Background and Importance Data concerning drugs’ dose, efficacy and safety in paediatric are very limited and this gap of knowledge induces the off-label (OL) drug use. A study showed that 60% of paediatric prescriptions were OL and the main OL-drug classes were antibacterials/antiasthmatics/analgesics. Over the last 30 years the Drug-Agency has approved laws to ensure an appropriate use of OL-medications (Law 648/96, Law 94/98, Law 326/03, Law 7/9/2017).

Aim and Objectives The aim of this work was to evaluate the paediatric OL-drug use and safety in our hospital in the last 2 years according to the Law 94/98 and the Law 326/03.

Material and Methods We analysed OL-prescriptions evaluated by the Hospital-OL-Committee (HOLC) (composed by a Hospital-Pharmacist/Pharmacologist/Clinician) from January-2021/December-2022. We calculated how many paediatric patients were involved, which OL-drug was the most prescribed and for what type of disease (if rare disease according to the national-rare-disease-database), how many patients presented an Adverse-Drug-Reaction (ADR). We considered OL all the Intravenous-Immunoglobulins (IgIv) that were not prescribed according to our regional ‘Operative-Procedure-for-the-appropriate-use-of-IgIv’.

Results The HOLC evaluated 258 OL prescriptions according to the Law 94/98 and 69 (27%) administered to 49 paediatric patients (two patients received two OL-drugs). 25 different OL-drugs were used to treat 33 conditions (20 rare diseases); seven drugs(28%) did not have the paediatric license. The most prescribed OL drug (second-level ATC) was J06-Immune-Serum-and-Immunoglobulins (20%) represented by IgIv to treat Idiopathic-Dermatomyositis/Giant-cell-Hepatitis with Autoimmune-Haemolytic-Anemia/Chronic-Polyradiculoneuritis (with or without anti-MOG antibodies)/Autoimmune-Encephalitis/Rasmussen-Syndrome/Opsoclone-Myoclone-Syndrome followed by L01-Cytostatic (17,5%) represented by bevacizumab to treat glioma and L04-immunosuppressant (17,5%) represented by adalimumab to treat Bechet-Syndrome/Systemic-Vasculitis. In the same period six patients received OL drugs according to the Law 326/03 and 4(67%) were paediatric. Three OL-drugs were used to treat two rare conditions: two patients received ivacaftor/tezacaftor/elexacaftor+ivacaftor to treat cystic-fibrosis and two fenfluramine to treat Dravet-Syndrome. Four ADRs referred to four OL therapies were reported in four paediatric patients induced by Ponatinib, IgIv, Arsenic-Trioxide, Rituximab.

Conclusion and Relevance The paediatric OL drug use in a common practice and over the last 30 years several strategies were adopted to guarantee an early and safe access to paediatric OL-medications. For example in our hospital, since 2007, all drugs included in the Hospital-Therapeutic-Formulary can be prescribed (without the HOLC’s evaluation) if they are on-label for indication but off-label for age/dosage/frequency.

Conflict of Interest No conflict of interest.