Efficacy and safety of tofacitinib in polymyalgia rheumatica: a retrospective comparative study

Polymyalgia rheumatica (PMR) is a common inflammatory disorder in older adults, characterized by persistent muscle pain and stiffness. This study aimed to evaluate the efficacy and safety of tofacitinib as a potential therapeutic option in patients with PMR.

This single-centre retrospective study included 60 patients diagnosed with PMR between February 2021 and March 2025. Patients were categorized into three groups: tofacitinib monotherapy (n = 14), tofacitinib combined with prednisone (n = 20), and prednisone monotherapy (n = 26). The primary outcome was the change in the Polymyalgia Rheumatica Activity Score (PMR-AS) from baseline to month 6. Secondary outcomes included erythrocyte sedimentation rate (ESR), C-reactive protein (CRP), morning stiffness duration, elevation of upper limbs, physician- and patient-rated visual analogue scales (VAS), haemoglobin, platelet (PLT) and white blood cell counts, as well as 6 month changes in these parameters, cumulative prednisone dose, and incidence of adverse events.

After 6 months of treatment, all regimens produced significant within-group improvements in PMR-AS, ESR, VAS, haemoglobin, PLT, morning stiffness duration, and CRP levels (p < 0.01 for all). The mean changes in PMR-AS and other outcomes did not differ significantly among groups. Patients receiving tofacitinib combined with prednisone required lower daily and cumulative prednisone doses (p < 0.01). No serious adverse events were recorded during the 6 month follow-up.

Tofacitinib-containing regimens were associated with improvement in PMR disease activity, and combination treatment was associated with lower prednisone exposure. These findings support further prospective evaluation of tofacitinib as a potential glucocorticoid-sparing strategy in PMR.