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Abstract Number: 1039

Tofacitinib as a Steroid-sparing Therapy in Pulmonary Sarcoidosis: Two Prospective Cases and Molecular Analysis

Marcia Friedman1, Stevens Janelle2, Julianna Desmarais3, Daniel Seifer3, Brian Le3, Kimberly Ogle3, Christina Harrington3, Peter Jackson4, Dongseok Choi3 and James Rosenbaum3, 1Oregon Health and Science University, Portland, OR, 2Tuality Healthcare, Hillsboro, OR, 3Oregon Health & Science University, Portland, OR, 4Johns Hopkins University, Baltimore, MD

Meeting: ACR Convergence 2020

Keywords: Biologicals, clinical trial, Gene Expression, Intervention, pulmonary

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Session Information

Date: Sunday, November 8, 2020

Session Title: Miscellaneous Rheumatic & Inflammatory Diseases Poster II: Sarcoidosis, Interstitial Lung Disease, & Inflammatory Eye Disease

Session Type: Poster Session C

Session Time: 9:00AM-11:00AM

Background/Purpose: Patients with pulmonary sarcoidosis often require prolonged corticosteroids to treat their disease. There are currently no FDA approved steroid-sparing therapies for sarcoidosis. We report here on the first two patients who have completed a prospective open-label study evaluating tofacitinib as a steroid-sparing therapy in pulmonary sarcoidosis.

Methods: Patients with pulmonary sarcoidosis who were unable to taper below 15-30mg/day of prednisone were invited to participate in this open-label proof of concept study. Patients were started on tofacitinib 5mg twice daily. After 4 weeks, prednisone was tapered according to a specified protocol. The primary endpoint was a ≥ 50% reduction in corticosteroids at week 16 with no worsening of respiratory symptoms by Saint George Respiratory Questionnaire (SGRQ) or pulmonary function; patients who met the primary endpoint were invited to enroll in a one-year extension study.

Results: The first two patients have completed this open-label study. By week 16 each had tapered to ≤ 5mg/day prednisone and thus met the primary endpoint. During the one-year extension study, both patients tapered fully off prednisone. There was no significant change in spirometry. Respiratory symptoms improved (average total SGRQ score change of -24.1 points). Chest imaging was stable or mildly improved.

Conclusion: We report here the first two patients enrolled as part of an open-label prospective pilot study of tofacitinib as a steroid-sparing agent in pulmonary sarcoidosis. Both patients were able to taper steroids successfully with no disease worsening or significant adverse events. Tofacitinib is a promising therapy for pulmonary sarcoidosis.

Figure 1: Changes in clinical data from baseline to week 16: A) Prednisone was tapered according to a specified protocol; there were no deviations from the planned taper. Both patients tapered prednisone to < 5mg by week 16 and continued to taper during 1-year extension. B) Saint George Respiratory Questionnaire (SGRQ) scores all improved during the trial; a clinically significant difference for SGRQ is 4 units, thus all SGRQ changes were clinically significant. C) The chest x-ray for patient 1 remained stable over 16 weeks, the chest x-ray for patient 2 showed improvement in mild bronchovascular nodularity (arrow indicates area of improved nodularity). D) Pulmonary function was stable for both patients given established 10% between-test margin of variability for spirometry measurement.

Table 1: Differentially expressed genes in whole blood RNA at week 16 (on tofacitinib) compared to baseline (on higher dose prednisone): A total of 41 genes (23 upregulated and 18 downregulated) were differentially expressed, each with a fold change (FC) >1.5 and a false discovery rate (FDR) p-value < 0.05. A Kyoto Encyclopedia of Genes and Genomes (KEGG) pathway analysis revealed 13 pathways significantly associated with differentially expressed genes.


Disclosure: M. Friedman, None; S. Janelle, None; J. Desmarais, None; D. Seifer, None; B. Le, None; K. Ogle, None; C. Harrington, None; P. Jackson, None; D. Choi, None; J. Rosenbaum, Gilead, 1, Eli Lilly, 1, Abbvie, 5, UCB Pharma, 5, Roche, 1, Santen, 1, Corvus, 1, Celldex, 1, Horizon, 1, Novartis, 1, Eyevensys, 5, Janssen, 5, UpToDate, 7.

To cite this abstract in AMA style:

Friedman M, Janelle S, Desmarais J, Seifer D, Le B, Ogle K, Harrington C, Jackson P, Choi D, Rosenbaum J. Tofacitinib as a Steroid-sparing Therapy in Pulmonary Sarcoidosis: Two Prospective Cases and Molecular Analysis [abstract]. Arthritis Rheumatol. 2020; 72 (suppl 10). https://acrabstracts.org/abstract/tofacitinib-as-a-steroid-sparing-therapy-in-pulmonary-sarcoidosis-two-prospective-cases-and-molecular-analysis/. Accessed January 22, 2021.
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