Gabriella Guarnieri

With a prevalence between 1.3 and 2.1 per 10 000 live male births, Duchenne Muscular Dystrophy (DMD) is the most common inherited childhood neuromuscular disease.1 Prior to overt ventilatory failur...

Background: Despite optimized pharmacological therapy, a significant subset of severe asthma patients remains poorly controlled. Lack of control is often driven by complex, non-inflammatory “treatable traits” that require a multidimensional management strategy beyond traditional airway targeting. Objective: To characterize clinical, functional, and inflammatory profile of severe asthma patients at the time of prescribing biologic therapy and identify potential candidates to ARM (Assessment-Rehabilitate-Maintenance) pulmonary rehabilitation (PR) program. Methods: This retrospective study analyzed 195 consecutive adults meeting GINA criteria for severe asthma admitted to our outpatient clinic between January 1, 2015, and December 31, 2025. ARM-program candidacy was predefined by the coexistence of: FEV1 < 80% predicted, mMRC ≥ 2, ACT < 20, and ≥2 annual exacerbations. Results: The median age of the cohort was 55 years and 60% of patients were female. A substantial disease burden was detected (median ACT: 16; annual exacerbations: 3). Females showed significantly higher maintenance oral corticosteroid (OCS) dependence (35.9% vs. 21.8%, p = 0.036) and increased risk of air trapping (RV/TLC: 42.0% vs. 38.5%, p = 0.035). 47 patients (24%) met ARM candidacy criteria, showing higher OCS use (42.6% vs. 26.4%; p = 0.035), and a four-fold higher prevalence of anxiety/depression (12.8% vs. 3.4%; p = 0.015) compared with non-candidates. Conclusions: At the time of biologic prescription, a significant proportion of severe asthma patients present a multidimensional burden, including dyspnea, frequent exacerbations, and psychological comorbidities and are potential candidates to ARM PR program. Prospective studies are needed to validate patient selection criteria and support whether pulmonary rehabilitation—may improve clinical and functional outcomes in target subgroup.

Background: Despite optimal pharmacological and biologic therapies, many patients with severe asthma (SA) remain symptomatic and functionally impaired, experiencing decreased quality of life and increased healthcare utilization. Whilst physical inactivity can exacerbate symptoms, structured exercise was found to improve functional capacity and immune response in asthmatic patients, highlighting the potential role of pulmonary rehabilitation (PR). The availability of specific, standardized PR protocols for SA patients is nevertheless limited. Objective: We propose a comprehensive, phenotype-informed PR program tailored for patients with SA. This intervention aims to enhance physical function, optimize symptom control, promote self-management, and foster long-term health behaviors. Program overview: The A-R-M (assessment-rehabilitate-maintenance) PR model is articulated as follows: multidisciplinary assessment, 8-week rehabilitation phase, end-program evaluation, longitudinal follow-up. Key components include: (1) assessment: baseline evaluations by a multidisciplinary team using validated instruments for quality of life, disease control, exercise capacity, lung function, mood, and educational needs; (2) rehabilitation (education + exercise): education: eight sessions covering topics such as asthma pathophysiology, pharmacological treatment, self-management strategies, nutrition and satisfaction assessment. Exercise prescription: an individualized, physiotherapist-supervised regimen incorporating aerobic exercise, strength training, and inspiratory muscle training; and (3) maintenance and monitoring: personalized plans to support sustained physical activity and behavior change. Expected outcomes include significant improvement in exercise capacity, symptom control and quality of life, along with reduced use of oral corticosteroid, exacerbation's numbers and unplanned specialist visits. Conclusion: The A-R-M program offers a practical, evidence-based approach to improve outcomes of SA patients by addressing symptoms, function, and self-management, ultimately reducing healthcare costs and enhancing patients' wellbeing.

