Platelet-Rich Plasma Versus Saline for the Treatment of Vulvar Lichen Sclerosus: Protocol for a Randomized Controlled Trial.

Background: Vulvar lichen sclerosus (LS) is a chronic relapsing dermatosis commonly affecting the anogenital region in postmenopausal women, though it can affect people of any age and sex. The current gold standard treatment is lifelong topical steroid application to reduce symptoms and prevent the progression of disease, causing irreversible architectural change to the vulval tissue. LS is associated with decreased quality of life and increased risk of vulvar neoplasia. Alternatives to current treatments are highly desired by both clinicians and patients. Platelet-rich plasma (PRP) is an autologous blood product containing high concentrations of platelets and growth factors and is hypothesized to promote wound healing. PRP has been reported to improve symptoms of LS in several case reports and uncontrolled trials.

Objective: This study aims to evaluate the efficacy of PRP versus saline injections to improve the clinical signs and symptoms of LS.

Methods: This is a protocol for a 12-month, single-center, parallel-group, double-blind, randomized controlled trial evaluating the efficacy of PRP for LS compared to saline. Adult female patients with biopsy-proven LS who are otherwise in good health but are nonresponders to topical steroid treatment prior to inclusion are eligible for the trial. A total of 68 participants will be randomly allocated via a random number generator to receive either PRP or saline injections to areas of the vulva with LS. All participants will be blinded to the intervention received. Participants will be followed up with assessments of clinical LS signs, subjective symptom scores, and quality of life questionnaires by blinded assessors at 4 timepoints: baseline, 6 weeks, 6 months, and 12 months. The primary outcome is the clinical scoring system for LS-the total LS score.

Results: Recruitment commenced in May 2020 and concluded in November 2023. This study closed in September 2024 due to slow recruitment. Data are being analyzed in 2025, and results are expected to be published in late 2025.

Conclusions: This study will evaluate the safety and efficacy of PRP injections compared to those of saline injections for the treatment of vulvar LS, potentially providing a novel therapeutic option for patients who do not respond to topical steroids.

Trial Registration: Australia and New Zealand Clinical Trials Registry ACTRN12618001321235p; Universal Trial Number: U1111-1207-4893; anzctr.org.au/Trial/Registration/TrialReview.aspx?id=375536.

International Registered Report Identifier (irrid): DERR1-10.2196/68871.