TY - JOUR
T1 - Tofacitinib as a Steroid-Sparing Therapy in Pulmonary Sarcoidosis, an Open-Label Prospective Proof-of-Concept Study
AU - Friedman, Marcia A.
AU - Le, Brian
AU - Stevens, Janelle
AU - Desmarais, Julianna
AU - Seifer, Daniel
AU - Ogle, Kimberly
AU - Choi, Dongseok
AU - Harrington, Christina A.
AU - Jackson, Peter
AU - Rosenbaum, James T.
N1 - Funding Information:
We wish to acknowledge the OHSU Massively Parallel Sequencing Shared Resource and the Gene Profiling Shared Resource, which performed all RNA extraction and sequencing for this work; Manny Rodriguez who assisted with the laboratory sample processing; and Puthyda Keath who assisted with patient coordination for this study. We additionally wish to thank the patients who participated in this study.
Funding Information:
This is an investigator-initiated study with expenses paid by Pfizer. The pharmaceutical company played no role in the interpretation of data or the writing of this report. Funding also including NIH grants RO1 EY020249, RO1 EY026572, P30 EY010572, KL2TR002370, and 3T32HL094294-08S1. Funding was also provided by an unrestricted grant from Research to Prevent Blindness, the Grandmaison Fund for Autoimmunity Research, the William and Mary Bauman Foundation, the Stan and Madelle Rosenfeld Family Trust, and the OHSU Wheels Up Program.
Publisher Copyright:
© 2021, The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature.
PY - 2021/4
Y1 - 2021/4
N2 - This is a prospective, open-label, proof-of-concept study of tofacitinib, a Janus kinase inhibitor, as a steroid-sparing therapy in corticosteroid-dependent pulmonary sarcoidosis. Five patients with corticosteroid-dependent pulmonary sarcoidosis were treated with tofacitinib 5 mg twice daily. The primary endpoint was a ≥ 50% reduction in corticosteroids at week 16 with no worsening in pulmonary function or respiratory symptoms. 60% of patients (3/5) met the primary endpoint. One patient was lost to follow up prior to steroid taper, and another was withdrawn due to worsening of known neurosarcoidosis. The three patients who met the primary endpoint each tapered to ≤ 5 mg/day prednisone, respiratory symptoms improved, and spirometry remained stable. In this proof-of-concept study, the addition of a JAK-inhibitor allowed 60% of patients with pulmonary sarcoidosis to successfully taper corticosteroids. JAK-inhibitors are a promising therapy for pulmonary sarcoidosis, which require further investigation in randomized trials. Trial Registration clinicaltrials.gov NCT03793439; registered Jan 4, 2019.
AB - This is a prospective, open-label, proof-of-concept study of tofacitinib, a Janus kinase inhibitor, as a steroid-sparing therapy in corticosteroid-dependent pulmonary sarcoidosis. Five patients with corticosteroid-dependent pulmonary sarcoidosis were treated with tofacitinib 5 mg twice daily. The primary endpoint was a ≥ 50% reduction in corticosteroids at week 16 with no worsening in pulmonary function or respiratory symptoms. 60% of patients (3/5) met the primary endpoint. One patient was lost to follow up prior to steroid taper, and another was withdrawn due to worsening of known neurosarcoidosis. The three patients who met the primary endpoint each tapered to ≤ 5 mg/day prednisone, respiratory symptoms improved, and spirometry remained stable. In this proof-of-concept study, the addition of a JAK-inhibitor allowed 60% of patients with pulmonary sarcoidosis to successfully taper corticosteroids. JAK-inhibitors are a promising therapy for pulmonary sarcoidosis, which require further investigation in randomized trials. Trial Registration clinicaltrials.gov NCT03793439; registered Jan 4, 2019.
KW - Janus kinase inhibitors
KW - Pulmonary sarcoidosis
KW - Sarcoidosis
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U2 - 10.1007/s00408-021-00436-8
DO - 10.1007/s00408-021-00436-8
M3 - Article
C2 - 33825964
AN - SCOPUS:85103892884
VL - 199
SP - 147
EP - 153
JO - Pneumonologie. Pneumonology
JF - Pneumonologie. Pneumonology
SN - 0341-2040
IS - 2
ER -