BackgroundThe transition from hospital to home care in pediatric hematology/oncology brings important benefits for families, but it can also generate anxiety regarding the management of symptoms at home. Although hospitals offer 24/7 telephone support, there is currently no standardized telephone triage tool in Italy for pediatric hematology/oncology, resulting in variability in inconsistent practices. This study aims to carry out the linguistic validation in Italian of the "Oncology/Hematology Telephone Triage Toolkit for Children and Young People V2 (2020)," a tool designed to standardize and improve the telephone triage process.MethodAfter obtaining approval from the tool's authors, the linguistic validation process was developed in 10 steps, according to international guidelines: After forward and backward translation, a preliminary version was created and evaluated through the Item and Scale Content Validity Indices to obtain an Italian version of the tool.ResultsThe working group of native-speaking professionals and a panel of nine experts from various Italian hematology and oncology institutions reviewed and approved the translation, achieving item content validity scores between 0.8 and 1 and scale content validity scores between 0.88 and 1. Some linguistic adjustments were made to adapt terms to the Italian context while maintaining the original meaning. The final Italian version preserved the original meaning and was deemed conceptually equivalent to the source tool.DiscussionA validated methodology in the translation and validation process successfully adapted the tool to the Italian context, maintaining its content and design. The final Italian version is now ready for usability testing.

IntroductionIn 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. MethodsThe 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 off-label 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. ResultsTotal 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. ConclusionThis 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 adult oncology, the practice of tracking symptoms and toxicities using patient-reported outcomes (PROs) has increased and correlates with increased survival. In contrast, symptom monitoring using PROs is not common in pediatric oncology. Only in the last couple of years attention has also been paid to the patient’s perception in pediatrics and listening to the voice of children and to making them participate in the treatment. Methods: A comprehensive literature search was conducted in MEDLINE/PubMed and PsycINFO to identify relevant articles published through December 2022. Results: From 58 non-duplicate articles, 33 met our eligibility criteria. Of these, 17 were used in clinical trials. Conclusions: The dissemination and use of these tools will therefore have surprising repercussions on the control of pain and physical symptoms of small patients as well as on physical and psychological aspects. The administration and use of the PROs ensures optimal use of the drugs currently present in clinical trials by researcher and nurse and aims at a safer and more controlled approval of new drugs.

A 12-year-old male being treated for a high-risk relapsed T-acute lymphoblastic leukemia presented progressive weakness and numbness of both legs after having received a chemotherapy regimen that included bortezomib. Diagnosis of acute Guillain-Barré syndrome-like inflammatory demyelinating polyneuropathy was made following clinical examination, cerebrospinal fluid analysis, electrodiagnostic studies, magnetic resonance imaging, and serum immunoglobulin antibodies to anti-ganglioside. Intravenous immunoglobulin treatment was started, resulting in complete clinical recovery. Although in rare cases, Guillain-Barré syndrome after bortezomib therapy has been reported; this paper suggests that GBS may occur when bortezomib is administered and high‑dose intravenous immunoglobulin lead to a resolution of the symptoms.

Over the past few years, clinical research has developed considerably. To achieve adequate quality standards in its design and management, it was necessary to set up dedicated and qualified multi-professional teams, made up not only of medical personnel. This has led to the emergence of new professional roles, such as that of the research nurse (clinical research nurse, study nurse or clinical trial nurse). While in Europe this professional role is defined, in Italy it has started to assert itself only recently, with a profile linked mainly to practical rather than managerial tasks. The role is not yet recognized at institutional level and is not registered. The goal of our research was to understand how many research centers can rely on the presence of a research nurse in their staff and what was the research nurse's contribution to achieving the satisfaction of patients enrolled in clinical studies and on the work of the research team.

Nel corso degli ultimi anni si è potuto assistere allo sviluppo, su larga scala, della ricerca clinica. Per raggiungere standard qualitativi adeguati nella progettazione e conduzione, si è resa necessaria la costituzione di team multiprofessionali dedicati e qualificati, composti non solo da personale medico. Questo ha portato alla nascita di nuove figure professionali, come quella dell’infermiere di ricerca (clinical research nurse, study nurse o clinical trial nurse). Mentre in Europa questa figura professionale è ben definita, in Italia ha iniziato ad affermarsi solo di recente, con un profilo legato prevalentemente a mansioni pratiche piuttosto che gestionali. Il ruolo non è ancora riconosciuto a livello istituzionale e non è censito. Obiettivo della nostra ricerca è stato comprendere quanti centri di ricerca possono contare sulla presenza di un infermiere di ricerca nel proprio staff e quale fosse il suo contributo al raggiungimento della soddisfazione dei pazienti arruolati in studi clinici e sull’operato del team di ricerca.