Long-Term Ventilation in Children with Medical Complexity: A Challenging Issue.

Children with medical complexity (CMCs) represent a subgroup of children who may have congenital or acquired multisystemic disease. CMCs are frequently predisposed to respiratory problems and often require long-term mechanical ventilation (LTMV). The indications for LTMV in CMCs are increasing, but gathering evidence about indications, titration, and monitoring is currently the most difficult challenge due to the absence of validated data. The aim of this review was to examine the clinical indications and ethical considerations for the initiation, continuation, or withdrawal of LTMV among CMCs. The decision to initiate long-term ventilation should always be based on clinical and ethical considerations and should be shared with the parents.

Prevention of recurrent respiratory infections : Inter-society Consensus.

Recurrent respiratory infections (RRIs) are a common clinical condition in children, in fact about 25% of children under 1 year and 6% of children during the first 6 years of life have RRIs. In most cases, infections occur with mild clinical manifestations and the frequency of episodes tends to decrease over time with a complete resolution by 12 years of age. However, RRIs significantly reduce child and family quality of life and lead to significant medical and social costs.Despite the importance of this condition, there is currently no agreed definition of the term RRIs in the literature, especially concerning the frequency and type of infectious episodes to be considered. The aim of this consensus document is to propose an updated definition and provide recommendations with the intent of guiding the physician in the complex process of diagnosis, management and prevention of RRIs.

Inter-society consensus for the use of inhaled corticosteroids in infants, children and adolescents with airway diseases.

BACKGROUND: In 2019, a multidisciplinary panel of experts from eight Italian scientific paediatric societies developed a consensus document for the use of inhaled corticosteroids in the management and prevention of the most common paediatric airways disorders. The aim is to provide healthcare providers with a multidisciplinary document including indications useful in the clinical practice. The consensus document was intended to be addressed to paediatricians who work in the Paediatric Divisions, the Primary Care Services and the Emergency Departments, as well as to Residents or PhD students, paediatric nurses and specialists or consultants in paediatric pulmonology, allergy, infectious diseases, and ear, nose, and throat medicine. METHODS: Clinical questions identifying Population, Intervention(s), Comparison and Outcome(s) were addressed by methodologists and a general agreement on the topics and the strength of the recommendations (according to the GRADE system) was obtained following the Delphi method. The literature selection included secondary sources such as evidence-based guidelines and systematic reviews and was integrated with primary studies subsequently published. RESULTS: The expert panel provided a number of recommendations on the use of inhaled corticosteroids in preschool wheezing, bronchial asthma, allergic and non-allergic rhinitis, acute and chronic rhinosinusitis, adenoid hypertrophy, laryngitis and laryngospasm. CONCLUSIONS: We provided a multidisciplinary update on the current recommendations for the management and prevention of the most common paediatric airways disorders requiring inhaled corticosteroids, in order to share useful indications, identify gaps in knowledge and drive future research.

Relationship between quality of life and behavioural disorders in children with persistent asthma: a Multiple Indicators Multiple Causes (MIMIC) model.

Knowledge on multiple interdependences between quality of life (QoL) and behavioural problems in relation to asthma severity and control is undetermined. The aims of the study were: (i) to assess the relationship of QoL and behavioural problems with asthma severity and control (ii) to predict children's "abnormal/borderline" status with variation in QoL. For these purposes a multicenter case-control study on 47 Severe Asthma (SA) and 94 Moderate Asthma (MA) children was performed. The MIMIC approach was applied to investigate the effect of SA and non-controlled asthma (NC) on QoL and behavioural disorders. Logistic regression was used to estimate probabilities of having an "abnormal/borderline" status with variation in QoL. The MIMIC model showed that the magnitude of the effect of SA and NC was larger on QoL (ß = -0.37 and ß = -0.30, respectively) than on behavioural problems (ß = 0.27). With regards to the probability of having a borderline status, in MA a QoL of 1 returned a probability of 0.81, whereas in SA a QoL of 1 returned a probability of 0.89. In conclusion, SA children are highly affected by impaired QoL and behavioural problems. The MIMIC model allowed us to obtain a comprehensive assessment of QoL and behavioural problems with asthma severity and control.

