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Practical consideration for successful sequential tumor biopsies in first-in-human trials
In first-in-human (FIH) trials, sequential tumor biopsies, i.e., two consecutive tumor biopsies, the first performed at baseline (pretreatment) and the second during the early treatment period (on-treatment), provide proof of concept in investigational new drugs. We evaluated the success of sequenti...
Autores principales: | , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer US
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9288361/ https://www.ncbi.nlm.nih.gov/pubmed/35404018 http://dx.doi.org/10.1007/s10637-022-01236-4 |
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author | Koyama, Takafumi Shimizu, Toshio Sato, Jun Katsuya, Yuki Iwasa, Satoru Kondo, Shunsuke Yoshida, Tatsuya Sudo, Kazuki Nishino, Makoto Takiguchi, Yuichi Yonemori, Kan Yamamoto, Noboru |
author_facet | Koyama, Takafumi Shimizu, Toshio Sato, Jun Katsuya, Yuki Iwasa, Satoru Kondo, Shunsuke Yoshida, Tatsuya Sudo, Kazuki Nishino, Makoto Takiguchi, Yuichi Yonemori, Kan Yamamoto, Noboru |
author_sort | Koyama, Takafumi |
collection | PubMed |
description | In first-in-human (FIH) trials, sequential tumor biopsies, i.e., two consecutive tumor biopsies, the first performed at baseline (pretreatment) and the second during the early treatment period (on-treatment), provide proof of concept in investigational new drugs. We evaluated the success of sequential tumor biopsies in FIH trials, and explored approaches for improved success rates. We retrospectively reviewed the sequential tumor biopsies required in 17 of 52 FIH trials conducted from 2015 to 2020. One hundred and thirty-eight patients were identified. Success of either pretreatment or on-treatment biopsy alone, and of sequential tumor biopsies, was defined as the acquisition of viable tumor cells and as obtaining tumor cells from both biopsy specimens, respectively. The success rates of pretreatment and on-treatment biopsy were 98.6% and 94.2%, respectively, and of sequential tumor biopsies was 70.3%. Adverse events associated with the pretreatment biopsies (33.3% positive; 72.0% negative) and timing of the first imaging assessment (before on-treatment biopsy = 40.0%; after on-treatment biopsy = 82.7%) correlated with successful sequential tumor biopsies. The reasons for unsuccessful sequential tumor biopsies could be categorized into two groups: 1) patient refusal of the on-treatment biopsy (most frequently due to early disease progression); and 2) absence of tumor cells in the pretreatment or on-treatment biopsy specimen. We propose an approach to achieving greater success in sequential tumor biopsies in FIH trials; the first imaging assessment during the study should be scheduled after on-treatment biopsy. (Registration number UMIN000042487, Date of registration November 18, 2020). |
format | Online Article Text |
id | pubmed-9288361 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | Springer US |
record_format | MEDLINE/PubMed |
spelling | pubmed-92883612022-07-18 Practical consideration for successful sequential tumor biopsies in first-in-human trials Koyama, Takafumi Shimizu, Toshio Sato, Jun Katsuya, Yuki Iwasa, Satoru Kondo, Shunsuke Yoshida, Tatsuya Sudo, Kazuki Nishino, Makoto Takiguchi, Yuichi Yonemori, Kan Yamamoto, Noboru Invest New Drugs Short Report In first-in-human (FIH) trials, sequential tumor biopsies, i.e., two consecutive tumor biopsies, the first performed at baseline (pretreatment) and the second during the early treatment period (on-treatment), provide proof of concept in investigational new drugs. We evaluated the success of sequential tumor biopsies in FIH trials, and explored approaches for improved success rates. We retrospectively reviewed the sequential tumor biopsies required in 17 of 52 FIH trials conducted from 2015 to 2020. One hundred and thirty-eight patients were identified. Success of either pretreatment or on-treatment biopsy alone, and of sequential tumor biopsies, was defined as the acquisition of viable tumor cells and as obtaining tumor cells from both biopsy specimens, respectively. The success rates of pretreatment and on-treatment biopsy were 98.6% and 94.2%, respectively, and of sequential tumor biopsies was 70.3%. Adverse events associated with the pretreatment biopsies (33.3% positive; 72.0% negative) and timing of the first imaging assessment (before on-treatment biopsy = 40.0%; after on-treatment biopsy = 82.7%) correlated with successful sequential tumor biopsies. The reasons for unsuccessful sequential tumor biopsies could be categorized into two groups: 1) patient refusal of the on-treatment biopsy (most frequently due to early disease progression); and 2) absence of tumor cells in the pretreatment or on-treatment biopsy specimen. We propose an approach to achieving greater success in sequential tumor biopsies in FIH trials; the first imaging assessment during the study should be scheduled after on-treatment biopsy. (Registration number UMIN000042487, Date of registration November 18, 2020). Springer US 2022-04-11 2022 /pmc/articles/PMC9288361/ /pubmed/35404018 http://dx.doi.org/10.1007/s10637-022-01236-4 Text en © The Author(s) 2022 https://creativecommons.org/licenses/by/4.0/Open AccessThis article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) . |
spellingShingle | Short Report Koyama, Takafumi Shimizu, Toshio Sato, Jun Katsuya, Yuki Iwasa, Satoru Kondo, Shunsuke Yoshida, Tatsuya Sudo, Kazuki Nishino, Makoto Takiguchi, Yuichi Yonemori, Kan Yamamoto, Noboru Practical consideration for successful sequential tumor biopsies in first-in-human trials |
title | Practical consideration for successful sequential tumor biopsies in first-in-human trials |
title_full | Practical consideration for successful sequential tumor biopsies in first-in-human trials |
title_fullStr | Practical consideration for successful sequential tumor biopsies in first-in-human trials |
title_full_unstemmed | Practical consideration for successful sequential tumor biopsies in first-in-human trials |
title_short | Practical consideration for successful sequential tumor biopsies in first-in-human trials |
title_sort | practical consideration for successful sequential tumor biopsies in first-in-human trials |
topic | Short Report |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9288361/ https://www.ncbi.nlm.nih.gov/pubmed/35404018 http://dx.doi.org/10.1007/s10637-022-01236-4 |
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