Avina Rami, Kira Bona, Suzanne Shusterman, Karen Wright, Andrew E. Place, Puja J. Umaretiya, Ketki Bhushan, Steven G. DuBois, Kevin Campbell
{"title":"参与早期临床试验的儿科肿瘤患者的当代特征和结果","authors":"Avina Rami, Kira Bona, Suzanne Shusterman, Karen Wright, Andrew E. Place, Puja J. Umaretiya, Ketki Bhushan, Steven G. DuBois, Kevin Campbell","doi":"10.1002/cam4.71222","DOIUrl":null,"url":null,"abstract":"<div>\n \n \n <section>\n \n <h3> Background</h3>\n \n <p>Phase 1 or phase 1/2 trials are a first step in pediatric cancer drug development. Currently, there is a paucity of information regarding contemporary outcomes for pediatric patients enrolled in these trials. We describe characteristics and outcomes of patients enrolled in pediatric phase 1 clinical trials over a 9-year period at a single institution.</p>\n </section>\n \n <section>\n \n <h3> Methods</h3>\n \n <p>We queried our clinical trials management system to generate a list of patients enrolled and treated on pediatric phase 1 or phase 1/2 trials from 2011 to 2019. We collected baseline demographics, clinical data, efficacy, and safety endpoints post-enrollment including: time to death, objective response to therapy, duration on therapy, need for dose modification, and occurrence of dose-limiting toxicity. Overall survival was calculated using Kaplan–Meier methods.</p>\n </section>\n \n <section>\n \n <h3> Results</h3>\n \n <p>A total of 224 unique patients accounted for 259 enrollments and 242 treatment episodes. The median age at enrollment was 11 years (range 0–27 years) and 56.2% were male. The majority were White (85.7%) and Non-Hispanic (88.2%). English was the primary language for 86.3% of patients, and 54.9% had private insurance. Solid tumors were the most common malignancy (41.0%), followed by brain tumors (34.1%), and hematologic malignancies (24.9%). Among treatment episodes, 49.3% received targeted monotherapy. After first enrollment, 27.6% of patients had an objective response to therapy (52.9% for hematologic malignancies, 20.5% for brain tumors, and 15.8% for solid tumors). The median duration of therapy was 1.5 months. Median overall survival from first enrollment for 218 patients treated with available vital status was 13.1 months. Toxicity outcomes included 27 patients (11.2%) requiring dose modification and 22 patients (9.0%) having a DLT.</p>\n </section>\n \n <section>\n \n <h3> Conclusions</h3>\n \n <p>Overall survival is poor for patients in pediatric oncology early phase trials, despite approximately a quarter having an initial response. These data are informative for discussions between providers and families regarding outcomes after phase 1 trial participation.</p>\n </section>\n </div>","PeriodicalId":139,"journal":{"name":"Cancer Medicine","volume":"14 17","pages":""},"PeriodicalIF":3.1000,"publicationDate":"2025-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/cam4.71222","citationCount":"0","resultStr":"{\"title\":\"Contemporary Characteristics and Outcomes of Pediatric Oncology Patients Participating in Early Phase Clinical Trials\",\"authors\":\"Avina Rami, Kira Bona, Suzanne Shusterman, Karen Wright, Andrew E. Place, Puja J. Umaretiya, Ketki Bhushan, Steven G. DuBois, Kevin Campbell\",\"doi\":\"10.1002/cam4.71222\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div>\\n \\n \\n <section>\\n \\n <h3> Background</h3>\\n \\n <p>Phase 1 or phase 1/2 trials are a first step in pediatric cancer drug development. Currently, there is a paucity of information regarding contemporary outcomes for pediatric patients enrolled in these trials. We describe characteristics and outcomes of patients enrolled in pediatric phase 1 clinical trials over a 9-year period at a single institution.</p>\\n </section>\\n \\n <section>\\n \\n <h3> Methods</h3>\\n \\n <p>We queried our clinical trials management system to generate a list of patients enrolled and treated on pediatric phase 1 or phase 1/2 trials from 2011 to 2019. We collected baseline demographics, clinical data, efficacy, and safety endpoints post-enrollment including: time to death, objective response to therapy, duration on therapy, need for dose modification, and occurrence of dose-limiting toxicity. Overall survival was calculated using Kaplan–Meier methods.