Consensus communication strategies to improve doctor-patient relationship in paediatric severe asthma.

BACKGROUND: Asthma is a chronic inflammatory disease that is very common among youth worldwide. The burden of this illness is very high not only considering financial costs but also on emotional and social functioning. Guidelines and many researches recommend to develop a good communication between physicians and children/caregiver and their parents. Nevertheless, a previous Italian project showed some criticalities in paediatric severe asthma management. The consensus gathered together experts in paediatric asthma management, experts in narrative medicine and patient associations with the aim of identify simple recommendation to improve communication strategies. METHODS: Participants to the consensus received the results of the project and a selection of narratives two weeks before the meeting. The meeting was structured in plenary session and in three working groups discussing respectively about communication strategies with children, adolescents and parents. The task of each working group was to identify the most effective (DO) and least effective practices (DON' T) for 5 phases of the visit: welcome, comprehension of the context, emotions management, duration and end of the visit and endurance of the relationship. RESULTS: Participants agreed that good relationships translate into positive outcomes and reached consensus on communication strategies to implement in the different phase of relationships. CONCLUSIONS: The future challenges identified by the participants are the dissemination of this Consensus document and the implementation of effective communication strategies to improve the management of pediatric asthma.

Researching children's perspectives in pediatric palliative care: A systematic review and meta-summary of qualitative research.

OBJECTIVE: Qualitative research is pivotal in gaining understanding of individuals' experiences in pediatric palliative care. In the past few decades, the number of qualitative studies on pediatric palliative care has increased slightly, as has interest in qualitative research in this area. Nonetheless, a limited number of such studies have included the first-person perspective of children. The aim of this article is to understand the contribution of previous qualitative research on pediatric palliative care that included the voices of children. METHOD: A systematic review of qualitative studies and a meta-summary were conducted. MEDLINE, CINAHL, PsycINFO, PsycARTICLES, and ERIC were searched without limitations on publication date or language. Eligible articles were qualitative research articles in which the participants were children ranging in age from 3 to 18 years.ResultWe retrieved 16 qualitative research articles reporting on 12 unique studies, and we selected two mixed-method articles. The meta-summary shows eight themes: the relationship with professional caregivers, pain and its management, "living beyond pain," the relationship between pediatric patients and their families, children's view on their treatment and service provision, meanings children give to their end-of-life situation, consequences of clinical decisions, and the relationships among children in pediatric palliative care and their peers.Significance of resultsThis meta-summary presents the "state of the art" of pediatric palliative care qualitative research on children and highlights additional research areas that warrant qualitative study.

Sleep disordered breathing in patients with Prader-Willi syndrome: A multicenter study.

OBJECTIVES: Sleep disordered breathing (SDB) is common in patients with Prader-Willi syndrome (PWS) and systematic screening is recommended, especially before growth hormone treatment. The aim of the study was to describe the baseline SDB and therapeutic interventions in a large cohort of patients. STUDY DESIGN: Retrospective study. SUBJECT SELECTION: Eighty-eight patients with PWS, median [interquartile range] age of 5.1 [1.0-14.5] years old (range 0.3-44.3), who were followed in three centers (France, Italy). METHODOLOGY: Anthropometrics, polygraphy (PG), and gas exchange data were analyzed. RESULTS: Median body mass index (BMI) was 20 [16-34] kg/m(2), BMI z-score for patients aged 2-20 years old was 2.1 [1.2-2.8] SD, mixed-obstructive apnea-hypopnea index (MOAHI) 1.8 [0.6-5.0] events/hr, and central apnea index (CAI) 0.1 [0.0-0.6] events/hr. Minimum pulse oximetry (SpO2) was 88 [84-91]%, percentage of time with SpO2 <90% 0.1 [0.0-1.0]%, and oxygen desaturation index 2 [1-4]/hr. An apnea-hypopnea index (AHI) ≥ 1.5 and ≥ 5 events/hr was observed in 53% of children and 41% of adults, respectively. No correlations were observed between MOAHI and anthropometrics data (age, BMI, BMI z-score), while MOAHI significantly correlated with SpO2 indexes. Age and BMI only weakly correlated with SpO2 indexes. Growth hormone could be initiated in 48 patients. Regarding post-PG therapy, 9 patients had upper airway surgery, and noninvasive CPAP/bilevel ventilation was started in 16 patients. CONCLUSIONS: Patients with PWS exhibit a high prevalence of SDB. The lack of association between obesity and SDB leads to hypothesize that hypotonia and/or facial dysmorphic features may play a major role in the occurrence of SDB.