An assessment of pulmonary function provides information that can contribute to establishing the severity of respiratory impairment, to predicting the onset of respiratory symptoms, and ultimately to optimizing the care of Neuromuscular Disease (NMD) patients. It is nevertheless well known that conventional Pulmonary Function Tests (PFTs) have several technical limitations and that their accuracy depends to some extent on the patient’s ability to cooperate. For this reason, it is essential to move beyond traditional pulmonary function evaluation in individuals with NMD. A relatively new technology, electrical impedance tomography (EIT) is an easy-to-use, radiation-free imaging technique that may overcome many of the limitations of conventional PFTs by producing real-time images of regional ventilation and tidal volume distribution. As it is safe and independent from patient cooperation, EIT is expected to improve the diagnosis of respiratory compromise and facilitate the implementation of timely, personalized treatments for NMD patients. There are nevertheless technical problems that need to be addressed to facilitate its diffusion in clinical practice.

Background: The impact of Long-Term Non-Invasive Ventilation (LT-NIV) on patient-relevant outcomes has yet to be clearly established in types 2 and 3 Spinal Muscular Atrophy (SMA). Objectives: The current study aimed to assess LT-NIV effect(s) on tracheostomy-free survival and the need for hospitalization. Its secondary aim was to identify patient characteristics that can be considered risk factors for an unsatisfactory response to treatment. Design: This study is a retrospective analysis of long-term outcomes in types 2 and 3 SMA patients receiving home LT-NIV. Methods:Patients and interventions: Eighteen types 2 and 3 SMA patients who were receiving home LT-NIV between 1 January 1990 and 31 March 2025 were included. Primary study endpoint: The endpoint comprised a composite of tracheostomy-free survival time and hospitalization rate. Results: Twelve patients (66.7%) had a positive response to LT-NIV (group A); the response was unsatisfactory in the remaining 6 (33.3%) (group B). Tracheostomy-free survival time was significantly reduced in the latter compared to the former [66 (range: 2–172) vs. 280 (range: 67–407) months; p = 0.010] and the hospitalization rate was significantly higher [0.35 (range: 0.06–1.44) vs. 0.07 (range: 0.00–0.18) episodes/yr; p = 0.007]. A correlation was found between an unsatisfactory response to LT-NIV and treatment initiation following an episode of acute respiratory failure (OR: 7.90; 95% CI, 0.99–123.2; p = 0.051). Conclusions: LT-NIV has a positive impact on tracheostomy-free survival and hospitalizations in types 2 and 3 SMA patients. The risk of an unsatisfactory response appears to be higher when the treatment is initiated in clinically unstable patients.

Benralizumab emerged as a promising treatment option for eosinophilic granulomatosis with polyangiitis (EGPA). This study assessed the long-term effectiveness and safety of benralizumab in patients with severe asthma and relapsing-refractory EGPA. This retrospective, single-center study evaluated patients treated with benralizumab (30 mg/8 weeks), followed for up to 36 months. Primary outcome included disease remission (defined as Birmingham Vasculitis Activity Score version 3 = 0 and prednisone dose ≤4 mg/day). Secondary endpoints were corticosteroid tapering, lung function, relapses, treatment failure and drug retention rates. The study included 33 EGPA patients (17 [51.5%] male; median age at benralizumab initiation 56 years [IQR: 47-62]). Before starting benralizumab, most patients were on corticosteroids (90.9%), prior treatments included mepolizumab (24.2%). Benralizumab showed effectiveness, with clinical remission rates increasing from 39.4% (95% CI: 22.9-57.9) at 3 months to 65.0% (95% CI: 40.8-84.6) at 36 months (p < 0.001). Corticosteroid use declined from 90.9% to 15.4%, eosinophil counts dropped from 850 (515-1367) to 0 (0-0) cells/µL, and BVASv3 decreased from 2 (2-5) to 0 (0-0), showing significant improvements (p = 0.002 and p < 0.001, respectively). Proportion of patients experiencing asthma exacerbations reduced, alongside improved lung function. Retention rates were 81.8% at 1 year, 72.6% at 2 years, and 62.4% at 3 years, with secondary failure due to uncontrolled sinonasal symptoms. Mild adverse events were observed in 21.2% of patients. These findings support the long-term effectiveness and safety of benralizumab for EGPA, highlighting its role in inducing clinical remission, reducing corticosteroid dependence, and controlling disease activity.

