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Modern treatment strategies in pediatric oncology and hematology.

ABSTRACT: Every year, approximately 400 00 children worldwide are diagnosed with cancer. Although treatment results in most types of childhood neoplasms are excellent with survival more than 80%, there are some with poor prognosis. Also recurrent and resistant to treatment childhood cancer remain a therapeutic challenge. Besides chemotherapy, which has been the basis of cancer therapy for years, molecular methods and precisely targeted therapies have recently found their usage. As a result of that, survival has improved and has positively impacted the rate of toxicities associated with chemotherapy (Butler et al. in CA Cancer J Clin 71:315-332, 2021). These achievements have contributed to better quality of patients' lives. Current methods of treatment and ongoing trials give hope for patients with relapses and resistance to conventional chemotherapy. This review focuses on the most recent progress in pediatric oncology treatments and discusses specific therapy methods for particular cancers types of cancer. Targeted therapies and molecular approaches have become more beneficial but research need to be continued in this field. Despite significant breakthroughs in pediatric oncology in the last few years, there is still a need to find new and more specific methods of treatment to increase the survival of children with cancer.

SUBMITTER: Adamczewska-Wawrzynowicz K

PROVIDER: S-EPMC10267092 | biostudies-literature | 2023 Jun

REPOSITORIES: biostudies-literature

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Modern treatment strategies in pediatric oncology and hematology.

Adamczewska-Wawrzynowicz Katarzyna K Wiącek Anna A Kozłowska Aleksandra A Mikosza Klaudia K Szefler Lidia L Dudlik Weronika W Dey Shreya S Varghese Noel N Derwich Katarzyna K

Discover oncology 20230614 1

Every year, approximately 400 00 children worldwide are diagnosed with cancer. Although treatment results in most types of childhood neoplasms are excellent with survival more than 80%, there are some with poor prognosis. Also recurrent and resistant to treatment childhood cancer remain a therapeutic challenge. Besides chemotherapy, which has been the basis of cancer therapy for years, molecular methods and precisely targeted therapies have recently found their usage. As a result of that, surviv ...[more]

PMID: 37314524

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Project description:Background/aimsThe Pediatric Research Equity Act and Best Pharmaceuticals for Children Act are intended to promote the conduct of clinical trials that generate pediatric-specific evidence about drug safety and efficacy. This study assesses the quality of evidence generated through Pediatric Research Equity Act-mandated and Best Pharmaceuticals for Children Act-incentivized clinical trials of hematology/oncology drugs and characterizes subsequent changes in pediatric drug utilization rates.MethodsTrial characteristics (blinding, randomization, and comparator group) were determined for clinical trials that supported pediatric label changes. Using data from OptumLabs® Data Warehouse, a de-identified administrative claims database, we calculated pediatric utilization rates for each drug. We calculated monthly utilization rates from January 2003 (or from the first month in which data were available) to December 2018.ResultsWe identified 11 hematology/oncology drugs that underwent pediatric label changes under the Pediatric Research Equity Act Pediatric Research Equity Act and/or Best Pharmaceuticals for Children Act, and we identified 15 trials supporting these changes. Of these trials, 36% (5/14) were randomized, 31% (4/13) were blinded, and 36% (5/14) used a comparator group. A median of 49 children (interquartile range 29.5) received the drug under investigation across these trials. Pediatric label changes were not associated with subsequent changes in pediatric drug utilization. Although some drugs saw increased pediatric use after gaining new pediatric indications, this pattern was not consistently observed. In addition, there was no evidence to suggest that drugs were utilized less frequently after they failed to receive pediatric indications.ConclusionsClinical trials of hematology/oncology drugs conducted under the Pediatric Research Equity Act Pediatric Research Equity Act and Best Pharmaceuticals for Children Act generally have low methodological rigor, and the resulting label changes are not consistently associated with changes in pediatric utilization. Alternative regulatory strategies and study designs may be necessary to maximize the impact of newly generated knowledge on drug utilization.

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Modern treatment strategies in pediatric oncology and hematology.

Publications

Modern treatment strategies in pediatric oncology and hematology.

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