Use of vismodegib for the treatment of multiple basal cell carcinomas in a patient with xeroderma pigmentosum

Pediatr Dermatol. 2018 Nov;35(6):e334-e336. doi: 10.1111/pde.13610. Epub 2018 Sep 3.

Abstract

A female patient with xeroderma pigmentosum and multiple basal cell carcinomas was treated with a hedgehog pathway inhibitor (vismodegib), which successfully treated the majority of her basal cell carcinomas while preventing the appearance of new lesions. The sum diameter of lesions showed a 61% decrease after 16.5 months of treatment, although after 18.5 months of treatment, a persistent lesion showed progression and metatypical characteristics; adverse events included persistent alopecia muscle cramps, dysgeusia, and amenorrhea. Despite these limitations, vismodegib may have a role in the treatment of some patients with xeroderma pigmentosum.

Keywords: alopecia; heghehog pathway inhibitors; metatypical BCC; vismodegib; xeroderma pigmentosum.

Publication types

  • Case Reports

MeSH terms

  • Adult
  • Anilides / adverse effects
  • Anilides / therapeutic use*
  • Antineoplastic Agents / adverse effects
  • Antineoplastic Agents / therapeutic use*
  • Carcinoma, Basal Cell / complications
  • Carcinoma, Basal Cell / drug therapy*
  • Female
  • Hamartoma Syndrome, Multiple / complications
  • Hamartoma Syndrome, Multiple / drug therapy*
  • Humans
  • Pyridines / adverse effects
  • Pyridines / therapeutic use*
  • Skin Neoplasms / complications
  • Skin Neoplasms / drug therapy*
  • Skin Neoplasms / pathology
  • Xeroderma Pigmentosum / complications*

Substances

  • Anilides
  • Antineoplastic Agents
  • HhAntag691
  • Pyridines

Supplementary concepts

  • Basal cell carcinoma, multiple