Multicenter Analysis of Genomically Targeted Single Patient Use Requests for Pediatric Neoplasms.

ABSTRACT:

Purpose

The US Food and Drug Administration-expanded access program (EAP) uses a single patient use (SPU) mechanism to provide patient access to investigational agents in situations where no satisfactory or comparable therapy is available. Genomic profiling of de novo and relapsed or refractory childhood cancer has led to increased identification of new drug targets in the last decade. The aim of this study is to examine the SPU experience for genomically targeted therapies in patients with pediatric cancer.

Patients and methods

All genomically targeted therapeutic SPUs obtained over a 5-year period were evaluated at four large pediatric cancer programs. Data were collected on the type of neoplasm, agents requested, corresponding molecularly informed targets, and clinical ou

SUBMITTER: Sabnis HS 

PROVIDER: S-EPMC9851705 | biostudies-literature | 2021 Dec

REPOSITORIES: biostudies-literature