Introduction:In Europe, despite recent advances in clinical development, most of the drugs currently used to treat childhood cancers are adult medicines, prescribed outside of the authorized indication. In this context, a monocentric retrospective cohort analysis was conducted, evaluating pediatric, adolescent, and young adult patients affected by onco-hematologic disease, treated with targeted therapies used off-label or as compassionate use. Methods:The analysis was conducted on 45 patients aged less than or equal to 30 years with cancer, having received at least one targeted therapy prescribed as offlabel or compassionate use at a large Italian pediatric center between January 1, 2016 and June 30, 2021. Data collected included information on the patient and tumor, data on off-label/compassionate treatment, and data on safety and efficacy.Results: Total 25 out of 45 patients treated with off-label or compassionate targeted therapies were affected by onco-hematological diseases. Overall, 22 out of the 52 agents (42%) were prescribed in patients with relapsed neoplasm and 39% (20/52) in patients with refractory diseases. Complete response was observed in more than half (27/52) of treatments. At least one adverse reaction occurred in 76% (n = 22) of agents administered to patients with onco-hematological tumor and in 43% (n = 10) of agents prescribed to patients with solid tumor. Conclusion:This work aims to provide a snapshot of off-label and compassionate use prescriptions in a large Italian pediatric cancer center. This study confirms that targeted agents for unauthorized indications are often prescribed in pediatric patients with cancer, especially after disease relapse and that these treatments are mostly tolerable and effective.

Background: In an era where clinical trials have become more and more complex and regulatory authorities impose very high quality standards, the education of clinical research professionals becomes crucial. As one of ICH-GCP guiding principles, adequate training should be ensured and included in educational programs. Methods: In 2021, the Italian Group of Data Managers and Clinical Research Coordinators shared among professionals involved in clinical research an online survey aimed at investigating quality and characteristics of clinical research training provided during undergraduate and postgraduate Italian programs. Results: The survey was completed by 280 professionals: 178 study coordinators, 29 clinical research associates, 20 project managers, 7 study nurses, and 44 others. The majority were 25-45 years old (n = 242, 86.4%), worked at experimental sites (n = 211, 75.4%), and almost all (n = 252, 90.0%) had at least a master’s degree, mainly in biology/biotechnology (n = 162, 57.9%) and pharmacy (n = 64, 22.9%). Clinical research education during the degree courses was considered poor by 73.6% (n = 206). The knowledge on clinical research professional world at the time of graduation was considered poor by 71.1% of participants (n = 199), like the knowledge of related career opportunities (71.1%, n = 199, poor). According to 85.0% of professionals (n = 238) additional postgraduate trainings were needed, mainly university master courses (47.50%, n = 133) and private institution courses (47.86%, n = 134). Postgraduate trainings were considered very useful by 71.4% (n = 200) of responders. Conclusion: Our data suggest undergraduate programs on clinical research education failing at providing even the basic information on clinical research. Therefore, most professionals resort to specific additional postgraduate courses.

Cardiovascular disease is the leading cause of non‐malignant morbidity and mortality in childhood cancer survivors (CCSs). Anthracyclines are included in many treatment regimens for paediatric cancer, but unfortunately, these compounds are cardiotoxic. One in 10 CCSs who has received an anthracycline will develop a symptomatic cardiac event over time. Given the crucial need to mitigate anthracycline‐related cardiotoxicity (ARC), the authors critically examined published data to identify effective cardioprotective strategies. Based on their expert analysis of contemporary literature data, it was concluded that consideration should be given for routine use of dexrazoxane in children with cancer who are at risk of ARC.