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基于thalidomide的疗法在大型颗粒性淋巴细胞白血病中显示出有希望的疗效:多中心II期研究

Ying Yu^1,2, Yuxi Li^1,2, Rui Cui^1,2,3

¹State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.

Signal transduction and targeted therapy

|March 12, 2025

概括

塔利多米德,普雷尼松和甲状腺素 (TPM) 治疗方案在治疗大粒状淋巴细胞白血病 (LGLL) 中显示出高效率. 这种治疗在患者中实现了75%的完全缓解率,为LGLL管理提供了一个有前途的新选择.

科学领域:

血液学血液学血液学
免疫学免疫学免疫学
在瘤学瘤学.

背景情况:

大粒状淋巴细胞白血病 (LGLL) 涉及细胞毒性T淋巴细胞或NK细胞的克隆扩张.
目前针对LGLL的免疫抑制疗法存在局限性,完全缓解率约为50%.
免疫失调是LGLL病变的关键因素.

研究的目的:

在LGLL患者中评估thalidomide,prednisone和甲状腺素 (TPM) 治疗方案的疗效和安全性.
确定TPM治疗方案的完全缓解率 (CR) 在症状,未经治疗的LGLL患者中.

主要方法:

一项涉及52名LGLL患者的多中心研究,从2020年6月到2022年8月.
患者接受了沙利多米德 (每天100毫克),普雷尼松 (每隔一天0.5-1.0毫克/公斤) 和甲状腺素 (每周10毫克/米2).
thalidomide的治疗持续时间高达24个月,在3个月后降低了普得尼松,在12个月后降低了甲状腺素.

主要成果:

血液和症状改善的高整体响应率为90.4% (47/52名患者).
实现了75.0% (39/52名患者) 的完全缓解率 (CR).
最常见的不良事件是外围神经病变 (24.1%),主要是低级 (1-2).

结论:

TPM疗法是对症状性LGLL的有效和安全治疗.
在LGLL患者中,TPM疗法显示显著高的CR率.
这项研究提供了TPM作为LGLL有价值的治疗选择的证据.

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