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Table · dataset · 2026

Supplemental Material for: Complete Response to Neoadjuvant Ipilimumab-Nivolumab in Mucosal Melanoma: Two Case Reports

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<p dir="ltr">Introduction</p><p dir="ltr">Mucosal melanoma (MM) is a rare and biologically aggressive melanoma subtype associated with poor outcomes, even when diagnosed at an early stage.

Description

Unlike cutaneous melanoma (CM), the role of perioperative immunotherapy in patients with localized or locoregional MM remains poorly defined, and the benefit from adjuvant immunotherapy has yet to be established. Although neoadjuvant immunotherapy has demonstrated clear benefit in CM, evidence supporting its use in MM remains limited.</p><p dir="ltr">Case Presentation</p><p dir="ltr">We report two patients with MM treated with neoadjuvant ipilimumab plus nivolumab, both of whom achieved complete responses.

The first patient, diagnosed with maxillary sinus MM, achieved pathological complete response (pCR) after three cycles of combined immunotherapy, despite the development of immune-related hypophysitis and focal myositis involving the lateral rectus muscle. The patient subsequently underwent right maxillary resection and cervical level II lymphadenectomy, followed by adjuvant radiotherapy and nivolumab for a total of 12 months, and remained disease-free at the most recent follow-up, 40 months after surgery.

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The second patient presented with multifocal urethral MM with satellite lesions in the vagina and achieved complete clinical response following four cycles of ipilimumab and nivolumab, with no residual melanoma identified on multiple biopsies of the urethral and vaginal mucosa. Given the anticipated morbidity associated with radical surgery, definitive resection was omitted, and the patient continued maintenance nivolumab for 18 months, which was discontinued following the development of bullous pemphigoid.

The patient remained in complete response at the most recent follow-up, 23 months after documented complete response.</p><p dir="ltr">Conclusion</p><p dir="ltr">These cases highlight the potential of neoadjuvant combination immunotherapy as a promising perioperative strategy for localized MM, capable of inducing deep and durable responses and long-term benefits. They also support the need for prospective studies to better define the role of neoadjuvant immunotherapy and identify predictive biomarkers to select patients most likely to benefit in this therapeutically challenging disease.</p>

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Disease 75% · Immunology 69%
Provenance · 1 source records, 18 field assertions
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