FRI0328 BRANCHED CHAIN AMINO ACIDSIN THE TREATMENT OF POLYMYOSITIS AND DERMATOMYOSITIS: RESULTS FROM THE BTOUGH STUDY

Abstract

Background Muscle functions of patients with polymyositis and dermatomyositis (PM/DM) remain often impaired even after successful control of the immune-mediated muscle injury by immunosuppressive therapy. The only effort at the present to regain muscle functions except for the immunosuppression is rehabilitation, which is carried out systematically in limited institutes. No medicines for rebuilding muscles have been approved. Branched chain amino acids (BCAA) promote skeletal muscle protein synthesis and inhibit muscle atrophy. They thus have positive effects on muscle power, but have never been examined for the effects on PM/DM patients. Objectives To assess the efficacy and safety of BCAA in the treatment of PM/DM for official approval of their use in Japan. Methods Untreated adults with PM/DM were enrolled in a randomized, double-blind trial to receive either TK-98 (drug name of BCAA) or placebo in addition to the conventional immunosuppressive agents. One package of TK-98 (4.15g) contained L-isoleucine 952mg, L-leucine 1904mg, and L-valine 1144mg (molar ratio is 1:2:1.35), and 6 packages were administered daily in 3 divided doses. After 12 weeks, patients with average manual muscle test (MMT) score less than 9.5 were enrolled in an open label extension study for 12 weeks. The primary end point was the change of the MMT score at 12 weeks. The secondary end points were the disease activity evaluated with myositis disease activity core set (MDACS) and the change of functional index (FI), which evaluates dynamic repetitive muscle functions. Results Forty-seven patients were randomized to the TK-98 (24 patients [12 with PM and 12 with DM]) and placebo (23 patients [11 with PM and 12 with DM]) groups. The baseline MMT scores were equivalent (7.97±0.92 [mean±SD] in the TK-98 group and 7.84±0.86 in the placebo group). The change of MMT scores at 12 weeks were 0.70±0.19 (mean±SEM) and 0.69±0.18, respectively (P = 0.98). Thirteen patients from the TK-98 group and 12 from the placebo group were enrolled in the extension study. The MMT scores in both groups improved comparably throughout the extension study. The increase of the FI scores of the shoulder flexion at 12 weeks was significantly larger in the TK-98 group (27.9±5.67 and 12.8±5.67 in the right shoulder flexion [P Conclusion Although BCAA exerted no effects in the improvement of the muscle strength evaluated with MMT, they were effective in the improvement of dynamic repetitive muscle functions in patients with PM/DM without significant increase of adverse events. Disclosure of Interests None declared