Italian Network Research Articles

Therapeutic Inertia in Dyslipidemia Management for Secondary Cardiovascular Prevention: Results from the Italian ITACARE-P Network

Background/Objectives: This study assessed the proportion of secondary cardiovascular prevention patients who achieved low-density lipoprotein (LDL) cholesterol targets as per the 2019 ESC/EAS Dyslipidemia Guidelines. We also evaluated whether lipid-lowering therapies (LLTs) were adjusted in patients not meeting targets and analyzed the likelihood of these modifications achieving recommended levels. Methods: A multicenter, cross-sectional observational study retrospectively reviewed medical records of 1909 outpatients in 9 Italian cardiac rehabilitation/secondary prevention clinics from January 2023 to June 2024. Inclusion criteria included prior atherosclerotic cardiovascular disease (ASCVD) and recent LDL-cholesterol levels. Data included demographics, ASCVD presentation, lipid profiles, and LLTs. Patients at very high risk had LDL targets of ≤55 mg/dL, or ≤40 mg/dL for recurrent events within 2 years. Clinicians’ approaches to LLT modification in patients not at target were recorded, with LLT efficacy estimated based on percentage distance from LDL-cholesterol targets. Results: Of the 1909 patients, 41.3% met the LDL-cholesterol target. Predictors of achieving targets included male gender, cardiac rehabilitation, recent acute coronary syndrome, diabetes, and triple therapy (statin + ezetimibe + PCSK9 inhibitors). Conversely, a target of ≤40 mg/dL, lack of therapy, and monotherapy were negative predictors. Among 1074 patients not at target, LLT modifications were proposed for 48.6%. Predictors of LLT modification included recent ASCVD events, cardiac rehabilitation, and greater percentage distance from the LDL target, while advanced age and an LDL target of ≤40 mg/dL were negative predictors. However, only 42.3% of modified therapies were predicted to be effective in reaching LDL targets. Conclusions: Despite 2019 ESC/EAS guidelines, a significant proportion of high-risk patients did not achieve LDL targets, and proposed LLT modifications were often insufficient. More intensive LLT regimens are needed to improve outcomes in this population.

Effect of the COVID-19 lockdown on background noise levels in Italian strong motion network

Effect of the COVID-19 lockdown on background noise levels in Italian strong motion network

STUDYING EFFICIENCY OF DATA CACHING STRATEGIES IN INFORMATION-CENTRIC NETWORKS

Today, the architecture of computer networks using IP addressing is focused on interaction between network devices with «binding» of data objects to specific sources, which leads to a decrease in bandwidth and network congestion. In information-centric networks (ICN), routing and caching of data objects on intermediate network nodes during transmission from source to recipient is performed using unique names. The purpose of the study is to analyze the performance of various caching strategies in terms of cache hit rate and request response time for data objects. Materials and methods. The following caching strategies were selected for the study: Leave Copy Everywhere, Leave Copy Down, Probabilistic Cache, random caching, Bernoulli probabilistic caching, symmetric hash routing scheme, asymmetric hash routing scheme, multicast scheme. The following metrics were calculated: cache hit rate, time delay in delivering the requested data object to the recipient, channel load when transmitting the data object, and the length of the data object’s path. The metrics were measured taking into account the following characteristics: asymmetry in the distribution of data object popularity, the total size of network caches of intermediate nodes as a share of the total number of data objects. The simulation was performed for three network topologies: GARR (Italian academic network), GEANT (European academic network), WIDE (Japanese academic network) using the Icarus 8.0 simulator. Results. The use of symmetric hash routing and multicast schemes allowed us to obtain the highest cache hit rate. The lowest cache hit rate in most tests is observed when using the asymmetric hash routing scheme, since the path of a data object to the recipient may not pass through the router responsible for caching. When using symmetric hash routing and multicast schemes, it was possible to obtain a lower delivery delay compared to other strategies. When using the Leave Copy Down caching strategy, the delivery delay with an increase in the data object popularity asymmetry indicator takes smaller values ​​than that of the hash routing schemes. The highest delay among the strategies under consideration is observed when using the asymmetric hash routing scheme. The use of the hash routing scheme leads to a relatively high value of the communication channel load parameter. The channel load indicator values ​​for such strategies as Leave Copy Everywhere (LCE), Leave Copy Down (LCD), Probabilistic Cache (ProbCache), random choice caching (RAND_CHOICE), Bernoulli caching (RAND_BERNOULLI) do not differ much from each other. The use of hash routing schemes leads to an increase in the length of the data delivery path, especially when using symmetric hash routing schemes and multicasting, since the path of the data object to the recipient may pass through the router responsible for caching, which is not always the shortest. Conclusions. Hash routing schemes with caching in some cases are a completely reasonable alternative to methods that are focused on caching along the request path and do not involve the use of hash functions to match the unique name of the data object and the router intended for caching. The practical significance of this work lies in the possibility of using the results of the study when choosing a particular caching strategy on intermediate network devices depending on the characteristics of the communication channel, in particular, the network topology and the degree of popularity of the transmitted data object.