Background: Severe asthma remains a major problem despite pharmacological advances. Pulmonary rehabilitation (PR) is established in chronic respiratory disease but its role in severe asthma is unclear. Objectives: Summarise evidence on PR in severe and uncontrolled asthma, describe PR-modalities, and outline implementation and research priorities. Methods: Narrative review of systematic reviews and clinical studies of multidimensional PR programmes and isolated components [aerobic training, inspiratory muscle training (IMT), breathing retraining, neuromuscular electrical stimulation (NMES), telerehabilitation]. Outcomes included asthma control, HRQoL, exercise capacity and healthcare utilisation. Results: Multicomponent PR improves exercise capacity and multiple QoL domains; pooled data show substantial increases in six-minute walk distance. Combined exercise, education and self-management produced clinically meaningful improvements in asthma control and symptoms, notably patients with uncontrolled disease and functional impairment. IMT, NMES and breathing retraining improved inspiratory strength, peripheral muscle function and hyperventilation symptoms. Telerehabilitation expands access but requires attention to digital literacy and adherence. Heterogeneity, small samples and attrition limit generalisability. Conclusion: PR is a promising personalised, multidisciplinary adjunct for severe asthma. Larger phenotype-stratified trials, harmonised outcome sets and implementation research are needed to define candidate selection, optimal dose and cost-effectiveness; embedding PR within severe asthma centres may optimise outcomes and reduce healthcare use.

An accurate diagnosis of occupational allergic respiratory diseases is crucial because they are related to disability, loss of work and worsening of psychosocial and economic status of workers. Early diagnosis is therefore essential to prevent the progression of allergic respiratory diseases and to implement preventive measures. The most frequent occupational allergic respiratory diseases are asthma (OA) and rhinitis (OR), less frequently hypersensitivity pneumonia (HP) and non-asthmatic eosinophilic bronchitis (NAEB). The diagnosis is based on the objective evidence of the disease, on a clear correlation with the work activity and the identification of a specific sensitizing agent in the workplace. Reference standard methods are available, such as Specific Inhalation Challenge in the laboratory or in the workplace. A multidisciplinary approach in highly-specialized centres is always recommended for diagnostic confirmation, as it is accompanied by greater diagnostic sensitivity and specificity.

Background: Patients suffering from idiopathic pulmonary fibrosis (IPF) may experience acute exacerbation (AE-IPF), which frequently results in acute respiratory failure (ARF) requiring hospitalization. Objective: This study aims to determine if survival has improved over the last decade in patients hospitalized for ARF consequent to AE-IPF, in view of the progress recently made in pharmacological and supportive treatment strategies. Methods: This was an observational retrospective single-center study. The data of 14 patients admitted to an Intermediate Respiratory Care Unit (IRCU) between 1 January 2004 and 31 December 2013 (group A) were compared with those of 26 patients admitted between 1 January 2014 and 31 December 2023 (group B). This study’s primary endpoint was survival following IRCU admission. Results: Survival time was significantly longer in the second group of patients compared to the first one [median survival time: 134 (31–257) vs. 25.5 (20–50) days; p < 0.001]. Group B patients also had a lower IRCU mortality rate (6/26 vs. 10/14; p = 0.003) and a significantly shorter stay in the IRCU [6 (1–60) vs. 14 (1–43) days; p = 0.039]. Conclusions: Innovative pharmacologic treatments and supportive therapeutic strategies are able to prolong survival and reduce the risk of in-hospital mortality in patients with AE-IPF hospitalized for ARF.