From the Guidelines
Tofacitinib has been used in a limited number of cases for treating refractory sarcoidosis, with some evidence suggesting its potential efficacy in improving skin lesions and lung function, although it remains an off-label, experimental treatment. The use of tofacitinib in sarcoidosis is based on its mechanism as a JAK inhibitor, which blocks cytokine signaling pathways involved in granuloma formation and inflammation central to sarcoidosis pathophysiology 1.
Key Points
- There are only a few documented cases in the medical literature of tofacitinib being used to treat sarcoidosis, with varying degrees of success.
- In one case, a patient with pulmonary and cutaneous sarcoidosis who failed multiple therapies received tofacitinib 5 mg twice daily, showing significant improvement in skin lesions and lung function after 6 months.
- Another case involved a patient with neurosarcoidosis who was treated with tofacitinib 5 mg twice daily after failing conventional treatments, with reported improvement in neurological symptoms.
- A small case series documented patients with cutaneous sarcoidosis who received tofacitinib 5-10 mg daily with partial to complete resolution of skin lesions over 3-6 months.
Considerations
- Tofacitinib remains an off-label, experimental treatment for sarcoidosis with no established guidelines for dosing or duration.
- Patients should be monitored for potential adverse effects including infection risk, cytopenias, and elevated lipids.
- The Delphi consensus recommendations for a treatment algorithm in pulmonary sarcoidosis suggest glucocorticoids as initial therapy, with non-biologics and biologic therapies considered in severe or extrapulmonary disease requiring prolonged treatment 1.
- However, the most recent and highest quality study, which is the 2025 systematic literature review, provides the most relevant evidence for the use of tofacitinib in sarcoidosis, and should be prioritized in clinical decision-making 1.
From the Research
Cases of Tofacitinib Being Used to Treat Sarcoidosis
- Tofacitinib has been used to treat various forms of sarcoidosis, including:
- Multiorgan sarcoidosis: A patient with long-standing multiorgan sarcoidosis who was unresponsive to other therapies was treated with tofacitinib, resulting in clinical remission of cutaneous sarcoidosis lesions and resolution of positron emission tomography avid lesions in internal organs after 6 months 2.
- Pulmonary sarcoidosis: A prospective, open-label, proof-of-concept study found that 60% of patients with corticosteroid-dependent pulmonary sarcoidosis were able to taper corticosteroids by at least 50% at week 16 with no worsening in pulmonary function or respiratory symptoms while being treated with tofacitinib 3.
- Cutaneous sarcoidosis: An open-label trial found that all patients with cutaneous sarcoidosis experienced improvement in their skin, with 6 patients showing a complete response, after being treated with tofacitinib for 6 months 4.
- Severe laryngeal sarcoidosis: A case report found that tofacitinib prevented critical laryngeal stenosis from progressing to tracheostomy, induced regression of cutaneous lesions, and improved pulmonary function in a patient with severe multi-systemic sarcoidosis 5.
- Refractory sarcoidosis: A study found that sarcoidosis patients treated with tofacitinib had long drug survival, achieved high response rates, and represented a good and safe therapeutic option in patients refractory to previous TNFi therapy 6.
Types of Sarcoidosis Treated with Tofacitinib
- Tofacitinib has been used to treat various types of sarcoidosis, including:
- Corticosteroid-dependent pulmonary sarcoidosis
- Cutaneous sarcoidosis
- Multiorgan sarcoidosis
- Severe laryngeal sarcoidosis
- Refractory sarcoidosis
Efficacy of Tofacitinib in Treating Sarcoidosis
- The efficacy of tofacitinib in treating sarcoidosis has been demonstrated in several studies, with response rates ranging from 60% to 90% 2, 3, 4, 6.
- Tofacitinib has been shown to improve symptoms, reduce corticosteroid use, and induce regression of lesions in patients with sarcoidosis 2, 3, 4, 5, 6.