Twenty-year trends in grade 5 toxicity and response in phase I trials in hematologic malignancy

In the past two decades, cancer drug development has largely shifted from cytotoxic chemotherapy to targeted treatment. Analysis of the Cancer Therapy Evaluation Program at the National Cancer Institute evaluated the toxicity and response in phase I trials for hematologic malignancies and determined how these changed over time. The analysis showed that the response rate in these trials has increased significantly, from 18.5% in 2000-2005 to 50.6% in 2013-2019 (p<0.001). Grade 5 toxicity remained stable over this period with 1.8% overall. Trials that evaluated combination treatment focused on hematologic malignancy of specific disease showed higher response rates.