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Sarcoidosis Treatment Patterns in the United States: 2016-2022.
Chest ( IF 9.5 ) Pub Date : 2024-11-08 , DOI: 10.1016/j.chest.2024.10.040
Ruchika Sangani,Nicholas A Bosch,Praveen Govender,Brittany Scarpato,Allan J Walkey,Julia Newman,Anica C Law,Kari R Gillmeyer,Divya A Shankar

BACKGROUND There are limited FDA-approved medications and real-world data on sarcoidosis treatment in the U.S. and concordance of practice patterns with guideline recommendations have not been well characterized. RESEARCH QUESTION What are the practice patterns and factors associated with treatment for patients with sarcoidosis in the year following diagnosis? STUDY DESIGN AND METHODS We conducted a retrospective analysis of patients with sarcoidosis from 2016 - 2022 using a multicenter, all-payer, claims database (TriNetX). We ascertained treatments with corticosteroids and/or non-steroidal immunosuppressive medications (methotrexate, mycophenolate, leflunomide, hydroxychloroquine, cyclophosphamide, infliximab, adalimumab, azathioprine, rituximab, and janus kinase inhibitors) within one year of diagnosis. We summarized treatment rates, sequence of prescribed medications by mean rank, and used multivariable logistic regression analyses to identify factors associated with treatment. RESULTS Out of 13,330 patients with sarcoidosis meeting inclusion, 5,671 (42.5%) received treatment within a year of diagnosis. Of those treated, 60% received steroids alone, 13% received non-steroidal immunosuppressives alone, and 27% received both. Further, 25% of treated patients received a non-steroidal immunosuppressive as their first medication. Corticosteroids had the lowest mean rank order, indicating they were, on average, the first medication initiated. Among those with pulmonary or cutaneous involvement, the second medication initiated on average was hydroxychloroquine, while in those with cardiac or neurologic involvement it was adalimumab and mycophenolate, respectively. Factors associated with higher odds of treatment were Black race, organ involvement at baseline (pulmonary, cardiac, and neurologic), and comorbid diagnoses (fatigue, hypercalcemia, and ILD). INTERPRETATION Within the first year of diagnosis, 43% of patients with sarcoidosis were started on treatment. Non-steroidal immunosuppressives were used in 40% of treated patients. While factors associated with treatment initiation aligned with guideline recommendations, practice patterns of treatment were variable, particularly in choice and sequence of non-steroidal immunosuppressive therapy, underscoring the need for future trials and comparative effectiveness studies.

中文翻译:


美国结节病治疗模式:2016-2022 年。



背景 美国 FDA 批准的药物和关于结节病治疗的真实世界数据有限,并且实践模式与指南建议的一致性尚未得到很好的表征。研究问题 诊断后一年内与结节病患者治疗相关的实践模式和因素是什么?研究设计和方法 我们使用多中心、全付款人索赔数据库 (TriNetX) 对 2016 年至 2022 年的结节病患者进行了回顾性分析。我们确定了在诊断后 1 年内使用皮质类固醇和/或非甾体免疫抑制药物(甲氨蝶呤、麦考酚酯、来氟米特、羟氯喹、环磷酰胺、英夫利昔单抗、阿达木单抗、硫唑嘌呤、利妥昔单抗和 janus 激酶抑制剂)的治疗。我们按平均秩总结了治疗率、处方药顺序,并使用多变量 logistic 回归分析来确定与治疗相关的因素。结果在符合纳入标准的 13,330 名结节病患者中,5,671 名 (42.5%) 在诊断后一年内接受了治疗。在接受治疗的患者中,60% 仅接受类固醇治疗,13% 仅接受非甾体免疫抑制剂治疗,27% 接受两者治疗。此外,25% 的接受治疗的患者接受了非甾体免疫抑制药物作为他们的第一种药物。皮质类固醇的平均排名顺序最低,表明它们平均是第一种开始使用的药物。在肺部或皮肤受累的患者中,平均开始使用的第二种药物是羟氯喹,而在心脏或神经系统受累的患者中,分别是阿达木单抗和麦考酚酯。 与较高治疗几率相关的因素是黑人种族、基线器官受累(肺、心脏和神经系统)和合并症诊断(疲劳、高钙血症和 ILD)。解释 在诊断的第一年内,43% 的结节病患者开始接受治疗。40% 的接受治疗的患者使用了非甾体免疫抑制剂。虽然与治疗开始相关的因素与指南建议一致,但治疗实践模式是可变的,特别是在非甾体免疫抑制治疗的选择和顺序方面,这强调了未来试验和比较有效性研究的必要性。
更新日期:2024-11-08
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