Fostamatinib for the treatment of Japanese patients with primary immune thrombocytopenia: A phase 3, placebo‐controlled, double‐blind, parallel‐group study. (5th December 2022)

• Record Type: Journal Article
• Authors: Kuwana, Masataka; Ito, Tomoki; Kowata, Shugo; Hatta, Yoshihiro; Fujimaki, Katsumichi; Naito, Kensuke; Kurahashi, Shingo; Kagoo, Toshiya; Tanimoto, Kazuki; Saotome, So; Tomiyama, Yoshiaki
• Other Names: Koike Michiaki investigator.; Nakajima Yuki investigator.; Harada Hiroshi investigator.; Hangaishi Akira investigator.; Yokoyama Kenji investigator.; Cho Ryuko investigator.; Kyoda Katsunori investigator.; Kakinoki Yasutaka investigator.; Yoshida Masahiro investigator.; Shimizu Seiichi investigator.; Kashiwagi Hirokazu investigator.; Kirito Keita investigator.; Yokota Akira investigator.; Kikuchi Takahide investigator.; Harada Naoki investigator.; Imamura Yutaka investigator.; Yano Tomofumi investigator.; Kosugi Hiroshi investigator.; Ogawa Ryosuke investigator.; Okamura Atsuo investigator.; Yamaguchi Masaki investigator.; Yoshimitsu Makoto investigator.; Motomura Sayuri investigator.; Ito Toshiro investigator.; Yamanouchi Jun investigator.; Katsutani Shinya investigator.; Hiramatsu Yasushi investigator.; Asagoe Kohsuke investigator.
• Is Part Of: British journal of haematology. Volume 200:Number 6(2023)
• Page Start: 802
• Page End: 811
• Publication Date: 2022-12-05
• Subjects: Fostamatinib -- phase 3 study -- primary immune thrombocytopenia -- spleen tyrosine kinase -- treatment; Hematology -- Periodicals; Blood -- Diseases -- Periodicals; 616.15
• DOI: 10.1111/bjh.18582
• Languages: English
• ISSNs: 0007-1048

Abstract

Summary: Fostamatinib, a spleen tyrosine kinase inhibitor, has been approved for the treatment of chronic primary immune thrombocytopenia (ITP) in the United States, Canada and some European countries. We conducted a phase 3, placebo‐controlled, double‐blind, parallel‐group study to evaluate the efficacy and safety of fostamatinib in Japanese patients with primary ITP. Thirty‐four patients were randomised to fostamatinib ( n = 22) or placebo ( n = 12) at 100–150 mg twice a day for 24 weeks. Stable responses (platelet ≥50 000/μl at ≥4 of the 6 visits from weeks 14 to 24) were observed in eight (36%) patients on fostamatinib and in none of the patients on placebo ( p = 0.030). Overall responses (platelet ≥50 000/μl at ≥1 of the 6 visits from weeks 2 to 12) were seen in 10 (45%) patients on fostamatinib and in none of the patients on placebo ( p = 0.006). Patients on fostamatinib required rescue medication less often and experienced fewer bleeding symptoms than patients on placebo. Adverse events observed were mild or moderate and were manageable. No new safety signals were identified in Japanese patients with ITP.