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皮肤 T 细胞淋巴瘤患者的治疗格局变化、疾病负担和未满足的需求。

The changing therapeutic landscape, burden of disease, and unmet needs in patients with cutaneous T-cell lymphoma.

机构信息

University Hospital Birmingham, Birmingham, UK.

Hôpital Saint Louis, Université de Paris, Paris, France.

出版信息

Br J Haematol. 2021 Feb;192(4):683-696. doi: 10.1111/bjh.17117. Epub 2020 Oct 23.

Abstract

Cutaneous T-cell lymphomas (CTCLs) have a chronic, relapsing course, and the most common subtypes are mycosis fungoides and Sézary syndrome. The disease causes visible skin alterations and can also cause alopecia, pruritus and pain, all of which can impact patients' health-related quality of life (HRQoL). The goal of treatment is to reduce symptoms and prevent disease progression. However, treatment recommendations are often based on low levels of evidence due to the lack of well-designed randomised clinical trials and treatment guidelines, and approved drugs vary considerably across different countries and regions. Currently, available treatments rarely lead to durable remissions and eventually become less effective, meaning patients often require multiple therapy changes. Skin-directed therapies (SDTs) are first-line treatments for early-stage CTCL, whereas systemic therapies may be needed for early-stage disease that does not respond to SDT or for advanced-stage disease. However, patients can experience significant side-effects with these treatments or may be unable to tolerate them. Hence, there is an unmet need for effective therapies with good safety profiles for the treatment of early- and late-stage CTCL. Here, we review current treatment guidelines, investigational and approved treatments, the impact of CTCL on patients' HRQoL, and the treatment of pruritus.

摘要

皮肤 T 细胞淋巴瘤 (CTCLs) 具有慢性、复发性病程,最常见的亚型是蕈样真菌病和 Sezary 综合征。该疾病会导致可见的皮肤改变,还可能导致脱发、瘙痒和疼痛,所有这些都会影响患者的健康相关生活质量 (HRQoL)。治疗的目标是减轻症状和预防疾病进展。然而,由于缺乏精心设计的随机临床试验和治疗指南,治疗建议通常基于低水平的证据,并且在不同国家和地区,批准的药物差异很大。目前,可用的治疗方法很少能导致持久缓解,而且最终效果会降低,这意味着患者通常需要多次改变治疗方案。皮肤定向治疗 (SDTs) 是 CTCL 早期的一线治疗方法,而对于早期 SDT 治疗无反应或晚期疾病,可能需要全身治疗。然而,这些治疗方法可能会导致患者出现严重的副作用,或者患者无法耐受这些治疗方法。因此,对于早期和晚期 CTCL,人们迫切需要具有良好安全性的有效治疗方法。在此,我们回顾了当前的治疗指南、研究性和批准的治疗方法、CTCL 对患者 HRQoL 的影响,以及瘙痒的治疗。

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