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Case Report/Case Series |

The Use of Vismodegib to Shrink Keratocystic Odontogenic Tumors in Patients With Basal Cell Nevus Syndrome

Mina S. Ally, BSc, MBBS1; Jean Y. Tang, MD, PhD1; Timmy Joseph, MD2; Bobbye Thompson, MD3; Joselyn Lindgren, MS4; Maria Acosta Raphael, BA4; Grace Ulerio, BA3; Anita M. Chanana, BS4; Julian M. Mackay-Wiggan, MD3; David R. Bickers, MD3; Ervin H. Epstein Jr, MD4
[+] Author Affiliations
1Department of Dermatology, Stanford University, Redwood City, California
2Department of Radiology, Stanford University, Redwood City, California
3Herbert Irving Comprehensive Cancer Center, Department of Dermatology, Columbia University Medical Center, New York, New York
4Children’s Hospital Oakland Research Institute, Oakland, California
JAMA Dermatol. 2014;150(5):542-545. doi:10.1001/jamadermatol.2013.7444.
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Importance  Keratocystic odontogenic tumors (KCOTs) of the jaw affect more than 65% of patients with basal cell nevus syndrome (BCNS). Surgery frequently causes facial disfigurement and is not always curative. Most BCNS-related and some sporadic KCOTs have malignant activation of the Hedgehog signaling pathway.

Observations  We examined the effect of vismodegib (an oral Hedgehog pathway inhibitor) on KCOT size in patients with BCNS enrolled in a clinical trial testing vismodegib for basal cell carcinoma prevention (NCT00957229), using pretreatment and posttreatment magnetic resonance imaging. Four men and 2 women had pretreatment KCOTs (mean longest diameter, 2.0 cm; range, 0.7-3.3 cm), occurring primarily in the mandible. Patients were treated with vismodegib, 150 mg/d, for a mean (SD) of 18.0 (4.8) months (range, 11-24 months). Four patients experienced a size reduction and 2 had no change. Vismodegib reduced the mean longest diameter of KCOTs in all patients by 1.0 cm (95% CI, 0.03-1.94; P = .02) or 50% from baseline. We observed no enlargement of existing KCOTs or new KCOT development.

Conclusions and Relevance  Vismodegib shrinks some KCOTs in patients with BCNS and may offer an alternative to surgical therapy. These effects were maintained for at least 9 months after drug cessation in 1 patient. Further studies assessing long-term efficacy and optimal maintenance regimens should be performed.

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Figure 1.
Consolidated Standards of Reporting Trials (CONSORT) Diagram

A CONSORT flow diagram of patient selection highlighting reasons for exclusion from the case series, as well as baseline and posttreatment magnetic resonance imaging (MRI) results. KCOT indicates keratocystic odontogenic tumor.

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Figure 2.
Magnetic Resonance Images of Patient 4

Serial sagittal T2-weighted magnetic resonance images demonstrating the size change in the left maxillary keratocystic odontogenic tumor (arrows) (A) from baseline, (B) after 11 months of vismodegib, and (C) 9 months after drug discontinuation.

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