Investigators Assessed the Long-Term Safety and Effectiveness of Larotrectinib Use in Children With TRK Fusion Sarcoma
Investigators at Cedars-Sinai and other institutions examined the long-term safety and effectiveness of a prescription drug for children with a rare form of cancer who chose to stop taking the drug when their disease hadn't progressed.
The study, published in the Journal of Clinical Oncology, evaluated the long-term efficacy of larotrectinib, a drug that specifically targets the TRK fusion protein found in some children under 18 with cancer, more specifically a type of sarcoma. Researchers wanted to understand what happens when young patients stop taking the medicine through a “wait-and-see” protocol.
Leo Mascarenhas, MD, MS
“Some children were able to stop taking the medicine and still had good results,” said Leo Mascarenhas, MD, MS, corresponding author of the study, director of Pediatric Hematology and Oncology at Cedars-Sinai Guerin Children’s, and medical director of the Sarcoma Program at Cedars-Sinai Cancer. “If their cancer came back, they responded well when they started taking larotrectinib again.”
The study included 91 patients from two clinical trials. Investigators found that out of 47 who stopped taking larotrectinib, 16 had their cancer come back. When these 16 patients started retaking larotrectinib, most responded to the drug and 94% of them benefited.
These findings suggest that doctors can confidently consider stopping larotrectinib treatment in some children with TRK fusion sarcomas, knowing that the same treatment will be effective if the cancer comes back. This study also paves the way for further research into whether stopping targeted treatment could be beneficial for other cancers currently treated with similar medicines.
Other authors include: Steven G. DuBois, MD, MS; Catherine M. Albert, MD; Stefan Bielack, MD; Daniel Orbach, MD; Noah Federman, MD; Birgit Geoerger, MD, PhD; Ramamoorthy Nagasubramanian, MD; Yizhou Zhang, MD; Julia Chisholm, BMBCh, PhD; Soledad Gallego Melcon, MD, PhD; Hiroaki Goto, MD, PhD; Daniel A. Morgenstern, MB, BChir, PhD; Cormac Owens, MD; Alberto S. Pappo, MD; Sébastien Perreault, MD; Johannes H. Schulte, MD; Neerav Shukla, MD; Christian Michel Zwaan, MD, PhD; Natascha Neu, MS; Vadim Bernard-Gauthier, PhD; Esther De La Cuesta, MD, MBA; Cornelis M. van Tilburg, MD, PhD; Theodore W. Laetsch, MD.
Funding: The study was funded by Bayer Healthcare and Loxo Oncology Inc., a wholly owned subsidiary of Eli Lilly and Company.
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