Department of Dermatology, University of Tokyo Graduate School of Medicine, 7-3-1 Hongo, Bunkyo-ku, Tokyo, 113-8655, Japan.
Department of Dermatology, St. Marianna University School of Medicine, Kawasaki, Kanagawa, Japan.
Curr Treat Options Oncol. 2021 Jan 7;22(2):10. doi: 10.1007/s11864-020-00809-w.
While most patients with early-stage mycosis fungoides (MF) follow an indolent course, patients with advanced-stage MF/Sézary syndrome (SS) have a poor prognosis with a median survival of less than 5 years. Although there are a number of treatments currently available, achieving and maintaining a durable response remain challenging, especially in advanced-stage MF/SS. The choice of frontline therapy is dependent on the stage of disease. For early-stage MF, the treatment concept is to control skin lesions mainly by skin-directed therapies, such as topical therapies, phototherapies, and radiotherapies. For advanced-stage MF/SS, systemic treatments by biological or targeted therapies including bexarotene and interferon either alone or in combination are tried first, with more immunosuppressive chemotherapies being reserved for refractory or rapidly progressive disease. Recent improvements in biological or targeted therapies include brentuximab vedotin and mogamulizumab. When biopsy samples have 10% or more CD30-positive malignant cells, brentuximab vedotin, an anti-CD30 antibody conjugated to monomethyl auristin E, can be a desirable treatment option. For cases with blood involvement, mogamulizumab, an antibody binding to C-C chemokine receptor 4, is effective with high response rates. In the refractory setting, alemtuzumab, histone deacetylase inhibitors, pralatrexate, gemcitabine, and doxorubicin are considered as the treatment option. Because only allogeneic hematopoietic stem cell transplantation can offer a chance of cure with durable complete remission, advanced-stage patients with a markedly short life expectancy should be evaluated for eligibility. Given that there are few randomized controlled studies in the literature, it is necessary to investigate which therapy is preferable for each patient with MF/SS by comparative prospective trials.
虽然大多数早期蕈样真菌病（MF）患者病情进展缓慢，但晚期 MF/Sezary 综合征（SS）患者预后较差，中位生存期不足 5 年。尽管目前有多种治疗方法，但实现并维持持久缓解仍然具有挑战性，尤其是在晚期 MF/SS 中。一线治疗的选择取决于疾病的阶段。对于早期 MF，治疗理念主要是通过皮肤定向治疗（如局部治疗、光疗和放疗）来控制皮肤病变。对于晚期 MF/SS，首先尝试使用生物制剂或靶向治疗（包括贝沙罗汀和干扰素）进行全身治疗，单独或联合使用，对于难治性或快速进展性疾病则保留更具免疫抑制作用的化疗。最近在生物制剂或靶向治疗方面的进展包括 Brentuximab vedotin 和 Mogamulizumab。当活检样本中 CD30 阳性恶性细胞占 10%或更多时，Brentuximab vedotin（一种与单甲基奥瑞他汀 E 偶联的抗 CD30 抗体）可能是一种理想的治疗选择。对于有血液受累的病例，Mogamulizumab（一种与 C-C 趋化因子受体 4 结合的抗体）有效，应答率高。在难治性情况下，可考虑使用阿仑单抗、组蛋白去乙酰化酶抑制剂、普拉曲沙、吉西他滨和多柔比星作为治疗选择。由于只有同种异体造血干细胞移植才能提供治愈机会和持久完全缓解的机会，因此应评估预期寿命明显缩短的晚期患者是否有资格进行移植。鉴于文献中很少有随机对照研究，有必要通过比较前瞻性试验来研究哪种治疗方法对每个 MF/SS 患者更优。
