An investigator-initiated open-label clinical trial of vismodegib as a neoadjuvant to surgery for high-risk basal cell carcinoma

Mina Sarah Ally, Sumaira Aasi, Ashley Wysong, Claudia Teng, Eric Anderson, Irene Bailey-Healy, Anthony Oro, Jinah Kim, Anne Lynn Chang, Jean Yuh Tang

Research output: Contribution to journalArticle

69 Citations (Scopus)

Abstract

Background: Vismodegib is an oral hedgehog-pathway inhibitor approved for advanced basal cell carcinoma (BCC). Although most BCCs are amenable to surgery, excision of large tumors in aesthetically sensitive sites may compromise function or cosmesis. Objective: We sought to evaluate the reduction in BCC surgical defect area after 3 to 6 months of neoadjuvant vismodegib. Methods: This was an open-label, single-arm intervention trial with a primary outcome of change in targettumor surgical defect area pre- and post-vismodegib (150 mg/d). Secondary outcomes were change in tumor area and tolerability. Results: Eleven of 15 enrolled patients, aged 39 to 100 years, completed the trial. Thirteen target tumors were excised after a mean of 4 ± 2 months of vismodegib. In all, 29% (4 of 14 patients) could not complete more than 3 months because of vismodegib-related side effects. The mean baseline target-tumor diameter was 3.2 cm, and 10 of 13 tumors occurred on the face. Overall, vismodegib reduced the surgical defect area by 27% (95% confidence interval e45.7% to e7.9%; P =.006) from baseline. Vismodegib was not effective in patients who received less than 3 months. Over a mean follow-up of 11.5 (range 4-21) months for all tumors, only 1 tumor recurred at 17 months post-Mohs micrographic surgery. Limitations: Short follow-up time and no placebo control are limitations. Conclusion: Neoadjuvant vismodegib appears to reduce surgical defect area when taken for 3 months or longer for nonrecurrent BCCs in functionally sensitive locations. Further studies with larger sample sizes and long-term follow-up are warranted.

Original languageEnglish (US)
Pages (from-to)904-911.e1
JournalJournal of the American Academy of Dermatology
Volume71
Issue number5
DOIs
StatePublished - Jan 1 2014

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HhAntag691
Basal Cell Carcinoma
Research Personnel
Clinical Trials
Neoplasms
Mohs Surgery
Sample Size

Keywords

  • Basal cell carcinoma
  • Mohs
  • Neoadjuvant
  • Surgery
  • Surgical defect
  • Vismodegib

ASJC Scopus subject areas

  • Dermatology

Cite this

An investigator-initiated open-label clinical trial of vismodegib as a neoadjuvant to surgery for high-risk basal cell carcinoma. / Ally, Mina Sarah; Aasi, Sumaira; Wysong, Ashley; Teng, Claudia; Anderson, Eric; Bailey-Healy, Irene; Oro, Anthony; Kim, Jinah; Chang, Anne Lynn; Tang, Jean Yuh.

In: Journal of the American Academy of Dermatology, Vol. 71, No. 5, 01.01.2014, p. 904-911.e1.

Research output: Contribution to journalArticle

Ally, Mina Sarah ; Aasi, Sumaira ; Wysong, Ashley ; Teng, Claudia ; Anderson, Eric ; Bailey-Healy, Irene ; Oro, Anthony ; Kim, Jinah ; Chang, Anne Lynn ; Tang, Jean Yuh. / An investigator-initiated open-label clinical trial of vismodegib as a neoadjuvant to surgery for high-risk basal cell carcinoma. In: Journal of the American Academy of Dermatology. 2014 ; Vol. 71, No. 5. pp. 904-911.e1.
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T1 - An investigator-initiated open-label clinical trial of vismodegib as a neoadjuvant to surgery for high-risk basal cell carcinoma

AU - Ally, Mina Sarah

AU - Aasi, Sumaira

AU - Wysong, Ashley

AU - Teng, Claudia

AU - Anderson, Eric

AU - Bailey-Healy, Irene

AU - Oro, Anthony

AU - Kim, Jinah

AU - Chang, Anne Lynn

AU - Tang, Jean Yuh

PY - 2014/1/1

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N2 - Background: Vismodegib is an oral hedgehog-pathway inhibitor approved for advanced basal cell carcinoma (BCC). Although most BCCs are amenable to surgery, excision of large tumors in aesthetically sensitive sites may compromise function or cosmesis. Objective: We sought to evaluate the reduction in BCC surgical defect area after 3 to 6 months of neoadjuvant vismodegib. Methods: This was an open-label, single-arm intervention trial with a primary outcome of change in targettumor surgical defect area pre- and post-vismodegib (150 mg/d). Secondary outcomes were change in tumor area and tolerability. Results: Eleven of 15 enrolled patients, aged 39 to 100 years, completed the trial. Thirteen target tumors were excised after a mean of 4 ± 2 months of vismodegib. In all, 29% (4 of 14 patients) could not complete more than 3 months because of vismodegib-related side effects. The mean baseline target-tumor diameter was 3.2 cm, and 10 of 13 tumors occurred on the face. Overall, vismodegib reduced the surgical defect area by 27% (95% confidence interval e45.7% to e7.9%; P =.006) from baseline. Vismodegib was not effective in patients who received less than 3 months. Over a mean follow-up of 11.5 (range 4-21) months for all tumors, only 1 tumor recurred at 17 months post-Mohs micrographic surgery. Limitations: Short follow-up time and no placebo control are limitations. Conclusion: Neoadjuvant vismodegib appears to reduce surgical defect area when taken for 3 months or longer for nonrecurrent BCCs in functionally sensitive locations. Further studies with larger sample sizes and long-term follow-up are warranted.

AB - Background: Vismodegib is an oral hedgehog-pathway inhibitor approved for advanced basal cell carcinoma (BCC). Although most BCCs are amenable to surgery, excision of large tumors in aesthetically sensitive sites may compromise function or cosmesis. Objective: We sought to evaluate the reduction in BCC surgical defect area after 3 to 6 months of neoadjuvant vismodegib. Methods: This was an open-label, single-arm intervention trial with a primary outcome of change in targettumor surgical defect area pre- and post-vismodegib (150 mg/d). Secondary outcomes were change in tumor area and tolerability. Results: Eleven of 15 enrolled patients, aged 39 to 100 years, completed the trial. Thirteen target tumors were excised after a mean of 4 ± 2 months of vismodegib. In all, 29% (4 of 14 patients) could not complete more than 3 months because of vismodegib-related side effects. The mean baseline target-tumor diameter was 3.2 cm, and 10 of 13 tumors occurred on the face. Overall, vismodegib reduced the surgical defect area by 27% (95% confidence interval e45.7% to e7.9%; P =.006) from baseline. Vismodegib was not effective in patients who received less than 3 months. Over a mean follow-up of 11.5 (range 4-21) months for all tumors, only 1 tumor recurred at 17 months post-Mohs micrographic surgery. Limitations: Short follow-up time and no placebo control are limitations. Conclusion: Neoadjuvant vismodegib appears to reduce surgical defect area when taken for 3 months or longer for nonrecurrent BCCs in functionally sensitive locations. Further studies with larger sample sizes and long-term follow-up are warranted.

KW - Basal cell carcinoma

KW - Mohs

KW - Neoadjuvant

KW - Surgery

KW - Surgical defect

KW - Vismodegib

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