Off-label and compassionate use of targeted anticancer therapies: The experience of an Italian pediatric cancer center.

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 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. 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.

Guillain-Barré syndrome after bortezomib therapy in a child with relapsed acute lymphoblastic leukemia.

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 high­dose intravenous immunoglobulin lead to a resolution of the symptoms.

[Clinical research nurse in italian centers: a mandatory figure?] / Infermieri di ricerca in Italia: una figura indispensabile?

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.