New Study on Management and Treatment of Vaginal Mucosal Melanoma

Mucosal melanoma is a very rare form of melanoma, affecting about 1% of melanoma cases. Mucosal melanoma may affect certain areas of the body, including:

• Oral mucosal melanoma 
• Vulvar mucosal melanoma
• Anorectal mucosal melanoma
• Mucosal melanoma of the head and neck
• Vaginal mucosal melanoma

Interestingly, this type of melanoma is not related to sun exposure and has been identified to be linked to a genetic link with DNA mutation of the KIT gene. As researchers continue to investigate the cause of mucosal melanoma, our Moffitt physicians are making novel strides in the management and treatment of vaginal mucosal melanoma.

Historically, patients diagnosed with locally advanced vaginal mucosal melanoma have resulted in poor prognoses despite aggressive management approaches including pelvic exenteration and adjuvant radiation that carry major morbidities. Moffitt’s research team conducted an immune monitoring study to determine immunotherapy response using ipilimumab and nivolumab.

Moffitt physicians reported a case including a patient in her early 40s who experienced complete pathologic response and organ preservation following immunotherapy consisting of three cycles of ipilimumab and nivolumab. The treatment presented obstacles such as a high-grade immune mediated hepatitis that ultimately resolved with immunosuppressive therapy. Immune monitoring studies utilizing vaginal tumor biopsies showed evidence of enhanced infiltration by CD3+/CD8+ cytotoxic T-cells and increased expression of MHC-I/PD-L1 within the tumor microenvironment following immunotherapy.

Today, the patient's results show no evidence of disease recurrence by radiologic and gynecologic examinations after more than two years of follow-up from the time of immunotherapy initiation. These are exciting outcomes and to our knowledge, this is the only case reported in which a patient with locally advanced vaginal mucosal melanoma experienced complete pathologic response and organ preservation following immune checkpoint blockade as the only treatment approach.

Learn more about Lena’s story.

If you'd like to refer a patient to Moffitt Cancer Center, complete our online form or contact a physician liaison for assistance. As part of our efforts to shorten referral times as much as possible, online referrals are typically responded to within 24 - 48 hours.