OP0172 BRENTUXIMAB VEDONTIN FOR SKIN INVOLVEMENT IN REFRACTORY DIFFUSE CUTANEOUS SYSTEMIC SCLEROSIS, INTERIM RESULTS OF A PHASE IIB OPEN-LABEL TRIAL

Background: Systemic sclerosis (SSc) is an autoimmune disease affecting multiple organs causing morbidity and mortality. Treatments targeting SSc skin often have limited success. The presence of CD30+ lymphocytes in biopsies increased levels serum CD30 been reported patients 1 . This could constitute a new therapeutic target. Objectives: To explore the efficacy safety brentuximab vedotin, chimeric anti-CD30 antibody drug conjugate, with severe active diffuse cutaneous who failed treatments. Methods: Phase IIb, single center, open-label, investigator-initiated trial will recruit 10 patients. Brentuximab vedotin 0.6 mg/Kg was infused intravenously every 3 weeks for 48 weeks. Inclusion criteria were age ≥18 years, meeting 2013 ACR/EULAR classification criteria, modified Rodnan score (mRSS) ≥15 <5 years since first non-Raynaud’s symptom and/or worsening despite immunosuppression. Patients allowed to continue their standard care medications except rituximab. cardiac or pulmonary involvement, infections, significant peripheral neuropathy, malignancy excluded. primary objective decrease mRSS ≥8 at main secondary endpoint MRSS 24 Differences assessed by paired t tests. Data compared 16 age, duration, past/present use immunosuppressors-matched controls (ratio 2-3:1) from Canadian Scleroderma Research Group (CSRG) registry. Results: Eight recruited date; two are 8 one withdrawn her request after developing influenza week 12. Five subjects (60% female) reached 24, completed shown Table 1. ΔMRSS treated between 0 8.2 ([CI 95% 2.8, 13.6], p = 0.013) 15.3 8.2, 22.5], 0.012). Whereas, CSRG 3.1 -2, 8.2], 0.211) Assuming that would least be same 2 cases brentuximab, we 5 vs (Figure 1). 12.2 5.9, 18.5], 0.006. No developed neuropathy only SAE (influenza). N (SD ) Age Disease duration 48** Case 60.2 (9.3) 4.5 (2.1) 33 (5.2) 24.8 (6) 15.7 (3) 20.8 (8.3) Control 58.5 4.9 31.3 (5.9) N/D 28.1 (7.5) 0.731 0.775 0.559 0.013 0.079 score, no data, ** comparisons including cases, assuming stability 6 Figure Conclusion: vedontin already achieved weeks, half intended recruitment sample this landmark. A comparison showed decreased significantly brentuximab. interim report suggests might effectively improve involvement involvement. References: [1]Mavalia C, Scaletti Romagnani P, et al. Type helper T-cell predominance high expression systemic sclerosis. Am J Pathol. 1997;151(6):1751-8. Acknowledgements: We like acknowledge Group, Louise Vanderhoek, Sara Macdonald Hewitt Jillian Bylsma collaboration Disclosure Interests: Andreu Fernandez-Codina Consultant of: Bayer, Boehringer Ingelheim, Atheneum consulting, Tatiana Nevskaya: None declared, Janet Pope Speakers bureau: Actelion, Amgen, Abbie, BMS, Eli Lilly, Galapagos, Gilead, Janssen, Medexus, Merck, Novartis, Pfizer, Roche, Samsung, Sandoz, Sanofi, Teva, UCB., Grant/research support from: UCB.