Endoscopic Epidurolysis for the Management of Chronic Spinal Pain: A Delphi-Based Italian Experts Consensus.

Endoscopic epidurolysis (EE) is a minimally invasive procedure used to manage chronic spinal pain, particularly in cases unresponsive to traditional treatments. Despite its growing recognition, the literature lacks comprehensive guidelines on its optimal use. This study utilized a modified Delphi approach to gather expert consensus on best practices for EE in the Italian pain therapy network. The study's scientific board conducted an extensive literature review to define key investigation topics, including clinical indications, preoperative assessments, and technical aspects of EE. A semi-structured questionnaire was developed and administered to a panel of experts. A two-round Delphi process was implemented, with consensus defined as at least 70% agreement on a 7-point Likert scale (agree or strongly agree). Statements that did not reach consensus in the first round were rephrased and resubmitted in the second round. Twenty-six clinicians participated in the study, with a 100% response rate in both rounds. In the first round, consensus was achieved for 9 out of 19 statements. In the second round, 8 out of 10 rephrased statements reached the consensus threshold. Key areas of agreement included the clinical indications for EE, the importance of preoperative imaging and anesthetic assessments, and the use of specific techniques and tools for EE. However, consensus was not reached on the use of EE for disc herniation with radicular pain and the safety of interlaminar access compared to sacral hiatus access. The study highlights the need for standardized protocols in EE to ensure consistent and effective treatment of chronic spinal pain. The consensus reached by the expert panel provides a framework for best practices, which can guide clinical decision-making and improve patient outcomes. Further research is necessary to validate these findings and address areas where consensus was not achieved.

Real-world adoption of left bundle branch area pacing: Insights from the conduction-system pacing Italian network group (C-SING)

Background and objectiveLeft bundle branch area pacing (LBBAP) is increasingly used for treating bradycardia and heart failure. However, real-world data are limited. The Conduction-System Pacing Italian Network Group (C-SING) collected prospective data on LBBAP procedures in a nationwide context. MethodsObservational data from 28 Italian sites were analysed for consecutive LBBAP procedures, comparing outcomes based on operator experience levels. ResultsFrom January 2022 to December 2023, 1250 patients (median age 78, 66.2 % male) underwent LBBAP attempt. Most frequent indications were atrioventricular block (40.8 %) and heart failure (25.6 %). Successful lead implantation was achieved in 1207 procedures (96.6 %), with a median fluoroscopy time of 6.2 min. Significant QRS duration reduction was observed in patients with interventricular conduction delay (144 ms to 120 ms, p < 0.001). Compared to low-experience operators (0–10 previous cases), high-experience operators (>50 cases) had significantly better outcomes, with reduced fluoroscopy time (5.9 min versus 9.0 min, p = 0.005) and an 86 % lower risk of lead implantation failure (adjusted odds ratio 0.14, p = 0.002). Periprocedural complications occurred in 6.2 % of patients, unaffected by operator experience. Follow-up data for 794 patients over a median of 93 days showed stable LBBAP pacing parameters and a 1.3 % loss of LBBAP capture. ConclusionsLBBAP is routinely adopted for bradycardia and heart failure indication, with high success and acceptable complication rates, even when performed by less experienced operators. However, procedure outcomes improved significantly as operators gained experience with at least 50 prior cases. Electrical parameters remained stable with a low rate of LBBAP loss during short-term follow-up.

