Tofacitinib as monotherapy in cutaneous polyarteritis nodosa: a case series

Abstract

Objectives Cutaneous polyarteritis nodosa (CPAN) is a distinct clinical entity represented by a chronic, relapsing, benign course with a rare systemic involvement [1]. Treatment is with steroids, cyclophosphamide or other conventional synthetic disease-modifying antirheumatic drugs (csDMARDS)[2]. In this case series, we aimed to share our varied clinical experience of successfully treating patients with CPAN, with tofacitinib in refractory/relapsing course as well as upfront monotherapy without steroids/csDMARDS. Methods We report this retrospective case series managed at our rheumatology centre in Bangalore from 2019–2022. Four patients identified as CPAN on biopsy were able to achieve disease-free remission with tofacitinib as part of treatment, with no relapse on further follow up. Our patients presented with subcutaneous nodules and cutaneous ulcers. After systemic evaluation, all the patients underwent skin biopsy which showed fibrinoid necrosis in the vessel walls of the dermis with histopathological impression of CPAN. They were initially treated with a conventional approach of steroids with/without csDMARDS. On experiencing a refractory/relapsing course, Tofacitinib was tried in all the patients as either steroid sparing/upfront monotherapy without concomitant csDMARDS. Results Use of tofacitinib resulted in improvement of ulcers and paresthesia, and gradual healing of skin lesions, although with scarring, with no further recurrence or relapse over a period of follow-up of 6 months for all the patients. The therapeutic effect of tofacitinib was consistent when used either as steroid sparing or as upfront monotherapy thereby proving the drug to be a promising option that warrants larger trials in future to treat the subset of patients with established CPAN. Conclusion Tofacitinib could be used for disease-free remission as monotherapy for Cutaneous polyarteritis nodosa either upfront or as steroid-sparing, even without concomitant csDMARDS, in those patients who are dependent on steroids or multiple DMARDs. Lay summary What does this mean for patients? Cutaneous polyarteritis nodosa (CPAN) is a rare disease that causes frequently recurring and often chronic skin lesions. Steroids and other drugs are used to treat it, but some patients may not respond well or may have side effects. In this case series, it was found that tofacitinib, a medication used to treat people with rheumatoid arthritis, was effective in treating CPAN in four patients. When we followed up with the patients, they had achieved disease-free remission with no relapse. Some patients were able to stop taking steroids and other drugs after starting tofacitinib. It is hence suggested that tofacitinib could be used as a monotherapy (i.e. without combining it with other drugs) for CPAN to reduce the need for steroids and other drugs. It is hoped that future researchers will find these data encouraging and may conduct larger trials to investigate the use of tofacitnib to treat CPAN.