Results of the preclinical multicenter randomized controlled paclitaxel-induced neuropathy prevention replication study (PINPRICS)

Abstract Objective Chemotherapy-induced peripheral neuropathy (CIPN) is a frequent and serious side effect of many cytotoxic drugs, including paclitaxel. Despite the identification of treatment options in animal models, clinical trials for the treatment or prevention of CIPN have been negative. Majo...

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Main Authors: Wolfgang Boehmerle, Tim Hagenacker, Markus Leo, Linda-Isabell Schmitt, Helmar C. Lehmann, Ines Klein, Regina Stegherr, Frank Konietschke, Matthias Endres, Petra Huehnchen
Format: Article
Language:English
Published: BMC 2025-04-01
Series:BMC Research Notes
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Online Access:https://doi.org/10.1186/s13104-025-07206-2
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author Wolfgang Boehmerle
Tim Hagenacker
Markus Leo
Linda-Isabell Schmitt
Helmar C. Lehmann
Ines Klein
Regina Stegherr
Frank Konietschke
Matthias Endres
Petra Huehnchen
author_facet Wolfgang Boehmerle
Tim Hagenacker
Markus Leo
Linda-Isabell Schmitt
Helmar C. Lehmann
Ines Klein
Regina Stegherr
Frank Konietschke
Matthias Endres
Petra Huehnchen
author_sort Wolfgang Boehmerle
collection DOAJ
description Abstract Objective Chemotherapy-induced peripheral neuropathy (CIPN) is a frequent and serious side effect of many cytotoxic drugs, including paclitaxel. Despite the identification of treatment options in animal models, clinical trials for the treatment or prevention of CIPN have been negative. Major challenges for successful clinical translation of preclinical data include a lack of reproducibility and randomization, small sample sizes and insufficient statistical tests. We therefore conducted a confirmatory, preclinical multicenter randomized controlled replication trial to test the safety and efficacy of three drugs for preventing paclitaxel-induced polyneuropathy: (1) nilotinib, (2) lithium carbonate and (3) interleukin-6-neutralizing antibodies. We preregistered the data analysis plan as well as the two-step study protocol: the optimal doses of the three compounds were assessed first and then tested in a mouse breast cancer xenograft model to compare safety and efficacy. Results Unfortunately, toxicity of intraperitoneally administered nilotinib in combination with paclitaxel was observed, and higher-than-expected tumor growth resulted in a lack of power when the trial was analyzed. Thus, although lithium carbonate and IL-6-neutralizing antibodies tended toward neuroprotection, the differences between these groups were not statistically significant. However, the PINPRICS study ultimately still provides important lessons with regard to the planning and conduction of multicenter preclinical trials.
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spelling doaj-art-d941fe9b369749dbae1f88178dab6dc62025-08-20T02:11:57ZengBMCBMC Research Notes1756-05002025-04-011811910.1186/s13104-025-07206-2Results of the preclinical multicenter randomized controlled paclitaxel-induced neuropathy prevention replication study (PINPRICS)Wolfgang Boehmerle0Tim Hagenacker1Markus Leo2Linda-Isabell Schmitt3Helmar C. Lehmann4Ines Klein5Regina Stegherr6Frank Konietschke7Matthias Endres8Petra Huehnchen9Klinik und Hochschulambulanz für Neurologie, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu BerlinDepartment of Neurology and Center for Translational Neuro- and Behavioral Science, University Hospital EssenDepartment of Neurology and Center for Translational Neuro- and Behavioral Science, University Hospital EssenDepartment of Neurology and Center for Translational Neuro- and Behavioral Science, University Hospital EssenDepartment of Neurology, Medical Faculty, University Hospital CologneDepartment of Neurology, Medical Faculty, University Hospital CologneInstitut für Biometrie und Klinische Epidemiologie, Charité – Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu BerlinInstitut für Biometrie und Klinische Epidemiologie, Charité – Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu BerlinKlinik und Hochschulambulanz für Neurologie, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu BerlinKlinik und Hochschulambulanz für Neurologie, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu BerlinAbstract Objective Chemotherapy-induced peripheral neuropathy (CIPN) is a frequent and serious side effect of many cytotoxic drugs, including paclitaxel. Despite the identification of treatment options in animal models, clinical trials for the treatment or prevention of CIPN have been negative. Major challenges for successful clinical translation of preclinical data include a lack of reproducibility and randomization, small sample sizes and insufficient statistical tests. We therefore conducted a confirmatory, preclinical multicenter randomized controlled replication trial to test the safety and efficacy of three drugs for preventing paclitaxel-induced polyneuropathy: (1) nilotinib, (2) lithium carbonate and (3) interleukin-6-neutralizing antibodies. We preregistered the data analysis plan as well as the two-step study protocol: the optimal doses of the three compounds were assessed first and then tested in a mouse breast cancer xenograft model to compare safety and efficacy. Results Unfortunately, toxicity of intraperitoneally administered nilotinib in combination with paclitaxel was observed, and higher-than-expected tumor growth resulted in a lack of power when the trial was analyzed. Thus, although lithium carbonate and IL-6-neutralizing antibodies tended toward neuroprotection, the differences between these groups were not statistically significant. However, the PINPRICS study ultimately still provides important lessons with regard to the planning and conduction of multicenter preclinical trials.https://doi.org/10.1186/s13104-025-07206-2Chemotherapy-induced polyneuropathyNeuropathic painNeuroprotectionPreclinical replication study
spellingShingle Wolfgang Boehmerle
Tim Hagenacker
Markus Leo
Linda-Isabell Schmitt
Helmar C. Lehmann
Ines Klein
Regina Stegherr
Frank Konietschke
Matthias Endres
Petra Huehnchen
Results of the preclinical multicenter randomized controlled paclitaxel-induced neuropathy prevention replication study (PINPRICS)
BMC Research Notes
Chemotherapy-induced polyneuropathy
Neuropathic pain
Neuroprotection
Preclinical replication study
title Results of the preclinical multicenter randomized controlled paclitaxel-induced neuropathy prevention replication study (PINPRICS)
title_full Results of the preclinical multicenter randomized controlled paclitaxel-induced neuropathy prevention replication study (PINPRICS)
title_fullStr Results of the preclinical multicenter randomized controlled paclitaxel-induced neuropathy prevention replication study (PINPRICS)
title_full_unstemmed Results of the preclinical multicenter randomized controlled paclitaxel-induced neuropathy prevention replication study (PINPRICS)
title_short Results of the preclinical multicenter randomized controlled paclitaxel-induced neuropathy prevention replication study (PINPRICS)
title_sort results of the preclinical multicenter randomized controlled paclitaxel induced neuropathy prevention replication study pinprics
topic Chemotherapy-induced polyneuropathy
Neuropathic pain
Neuroprotection
Preclinical replication study
url https://doi.org/10.1186/s13104-025-07206-2
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