Metrological traceability for the absolute gravity Italian network

Metrological traceability for the absolute gravity Italian network

Critical Convex-Type ST Elevation Correlate With Ventricular Tachyarrhythmia in Takotsubo Cardiomyopathy.

Ventricular tachyarrhythmia (VT) occasionally occurred in patients with Takotsubo cardiomyopathy (TC). Two convex-type ST elevations were significantly related to VT in coronary artery disease. This study assessed the correlation between VT and critical ECG patterns, as well other independent predictive factors of in-hospital outcome. Fifty-five consecutive patients fulfilled the diagnostic criteria of Takotsubo Italian Network (TIN) were retrospectively enrolled. The patients were classified into two groups according to their critical ECG patterns and VT occurrence. In-hospital outcomes and influencing factors were analyzed. The incidence of VT was higher in the critical ECG group than in the Noncritical ECG group (43.8% vs. 2.6%, p < 0.001). In-hospital death was more common in the critical ECG group than in the Noncritical ECG group (25.0% vs. 5.1%, p = 0.032). The composite end-point (combined VT and in-hospital death) revealed significant differences between these two groups (50.0% vs 7.7%, p < 0.001). Multi-variate analysis proved critical ECG type as one independent risk factor of VT (odds ratio [OR] = 61.8, p = 0.009) and the composite end-point (OR = 12.4, p = 0.007). The prolong QRS width ( ≥ 105 ms) was another independent factor for predicting VT (OR = 1.06, p = 0.022) and composite end-point (OR = 1.05, p = 0.017). Critical ECG types including tombstoning ST elevation and lambda-wave ST elevation have strong impact on short-term outcomes. Additionally, conduction disturbance with prolong QRS ≥ 105 ms also has independent predicting role for poor prognosis.

Severe bronchiolitis before and after the COVID-19 pandemic: a retrospective database analysis by the Italian Network of PICU study group (TIPNet)

BackgroundThe first post-COVID-19 pandemic year demonstrated an unusual bronchiolitis epidemic in both hemispheres and has been attributed to the removal of barriers implemented during SARS-CoV-2 infection. Several countries reported an increase in respiratory syncytial virus (RSV) bronchiolitis, with more hospitalizations and a greater need for respiratory support. We aimed to evaluate the consequences of the COVID-19 pandemic on the epidemiology and management of severe bronchiolitis in pediatric intensive care units (PICUs) in Italy.MethodsMulticenter, retrospective, cohort database analysis. All children younger than 24 months admitted to 7 PICUs from October 2017 to April 2023 diagnosed with bronchiolitis were included. We compared patients from pre-COVID and post-COVID eras, excluding patients from the 2020–2021 season due to low numbers. Logistic regression models were used to assess the impact of the pre-/post-COVID period on the need for invasive ventilation.ResultsSeven hundred fifteen patients were admitted to PICU during the study period, 451 patients pre-COVID and 251 patients post-COVID. Patients in the post-COVID group were older, had more comorbidities, and had higher Pediatric Index of Mortality scores at admission but the need for respiratory support was not significantly different. There was high variability in bronchiolitis management across centers. Presenting pre-COVID was protective against the risk of mechanical ventilation, adjusted for age and disease severity at admission (OR 0.38, 95% CI 0.16–0.89), while RSV infection increased the risk of intubation (OR 2.49, 95% CI 1.1–5.63).ConclusionsPICUs have faced an unexpected peak of significantly more severe cases of bronchiolitis after the COVID-19 pandemic, which did not require increased respiratory support.

