OBJECTIVE: To investigate the short and long-term efficacy and complications, as well as the influential factors of focused ultrasound for the treatment of vulva dystrophy. METHODS: Seventy-six eligible patients with vulva dystrophy were randomized and treated with focused ultrasound between 1999 and 2002. Among them, 45 patients were with squamous hyperplasia (SH) and 31 patients were with lichen sclerosus (LS). Colposcopic examination and biopsies were used to monitor and evaluate the changes. Statistical analysis was performed using chi(2) (McNemar chi(2)) test. RESULTS: The median follow-up period was 28.3 month (range 24 months to 60 months). Complete remission (CR) occurred in 39 of 76 patients (27 SH and 12 LS). The cure rate was 51% at four years. The response rate was 95% (72/76). Four of the 76 patients (2 SH and 2 LS) had slight skin burn and a few blisters around the labia 2-4 hours after treatment. Moreover, two patients (1 SH and 1 LS) had superficial ulcers on the treated vulva skin two weeks after ultrasound treatment, which were cured without any complications with local anti-inflammatory drugs for 2-3 weeks. No other long-term side effects were found after a follow up for 24-60 months. The total recurrence rate at four years was 36% (26/72), all of whom were treated again with ultrasound therapy with good results and no complications. There was no obvious difference among the different anaesthesia types and pathological types of the lesions (P > 0.05). However, the shorter the history of the disease, the better the efficacy of the treatment. The younger the patient was, the better the efficacy of the treatment. CONCLUSIONS: Vulva dystrophy can be treated with focused ultrasound effectively and safely. This approach appears to be a new promising treatment method.