2239: Effect of Treatment with IVIG (octagam10%) on Skin Symptoms and Quality of Life in Subjects with Dermatomyositis. Results of a Large, Randomized, Placebo-controlled International Phase III Trial

Rohit Aggarwal¹, Christina Charles-Schoeman², joachim Schessl³, Victoria Werth⁴, Zsuzsanna Bata-Csorgo⁵, Mazen Dimachkie⁶, Zoltan Griger⁷, Sergey Moiseev⁸, Chester Oddis⁹, Elena Schiopu¹⁰, Jiří Vencovský¹¹, Irene Beckmann¹², Elisabeth Clodi¹³, Todd Levine¹⁴ and and the ProDERM investigators¹⁵, ¹Division of Rheumatology and Clinical Immunology, Department of Medicine, University of Pittsburgh, Pittsburgh, PA, ²Division of Rheumatology, University of California, Los Angeles, Santa Monica, CA, ³Friedrich-Baur-Institut/Medical University Munich, Mainkofen, Germany, ⁴Philadelphia VAMC, Philadelphia, PA, USA and Department of Dermatology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, ⁵University of Szeged, Faculty of Medicine, Szeged, Hungary, ⁶University of Kansas Medical Center, Kansas City, KS, ⁷University of Debrecen, Debrecen, Hungary, ⁸First Moscow State Medical University, Moscow, Russia, ⁹University of Pittsburgh, Pittsburgh, PA, ¹⁰Michigan Medicine Rheumatology Clinic – Taubman Center, Ann Arbor, MI, ¹¹Institute of Rheumatology and Department of Rheumatology, First Faculty of Medicine, Charles University, Prague, Czech Republic, ¹²Octapharma PharmazeutikaProduktionsGesmbH, Vienna, Austria, ¹³Octapharma Pharmazeutika ProduktionsgesmbH, Vienna, Austria, ¹⁴Phoenix Neurological Associates, Ltd., Phoenix, AZ, ¹⁵Institutions in Europe and North America, Vienna, Austria

Background/Purpose: Dermatomyositis (DM) is a rare chronic systemic autoimmune disease with characteristic skin rashes and progressive proximal muscle weakness. The efficacy of IVIg treatment in improving muscle symptoms was recently shown. Here we focus on the effect of IVIg treatment on skin involvement and QoL.

Methods: The ProDERM trial was a double-blind, randomized, placebo-controlled, international, phase III clinical trial and consisted of the First Period (16 weeks; double-blind, placebo-controlled) and subsequent Extension Period (24 weeks; open-label – all IVIG). Patients received a total of 10 infusions (only IVIG or placebo-IVIG) in 4-weekly administrations.

The primary endpoint was the proportion of responders in TIS in IVIG vs. placebo arm at week 16. Secondary endpoints included mean change in CDASI from baseline (Week 0) to end of First Period (Week 16) as well as mean change from the end of First Period (Week 16) to end of Extension Period (Week 40) in CDASI. Mean change from Baseline (Week 0) to end of First Period (Week 16) and Extension Period (Week 40) in SF-36v2 Health Survey was also evaluated.

Results: Ninety-five adult DM patients (mean age: 53 years; 75% females) were enrolled (47- in the IVIG arm and 48 in the placebo arm). Clinical baseline characteristics (e.g. mean CDASI scores) were similar in both arms.

At week 16, the number of TIS-responders was significantly higher in the IVIG (37/47; 78.7%) than in the placebo arm (21/48; 43.8%; p-value 0.0008).

The least square means for change from Baseline to Week 16 were -10.3 and -2.3 for IVIG versus placebo in CDASI total activity score and -0.7 versus -0.1 for total damage. Differences between arms were statistically significant for activity (-8.0: 95% CI -11.5, -4.6; p < 0.0001) and damage (-0.6: 95% CI -1.1, -0.1; p=0.0304). After switching to IVIG, placebo patients attained similarly improved disease control at Week 40.

For SF-36v2 Health Survey a statistically significant effect of treatment (p=0.0057) for physical functioning was detected at Week 16. All other values increased from baseline to the end of the placebo-controlled period in both arms. The largest changes from baseline were seen at Week 16 in the octagam 10% group and at Week 40 in the placebo-IVIG group.

Conclusion: This large international, randomized, placebo-controlled phase III trial demonstrated the efficacy of IVIG treatment to improve skin symptoms in dermatomyositis patients. It also showed a significant difference in the improvement of physical functioning between patients treated with IVIG versus placebo.

Disclosures: R. Aggarwal, Mallinckrodt, Bristol Myers Squibb, EMD Serono, Pfizer, Octapharma, CSL Behring, Q32, Kezar, AstraZeneca, Alexion, Argenx, Boehringer Ingelheim, Corbus, Janssen, Kyverna, Roivant, AbbVie, Jubilant, Orphazyme, Genentech; C. Charles-Schoeman, AbbVie, Bristol Myers Squibb (BMS), Pfizer, Regeneron-Sanofi, Gilead; j. Schessl, Octapharma; V. Werth, GlaxoSmithKline, CLASI; Z. Bata-Csorgo, Sanofi-Genzyme, Berlin-Chemie; M. Dimachkie, Janssen, argenx, Amazentis, Catalyst, Cello, LabcorpCovance, CSL-Behring, EcoR1, Kezar, Medlink, Momenta, NuFactor, Octapharma, RaPharma/UCB, Roivant Sciences Inc, Sanofi Genzyme, Shire Takeda, Scholar Rock, Spark Therapeutics, Abata/Third Rock, , UCB Biopharma, UpToDate; Z. Griger, AbbVie/Abbott, Pfizer, Novartis, Eli Lilly, Boehringer-Ingelheim, Roche, Octapharma, CSL-Behring, Corbus; S. Moiseev, None; C. Oddis, None; E. Schiopu, Janssen; J. Vencovský, Abbvie, Biogen, Boehringer, Eli Lilly, Gilead, Kezar, Merck, Novartis, Octapharma, Pfizer, Takeda, UCB, Werfen, Argenx; I. Beckmann, Octapharma PharmazeutikaProduktionsGesmbH; E. Clodi, Octapharma PharmazeutikaProduktionsGesmbH; T. Levine, Octapharma; a. ProDERM investigators, None.