Sokolowska-Wojdylo Malgorzata, Florek Aleksandra, Zaucha Jan Maciej, Chmielowska Ewa, Giza Agnieszka, Knopinska-Posluszny Wanda, Kulikowski Waldemar, Prejzner Witold, Romejko-Jarosinska Joanna, Paszkiewicz-Kozik Ewa, Osowiecki Michal, Walewski Jan, Rogowski Wojciech, Grzanka Aleksandra, Placek Waldemar, Lugowska-Umer Hanna, Kowalczyk Anna, Nowicki Roman, Jurczak Wojciech
1Department of Dermatology, Venereology and Allergology, Medical University of Gdansk, Gdansk, Poland; 2Department of Internal Medicine, New York Medical College, Valhalla, NY; 3Department of Propedeutic Oncology, Medical University of Gdansk, Gdansk, Poland; 4Department of Oncology, Center of Oncology of Professor Franciszek Lukaszczyk, Bydgoszcz, Poland; 5Department of Hematology, Collegium Medicum of the Jagiellonian University, Krakow, Poland; 6Department of Oncology, University of Warmia and Mazury, Olsztyn, Poland; 7Department of Hematology, Medical University of Gdansk, Gdansk, Poland; 8Department of Lymphoid Malignancies, Maria Sklodowska-Curie Institute and Oncology Center, Warsaw, Poland; 9Department of Dermatology, Collegium Medicum in Bydgoszcz, Bydgoszcz, Poland; 10Department of Dermatology, University of Warmia and Mazury, Olsztyn, Poland; 11Department of Oncology and Radiotherapy, Medical University of Gdansk, Gdansk, Poland.
Am J Ther. 2016 May-Jun;23(3):e749-56. doi: 10.1097/MJT.0000000000000056.
Bexarotene, a synthetic retinoid licensed for the treatment of refractory cutaneous T-cell lymphoma (CTCL), has been used clinically in Poland since 2007 in 21 patients. The objective of our retrospective, multicenter study was to evaluate our experience with bexarotene therapy, including efficacy, safety, and survival outcomes. We retrospectively identified 21 adult patients who were treated with bexarotene between the years 2007 and 2012. Starting dose of bexarotene was 300 mg/m per day. The analysis included 3 patients with early-stage mycosis fungoides (MF), 16 patients with advanced-stage MF, and 2 patients with Sézary syndrome (SS). The mean duration of therapy with bexarotene was 14.5 months. Use of bexarotene resulted in an overall response rate of 81.0%, although the overall mortality rate was 52.8%. In our study, early-stage CTCL responded better than advanced-stage CTCL (100.0% vs. 77.8%, respectively). The mean time to observable response was 1.8 months, and the mean duration of the response was 16.4 months. Most significant side effects were hyperlipidemia, hypothyroidism, and a bleeding gastric ulcer. Based on the results of our analysis, bexarotene is a valuable tool in the treatment of refractory early-stage CTCL. Although a majority of patients initially responded to therapy, the high mortality rate in the advanced-stage group suggests that bexarotene does not completely resolve the therapeutic problems in all stages of CTCL. Patient stratification for bexarotene treatment may need a thorough reassessment, in that bexarotene may not be an effective drug in the very advanced stages of CTCL.
贝沙罗汀是一种已获许可用于治疗难治性皮肤T细胞淋巴瘤(CTCL)的合成维甲酸,自2007年起在波兰临床上用于21例患者。我们这项回顾性多中心研究的目的是评估我们使用贝沙罗汀治疗的经验,包括疗效、安全性和生存结果。我们回顾性地确定了2007年至2012年间接受贝沙罗汀治疗的21例成年患者。贝沙罗汀的起始剂量为每天300mg/m²。分析包括3例早期蕈样肉芽肿(MF)患者、16例晚期MF患者和2例塞扎里综合征(SS)患者。贝沙罗汀的平均治疗持续时间为14.5个月。使用贝沙罗汀导致总体缓解率为81.0%,尽管总死亡率为52.8%。在我们的研究中,早期CTCL的反应优于晚期CTCL(分别为100.0%和77.8%)。可观察到反应的平均时间为1.8个月,反应的平均持续时间为16.4个月。最显著的副作用是高脂血症、甲状腺功能减退和出血性胃溃疡。根据我们的分析结果,贝沙罗汀是治疗难治性早期CTCL的一种有价值的工具。尽管大多数患者最初对治疗有反应,但晚期组的高死亡率表明贝沙罗汀并不能完全解决CTCL所有阶段的治疗问题。贝沙罗汀治疗的患者分层可能需要彻底重新评估,因为贝沙罗汀在CTCL的极晚期可能不是一种有效的药物。