Targeted Inhibition of the Epidermal Growth Factor Receptor and Mammalian Target of Rapamycin Signaling Pathways in Olmsted Syndrome.

ABSTRACT:

Importance

Olmsted syndrome is a rare and disabling genodermatosis for which no successful treatment is currently available.

Objective

To evaluate the clinical response to the mammalian target of rapamycin (mTOR) inhibitor sirolimus and/or the epidermal growth factor receptor (EGFR) inhibitor erlotinib among patients with Olmsted syndrome.

Design, setting, and participants

This case series focused on 4 children with treatment-refractory Olmsted syndrome. These children received treatments (initiated in 2017 and 2018) at the outpatient dermatology clinic at the Children's Hospital of Wisconsin in Milwaukee, Wisconsin; Children's National Hospital in Washington, DC; and Hospital Infantil Pequeno Príncipe, Curitiba in Paraná, Brazil.

Exposures

Immunohistochemical

SUBMITTER: Zhang A 

PROVIDER: S-EPMC6990762 | biostudies-literature | 2020 Feb

REPOSITORIES: biostudies-literature