Toyosaki Masako, Machida Shinichiro, Tomizawa Daisuke, Okada Masaya, Sawa Masashi, Ueda Yasunori, Omi Ai, Koroki Yosuke, Teshima Takanori
Department of Hematology and Oncology, Tokai University Hospital, Isehara, Japan.
Division of Leukemia and Lymphoma, Children's Cancer Center, National Center for Child Health and Development, Tokyo, Japan.
Int J Hematol. 2025 Mar;121(3):388-396. doi: 10.1007/s12185-024-03882-1. Epub 2024 Dec 10.
The GVH3001 study assessed the efficacy and safety of ibrutinib in Japanese patients with steroid-dependent or -refractory chronic graft-versus-host disease (cGVHD). However, the effects of ibrutinib on lung function and reduction in corticosteroid dose, which is a measurable factor associated with improved quality of life, could not be adequately assessed in patients who initially presented with lung involvement. This post hoc analysis aimed to evaluate temporal changes in daily corticosteroid dose, as well as effectiveness outcomes based on lung function and symptom burden (percent predicted forced expiratory volume in 1 s [%FEV] and Lee cGVHD Symptom Scale lung subscale score, respectively) in the subgroup of patients with cGVHD who had lung involvement at baseline. Seven of the 19 patients in the GVH3001 study had lung involvement at baseline. The daily corticosteroid dose for cGVHD decreased in five of these patients, and %FEV remained relatively stable in two patients but increased to > 80% in one patient. Lee cGVHD Symptom Scale scores were relatively stable throughout the study in patients with lung involvement. Ibrutinib may allow corticosteroid dose reduction without worsening lung function or increasing symptom burden in previously treated patients with cGVHD and associated lung involvement.
GVH3001研究评估了依鲁替尼对日本类固醇依赖或难治性慢性移植物抗宿主病（cGVHD）患者的疗效和安全性。然而，对于最初出现肺部受累的患者，无法充分评估依鲁替尼对肺功能的影响以及皮质类固醇剂量的降低情况，而皮质类固醇剂量降低是与生活质量改善相关的一个可测量因素。这项事后分析旨在评估基线时存在肺部受累的cGVHD患者亚组中每日皮质类固醇剂量的时间变化，以及基于肺功能和症状负担（分别为1秒用力呼气量预测值百分比[%FEV]和Lee cGVHD症状量表肺部子量表评分）的有效性结果。GVH3001研究中的19例患者中有7例在基线时存在肺部受累。其中5例患者的cGVHD每日皮质类固醇剂量降低，2例患者的%FEV保持相对稳定，1例患者的%FEV增加至>80%。在整个研究过程中，肺部受累患者的Lee cGVHD症状量表评分相对稳定。对于先前接受治疗的cGVHD及相关肺部受累患者，依鲁替尼可能允许降低皮质类固醇剂量，而不会使肺功能恶化或增加症状负担。
