Objective: Growth hormone deficiency (GHD) is the most common pituitary hormone deficiency and is one of the main causes of short stature in children and adolescents. The aim of this study is to evaluate the epidemiology of pediatric GHD worldwide, since no other systematic review has been published so far. Methods: We searched PubMed, Embase, and Web of Science up to July 2023 to find epidemiological studies involving children with GHD. Two review authors independently screened articles, extracted data and performed the quality assessment. Results: We selected 9 epidemiological studies published from 1974 to 2022. The range of prevalence was 1/1107–1/8,646. A study based on a registry of GH users in the Piedmont region (Italy) reported the highest mean prevalence. In the included studies, the mean incidence ranged from 1/28,800 to 1/46,700 cases per year. One study reported a 20-year cumulative incidence of 127/100,000 for boys and 93/100,000 for girls. Studies were heterogeneous in terms of population (age and GHD etiology) and diagnostic criteria. As for the methodological quality of included studies, all but one study satisfied the majority of the checklist items. Conclusions: The included studies are mostly European, so the provided estimates cannot be considered global. International multicentre studies are needed to compare epidemiological estimates of GHD among different ethnical groups. Considering the considerable cost of human recombinant GH, the only available therapy to treat GHD, understanding accurate epidemiological estimates of GHD in each country is fundamental for resource allocation.

Fabry disease (FD) is a genetic disease included in the group of lysosomal storage disorders, caused by X-linked deficiency of the enzyme alpha-galactosidase A. The aim of this study was to evaluate different aspects related to the quality of life (QoL) of a multicentre cohort of Italian patients with FD. An observational survey was conducted to measure health-related quality of life (HR-QoL) in FD patients using the CAPI (Computer-Assisted Personal Interview) method: 106 patients (mostly women) responded to the questionnaire. Geographically, 53.7% of patients lived in northern Italy, 18.9% in central Italy and 27.4% in southern Italy or the Islands. All data were collected through a five-dimensional EuroQoL questionnaire referring to functional aspects (mobility, personal care, routine activities) and perception of physical/mental well-being (pain or discomfort, anxiety or depression). A descriptive analysis of responses was performed; FD patients were compared in terms of QoL with subjects suffering from other chronic diseases, such as Crohn’s disease, chronic hepatitis, cirrhosis and multiple sclerosis. Difficulty in normal daily activities was reported by 47.2% of FD patients. About one third of subjects also had mobility difficulties. Feelings of loneliness and isolation were reported by 33.3% of those being 60–69 years old. Anxiety was equally reported in both oldest and youngest patients (66.7%), while depression, relational problems, fear of other people’s judgement increased along with age, reaching 66.7% in the over-70-years group. Male patients were largely troubled about the risk of physical disability, particularly those aged 60 years or over. Furthermore, FD patients had a poorer QoL than people suffering from other chronic inflammatory disorders. Our study upholds that FD patients have a poor QoL, as already known, negatively impacting psychic well-being and social activities. Our survey has also found a worse QoL in FD patients compared with other severe chronic disorders.

Background: Understanding trends in pneumonia-associated hospitalizations can help to quantify the burden of disease and identify risk conditions and at-risk populations. This study evaluated characteristics of hospitalizations due to pneumonia that occurred in Italy in a 10-year period from 2010 to 2019. Methods: All hospitalizations with a principal or secondary diagnosis of pneumonia over the 10-year period were included, which were identified by hospital discharges for all-cause pneumonia and pneumococcal pneumonia in the anonymized hospital discharge database of the Italian Health Ministry. Results: A total of 2,481,213 patients were hospitalized for pneumonia between 2010 and 2019; patients aged 75–86 years accounted for 30.1% of hospitalizations. Most hospitalizations (88.1%) had an unspecified pneumonia discharge code. In-hospital death was recorded in 13.0% of cases. The cumulative cost for pneumonia hospitalizations of the 10-year period were EUR 11,303,461,591. Over the observation period, the incidence rate for hospitalized all-cause pneumonia in any ages increased from 100 per 100,000 in 2010 to over 160 cases per 100,000 per year in 2019 (p < 0.001). Overall, there was a significant increase in annual percent changes in hospitalization rates (+3.47 per year), in-hospital death (+4.6% per year), and costs (+3.95% per year) over the 10-year period. Conclusions: Our analysis suggests that hospitalizations for pneumonia are increasing over time in almost all age groups, especially in the elderly. Given the substantial burden of pneumonia in terms of mortality, healthcare resources, and economic costs, greater public health efforts should thus be made to promote vaccinations against influenza and pneumococcus, particularly in high-risk groups.

The availability of a new 20-valent pneumococcal conjugate vaccine (PCV) makes it appropriate to assess its cost-effectiveness. This was evaluated by adopting the Italian National Health Service perspective, using a cost consequences Markovian model. The expected effects of vaccination with 20-valent PCV were compared with the administration of 13-valent PCV and 15-valent PCV. Assuming a 100% vaccination of cohorts aged 65–74 years, in the (lifetime) comparison between 20-valent PCV and 13-valent PCV, the former is dominant (lower cost for a better health outcome). A reduction in disease events was estimated: −1208 deaths; −1171 cases of bacteraemia; −227 of meningitis; −9845 hospitalised all-cause nonbacteremic pneumonia cases (NBP) and −21,058 non-hospitalised. Overall, in the Italian population, a total gain of 6581.6 life years and of 4734.0 QALY was estimated. On the cost side, against an increase in vaccinations costs (EUR +40.568 million), other direct health costs are reduced by EUR 48.032 million, with a net saving of EUR +7.464 million. The comparison between 20-valent PCV and 15-valent PCV results in an Incremental Cost-Effectiveness Ratio (ICER) of EUR 66 per life year gained and EUR 91 per QALY gained. The sensitivity analyses confirm the robustness of the results. We can conclude that the switch to 20-valent PCV is a sustainable and efficient investment.

Background: Respiratory syncytial virus (RSV) affects 60–80% of children below 1 year and it’s the first cause of acute bronchiolitis. The aim of this study was to assess the trend and characteristics of hospitalizations for RSV infections in Italy. Methods: This is a retrospective study based on the Italian Hospital Discharge Record (HDR) database. We analysed HDRs from June 2015 to May 2019, considering two groups of infants: Group 1 had a confirmed diagnosis of RSV; Group 2 had a diagnosis of acute bronchiolitis not RSV-coded. Results: There were 67,746 overall hospitalizations (40.1% Group 1, and 59.9% Group 2). Hospitalization rate increased for Group 1 from 125 to 178 per 10,000 infants (+ 42.4%), and for Group 2 from 210 to 234 per 10,000 (+ 11.4%). The mean hospitalization length was 6.3 days in Group 1, longer than Group 2 (+ 1.0 day). A further analysis revealed that infants with heart disease or born premature had longer mean hospital stay compared to infants without risk factors (10.7 days versus 6.1 days, p < 0.0001; 34.0 days versus 6.1 days, p < 0.0001, respectively). Group 1 required more critical care (oxygen therapy and/or mechanical ventilation) than Group 2. We found that, in proportion to hospital admissions in pediatric and general hospitals, RSV was more frequently diagnosed in the first ones. The mean hospitalization cost increased for Group 1 (from € 2,483 to € 2,617) and Group 2 (from € 2,007 to € 2,180). Conclusions: Our results confirmed that RSV pulmonary disease in infants is seasonal and often requires hospitalization. Our study suggested that RSV is responsible for an increasing hospitalization rate and related costs during the study period.