An uncommon case of postpartum venous thrombosis in a patient with hereditary angioedema. Patient from the ITACA Cohort (Italian Network for Hereditary and Acquired Angioedema)

Hereditary angioedema (HAE) is a rare genetic condition characterized by episodes of cutaneous or submucosal edema, most commonly affecting the skin, the abdomen, and the upper respiratory tract. The most common cause of HAE is either a deficiency (type 1) or dysfunction (type 2) of the C1-inhibitor, leading to the overproduction of bradykinin and activation of bradykinin B2 receptors. This increases vascular permeability and results in angioedema attacks. Anatomic, physiological, and hormonal changes during pregnancy can have an impact on the manifestations of the disease and therefore its treatment. Here, we describe the case of a 30-year-old woman who experienced a significant worsening in both the number and severity of angioedema attacks during pregnancy. The cesarean section was complicated by thrombosis of the ovarian vein and inferior vena cava.

Spatial MILP optimization framework for siting Hydrogen Refueling Stations in heavy-duty freight transport

The need for deep decarbonization of the transport sector cannot be understated, as it accounts for about the 25% of greenhouse gas emissions in Europe. Developing hydrogen-based trucks is one of the viable solutions for exploiting green hydrogen and reaching climate neutrality. This work presents an optimization framework to optimally place Hydrogen Refueling Stations (HRS) for hydrogen-based trucks under technical, policy and regulatory constraints. It relies on an EU heavy-duty road freight transport database adapted to the latest publicly available statistics to update the demand intensity. A revised Node Capacitated Flow Refueling Location Model is proposed to minimize the number of HRS to be sited on the highway network. The node capacity constraint considers standard sized HRS with a maximum daily capacity ranging from 500 (S-sized) to 4000 kg (XL-sized). The framework can be a useful evaluation tool to strategically site HRS, both for policymakers and stakeholders. To this end, the Italian highway network was evaluated as a case study, finding that at least 78 HRS nodes are required across the road network if a 10% share of hydrogen vehicles is considered, as planned in the Italian National Recovery and Resilience Plan. The median utilization factor of the refueling stations is 67.5%, ranging from 49% for the S-sized to 86% for the XL-sized, which are located mainly in northern Italian regions. To effectively reduce emissions in road freight transport, results show that at least 368 MW of additional equivalent photovoltaic capacity is needed to produce entirely green hydrogen, reducing the greenhouse gases emissions associated to the road freight transport by 6.5%.

Clinical, Histopathologic, and Genetic Features of Patients With Myofibrillar and Distal Myopathies: Experience From the Italian Network.

Clinical, Histopathologic, and Genetic Features of Patients With Myofibrillar and Distal Myopathies: Experience From the Italian Network.

Predicting the impact of public events and mobility in Smart Cities

AbstractThe ubiquitous presence of smartphones and the ever‐expanding Internet of Things are generating a treasure trove of data on human movement. We harness the power of Artificial Intelligence to extract knowledge within this data, in particular for predicting people flows and density in a Smart City. This predictive ability holds immense potential for a multitude of applications, from optimising people flow to streamlining event planning, while offering a powerful tool for pre‐emptive identification of situations that may lead to crowd disasters. In this paper, we tackle two crucial aspects of people mobility using data from public events and an Italian mobile phone network: to predict both event attendance and future crowd density in specific areas. The event details (location, time etc.) are automatically gathered and stored in a structured format. Next, we handle these problems are treated in a “supervised learning” setting, and various state‐of‐art Machine Learning techniques are tested to find the best model for each task. The obtained models will be encapsulated into a Policy Support System contributing to foster planning actions of mobility services.

