Dr. Timothy S. Olson, Associate Professor of Pediatrics and Medical Director of the Blood & Marrow Transplant Program at the Children’s Hospital of Philadelphia (CHOP), provides an overview of the RUNX1-FPD stem cell harvesting and banking clinical trial he is leading at this site.

This RRP-sponsored study evaluates the safety and feasibility of collecting and storing blood stem cells from individuals with RUNX1 familial platelet disorder. Learn about:

• Why stem-cell–based gene therapy is becoming an important future option for inherited blood disorders

• How the trial collects, banks, and monitors patients’ autologous stem cells

• Who may be eligible to participate and what the process involves

• The role of RUNX1 mutations in leukemia risk and prevention

• How CHOP and MD Anderson are collaborating on parallel studies

This presentation is intended for patients and families, though clinicians and researchers may also find it useful.