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Peer-reviewedCC BY 4.0International Journal of Hematology

Real-world effectiveness of fostamatinib in patients with immune thrombocytopenia: a single-center retrospective study

  • Katsumichi Fujimaki1
  • Takaaki Takeda1
  • Kota Kawabata1
  • Ayako Bouno1
  • Yuki Nakajima1
  • Hideaki Nakajima2
  1. 1Department of Hematology, Fujisawa City Hospital, 2-6-1, Fujisawa, Fujisawa, Kanagawa 251-8550 Japan
  2. 2Department of Hematology and Clinical Immunology, Yokohama City University School of Medicine, Kanagawa, Japan
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Abstract

Introduction: Immune thrombocytopenia (ITP) is an acquired autoimmune disease characterized by increased platelet destruction and thrombocytopenia. Fostamatinib inhibits spleen tyrosine kinase and suppresses platelet destruction. However, clinical evidence of its efficacy in Japanese patients with ITP remains limited.

Methods: In this study, we retrospectively evaluated the clinical outcomes of fostamatinib, with or without thrombopoietin receptor agonists (TPO-RAs), at a single center in Japan.

Results: The study included 11 patients. At week 1, all 3 evaluable patients (100.0%) achieved platelet count ≥ 30,000/μL and a twofold increase from baseline; at week 2, 10 of 11 (90.9%) met the criteria. Ultimately, 10 of 11 (90.9%) patients achieved the criteria at least once within 24 weeks. All 3 patients using concomitant TPO-RAs (100.0%) achieved the primary endpoint. After fostamatinib initiation, 9 of 11 (81.8%) patients discontinued or reduced their concomitant glucocorticoid (GC) dose. Thrombosis was not observed regardless of concomitant TPO-RA use.

Conclusions: This study demonstrates the real-world effectiveness and safety of fostamatinib in patients with ITP. Platelet count rose soon after fostamatinib initiation, within 1–2 weeks. Fostamatinib may enable reduction or discontinuation of GCs, even in patients with ITP refractory to TPO-RAs.

Trial registration: UMIN000057096

Supplementary Information: The online version contains supplementary material available at https://doi.org/10.1007/s12185-026-04243-w.

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Cite

BibTeXRIS
BibTeX
@article{fujimaki2026realworld,
  title = {{Real-world effectiveness of fostamatinib in patients with immune thrombocytopenia: a single-center retrospective study}},
  author = {Katsumichi Fujimaki and Takaaki Takeda and Kota Kawabata and Ayako Bouno and Yuki Nakajima and Hideaki Nakajima},
  journal = {International Journal of Hematology},
  year = {2026},
  month = aug,
  doi = {10.1007/s12185-026-04243-w},
  url = {https://doi.org/10.1007/s12185-026-04243-w},
  note = {CC BY 4.0},
}
RIS
TY  - JOUR
TI  - Real-world effectiveness of fostamatinib in patients with immune thrombocytopenia: a single-center retrospective study
AU  - Katsumichi Fujimaki
AU  - Takaaki Takeda
AU  - Kota Kawabata
AU  - Ayako Bouno
AU  - Yuki Nakajima
AU  - Hideaki Nakajima
PY  - 2026
DA  - 2026/08/04
JO  - International Journal of Hematology
DO  - 10.1007/s12185-026-04243-w
UR  - https://doi.org/10.1007/s12185-026-04243-w
AB  - Introduction: Immune thrombocytopenia (ITP) is an acquired autoimmune disease characterized by increased platelet destruction and thrombocytopenia. Fostamatinib inhibits spleen tyrosine kinase and suppresses platelet destruction. However, clinical evidence of its efficacy in Japanese patients with ITP remains limited.  Methods: In this study, we retrospectively evaluated the clinical outcomes of fostamatinib, with or without thrombopoietin receptor agonists (TPO-RAs), at a single center in Japan.  Results: The study included 11 patients. At week 1, all 3 evaluable patients (100.0%) achieved platelet count ≥ 30,000/μL and a twofold increase from baseline; at week 2, 10 of 11 (90.9%) met the criteria. Ultimately, 10 of 11 (90.9%) patients achieved the criteria at least once within 24 weeks. All 3 patients using concomitant TPO-RAs (100.0%) achieved the primary endpoint. After fostamatinib initiation, 9 of 11 (81.8%) patients discontinued or reduced their concomitant glucocorticoid (GC) dose. Thrombosis was not observed regardless of concomitant TPO-RA use.  Conclusions: This study demonstrates the real-world effectiveness and safety of fostamatinib in patients with ITP. Platelet count rose soon after fostamatinib initiation, within 1–2 weeks. Fostamatinib may enable reduction or discontinuation of GCs, even in patients with ITP refractory to TPO-RAs.  Trial registration: UMIN000057096  Supplementary Information: The online version contains supplementary material available at https://doi.org/10.1007/s12185-026-04243-w.
SN  - 0925-5710
SN  - 1865-3774
ER  -