Branched chain amino acids in the treatment of polymyositis and dermatomyositis: a phase II/III, multi-centre, randomized controlled trial.

Takashi Kanda, Hitoshi Kohsaka, Hideto Kameda, Akio Morinobu, Yasuhiko Nishioka, Masataka Kuwana, Hiroshi Nakajima, Yusuke Miwa, Keiju Hiromura, Tomonori Ishii, Masatoshi Jinnin, Atsushi Kawakami, Yoshiya Tanaka, Naoki Kimura, Yoshitaka Imura, Yoshinao Muro, Toshihiro Matsui, Yuko Kaneko, Yukiko Iwasaki, Hirofumi Amano, Yasushi Kawaguchi, Toshihde Mimura, Takayuki Sumida, Tatsuya Atsumi, Yukari Uemura, Takuya Kawahara

Journal: Rheumatology (Oxford, England) 2023;61(11):4445-4454

PMID: 35179548

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Abstract

OBJECTIVES

To assess the efficacy and safety of branched chain amino acids (BCAAs) in the treatment of PM/DM prior to official approval of their use in Japan.

METHODS

Treatment naïve adults with PM/DM were enrolled in a randomized, double-blind trial to receive either TK-98 (drug name of BCAAs) or placebo in addition to conventional treatment. After 12 weeks, patients with an average manual muscle test (MMT) score <9.5 were enrolled in an open label extension study for a further 12 weeks. The primary endpoint was the change of the MMT score at 12 weeks. The secondary endpoints were the clinical response and the change of functional index (FI).

RESULTS

Forty-seven patients were randomized either to the TK-98 (n = 24) or placebo (n = 23) group. The changes of MMT scores at 12 weeks were 0.70 (0.19) [mean (s.e.m.)] 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 similarly. The increase of the FI scores of the shoulder flexion at 12 weeks was significantly greater in the TK-98 group [27.9 (5.67) vs 12.8 (5.67) for the right shoulder flexion, and 27.0 (5.44) vs 13.4 (5.95) for the left shoulder; P < 0.05]. Frequencies of adverse events up to 12 weeks were similar.

CONCLUSION

BCAAs showed no effect on the improvement of the muscle strength evaluated by MMT and the clinical response. However, they were partly effective for improving dynamic repetitive muscle functions.

TRIAL REGISTRATION

UMIN-CTR Clinical Trial, https://center6.umin.ac.jp/, UMIN000016233.

Address: Department of Rheumatology.; Department of Lifetime Clinical Immunology, Graduate School of Medical and Dental Sciences, Tokyo Medical and Dental University (TMDU).; Clinical Research Promotion Center, The University of Tokyo Hospital.; Clinical Research Center, National Center for Global Health and Medicine, Tokyo.; Department of Rheumatology, Endocrinology and Nephrology, Faculty of Medicine, Hokkaido University, Hokkaido.; Department of Internal Medicine, Faculty of Medicine, University of Tsukuba, Ibaraki.; Department of Rheumatology and Applied Immunology, Faculty of Medicine, Saitama Medical University, Saitama.; Department of Rheumatology, School of Medicine, Tokyo Women's Medical University.; Department of Rheumatology and Internal Medicine, Juntendo University School of Medicine.; Department of Allergy and Rheumatology, Graduate School of Medicine, The University of Tokyo, Tokyo.; Department of Palliative Medicine, Faculty of Medicine, Saitama Medical University, Saitama.; Division of Rheumatology, Department of Internal Medicine, Keio University School of Medicine, Tokyo.; Department of Rheumatology, Clinical Research Center for Allergy and Rheumatology, National Hospital Organization Sagamihara National Hospital, Sagamihara, Kanagawa.; Department of Dermatology, Nagoya University Graduate School of Medicine, Nagoya.; Department of Rheumatology and Clinical Immunology, Graduate School of Medicine, Kyoto University, Kyoto.; Department of Clinical Immunology and Rheumatology, Tazuke-Kofukai Medical Research Institute, Kitano Hospital, Osaka.; Department of Neurology and Clinical Neuroscience, Yamaguchi University Graduate School of Medicine, Yamaguchi.; The First Department of Internal Medicine, School of Medicine, University of Occupational and Environmental Health, Kitakyushu.; Department of Immunology and Rheumatology, Division of Advanced Preventive Medical Sciences, Nagasaki University Graduate School of Biomedical Sciences, Nagasaki.; Department of Dermatology, Wakayama Medical University Graduate School of Medicine, Wakayama.; Clinical Research, Innovation and Education Center, Tohoku University Hospital, Sendai.; Department of Nephrology and Rheumatology, Gunma University Graduate School of Medicine, Maebashi.; Department of Medicine, Division of Rheumatology, Showa University School of Medicine, Tokyo.; Department of Allergy and Clinical Immunology, Chiba University Hospital, Chiba.; Department of Allergy and Rheumatology, Nippon Medical School Graduate School of Medicine, Tokyo.; Department of Respiratory Medicine and Rheumatology, Graduate School of Biomedical Sciences, Tokushima University, Tokushima.; Section of Rheumatology, Department of Internal Medicine, Kobe University Graduate School of Medicine, Kobe.; Department of Internal Medicine, Division of Rheumatology, Faculty of Medicine, Toho University, Tokyo.; Rheumatology Center, Chiba-Nishi General Hospital, Matsudo, Japan.

DOI: 10.1093/rheumatology/keac101

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MeSH Terms: Adult, Humans, Amino Acids, Branched-Chain, Dermatomyositis, Double-Blind Method, Muscle Strength, Polymyositis, Treatment Outcome