The importance of an accurate numerical model for the simulations of new generation district heating systems

Abstract The aim of this work is to present a fast and accurate district heating simulation tool conceived to support the implementation of successful integrations of renewable energy sources along district heating networks. New generation district heating systems are facing new challenges related to increasing integrations of distributed renewable energy sources and new type of customers, such as prosumers. These connections introduce a great variety of low temperature highly variables energy profiles in the network that traditional modelling tools cannot properly simulate. Therefore, a specific thermo-hydraulic simulation model has been built to simulate the complex dynamics of entire network and its components, combining accuracy and rapidity. The model uses different numerical approaches to properly taking into account the thermal inertia of the components capacity, namely the water network and the users. The rapidity and reduction of computational effort is reached by the use of a Lagrangian approach based on the method of characteristics applied to the piping elements. Compared with the commonly used finite-volume and the node method, the proposed model shows accurate results at a lower computational expense and reducing numerical diffusion and artificial smoothing. In order to properly simulate the effects of the user behaviour on energy demand, flow rates and return temperatures of the network, a simplified but accurate user substation model is built with a lumped capacity approach. The model accuracy is investigated under real operating conditions with one-year hourly monitoring data of an Italian district heating network.

Score-driven exponential random graphs: A new class of time-varying parameter models for temporal networks.

Motivated by the increasing abundance of data describing real-world networks that exhibit dynamical features, we propose an extension of the exponential random graph models (ERGMs) that accommodates the time variation of its parameters. Inspired by the fast-growing literature on dynamic conditional score models, each parameter evolves according to an updating rule driven by the score of the ERGM distribution. We demonstrate the flexibility of score-driven ERGMs (SD-ERGMs) as data-generating processes and filters and show the advantages of the dynamic version over the static one. We discuss two applications to temporal networks from financial and political systems. First, we consider the prediction of future links in the Italian interbank credit network. Second, we show that the SD-ERGM allows discriminating between static or time-varying parameters when used to model the U.S. Congress co-voting network dynamics.

Clinical characteristics and outcomes of patients with takotsubo syndrome and dynamic left ventricular outflow tract obstruction

Abstract Background Left ventricular outflow tract obstruction (LVOTO) is recognized as a serious complication of Takotsubo syndrome (TTS). However, its impact on patient prognosis remains insufficiently investigated. Purpose This study aimed to assess clinical characteristics and short- and long-term outcomes of patients with TTS complicated by LVOTO. Methods Patients with TTS were enrolled from the Takotsubo Italian Network (TIN). The primary outcome was the composite of acute heart failure, cardiogenic shock, and mortality during hospitalization. Secondary outcomes included the single components of the primary outcome and the occurrence of death or TTS recurrence during follow-up. Results Overall, 881 patients were included in the study [median age 72.0 (63.0–78.0) years; 91.3% female], with LVOTO detected in 59 patients (6.7%). Low diastolic blood pressure, ST-segment elevation, and moderate-to-severe mitral regurgitation were independently associated with occurrence of LVOTO. Propensity score weighting regression analysis revealed a significant higher risk of the primary outcome (OR, 2.99; 95% CI, 2.22–4.02), acute heart failure (OR, 2.19; 95% CI, 1.59–3.02), cardiogenic shock (OR, 4.56; 95% CI, 3.07–6.78), and in-hospital mortality (OR, 2.42; 95% CI, 1.13–5.21) in patients with TTS complicated by LVOTO. No significant association between LVOTO and long-term mortality or TTS recurrence was observed during follow-up. Conclusions In this real-world evidence study, LVOTO was detected in about 7% of TTS patients and was associated with a significantly higher risk of adverse events during hospitalization, including mortality. Early detection of LVOTO can aid in prognostic assessment and guide therapeutic management in patients with TTS.

