Clinical Utility of Functional Precision Medicine in the Management of Recurrent/Relapsed Childhood Rhabdomyosarcoma

Treatments for rhabdomyosarcoma (RMS) including sclerosing and spindle cell rhabdomyosarcoma (SRMS) remains challenging. Aggressive surgical resection and radiation therapy are generally recommended; however, there is no consensus on secondor third-line salvage chemotherapy. Therefore, developing novel therapeutic options for children with all types of RMS suffering relapse under standard protocols is vital. Here, we present results using our functional precision medicine platform in a heavily refractory pediatric SRMS patient who had exhausted all standard options. Our functional precision medicine platform integrates genomic profiling and ex vivo drug sensitivity testing (DST) to determine optimal individualized therapy for each patient (Fig 1).