阿那格雷联合或不联合羟基脲用于日本原发性血小板增多症患者的疗效和安全性的临床评估——一项35例患者的回顾性单中心研究

Clinical Evaluation of the Efficacy and Safety of Anagrelide Used with or without Hydroxycarbamide in Japanese Patients with Essential Thrombocythemia-A Retrospective Single-Center Study of 35 Cases.

作者信息

Sekiguchi Yasunobu, Wakabayashi Mutsumi, Takizawa Haruko, Iizuka Hiroko, Sakajiri Sakura, Sugimoto Keiji, Inano Tadaaki, Fukuda Yasutaka, Hamano Yasuharu, Tomita Shigeki, Izumi Hiroshi, Okubo Mitsuo, Nakamura Noriko, Sawada Tomohiro, Noguchi Masaaki

机构信息

Dept. of Hematology, Juntendo University Urayasu Hospital.

出版信息

Gan To Kagaku Ryoho. 2019 Jul;46(7):1141-1150.

PMID:31296820

Abstract

OBJECTIVE

The clinical features(CF), laboratory data, disease transformation pattern and drug metabolism in essential thrombocythemia(ET)differ between Japan and Western countries. The CF of ET in clinical practice(CP)are more diverse than in prospective clinical studies. We should conduct retrospective analyses in CP. The present study was aimed at evaluating the efficacy, safety and tolerability of anagrelide(ANA)monotherapy and combined ANA plus hydroxycarbamide(HC)in Japanese ET.

PATIENTS AND METHODS

We have a total of 35 cases. Sixteen patients received ANA monotherapy, 10 received ANA plus HC, and 9 received ANA plus other drugs.

RESULTS

Comparison among three groups revealed the absence of differences in response rate(platelet count C60×10 / / 4/mL, platelet count C40×104/mL)(43.8%, 6.3% vs. 50.0%, 10.0% vs. 44.4%, 11.1%), treatment continuation rate(81.3% vs. 40.0% vs. 55.6%), median daily dose of ANA(1.00 mg in all three groups)or median treatment period(days)(259 vs. 198.5 vs. 161.0), the treatment continuation rate tended to be lower in the combined ANA plus HC. The incidence of all adverse events(AEs)was higher in the ANA monotherapy(45.7%)than ANA plus HC(28.6%)or ANA plus other drugs(25.7%), the AEs were mild in all groups.

CONCLUSION

The tolerability of ANA monotherapy, ANA plus HC, and ANA plus other drugs were good.

摘要

目的

真性红细胞增多症（ET）在日本和西方国家之间的临床特征（CF）、实验室数据、疾病转化模式和药物代谢存在差异。临床实践（CP）中ET的CF比前瞻性临床研究中更多样化。我们应该在CP中进行回顾性分析。本研究旨在评估阿那格雷（ANA）单药治疗以及ANA联合羟基脲（HC）治疗日本ET患者的疗效、安全性和耐受性。

患者与方法

我们共纳入35例患者。16例患者接受ANA单药治疗，10例接受ANA联合HC治疗，9例接受ANA联合其他药物治疗。

结果

三组之间的比较显示，在缓解率（血小板计数≤60×10⁹/L、血小板计数≤40×10⁹/L）（43.8%，6.3% 对比50.0%，10.0% 对比44.4%，11.1%）、治疗持续率（81.3% 对比40.0% 对比55.6%）、ANA的中位日剂量（三组均为1.00 mg）或中位治疗期（天数）（259对比198.5对比161.0）方面没有差异，ANA联合HC组的治疗持续率往往较低。ANA单药治疗组的所有不良事件（AE）发生率（45.7%）高于ANA联合HC组（28.6%）或ANA联合其他药物组（25.7%），所有组的AE均为轻度。