COMPASS: A PeDRA-Piloted Multicenter Registry of Compounded Off-label Molecular Pathway Agents for Skin Syndromes
University of California, Irvine
University of California, Irvine
Rady Children’s Hospital of Orange County
Overview:
Some children have birthmarks or blood vessel conditions that can cause pain, bleeding, and visible skin changes that affect their confidence and daily life. Doctors are increasingly using medications originally developed for cancer as creams or gels applied directly to the skin. While these treatments show promise, we still do not know which work best, for which children, or at what dose.
We are building a national registry through PeDRA, a pediatric skin research network to collect information from children treated across the U.S. both past and ongoing. By learning from real-world experience, we aim to identify safer and more effective treatments. In the future, this approach may also help guide care for other genetic skin conditions.
Status:
This was funded through a 2026 PeDRA Research Fellowship Grant.