Polygraphic respiratory events during sleep in children treated with home continuous positive airway pressure: description and clinical consequences.

OBJECTIVE: Data are scarce on respiratory events during sleep for children treated at home with continuous positive airway pressure (CPAP). The present study aimed to characterize the respiratory events with CPAP during sleep and to analyze their clinical consequences. PATIENTS/METHODS: Consecutive polygraphies (PG) performed on stable children treated with CPAP were analyzed and scored using SomnoNIV Group definitions. For every respiratory event, the presence of a 3% oxygen desaturation and/or an autonomic arousal was systematically searched. Nocturnal gas exchange was assessed using summary data of oximetry and transcutaneous carbon dioxide pressure recordings. RESULTS: Twenty-nine consecutive polygraphies, performed on 26 children (mean age 7.8 ± 6.2 years, mean CPAP use 10.6 ± 14.4 months), were analyzed. The index of total respiratory events was low (median value 1.4/h, range 0-34). The mean number of different types of respiratory events per PG was 2 ± 1 (range 0-4), with always a predominant event. Partial or total upper airway obstruction without a decrease in ventilatory drive was the most frequent event and was the most frequently associated with an oxygen desaturation (in 30% of the events) and an autonomic arousal (in 55% of the events). Weak correlations were observed between nocturnal oximetry and PG results. CONCLUSIONS: The index of respiratory events during CPAP treatment for stable children is low. As these events may be associated with an oxygen desaturation or an autonomic arousal, and as nocturnal gas exchange cannot predict PG results, a systematic sleep study seems justified for the routine follow-up of children treated with CPAP.

Sleep architecture in infants with spinal muscular atrophy type 1.

OBJECTIVE: Few reports on sleep patterns of patients with spinal muscular atrophy type 1 (SMA1) have been published and none on sleep microstructure. The aim of this study was to analyze sleep architecture and microstructure in a group of infants with SMA1, compared with age- and sex-matched controls. METHODS: Twelve SMA1 patients (six males, mean age 5.9 months) and 10 controls (five males, mean age 4.8 months) underwent full polysomnography to evaluate their sleep architecture and microstructure by means of the cyclic alternating pattern (CAP). RESULTS: Compared with control children, SMA1 patients showed increased sleep latency and apnea/hypopnea index. CAP analysis revealed a significant increase in the percentage of A1 CAP subtypes, a reduction of that of A3 subtypes and of A2 and A3 indexes (number/h), indicating a dysfunction of the arousal system in these patients. CONCLUSION: The results indicate the presence of an abnormality of sleep microstructure in SMA1 patients, characterized by a reduction of A2 and A3 CAP subtypes. We hypothesize that SMA1 patients have reduced arousability during non-rapid eye movement sleep, which could be interpreted as additional evidence of central nervous system involvement in this disease.

Longitudinal course of lung function and respiratory muscle strength in spinal muscular atrophy type 2 and 3.

