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Evaluation of Efficacy and Safety of add-on Tofacitinib in Patients With Oral Lichen Planus

Evaluation of Efficacy and Safety of add-on Tofacitinib in Patients With Oral Lichen Planus

Recruiting
18 years and older
All
Phase 4

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Overview

Many of the patients with oral lichen planus (OLP) either fail to achieve complete remission or experience frequent relapses with conventional topical corticosteroid therapy, which is currently the mainstay of treatment. Long-term corticosteroid use is limited by local and systemic adverse effects, and many patients develop steroid resistance or intolerance. To overcome these limitations, combination therapy with agents having complementary mechanisms may improve therapeutic outcomes, reduce steroid requirements, and minimize associated adverse effects. Tofacitinib, a Janus kinase (JAK1/JAK3) inhibitor, modulates the JAK-STAT signaling pathway, thereby reducing inflammatory cytokine production involved in OLP pathogenesis. Preliminary case series and pilot trials have shown promising results with tofacitinib in OLP. However, to date, no randomized controlled trial has evaluated the efficacy and safety of add-on oral tofacitinib with standard topical steroid therapy in OLP. Hence, investigators considered tofacitinib to be a candidate drug for add-on therapy due to its anti-inflammatory and immunomodulatory properties. Adding tofacitinib to ongoing topical triamcinolone therapy may increase the response rate, reduce adverse drug reactions by lowering steroid dose requirements, or achieve a quicker therapeutic effect. Therefore, the present randomized controlled trial has been planned to evaluate the efficacy and safety of oral tofacitinib as an add-on therapy in patients with OLP.

Description

Lichen planus (LP) is a chronic, mucocutaneous inflammatory disorder that manifests as violaceous, polygonal, flat-topped papules and plaques, usually affecting the skin, oral mucosa, genital mucosa, and nails. Oral Lichen Planus (OLP) is the most persistent and symptomatic of its many forms, frequently impairing basic functions like eating, speaking, and swallowing. Oral Lichen Planus is an immune-mediated condition characterized by symmetrical, bilateral lesions most frequently seen on the tongue, gingiva, and buccal mucosa. Although the exact etiopathogenesis is unknown, antigen-specific T-cell-mediated cytotoxicity is thought to be a contributing factor. Early diagnosis, therapy, and follow-up are necessary because the disease has a relapsing-remitting course and, in a small percentage of patients, can potentially change into malignancies, particularly in its erosive and atrophic forms.

Oral Lichen Planus can be managed using a variety of treatment approaches, depending on its severity and clinical manifestation. Asymptomatic lesions can be controlled with observation and proper oral hygiene techniques without needing medication. Topical anesthetics and anti-inflammatory drugs can be used to treat mild cases of OLP. Because of their strong anti-inflammatory properties, topical corticosteroids continue to be the cornerstone of treatment for more severe atrophic or erosive forms. However, many patients have numerous relapses and are unable to attain complete remission with monotherapy. Adverse effects, such as oral candidiasis, mucosal thinning, and altered taste perception, are also linked to long-term usage of corticosteroids.

There is no definitive and proven treatment for OLP, despite the wide range of available options, including systemic immunosuppressants like cyclosporine and azathioprine and more recent techniques like laser therapy and photodynamic therapy. Furthermore, there aren't many well-designed, randomized controlled trials that direct standardized care. Therefore, the majority of therapy alternatives are empirical. Patients resistant to corticosteroids present a therapeutic dilemma, necessitating the exploration of newer, targeted options. Janus kinase (JAK) inhibitors, especially tofacitinib, have recently shown promise in treating immune-mediated dermatological disorders, including LP. Tofacitinib suppresses the generation of inflammatory cytokines associated with OLP pathogenesis by modulating the JAK-STAT signaling pathway through the selective inhibition of JAK1 and JAK3. According to preliminary case series and pilot trials, tofacitinib, when applied topically or systemically, can significantly improve erosive OLP, particularly in steroid-refractory cases.

However, to the best of investigator's knowledge, no randomized controlled trials have systematically evaluated the add-on effect of tofacitinib to standard topical steroid therapy in OLP patients. This represents a significant gap in current literature, particularly in defining the role of combination therapies that might offer enhanced efficacy, reduce cumulative steroid doses, and mitigate steroid-related adverse effects. Therefore, this study aims to evaluate the efficacy and safety of tofacitinib as an add-on to topical triamcinolone therapy in patients with oral lichen planus. This trial seeks to provide robust evidence that could potentially redefine treatment paradigms for OLP.

Eligibility

Inclusion Criteria:

  • Patients aged ≥18 of either sex with the clinical diagnosis of oral lichen planus.
  • Patients with a PGA score of ≥3 (moderate and severe oral LP).
  • Patients who are willing to give informed written consent.

Exclusion Criteria:

  • Treatment with a systemic corticosteroid within the last 4 weeks.
  • Patients on immunosuppressive agents such as azathioprine, cyclosporine, and others within one month of recruitment.
  • Patients with a clinical history and any lesion distribution suspicious of a lichenoid drug eruption, and patients with other skin diseases.
  • Past or current history of any malignancy, including moderate to severe dysplasia of the oral mucosa on oral biopsy.
  • Severe active infection, including active tuberculosis, hepatitis B, or C infection
  • Patients with cytopenia (Hb \<9g/dl, leukocyte count \<4000/mm3, platelet count \<100,000/mm3)
  • The patient with a history of alcohol abuse.
  • Decreased liver or renal function (creatinine \> 2.0mg/dl, total bilirubin \> 2.5 mg/dl).
  • Severe acute infection, uncontrolled diabetes mellitus, congenital or acquired immunodeficiency, severe cardiac disease (NYHA grade IV), MI in the last four weeks, severe schizophrenia, or depression.
  • Patient with a history of hypersensitivity to topical Triamcinolone or Tofacitinib.
  • Pregnancy and lactation, women of childbearing age without effective contraception.

Study details
    Oral Lichen Planus

NCT07131813

All India Institute of Medical Sciences, Bhubaneswar

1 February 2026

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