Ghanima Waleed, Lucas Boronat Francisco Javier, Carrai Valentina, Rackwitz Stefan
Department of Research, Sarpsborg and Department of Hematology, Institute of Clinical Medicine, Østfold Hospital Trust, Sarpsborg, Norway.
Hospital General Universitario Dr. Balmis de Alicante, Alicante, Spain.
Hematology. 2025 Dec;30(1):2456687. doi: 10.1080/16078454.2025.2456687. Epub 2025 Feb 2.
A goal of most primary immune thrombocytopenia (ITP) treatments is reducing or discontinuing treatment while maintaining a response including an absence of bleeding events. We present four cases describing treatment with the spleen tyrosine kinase (SYK) inhibitor, fostamatinib, that showed sustained response off treatment (SROT).
Case 1 was a 66-year-old male with chronic ITP. He was pre-treated with prednisone and rituximab before being in the FIT-2 clinical trial (placebo). He received fostamatinib in the FIT-3 open-label extension for seven weeks and maintained SROT for 2.5 years. Case 2 was a 54-year-old female patient with chronic, highly refractory ITP. SROT was achieved after 6 months of fostamatinib and was maintained for more than 16 months (in remission to date). Case 3 was a 60-year-old male with chronic ITP. He was successfully treated with cycles of corticosteroids for six years prior to fostamatinib. He was treated with fostamatinib plus prednisone for approximately two months. SROT was observed in this patient for one year. Case 4 was a 67-year-old male with persistent ITP. Before fostamatinib, he was unresponsive to high-dose dexamethasone, IVIG, eltrombopag and romiplostim. After 11 months of fostamatinib, his dose was tapered for three months and ultimately discontinued. SROT was observed for more than ten months (in remission to date).
These cases emphasize that SROT is achievable with fostamatinib in complex ITP cases unresponsive to multiple previous therapies. Additional research is needed to identify the magnitude of the underlying mechanisms, and the clinical factors associated with, and potentially predictive of, SROT.
大多数原发性免疫性血小板减少症（ITP）治疗的目标是在维持包括无出血事件在内的反应的同时减少或停止治疗。我们介绍了4例使用脾酪氨酸激酶（SYK）抑制剂福他替尼治疗的病例，这些病例显示出停药后持续缓解（SROT）。
病例1是一名66岁的慢性ITP男性。在参加FIT-2临床试验（安慰剂组）之前，他接受了泼尼松和利妥昔单抗的预处理。他在FIT-3开放标签扩展试验中接受了7周的福他替尼治疗，并维持SROT达2.5年。病例2是一名54岁的慢性、高度难治性ITP女性患者。接受福他替尼治疗6个月后实现SROT，并维持超过16个月（至今仍处于缓解期）。病例3是一名60岁的慢性ITP男性。在使用福他替尼之前，他成功接受了6年的糖皮质激素周期治疗。他接受了约两个月的福他替尼加泼尼松治疗。该患者观察到SROT达1年。病例4是一名67岁的持续性ITP男性。在使用福他替尼之前，他对高剂量地塞米松、静脉注射免疫球蛋白、艾曲泊帕和罗米司亭均无反应。接受福他替尼治疗11个月后，他的剂量逐渐减少3个月，最终停药。观察到SROT超过10个月（至今仍处于缓解期）。
这些病例强调，在对多种先前治疗无反应的复杂ITP病例中，使用福他替尼可实现SROT。需要进一步研究以确定潜在机制的程度，以及与SROT相关并可能预测SROT的临床因素。
