Department of Dermatology, Takamatsu Red Cross Hospital, Takamatsu, Japan.
Department of Geriatric and Environmental Dermatology, Nagoya City University Graduate School of Medical Sciences, Nagoya, Japan.
J Dermatol. 2022 Feb;49(2):253-262. doi: 10.1111/1346-8138.16201. Epub 2021 Oct 18.
To establish real-world evidence about the safety and efficacy of bexarotene for Japanese patients with cutaneous T-cell lymphoma, we conducted a nationwide cohort study using data from post-marketing surveillance for bexarotene treatment. In total, 294 patients with cutaneous T-cell lymphoma were identified between June 2016 and June 2018. Of these, 267 patients were included as the safety analysis set. Of the 267 patients, 175 were included in the efficacy analysis set. Of these, 139 patients had mycosis fungoides, including 46 with early stage disease and 93 with advanced stage disease. Among the 139 patients with mycosis fungoides, the objective response rate was 46.8%. A significant difference in objective response rate was detected between patients who started with bexarotene at 300 mg/m (61.6%) and patients who started with bexarotene at less than 300 mg/m (22.6%, p < 0.001). Of the 139 patients with mycosis fungoides, 92 were treated with a combination of bexarotene plus photo(chemo)therapy. A significant difference in objective response rate was seen between bexarotene with a combination of photo(chemo)therapy (57.6%) and bexarotene without a combination of photo(chemo)therapy (25.5%, p < 0.001). Starting bexarotene at 300 mg/m and combination with photo(chemo)therapy were detected as independent factors influencing response. Common treatment-related adverse events included hypothyroidism (85.8%), hypertriglyceridemia (68.5%), hypercholesterolemia (43.8%), and neutropenia (21.3%). Hypertriglyceridemia, hypercholesterolemia, and neutropenia occurred more frequently in patients who started with bexarotene at 300 mg/m than patients who started with bexarotene at less than 300 mg/m (hypertriglyceridemia, 76.4% vs. 57.0%, p = 0.001; hypercholesterolemia, 49.0% vs. 36.4%, p = 0.045; neutropenia, 28.0% vs. 12.1%, p = 0.002; respectively). The present study indicates that starting bexarotene at 300 mg/m and combination of photo(chemo)therapy offer a promising efficacy for the treatment of patients with mycosis fungoides. Efficacy of low-dose bexarotene plus photo(chemo)therapy should be evaluated in future.
为了在日本皮肤 T 细胞淋巴瘤患者中建立关于贝沙罗汀安全性和疗效的真实世界证据，我们使用贝沙罗汀上市后监测的数据进行了一项全国性队列研究。在 2016 年 6 月至 2018 年 6 月期间，共确定了 294 例皮肤 T 细胞淋巴瘤患者。其中，267 例患者被纳入安全性分析集。在这 267 例患者中，有 175 例被纳入疗效分析集。在这 175 例患者中，有 139 例患有蕈样真菌病，其中 46 例为早期疾病，93 例为晚期疾病。在这 139 例蕈样真菌病患者中，客观缓解率为 46.8%。在起始剂量为 300mg/m 的患者（61.6%）和起始剂量低于 300mg/m 的患者（22.6%，p<0.001）之间，客观缓解率有显著差异。在这 139 例蕈样真菌病患者中，有 92 例接受了贝沙罗汀联合光（化）疗治疗。在接受贝沙罗汀联合光（化）疗治疗的患者（57.6%）和未接受贝沙罗汀联合光（化）疗治疗的患者（25.5%）之间，客观缓解率有显著差异（p<0.001）。起始剂量为 300mg/m 和联合光（化）疗被检测为影响反应的独立因素。常见的治疗相关不良事件包括甲状腺功能减退症（85.8%）、高甘油三酯血症（68.5%）、高胆固醇血症（43.8%）和中性粒细胞减少症（21.3%）。起始剂量为 300mg/m 的患者中更常发生高甘油三酯血症、高胆固醇血症和中性粒细胞减少症，而起始剂量低于 300mg/m 的患者中分别为 76.4%和 57.0%（p=0.001）；49.0%和 36.4%（p=0.045）；28.0%和 12.1%（p=0.002）。本研究表明，起始剂量为 300mg/m 和联合光（化）疗为蕈样真菌病患者的治疗提供了有希望的疗效。低剂量贝沙罗汀联合光（化）疗的疗效应在未来进行评估。
