Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Randomized Controlled Trial
. 2023 Mar;200(6):802-811.
doi: 10.1111/bjh.18582. Epub 2022 Dec 5.

Fostamatinib for the treatment of Japanese patients with primary immune thrombocytopenia: A phase 3, placebo-controlled, double-blind, parallel-group study

Masataka Kuwana  1 , Tomoki Ito  2 , Shugo Kowata  3 , Yoshihiro Hatta  4 , Katsumichi Fujimaki  5 , Kensuke Naito  6 , Shingo Kurahashi  7 , Toshiya Kagoo  8 , Kazuki Tanimoto  9 , So Saotome  10 , Yoshiaki Tomiyama  11 ; R788-1301 Investigators
Collaborators, Affiliations
Randomized Controlled Trial

Fostamatinib for the treatment of Japanese patients with primary immune thrombocytopenia: A phase 3, placebo-controlled, double-blind, parallel-group study

Masataka Kuwana et al. Br J Haematol. 2023 Mar.

Abstract

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.

Keywords: Fostamatinib; phase 3 study; primary immune thrombocytopenia; spleen tyrosine kinase; treatment.

PubMed Disclaimer

References

REFERENCES

    1. Cines DB, Blanchette VS. Immune thrombocytopenic purpura. N Engl J Med. 2002;346(13):995-1008.
    1. Zufferey A, Kapur R, Semple JW. Pathogenesis and therapeutic mechanisms in immune thrombocytopenia (ITP). J Clin Med. 2017;6(2):16.
    1. Cohen YC, Djulbegovic B, Shamai-Lubovitz O, Mozes B. The bleeding risk and natural history of idiopathic thrombocytopenic purpura in patients with persistent low platelet counts. Arch Intern Med. 2000;160(11):1630-8.
    1. Hato T, Shimada N, Kurata Y, Kuwana M, Fujimura K, Kashiwagi H, et al. Risk factors for skin, mucosal, and organ bleeding in adults with primary ITP: a nationwide study in Japan. Blood Adv. 2020;4(8):1648-55.
    1. Neunert C, Noroozi N, Norman G, Buchanan GR, Goy J, Nazi I, et al. Severe bleeding events in adults and children with primary immune thrombocytopenia: a systematic review. J Thromb Haemost. 2015;13(3):457-64.

Publication types

LinkOut - more resources