Efficacy and safety of inclisiran in real-world clinical practice. Results from the CHOLINET Registry

Abstract Background/Introduction Inclisiran has been recently introduced in clinical practice. Thus, efficacy and safety data from real-world settings are limited. Purpose Cholinet (Cholesterol inclisiran Italian network) is an Italian multicenter prospective phase 4 registry including 36 Italian centers, designed to assess efficacy, safety, adherence and persistence of inclisiran treatment, and clinician’s behavior on LLT in very high CV risk patients with atherosclerotic CV disease and/or familial hypercholesterolemia (FH). Methods From November 2022 through February 2024, the Cholinet registry enrolled patients receiving inclisiran according to standard clinical practice. Clinical and demographic characteristics, concomitant therapies, blood chemistry, were recorded at the time of first prescription and at subsequent follow-up. Results We enrolled 668 patients in treatment with inclisiran (15.1% with FH, 30.7% female, mean age 63 years), Data on follow-up at 3 months (booster time) were available for 495 of them. At the time of inclisiran first prescription median LDL-C was 102 mg/dl and reached 51 mg/dl at the time of 3 months observation (50% mean reduction, 51 mg/dl absolute median reduction). None of the 495 patients reaching 3 months follow up missed the reinjection dose administration. Of 668 patients enrolled, 553 patients (82.7%) were on LLT, of them 107 (19.3%) were on ezetimibe alone, 67 (12.1%) on statin alone and 379 (68.5%) on a statin and ezetimibe combination. Of the enrolled patients, 45 (6.7%) were previously treated with evolocumab and 34 (5%) with alirocumab. Among the patients evaluated at the 3-month follow-up, 307 patients (62%) achieved the LDL cholesterol target; of them, 450 (91%) were treated with inclisiran and statin±ezetimibe. Evaluating the distribution of the percentage reduction in LDL cholesterol values, a heterogeneity emerged that was partly explainable by the concomitant lipid-lowering therapy and partly by the previous exposure to PCSK9 inhibitors. Conclusions These preliminary data from the Cholinet Italian registry show that inclisiran effectively reduces (50%) LDL-C in real-world settings. The LDL-C target was achieved in 62% of patients; of them 91% were on lipid-lowering therapy, showing that combination therapy is necessary in most of these patients.

ALICENET – an Italian network of automated lidar ceilometers for four-dimensional aerosol monitoring: infrastructure, data processing, and applications

Abstract. ​​​​​​​Vertically resolved information on aerosol particles represents a key aspect in many atmospheric studies, including aerosol–climate interactions and aerosol impacts on air quality and human health. This information is primarily derived by lidar active remote sensing, in particular with extensive networks currently in operation worldwide. In Italy, the Institute of Atmospheric Sciences and Climate (ISAC) of the National Research Council (CNR) established the ALICENET network of automated lidar ceilometers (ALCs) in 2015. Since then, ALICENET has grown as a cooperative effort of Italian institutions dealing with atmospheric science and monitoring, and it currently includes instruments run by regional environmental protection agencies, universities, research centres, and private companies. In the current configuration, the network makes use of both single-channel ALCs and dual-channel, polarisation-sensitive-system ALCs (referred to as PLCs). The systems operate in very different environments (urban, coastal, mountainous, and volcanic areas) from northern to southern Italy, thus allowing the continuous monitoring of the aerosol vertical distribution across the country. ALICENET also contributes to the EUMETNET programme E-PROFILE, filling an Italian observational gap compared to other EU member states, which generally run extended ALC networks through national meteorological services. In this work, we present the ALICENET infrastructure and the specifically developed data processing centralised at CNR-ISAC, converting raw instrumental data into quantitative, quality-controlled information on aerosol properties ranging from attenuated backscatter to aerosol mass and vertical stratifications. This setup allows us to get insights into the 4D aerosol field over Italy with applications from near-real-time monitoring to long-term analyses, examples of which are reported in this work. Specific comparisons of the ALICENET products to independent measurements obtained with different techniques, such as particulate matter (PM) concentrations from in situ samplers and aerosol optical depth (AOD) from sun photometers, are also included here, revealing the good performances of the ALICENET algorithms. Overall, ALICENET represents a valuable resource to extend the current aerosol observational capabilities in Italy and in the Mediterranean area, and it contributes to bridging the gap between atmospheric science and its application to specific sectors, among which are air quality, solar energy, and aviation safety.

