重组人血小板生成素加速低危骨髓增生异常综合征患者血小板恢复：一项概念验证研究。
Recombinant Human Thrombopoietin Accelerates the Recovery of Platelet in Patients With Lower-Risk Myelodysplastic Syndrome: A Proof-of-Concept Study.
作者信息
Yang Yuan, Tang Zengwei, Ji Jiang, Yang Chen, Chen Miao, Han Bing
机构信息
Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
Department of Hepatobiliary and Pancreatic Surgery, The First Affiliated Hospital, School of Medicine, Zhejiang University, Hangzhou, China.
出版信息
Front Oncol. 2021 Oct 28;11:721764. doi: 10.3389/fonc.2021.721764. eCollection 2021.
AIM
The effect of recombinant human thrombopoietin (rhTPO) is largely unknown in lower-risk myelodysplastic syndrome (LR-MDS). This study aimed at investigating the safety and efficacy of rhTPO in patients with LR-MDS.
METHODS
LR-MDS patients receiving stanozolol (2 mg, t.i.d.) and supportive care alone (non-rhTPO) or additional rhTPO were enrolled in this study prospectively. rhTPO was given at 15,000 U (q.d.) for 7 days/month for at least 3 months. Patients stopped rhTPO if the platelet count was higher than 50 × 10/L or had no effects after 3 months of treatment. The overall response (OR), complete response (CR), platelet response, side effects, clone evolution, and clinical outcome were evaluated.
RESULT
Thirty-five patients were enrolled: 20 (57.1%) patients in the rhTPO group and 15 (42.9%) patients in the non-rhTPO group. The demographic and baseline characteristics were balanced between the two groups. Platelet response was higher at 1 and 2 months as compared with that in the non-rhTPO group ( = 0.006 and = 0.001, respectively). Meanwhile, the rhTPO group had a shorter time to achieve a platelet transfusion-free state compared with the non-rhTPO group ( = 0.034). Hematologic response was higher at 1 and 2 months compared with that in the non-rhTPO group ( = 0.006 and = 0.001, respectively). There was no significant difference in the overall response or complete response at 1, 2, 3, 6, and 12 months between the two groups. One patient in the rhTPO group evolved into higher-risk MDS at 9 months. No significant difference in disease progression, infection, gastrointestinal disorders, or drug-related liver/renal injuries was found between the two groups ( > 0.05).
CONCLUSION
Adding short-term rhTPO can accelerate the early platelet response and decrease platelet transfusion, with no obvious side effects.
CLINICAL TRIAL REGISTRATION
https://clinicaltrials.gov/ct2/show/NCT04324060?cond=NCT04324060&draw=2, identifier NCT04324060.
目的
重组人血小板生成素（rhTPO）在低危骨髓增生异常综合征（LR-MDS）中的作用尚不清楚。本研究旨在探讨rhTPO对LR-MDS患者的安全性和疗效。
方法
前瞻性纳入单独接受司坦唑醇（2mg，每日3次）和支持治疗（非rhTPO组）或额外接受rhTPO的LR-MDS患者。rhTPO以15000U（每日1次）给药，每月7天，至少3个月。如果血小板计数高于50×10⁹/L或治疗3个月后无效，则停用rhTPO。评估总缓解率（OR）、完全缓解率（CR）、血小板反应、副作用、克隆演变和临床结局。
结果
共纳入35例患者：rhTPO组20例（57.1%），非rhTPO组15例（42.9%）。两组的人口统计学和基线特征均衡。与非rhTPO组相比，rhTPO组在第1个月和第2个月时血小板反应更高（分别为P = 0.006和P = 0.001）。同时，与非rhTPO组相比，rhTPO组达到无血小板输注状态的时间更短（P = 0.034）。与非rhTPO组相比，rhTPO组在第1个月和第2个月时血液学反应更高（分别为P = 0.006和P = 0.001）。两组在1、2、3、6和12个月时的总缓解率或完全缓解率无显著差异。rhTPO组1例患者在9个月时进展为高危MDS。两组在疾病进展、感染、胃肠道疾病或药物相关肝/肾损伤方面无显著差异（P>0.05）。
结论
短期添加rhTPO可加速早期血小板反应并减少血小板输注，且无明显副作用。
临床试验注册
https://clinicaltrials.gov/ct2/show/NCT04324060?cond=NCT04324060&draw=2，标识符NCT04324060 。
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