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Novel Combination Explored in Pediatric Rhabdomyosarcoma

Caroline Seymour
Published: Thursday, Nov 01, 2018

Dieter Zopf

Dieter Zopf

Among the 13 compounds investigated in combination with regorafenib (Stivarga), irinotecan seemed to produce the most favorable response in subcutaneous xenograft models of pediatric rhabdomyosarcomas (RMS), according to findings presented at the 2018 ESMO Congress.

during the 2018 ESMO Congress, Dieter Zopf, principal scientist, director, Innovation Sourcing Oncology Bayer Pharmaceuticals, Germany, discussed the identification of effective drug combinations with regorafenib for the treatment of patients with pediatric rhabdomyosarcomas.

OncLive: Please provide an overview of the study you presented on regorafenib combinations in pediatric rhabdomyosarcomas.

Zopf: In Europe, there is a requirement by the regulatory authorities that you have to do a pediatric investigation plan (PIP). Since regorafenib has an approved indication in gastrointestinal stromal tumors (GISTs), which also occur in children, Bayer was requested to submit a PIP. Regorafenib is approved in GISTs, but GISTs don’t often occur in pediatric patients. A trial was already running in patients with GISTs, but they could not recruit their official number of patients in time to achieve the PIP.
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