</p>\\n </section>\\n \\n <section>\\n \\n <h3> Results</h3>\\n \\n <p>A total of 224 unique patients accounted for 259 enrollments and 242 treatment episodes. The median age at enrollment was 11 years (range 0–27 years) and 56.2% were male. The majority were White (85.7%) and Non-Hispanic (88.2%). English was the primary language for 86.3% of patients, and 54.9% had private insurance. Solid tumors were the most common malignancy (41.0%), followed by brain tumors (34.1%), and hematologic malignancies (24.9%). Among treatment episodes, 49.3% received targeted monotherapy. After first enrollment, 27.6% of patients had an objective response to therapy (52.9% for hematologic malignancies, 20.5% for brain tumors, and 15.8% for solid tumors). The median duration of therapy was 1.5 months. Median overall survival from first enrollment for 218 patients treated with available vital status was 13.1 months. 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Contemporary Characteristics and Outcomes of Pediatric Oncology Patients Participating in Early Phase Clinical Trials
Background
Phase 1 or phase 1/2 trials are a first step in pediatric cancer drug development. Currently, there is a paucity of information regarding contemporary outcomes for pediatric patients enrolled in these trials. We describe characteristics and outcomes of patients enrolled in pediatric phase 1 clinical trials over a 9-year period at a single institution.
Methods
We queried our clinical trials management system to generate a list of patients enrolled and treated on pediatric phase 1 or phase 1/2 trials from 2011 to 2019. We collected baseline demographics, clinical data, efficacy, and safety endpoints post-enrollment including: time to death, objective response to therapy, duration on therapy, need for dose modification, and occurrence of dose-limiting toxicity. Overall survival was calculated using Kaplan–Meier methods.
Results
A total of 224 unique patients accounted for 259 enrollments and 242 treatment episodes. The median age at enrollment was 11 years (range 0–27 years) and 56.2% were male. The majority were White (85.7%) and Non-Hispanic (88.2%). English was the primary language for 86.3% of patients, and 54.9% had private insurance. Solid tumors were the most common malignancy (41.0%), followed by brain tumors (34.1%), and hematologic malignancies (24.9%). Among treatment episodes, 49.3% received targeted monotherapy. After first enrollment, 27.6% of patients had an objective response to therapy (52.9% for hematologic malignancies, 20.5% for brain tumors, and 15.8% for solid tumors). The median duration of therapy was 1.5 months. Median overall survival from first enrollment for 218 patients treated with available vital status was 13.1 months. Toxicity outcomes included 27 patients (11.2%) requiring dose modification and 22 patients (9.0%) having a DLT.
Conclusions
Overall survival is poor for patients in pediatric oncology early phase trials, despite approximately a quarter having an initial response. These data are informative for discussions between providers and families regarding outcomes after phase 1 trial participation.
期刊介绍：
Cancer Medicine is a peer-reviewed, open access, interdisciplinary journal providing rapid publication of research from global biomedical researchers across the cancer sciences. The journal will consider submissions from all oncologic specialties, including, but not limited to, the following areas:
Clinical Cancer Research
Translational research ∙ clinical trials ∙ chemotherapy ∙ radiation therapy ∙ surgical therapy ∙ clinical observations ∙ clinical guidelines ∙ genetic consultation ∙ ethical considerations
Cancer Biology:
Molecular biology ∙ cellular biology ∙ molecular genetics ∙ genomics ∙ immunology ∙ epigenetics ∙ metabolic studies ∙ proteomics ∙ cytopathology ∙ carcinogenesis ∙ drug discovery and delivery.
Cancer Prevention:
Behavioral science ∙ psychosocial studies ∙ screening ∙ nutrition ∙ epidemiology and prevention ∙ community outreach.
Bioinformatics:
Gene expressions profiles ∙ gene regulation networks ∙ genome bioinformatics ∙ pathwayanalysis ∙ prognostic biomarkers.
Cancer Medicine publishes original research articles, systematic reviews, meta-analyses, and research methods papers, along with invited editorials and commentaries. Original research papers must report well-conducted research with conclusions supported by the data presented in the paper.
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