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Treatment of Childhood NUT Carcinoma
Source: Childhood NUT Carcinoma Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: August 7, 2024 · Captured 2026-09-09.
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Treatment options for childhood NUT carcinoma include the following:
Chemotherapy.
Surgery.
Radiation therapy.
Treatment of childhood NUT carcinoma includes a multimodal approach with systemic chemotherapy, surgery, and radiation therapy. Cisplatin, taxanes, and alkylating agents have been used with some success. While early response to these agents is common, tumor progression occurs early in the course of the disease.[1]; [2][Level of evidence C1]
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In a report from the NUT Midline Carcinoma Registry, 40 patients with primary tumors in the head and neck were evaluable. The 2-year overall survival rate was 30%. The three long-term survivors (with survivals of 35, 72, and 78 months) underwent primary gross-total resection and received adjuvant therapy.[3]; [4][Level of evidence C1]
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Because of the presence of the BRD4::NUTM1 gene fusion in NUT carcinomas, there has been increased interest in evaluating BET bromodomain inhibitors for adults and children with this malignancy.[5] Unfortunately, activity for this class of agents has been limited in reported clinical trials:
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In a phase I study of the BET inhibitor molibresib, confirmed objective responses were observed in 2 of the 19 patients who were treated with a daily dose of 60 mg or higher.[6]
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A subsequent phase II study of molibresib used a daily dose of 75 mg in patients with NUT carcinomas. Only 1 of the 12 patients achieved an objective response, which did not meet the prespecified bar for activity.[7]
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In a phase I study of the BET inhibitor birabresib, three of the nine patients achieved responses, but only one response lasted longer than 2 months.[8]
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Publication references
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Preserved source evidence · Not medical advice