Sarcoidosis Treatment Patterns in the United States: 2016-2022

Abstract

Background: There are limited US Food and Drug Administration-approved medications and real-world data on sarcoidosis treatment in the United States. 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: A retrospective analysis was conducted of patients with sarcoidosis from 2016 to 2022 using a multicenter, all-payer claims database (TriNetX). Treatments with corticosteroids and/or nonsteroidal immunosuppressive medications (methotrexate, mycophenolate, leflunomide, hydroxychloroquine, cyclophosphamide, infliximab, adalimumab, azathioprine, rituximab, and Janus kinase inhibitors) within 1 year of diagnosis were ascertained. We summarized treatment rates and sequence of prescribed medications by mean rank, and used multivariable logistic regression analyses to identify factors associated with treatment.

Results: Of 13,330 patients with sarcoidosis meeting inclusion, 5,671 (42.5%) received treatment within 1 year of diagnosis. Of those treated, 60% received steroids alone, 13% received nonsteroidal immunosuppressives alone, and 27% received both. Furthermore, 25% of treated patients received a nonsteroidal 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; 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 interstitial lung disease).

Interpretation: Within the first year of diagnosis, 43% of patients with sarcoidosis were started on treatment. Nonsteroidal immunosuppressives were used in 40% of treated patients. Although factors associated with treatment initiation aligned with guideline recommendations, practice patterns of treatment were variable, particularly in choice and sequence of nonsteroidal immunosuppressive therapy, underscoring the need for future trials and comparative effectiveness studies.

Keywords: practice pattern research; sarcoidosis; treatment of sarcoidosis.