Perspective on donor-derived infections in Italy.

Expected and unexpected donor-derived infections (DDI) are a rare event in solid organ transplant (SOT) recipients but are potentially associated with significant morbidity and mortality. To assure the microbial safety of transplantation, both national guidelines and the current, regional, and local epidemiology of infectious diseases must be considered. In the present paper the strategies adopted by the Italian National Center for Transplantation (CNT) since 2003 and their evolution to guarantee the safety of organ transplantation are reported. Starting in 2012 mandatory reporting to the CNT of all adverse reactions was started. The number and type of DDI reported to the CNT are currently being analyzed. The infectious diseases second opinion has written and updated the guidelines on the safety of organs for transplantation and supported the Italian transplant network for the use of organs from donors with suspected or documented potentially transmissible infections. A transplant safety network was developed in Italy in 2003 and has been updated according to the evolving knowledge and the changing epidemiology. This is an evolving field, and a continuous update of the recommendation is needed.

Biliary drainage in patients with malignant distal biliary obstruction: results of an Italian consensus conference

BackgroundMalignant Distal Biliary Obstruction (MBDO) is a common event occurring along the natural history of both pancreatic cancer and cholangiocarcinoma. Epidemiological and biological features make MBDO one of the key elements of the clinical management of patients suffering for of pancreatic cancer or cholangiocarcinoma. The development of dedicated biliary lumen-apposing metal stents (LAMS) is changing the clinical work up of patients with MBDO. i-EUS is an Italian network of clinicians and scientists with a special interest in biliopancreatic endoscopy, EUS in particular.MethodsThe scientific methodology was chosen in line with international guidance and in a fashion similar to those applied by broader scientific associations. PICO questions were elaborated and subsequently voted by a broad panel of experts within a simplified Delphi process.Results and conclusionsThe manuscripts describes the results of a consensus conference organized by i-EUS with the aim of providing an evidence based-guidance for the appropriate use of the techniques in patients with MBDO.

Gene therapy for Duchenne Muscular Dystrophy: assessing the readiness of Italian centres of expertise.

Duchenne muscular dystrophy (DMD) is a heritable disorder that causes a rapid and progressive loss of ambulatory skills. There is no curative therapy for this pathology, that is currently managed with a combination of physiotherapy and pharmacological interventions limiting the progression of the disease (e.g. corticosteroids, cardiac medications). However, a new opportunity is represented by gene therapy, a promising treatment that, however, requires significant expertise during the whole delivery of care and a solid organisational infrastructure. An organisational strategy that could effectively support its delivery to DMD patients in Italy is the hub-and-spoke model. However, an accurate portrait of the present network of DMD centres of expertise in Italy and of their readiness in the delivery of gene therapy is paramount, to facilitate access to this experimental medicine in the future. In this context, the present study aimed to map the DMD centres of expertise in Italy and later evaluate their preparedness in terms of gene therapy delivery. For this purpose, a series of items was proposed to 30 centres in Italy, of which 20 responded. After assessing the readiness of the involved centres in terms of patient preparation, therapy infusion, close surveillance, and long-term follow-up, we proposed a suitable organizational model, namely a flexible hub-and-spoke model, for the delivery of gene therapy in the Italian DMD network and solutions to tackle the challenges emerged from the survey. Overall, the present study detected an adequate readiness of the Italian DMD centres of expertise, despite observing a significant room for improvement in digital infrastructures, culture, and training.