El Jammal Thomas, Jamilloux Yvan, Gerfaud-Valentin Mathieu, Valeyre Dominique, Sève Pascal
Department of Internal Medicine, Lyon University Hospital, Lyon, France.
Department of Pneumology, Assistance Publique - Hôpitaux de Paris, Hôpital Avicenne et Université Paris 13, Sorbonne Paris Cité, Bobigny, France.
Ther Clin Risk Manag. 2020 Apr 17;16:323-345. doi: 10.2147/TCRM.S192922. eCollection 2020.
Sarcoidosis is a multi-system disease of unknown etiology characterized by granuloma formation in various organs (especially lung and mediastinohilar lymph nodes). In more than half of patients, the disease resolves spontaneously. When indicated, it usually responds to corticosteroids, the first-line treatment, but some patients may not respond or tolerate them. An absence of treatment response is rare and urges for verifying the absence of a diagnosis error, the good adherence of the treatment, the presence of active lesions susceptible to respond since fibrotic lesions are irreversible. That is when second-line treatments, immunosuppressants (methotrexate, leflunomide, azathioprine, mycophenolate mofetil, hydroxychloroquine), should be considered. Methotrexate is the only first-line immunosuppressant validated by a randomized controlled trial. Refractory sarcoidosis is not yet a well-defined condition, but it remains a real challenge for the physicians. Herein, we considered refractory sarcoidosis as a disease in which second-line treatments are not sufficient to achieve satisfying disease control or satisfying corticosteroids tapering. Tumor necrosis alpha inhibitors, third-line treatments, have been validated through randomized controlled trials. There are currently no guidelines or recommendations regarding refractory sarcoidosis. Moreover, criteria defining non-response to treatment need to be clearly specified. The delay to achieve response to organ involvement and drugs also should be defined. In the past ten years, the efficacy of several immunosuppressants beforehand used in other autoimmune or inflammatory diseases was reported in refractory cases series. Among them, anti-CD20 antibodies (rituximab), repository corticotrophin injection, and anti-JAK therapy anti-interleukin-6 receptor monoclonal antibody (tocilizumab) were the main reported. Unfortunately, no clinical trial is available to validate their use in the case of sarcoidosis. Currently, other immunosuppressants such as JAK inhibitors are on trial to assess their efficacy in sarcoidosis. In this review, we propose to summarize the state of the art regarding the use of immunosuppressants and their management in the case of refractory or multidrug-resistant sarcoidosis.
结节病是一种病因不明的多系统疾病,其特征是在各个器官(尤其是肺和纵隔肺门淋巴结)中形成肉芽肿。超过半数的患者,疾病可自发缓解。如有必要,通常对一线治疗药物皮质类固醇有反应,但部分患者可能无反应或不耐受。治疗无反应的情况罕见,此时需确认无诊断错误、治疗依从性良好,且存在易对治疗产生反应的活动性病变,因为纤维化病变是不可逆的。这时应考虑二线治疗药物,即免疫抑制剂(甲氨蝶呤、来氟米特、硫唑嘌呤、霉酚酸酯、羟氯喹)。甲氨蝶呤是唯一经随机对照试验验证的一线免疫抑制剂。难治性结节病尚未有明确的定义,但对医生来说仍是一项切实的挑战。在此,我们将难治性结节病视为二线治疗不足以实现满意的疾病控制或满意的皮质类固醇减量的疾病。肿瘤坏死因子α抑制剂作为三线治疗药物,已通过随机对照试验验证。目前尚无关于难治性结节病的指南或建议。此外,需要明确规定治疗无反应的标准。还应确定对器官受累和药物产生反应的延迟时间。在过去十年中,难治性病例系列报道了几种先前用于其他自身免疫性或炎性疾病的免疫抑制剂的疗效。其中,抗CD20抗体(利妥昔单抗)、长效促肾上腺皮质激素注射液和抗JAK疗法抗白细胞介素-6受体单克隆抗体(托珠单抗)是主要报道的药物。不幸的是,尚无临床试验来验证它们在结节病中的应用。目前,其他免疫抑制剂如JAK抑制剂正在进行试验,以评估其在结节病中的疗效。在本综述中,我们建议总结难治性或多药耐药性结节病中免疫抑制剂的使用现状及其管理。