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结节病的治疗指征。

Indications for treatment of sarcoidosis.

机构信息

Service de Pneumologie, Hôpital Avicenne, Assistance Publique Hôpitaux de Paris.

Inserm UMR U1272, Université Paris 13, Paris, France.

出版信息

Curr Opin Pulm Med. 2019 Sep;25(5):505-518. doi: 10.1097/MCP.0000000000000604.

Abstract

PURPOSE OF REVIEW

To describe the current knowledge on indications for sarcoidosis treatment.

RECENT FINDINGS

Despite the lack of evidence-based recommendations, the sarcoidosis community has adopted the concept of starting systemic anti-inflammatory treatment because of potential danger (risk of severe dysfunction on major organs or death) or unacceptable impaired quality of life (QoL). On the contrary, while QoL and functionality are patients' priorities, few studies have evaluated treatment effect on patient-reported outcomes. The awareness of long-term corticosteroids toxicities and consequences on QoL and the emergence of novel drugs have changed therapeutic management. Second-line therapy, mainly methotrexate and azathioprine, are indicated for corticosteroids sparing or corticosteroids-resistant sarcoidosis. TNF-α inhibitors are a useful third-line therapy in chronic refractory disease. In addition to organ-targeted treatment, efforts should also be taken for treating nonorgan-specific symptoms, such as physical training for fatigue, and various disease complications.

SUMMARY

Clinicians should offer a tailored treatment for each patient and ensure a holistic multidisciplinary approach, including pharmacological and nonpharmacological interventions. Patient-centered communication is critical to drive shared decisions, in particular for the tricky situation of isolated impaired QoL as the unique therapeutic indication. Once treatment is decided, clinicians should define a clear therapeutic plan, including goals and instruments to assess response.

摘要

目的综述

描述目前对结节病治疗适应证的认识。

最近的发现

尽管缺乏循证推荐,但结节病领域已接受开始全身抗炎治疗的理念,因为存在潜在危险(主要器官严重功能障碍或死亡的风险)或无法接受的生活质量受损(生活质量)。相反,虽然生活质量和功能是患者的首要关注点,但很少有研究评估治疗对患者报告结局的影响。长期皮质类固醇毒性及其对生活质量的影响以及新型药物的出现改变了治疗管理。二线治疗,主要是甲氨蝶呤和硫唑嘌呤,适用于皮质类固醇保留或皮质类固醇耐药性结节病。TNF-α 抑制剂是慢性难治性疾病的一种有用的三线治疗药物。除了针对器官的治疗外,还应努力治疗非器官特异性症状,如疲劳的体能训练以及各种疾病并发症。

总结

临床医生应为每位患者提供个体化的治疗,并确保采取整体多学科方法,包括药物和非药物干预。以患者为中心的沟通对于推动共同决策至关重要,特别是对于唯一的治疗适应证孤立性生活质量受损这种棘手情况。一旦决定治疗,临床医生就应制定明确的治疗计划,包括目标和评估反应的工具。

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