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首次使用戈利替尼治疗伴有STAT3突变及溶血性贫血的难治性自然杀伤细胞大颗粒淋巴细胞白血病:1例报告

Golidocitinib was used for the first time to treat refractory NK-Large Granular lymphocytic leukemia with a STAT3 mutation, accompanied by hemolytic anemia: a case report.

作者信息

Hao Hongyuan, Yuan Chenglu, Zhang Chen, Jian Jimo

机构信息

Department of Hematology, Qilu Hospital of Shandong University, Qingdao, Shandong, China.

出版信息

Front Immunol. 2025 Aug 14;16:1632803. doi: 10.3389/fimmu.2025.1632803. eCollection 2025.

Abstract

BACKGROUND

NK-large granular lymphocytic leukemia (NK-LGLL) is a rare clonal lymphoproliferative disease of natural killer (NK) cells. In refractory cases, traditional chemotherapy regimens and immunosuppressive drugs often prove ineffective. Hematopoietic stem cell transplantation (HSCT) offers the opportunity to rebuild the immune system, but carries significant risks, including graft-versus-host disease (GVHD). Consequently, alternative therapeutic strategies need to be investigated. A recent study published in The Lancet reported on the use of golidocitinib in the treatment of relapsed or peripheral T-cell lymphoma. Notably, this represents the first clinical report demonstrating the efficacy of golidocitinib in managing intractable NK-large granular lymphocytic leukemia.

CASE PRESENTATION

In this study, we present a case of a 53-year-old male with NK-LGLL who achieved successful therapeutic outcomes with golidocitinib administration. The patient had a documented history of chronic gastritis and was admitted with symptoms of fatigue and chest tightness. Despite receiving various treatments such as methotrexate, cyclosporin, cyclophosphamide, and thalidomide, the patient exhibited treatment refractoriness. However, after initiating treatment with golidocitinib, the patient's blood count improved remarkedly after a single treatment cycle, obviating the need for blood transfusions. The patient maintained golidocitinib treatment without experiencing any serious complications. This is the first reported case demonstrating the efficacy of golidocitinib therapy in treating NK-LGLL.

CONCLUSION

This case highlights the clinical relevance of golidocitinib in developing novel therapeutic strategies for NK-LGLL. Moreover, this treatment option presents the potential as either a bridging therapy or alternative to allo-HSCT.

摘要

背景

自然杀伤细胞大颗粒淋巴细胞白血病(NK-LGLL)是一种罕见的自然杀伤(NK)细胞克隆性淋巴细胞增殖性疾病。在难治性病例中,传统化疗方案和免疫抑制药物往往无效。造血干细胞移植(HSCT)提供了重建免疫系统的机会,但存在重大风险,包括移植物抗宿主病(GVHD)。因此,需要研究替代治疗策略。最近发表在《柳叶刀》上的一项研究报道了戈利替尼在复发或外周T细胞淋巴瘤治疗中的应用。值得注意的是,这是第一份证明戈利替尼治疗难治性NK-LGLL有效性的临床报告。

病例介绍

在本研究中,我们报告了一例53岁的NK-LGLL男性患者,使用戈利替尼治疗取得了成功的治疗效果。该患者有慢性胃炎病史,因疲劳和胸闷症状入院。尽管接受了甲氨蝶呤、环孢素、环磷酰胺和沙利度胺等各种治疗,但患者仍表现出治疗抵抗性。然而,在开始使用戈利替尼治疗后,患者在一个治疗周期后血细胞计数显著改善,无需输血。患者持续接受戈利替尼治疗,未出现任何严重并发症。这是第一例报道戈利替尼治疗NK-LGLL有效性的病例。

结论

该病例突出了戈利替尼在开发NK-LGLL新治疗策略中的临床相关性。此外,这种治疗选择有可能作为异基因造血干细胞移植的桥接治疗或替代方案。

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