real-world” settings