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.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/tofacitinib-as-a-steroid-sparing-therapy-in-pulmonary-sarcoidosis-two-prospective-cases-and-molecular-analysis/