美国肉样瘤病治疗模式:2016-2022.

IF 9.5 1区 医学 Q1 CRITICAL CARE MEDICINE Chest 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
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引用次数: 0

摘要

背景:在美国,FDA 批准的药物和有关肉样瘤病治疗的真实世界数据都很有限,而且实践模式与指南建议的一致性也没有得到很好的体现:研究设计和方法:我们利用多中心、全付费者、理赔数据库(TriNetX)对2016-2022年期间的肉样瘤病患者进行了回顾性分析。我们确定了确诊后一年内皮质类固醇和/或非类固醇免疫抑制剂(甲氨蝶呤、霉酚酸、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)的治疗情况。我们总结了治疗率、按平均等级排列的处方药物顺序,并使用多变量逻辑回归分析来确定与治疗相关的因素:在 13,330 名符合纳入条件的肉样瘤病患者中,5,671 人(42.5%)在确诊后一年内接受了治疗。在接受治疗的患者中,60%只接受了类固醇治疗,13%只接受了非类固醇免疫抑制剂治疗,27%同时接受了这两种治疗。此外,25%接受治疗的患者首次用药是非类固醇免疫抑制剂。皮质类固醇的平均排序最低,这表明平均而言,皮质类固醇是最先开始使用的药物。在肺部或皮肤受累的患者中,第二种药物平均是羟氯喹,而在心脏或神经系统受累的患者中,第二种药物分别是阿达木单抗和霉酚酸酯。黑人种族、基线器官受累(肺部、心脏和神经系统)和合并诊断(疲劳、高钙血症和ILD)是导致更高的治疗几率的相关因素:在确诊后的第一年内,43%的肉样瘤病患者开始接受治疗。40%的患者使用了非类固醇免疫抑制剂。虽然与开始治疗相关的因素与指南建议一致,但治疗的实践模式却各不相同,尤其是在非类固醇免疫抑制剂治疗的选择和顺序方面,这说明今后需要进行试验和比较效果研究。
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Sarcoidosis Treatment Patterns in the United States: 2016-2022.

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.

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来源期刊
Chest
Chest 医学-呼吸系统
CiteScore
13.70
自引率
3.10%
发文量
3369
审稿时长
15 days
期刊介绍: At CHEST, our mission is to revolutionize patient care through the collaboration of multidisciplinary clinicians in the fields of pulmonary, critical care, and sleep medicine. We achieve this by publishing cutting-edge clinical research that addresses current challenges and brings forth future advancements. To enhance understanding in a rapidly evolving field, CHEST also features review articles, commentaries, and facilitates discussions on emerging controversies. We place great emphasis on scientific rigor, employing a rigorous peer review process, and ensuring all accepted content is published online within two weeks.
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