Tomoyuki Fujisawa1, Hironao Hozumi1, Yosuke Kamiya1, Yusuke Kaida2, Taisuke Akamatsu3, Hideki Kusagaya4, Yasuomi Satake5, Kazutaka Mori6, Masashi Mikamo7, Hiroyuki Matsuda8, Koshi Yokomura9, Naoki Koshimizu10, Mikio Toyoshima11, Shiro Imokawa12, Hideki Yasui1, Yuzo Suzuki1, Masato Karayama1, Kazuki Furuhashi1, Noriyuki Enomoto1, Yutaro Nakamura1, Naoki Inui1,13, Takafumi Suda1. 1. Second Division, Department of Internal Medicine, Hamamatsu University School of Medicine, Hamamatsu, Japan. 2. Department of Respiratory Medicine, Enshu Hospital, Hamamatsu, Japan. 3. Department of Respiratory Medicine, Shizuoka General Hospital, Shizuoka, Japan. 4. Department of Respiratory Medicine, Shizuoka Saiseikai General Hospital, Shizuoka, Japan. 5. Department of Respiratory Medicine, Shizuoka City Shizuoka Hospital, Shizuoka, Japan. 6. Department of Respiratory Medicine, Shizuoka City Shimizu Hospital, Shizuoka, Japan. 7. Department of Respiratory Medicine, Hamamatsu Medical Center, Hamamatsu, Japan. 8. Department of Respiratory Medicine, Japanese Red Cross Shizuoka Hospital, Shizuoka, Japan. 9. Department of Respiratory Medicine, Seirei Mikatahara General Hospital, Hamamatsu, Japan. 10. Department of Respiratory Medicine, Fujieda Municipal General Hospital, Fujieda, Japan. 11. Department of Respiratory Medicine, Hamamatsu Rosai Hospital, Hamamatsu, Japan. 12. Department of Respiratory Medicine, Iwata City Hospital, Iwata, Japan. 13. Department of Clinical Pharmacology and Therapeutics, Hamamatsu University School of Medicine, Hamamatsu, Japan.
Abstract
BACKGROUND AND OBJECTIVE: The efficacy of combination therapy with corticosteroids and CNI, TAC and CsA, for PM/DM-ILD has been described retrospectively. However, it remains unknown which CNI treatment regimens, TAC or CsA regimens, are more effective as initial treatments for patients with PM/DM-ILD. METHODS: We conducted a prospective multicentre, open-label, randomized, 52-week phase 2 trial. Patients with PM/DM-ILD were randomly allocated to receive PSL plus TAC (TAC group) or PSL plus CsA (CsA group). The primary endpoint was PFS rate in the intention-to-treat population at 52 weeks. The secondary endpoints were OS rate at 52 weeks, changes in pulmonary function tests from baseline to 52 weeks and AE. RESULTS:Fifty-eight patients were randomly assigned to the TAC group (n = 30) and the CsA group (n = 28). The PFS rates at 52 weeks were 87% in the TAC group and 71% in the CsA group (P = 0.16). The OS rates at 52 weeks were 97% in the TAC group and 93% in the CsA group (P = 0.50). The %FVC at 52 weeks in the per-protocol populations significantly increased in both groups. None of the patients discontinued the treatment due to AE. CONCLUSION:PSL plus TAC treatment may achieve a better short-term PFS rate compared with PSL plus CsA treatment. Further studies must be conducted to evaluate the long-term efficacy and safety of such treatment.
RCT Entities:
BACKGROUND AND OBJECTIVE: The efficacy of combination therapy with corticosteroids and CNI, TAC and CsA, for PM/DM-ILD has been described retrospectively. However, it remains unknown which CNI treatment regimens, TAC or CsA regimens, are more effective as initial treatments for patients with PM/DM-ILD. METHODS: We conducted a prospective multicentre, open-label, randomized, 52-week phase 2 trial. Patients with PM/DM-ILD were randomly allocated to receive PSL plus TAC (TAC group) or PSL plus CsA (CsA group). The primary endpoint was PFS rate in the intention-to-treat population at 52 weeks. The secondary endpoints were OS rate at 52 weeks, changes in pulmonary function tests from baseline to 52 weeks and AE. RESULTS: Fifty-eight patients were randomly assigned to the TAC group (n = 30) and the CsA group (n = 28). The PFS rates at 52 weeks were 87% in the TAC group and 71% in the CsA group (P = 0.16). The OS rates at 52 weeks were 97% in the TAC group and 93% in the CsA group (P = 0.50). The %FVC at 52 weeks in the per-protocol populations significantly increased in both groups. None of the patients discontinued the treatment due to AE. CONCLUSION:PSL plus TAC treatment may achieve a better short-term PFS rate compared with PSL plus CsA treatment. Further studies must be conducted to evaluate the long-term efficacy and safety of such treatment.