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芦可替尼治疗对日本骨髓纤维化患者生活质量的影响。

Effect of ruxolitinib therapy on the quality-of-life of Japanese patients with myelofibrosis.

作者信息

Oritani Kenji, Ohishi Kohshi, Okamoto Shinichiro, Kirito Keita, Komatsu Norio, Tauchi Tetsuzo, Handa Hiroshi, Saito Shigeki, Takenaka Katsuto, Shimoda Kazuya, Okada Hikaru, Amagasaki Taro, Wakase Shiho, Shimozuma Kojiro, Akashi Koichi

机构信息

a Department of Hematology, Graduate School of Medical Sciences , International University of Health and Welfare Hospital , Tochigi , Japan.

b Blood Transfusion Service, Mie University Hospital , Mie , Japan.

出版信息

Curr Med Res Opin. 2018 Mar;34(3):531-537. doi: 10.1080/03007995.2017.1415874. Epub 2018 Jan 4.

Abstract

OBJECTIVES

Myelofibrosis (MF) is associated with a significant symptom burden that severely impacts patient quality-of-life (QoL). Ruxolitinib, a potent Janus kinase 1 (JAK1)/JAK2 inhibitor, led to substantial improvements in splenomegaly, MF-associated symptoms, and QoL in the phase 3 COMFORT studies, proving superior to placebo and best available therapy. This study evaluated the effect of ruxolitinib on symptoms and QoL in Japanese patients with MF.

METHODS

A pooled analysis of studies A2202 (NCT01392443) and AJP01 (NCT02087059) of ruxolitinib in Japanese patients with MF (n = 81) was conducted. Changes in total symptom score (TSS) and the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 were summarized.

RESULTS

Most patients received a starting dose of 15 or 20 mg twice daily (BID) and had a final titrated dose of ≥10 mg BID. Overall, 67.7% (44/65) achieved a ≥50% reduction from baseline in TSS at week 24. Reductions in TSS were seen in every dose group; the greatest reductions occurred in patients with a final titrated dose of 20 or 25 mg BID. Improvements in QoL were seen in patients who achieved a ≥50% reduction in TSS. Generally, improvements in TSS and individual symptoms correlated with reductions in spleen size, with those having a ≥35% reduction in spleen volume having the greatest improvements.

CONCLUSIONS

Consistent with COMFORT-I, ruxolitinib provided substantial improvements in symptoms and QoL in Japanese patients with MF, with higher doses of ruxolitinib associated with better responses.

摘要

目的

骨髓纤维化(MF)伴有严重的症状负担,严重影响患者的生活质量(QoL)。芦可替尼是一种有效的Janus激酶1(JAK1)/JAK2抑制剂,在3期COMFORT研究中可显著改善脾肿大、MF相关症状和QoL,证明优于安慰剂和最佳可用疗法。本研究评估了芦可替尼对日本MF患者症状和QoL的影响。

方法

对芦可替尼治疗日本MF患者(n = 81)的研究A2202(NCT01392443)和AJP01(NCT02087059)进行汇总分析。总结了总症状评分(TSS)和欧洲癌症研究与治疗组织生活质量问卷核心30的变化。

结果

大多数患者起始剂量为每日两次(BID)15或20 mg,最终滴定剂量≥10 mg BID。总体而言,67.7%(44/65)的患者在第24周时TSS较基线降低≥50%。每个剂量组的TSS均有降低;最终滴定剂量为20或25 mg BID的患者降低幅度最大。TSS降低≥50%的患者QoL有所改善。一般来说,TSS和个体症状的改善与脾脏大小的减小相关,脾脏体积减小≥35%的患者改善最大。

结论

与COMFORT-I一致,芦可替尼使日本MF患者的症状和QoL有显著改善,较高剂量的芦可替尼反应更好。

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