Treatment comparison of hydroxyurea versus ruxolitinib in essential thrombocythaemia: A matched-cohort analysis

EJHaem Pub Date : 2024-07-19 DOI:10.1002/jha2.954

Michael R. Grunwald, Ellen K. Ritchie, Elisa Rumi, Albert Assad, J. E. Hamer-Maansson, Jingbo Yu, Tricia Kalafut, Evan Braunstein, Francesco Passamonti

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Abstract

Hydroxyurea is the preferred first-line cytoreductive treatment for high-risk essential thrombocythaemia (ET), but many patients are intolerant or refractory to hydroxyurea. Ruxolitinib has been shown to improve symptoms in patients with ET. This post hoc analysis compared the clinical outcomes of patients with ET who received hydroxyurea only with those who switched from hydroxyurea to ruxolitinib due to intolerance/resistance to hydroxyurea. Patients with ET refractory/intolerant to hydroxyurea treated with ruxolitinib in a completed phase 2 study (HU-RUX) were propensity score matched with patients who received hydroxyurea only in an observational study (HU). Changes in leukocyte and platelet counts were reported at 6-month intervals during the 48-month follow-up. Following propensity score matching, 37 patients were included for analysis in each cohort. Mean (standard deviation [SD]) leukocyte and platelet counts at index were higher for HU-RUX versus HU (leukocyte: 9.3 [5.1] vs. 6.8 [3.1] × 10⁹/L; platelet: 1027.4 [497.8] vs. 513.9 [154.7] × 10⁹/L), both of which decreased significantly from index to 6 months through to 48 months in HU-RUX (mean [SD] change from index at 6 months—leukocyte: −1.8 [4.6] × 10⁹/L; platelet: −391.7 [472.9] × 10⁹/L; at 48 months—leukocyte: −3.8 [5.3] × 10⁹/L; platelet: −539.0 [521.8] × 10⁹/L), but remained relatively stable in HU (mean [SD] change from index at 6 months—leukocyte: 0 [1.8] × 10⁹/L; platelet: −5.7 [175.3] × 10⁹/L; at 48 months—leukocyte: −0.1 [2.7] × 10⁹/L; platelet: −6.9 [105.1] × 10⁹/L). In conclusion, these results demonstrate that switching from hydroxyurea to ruxolitinib in patients with ET who are intolerant or refractory to hydroxyurea could improve abnormal haematologic values similar to those who receive first-line hydroxyurea.

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羟基脲与 Ruxolitinib 治疗重症血小板增多症的比较：匹配队列分析

羟基脲是治疗高危原发性血小板增多症（ET）的首选一线细胞再生疗法，但许多患者对羟基脲不耐受或难治。Ruxolitinib已被证明能改善ET患者的症状。这项事后分析比较了只接受羟基脲治疗的ET患者与因对羟基脲不耐受/耐药而从羟基脲转为服用Ruxolitinib的患者的临床疗效。在一项已完成的2期研究（HU-RUX）中，对羟基脲难治/不耐受的ET患者接受了鲁索利替尼治疗，并与一项观察性研究（HU）中仅接受羟基脲治疗的患者进行了倾向评分匹配。在 48 个月的随访期间，每 6 个月报告一次白细胞和血小板计数的变化。经过倾向评分匹配后，每个队列中有 37 名患者被纳入分析。HU-RUX与HU相比，指标时白细胞和血小板计数的平均值（标准差[SD]）更高（白细胞：9.3 [5.1] vs. 6.8 [3.1] ×109/L；血小板：1027.4 [497.8] vs. 513.9 [154.7] ×109/L），而HU-RUX从指标到6个月再到48个月的白细胞和血小板计数均显著下降（6个月时白细胞与指标相比的平均值[SD]变化：-1.8 [4.6] ×109/L）：-1.8[4.6]×109/L；血小板：-391.7[472.9]×109/L；48 个月时白细胞：-3.8[5.3]×109/L：-3.8[5.3]×109/L；血小板：白细胞：-3.8 [5.3] × 109/L；血小板：-539.0 [521.8] × 109/L），但 HU 保持相对稳定（6 个月时与指数相比的平均 [SD] 变化：白细胞：0 [1.8] × 109/L；血小板：-5.7 [175.8] × 109/L）：-5.7[175.3]×109/L；48 个月时白细胞白细胞：-0.1 [2.7] × 109/L；血小板：-6.9 [105.1] × 109/L：-6.9 [105.1] × 109/L).总之，这些结果表明，对羟基脲不耐受或难治的ET患者从羟基脲转用芦可利替尼可改善血液学异常值，其改善程度与接受一线羟基脲治疗的患者相似。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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