Treatment of primary cutaneous amyloidosis with tofacitinib: preliminary observations from a single-arm clinical trial.

BACKGROUND

Primary cutaneous amyloidosis (PCA) lacks consensus-based therapeutic guidelines, with conventional modalities demonstrating suboptimal clinical responses.

OBJECTIVES

To assess the efficacy and safety profile of oral tofacitinib, a Janus kinase 1/3 inhibitor, in PCA management.

METHODS

This single-centre, open-label, single-arm prospective interventional study enrolled 20 patients with PCA receiving tofacitinib 5 mg twice daily. Demographic parameters, disease characteristics [measured using the pruritus numeric rating scale (p-NRS), Dermatology Life Quality Index (DLQI) and Lesion Severity (LS) scores], and adverse events were systematically recorded at baseline and weeks 2, 6 and 10 post-treatment.

RESULTS

At week 10, significant reductions were observed across all endpoints: p-NRS from 6.9 (SD 2.9) to 0.4 (SD 0.6, P < 0.001); DLQI from 11.8 (SD 5.6) to 2.6 (SD 2.5, P < 0.001); and LS from 13.3 (SD 5.2) to 4.9 (SD 2.4, P < 0.001). Patients with lichen amyloidosis (8 of 15) and biphasic amyloidosis (3 of 3), but neither of the 2 patients with macular amyloidosis, achieved the primary efficacy threshold. Two treatment-emergent adverse events occurred; these were transient hepatobiliary enzyme elevations resolving with hepatoprotectants and one patient with a self-limiting upper respiratory infection.

CONCLUSIONS

Tofacitinib demonstrates rapid antipruritic effects and sustained lesion clearance in PCA, with favourable tolerability. Study limitations, including the small sample size (n = 20), 10-week duration and uncontrolled design, which may limit generalizability, warrant phase III trials for validation.