BACKGROUND: Spinal muscular atrophy (SMA) is a common genetic disorder that causes severe hypotonia and weakness, and often fatal restrictive lung disease. The aim of the study was to describe the natural history of the respiratory involvement in patients with SMA type 2 and 3 in order to assess the relevance of the clinical classification and identify the parameters associated with the earliest and most rapid decline over time. METHODS: Thirty-one patients aged 3-21 years were followed over a 10-year period. Lung function, blood gases, respiratory mechanics and muscle strength with recording of oesogastric pressures were measured during routine follow-up. RESULTS: At least two measurements were available in 16 patients (seven type 2 and nine type 3). Among all the volitional and non-volitional, invasive and non-invasive tests, forced vital capacity (FVC) and sniff nasal inspiratory pressure (SNIP) were shown to be the most informative parameters, showing lower values in SMA type 2, with however a similar rate of decline in patients with SMA type 2 and 3. CONCLUSION: Our results confirm an earlier decline in lung and respiratory muscle function in patients classified as SMA type 2 as compared with patients classified as type 3. This decline can be assessed by two simple non-invasive tests, FVC and SNIP, with the last maneuver being feasible and reliable in the youngest children, underlying its interest for the monitoring of children with SMA.

Polygraphic respiratory events during sleep with noninvasive ventilation in children: description, prevalence, and clinical consequences.

PURPOSE: The optimal monitoring during sleep with noninvasive positive pressure ventilation (NPPV) has not been validated in children. The aim of the study was to describe on polygraphic (PG) recordings the respiratory events and associated autonomic arousals (AA) and/or 3 % desaturations (DS3%) during nocturnal NPPV. METHODS: This was a prospective descriptive study performed in the pulmonology unit of a pediatric university hospital. Consecutive patients admitted for routine follow-up of long-term NPPV were enrolled. Nocturnal PG during sleep with NPPV was performed. A second PG was performed after adjustment of the ventilatory settings when a respiratory event occurred more than 50 times/h. RESULTS: The PG tracings of 39 patients (age range 1-18 years) were analyzed. Underlying diagnoses included neuromuscular disease (n = 13), obstructive sleep apnea (n = 15), and lung disease (n = 11). Unintentional leaks, patient-ventilator asynchronies, decrease in ventilatory drive, upper airway obstruction with or without reduction of ventilatory drive, and mixed events were observed in 27, 33, 10, 11, 12, and 3 % of the patients, respectively. A predominant respiratory event was observed in all patients. The mean duration spent with respiratory events was 32 ± 30 % (range 3-96 %) of total recording time. Unintentional leaks were the most frequently associated with AA, whereas patient-ventilator asynchronies were rarely associated with AA or DS3%. In eight re-evaluated patients, a decrease in the main event was observed (p = 0.005). CONCLUSION: Respiratory events during sleep with NPPV are common in children treated with long-term NPPV. Consequences of respiratory events vary according to the type of event with unintentional leaks being associated preferentially with AA.

Cardiac iron and cardiac disease in males and females with transfusion-dependent thalassemia major: a T2* magnetic resonance imaging study.

BACKGROUND: It has been repeatedly reported that female patients with thalassemia major survive longer than males and that the difference is due to a lower rate of cardiac disease in females. DESIGN AND METHODS: We compared the cardiac iron load as measured by T2* magnetic resonance imaging in 776 patients (370 males) examined at the National Research Council as part of an Italian cooperative study. We also established normal left ventricular ejection fraction values for our population. RESULTS: The prevalence of cardiac disease was higher in males than in females (105 males versus 69 females; P < 0.0001). Cardiac T2* was significantly lower in patients with heart dysfunction (P < 0.0001), but no difference was observed according to sex. Twenty males and five females had a history of cardiac arrhythmias. Their cardiac T2* was not significantly lower than that of patients without arrhythmias (24 ms versus 26 ms; P = 0.381), nor was there a difference between sexes. Liver T2* was significantly lower in males and females with heart dysfunction compared to those without. Ferritin levels were higher in patients of both sexes with heart dysfunction without significant differences between males and females. Conclusions Males and females are at the same risk of accumulating iron in their hearts, but females tolerate iron toxicity better, possibly as an effect of reduced sensitivity to chronic oxidative stress.