Explorative study of emerging blood biomarkers in progressive multiple sclerosis (EmBioProMS)

Objective: To assess whether serum GFAP and NFL might differentiate between progressive vs. non-progressive and active vs. non-active disease stages according to the Lublin criteria. Background: Defining clinical and subclinical progression in multiple sclerosis (MS) is challenging. Patient history, expanded disability status scale (EDSS) and MRI all have shortcomings and may underestimate disease dynamics. Emerging serum biomarkers such as NFL and GFAP proved useful in many cross-sectional studies. However, longitudinal data on progressive MS patients is scarce. Design/Methods: EmBioProMS is a pilot, observational, prospective, multicentric study funded by the German Multiple Sclerosis Society (DMSG). 200 patients with MS according to 2017 McDonald criteria and history of relapse-independent progression at any time, younger than 65 years, and with EDSS ≤ 6.5 will be recruited in 6 centers in Germany. At baseline, month 6 and 18, medical history, EDSS, Nine-Hole-Peg-Test (9-HPT), Timed-25-Foot-Walk-Test (T-25FW), Symbol-Digit-Modalities-Test, serum GFAP and NFL, MRI (at least baseline and month 18) and optional OCT will be performed. Disease progression before and during the study is defined by confirmed EDSS progression, increase by ≥ 20% in 9-HPT or T-25FW time Results: Between 06/2018 and 10/2019, 155 patients were recruited (54.4% PPMS, 45.6% SPMS; median age 55 years (IQR 49 – 61), disease duration 13 years (IQR 6 – 21), female-to-male ratio 1.1:1, median EDSS 5.0 (IQR 3.5 – 6.0)). Retrospectively, 57% of all patients had disease progression, and 35% had clinical or MRI disease activity within two years before baseline. 69 patients completed the month 6 visit. The interim clinical and biomarker analysis is scheduled for 12/2019 and will be available for the conference presentation. Conclusions: The PMS patients recruited in this study reflect the real-world population. The perspective of this multicentre study is to improve monitoring disease progression in concert with blood biomarkers in a real-world setting. Disclosure: Dr. Abdelhak has nothing to disclose. Dr. Huss has nothing to disclose. Dr. Stahmann has received research support from Biogen, Celgene, Merck and Novartis. Dr. Senel has received research support from Bayer, Biogen, Celgene, Roche, Sanofi Genzyme and TEVA. Dr. Krumbholz has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Genzyme, Merck, Roche. Dr. Kowarik has received research support from Biogen, Celgene, Merck, Novartis, Roche, and Sanofi-Genzyme. Dr. Havla has received research support from Merck, Novartis, Roche, Santhera, Biogen, Alexion, and Sanofi Genzyme.Dr. Kuempfel has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Bayer Healthcare, Teva Pharma, Merck, Novartis Pharma, Sanofi-Aventis/Genzyme, CLB Behring, Roche Pharma, and Biogen. Dr. Kuempfel has received research support from Bayer-Schering AG, Novartis, and Chugai Pharma. Dr. Kleiter has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Bayer Health Care, Biogen, Chugai, Merck, Novartis, Shire, Roche. Dr. Kleiter has received research support from Chugai, Diamed. Dr. Wuestinger has nothing to disclose. Dr. Zettl has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Almirall, Biogen Idec, Bayer, Merck Serono, Novartis, Roche, Sanofi and TEVA. Dr. schwartz has nothing to disclose. Dr. Friede has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Bayer, Boehringer Ingelheim, DaiichiSankyo, Feldmann Patent Attorneys, Galapagos, Janssen, Mediconomics, Novartis, Penumbra, Pharmalog, Roche, SGS, UCB. Dr. Ludolph has received research support from Albert C. Ludolph has received consultant fees and honoraria as a speaker from Desitin, GSK and Biogen.. Dr. Ziemann has received research support from Biogen Idec GmbH, Bayer Vital GmbH, Bristol Myers Squibb GmbH, Pfizer, CorTec GmbH, Medtronic. Dr. Kerschensteiner has nothing to disclose. Dr. Hohlfeld has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Actelion, Biogen, Genzyme-Sanofi, Novartis, and Roche. Dr. Hohlfeld has received research support from Biogen, Genzyme-Sanofi, Novartis, and Roche. Dr. Tumani has nothing to disclose.