Judson Marc A, Spagnolo Paolo, Stanfel Rebecca, Farrow Garrie, Tanase Ana-Maria, Perna Flavia, Baughman Robert P
Albany Medical College, Albany, New York, USA.
Department of Cardiac, Thoracic, Vascular Sciences and Public Health. University of Padova, Padova, Italy.
Respir Med. 2023 Apr-May;210:107174. doi: 10.1016/j.rmed.2023.107174. Epub 2023 Mar 3.
Sarcoidosis is a multisystem disease, characterised by the infiltration of various organs by non-necrotising granulomas. The disease's heterogeneity complicates the study of patients' experiences.
To gather insight into life experiences, unmet needs and views on hypothetically emerging treatment options among patients living with sarcoidosis.
Multinational, virtual, interactive, moderated discussion of specific questions between people with sarcoidosis, with experienced clinicians participating.
Nine patients with sarcoidosis from Australia, Denmark, Germany, Italy, Japan and the US, and three clinicians took part. All patients had pulmonary sarcoidosis, self-assessed as mild by five patients. The path to diagnosis was convoluted, with up to four physicians and a large number of tests involved. There was agreement that the process would be improved by earlier referral to specialists. The patients made a clear distinction between 'living with a condition' (adapting to the disease) and 'being ill'. The concept of remission was viewed sceptically as disease might develop in multiple organs. Panellists had a pragmatic attitude to therapies: side effects during a treatment course were accepted if overall symptoms improved. When considering hypothetical new therapies, improved quality of life (QoL) was the most important need; improved tolerability had lower priority. New therapies should be targeted on reducing disease progression and improving symptoms and QoL rather than corticosteroid withdrawal.
The interactive exchange provided insights into the need for earlier specialist referrals, distrust of the concept of remission in sarcoidosis, and the need for therapies targeted on reducing disease progression and improving symptoms and QoL.
结节病是一种多系统疾病，其特征是非坏死性肉芽肿浸润各种器官。该疾病的异质性使对患者经历的研究变得复杂。
深入了解结节病患者的生活经历、未满足的需求以及对假设出现的治疗选择的看法。
在结节病患者之间就特定问题进行跨国、虚拟、互动且有主持人引导的讨论，有经验丰富的临床医生参与。
来自澳大利亚、丹麦、德国、意大利、日本和美国的9名结节病患者以及3名临床医生参与了讨论。所有患者均患有肺部结节病，其中5名患者自我评估为轻度。诊断过程曲折，涉及多达4名医生和大量检查。大家一致认为，更早转诊至专科医生可改善诊断过程。患者明确区分了“带病生活”（适应疾病）和“生病”。由于疾病可能在多个器官发展，对缓解的概念持怀疑态度。小组成员对治疗持务实态度：如果总体症状改善，治疗过程中的副作用是可以接受的。在考虑假设的新疗法时，改善生活质量（QoL）是最重要的需求；提高耐受性的优先级较低。新疗法应旨在减少疾病进展、改善症状和生活质量，而不是停用皮质类固醇。
互动交流提供了一些见解，包括需要更早转诊至专科医生、对结节病缓解概念的不信任，以及需要旨在减少疾病进展、改善症状和生活质量的疗法。
