Fractional exhaled Nitric Oxide (FeNO) level as a predictor of COVID-19 disease severity.

OBJECTIVE: To assess the feasibility of Fractional exhaled Nitric Oxide (FeNO) as a simple, non-invasive, cost-effective and portable biomarker and decision support tool for risk stratification of COVID-19 patients. METHODS: We conducted a single-center prospective cohort study of COVID-19 patients whose FeNO levels were measured upon ward admission by the Vivatmo-me handheld device. Demographics, COVID-19 symptoms, and relevant hospitalization details were retrieved from the hospital databases. The patients were divided into those discharged to recover at home and those who died during hospitalization or required admission to an intensive care unit, internal medicine ward, or dedicated facility (severe outcomes group). RESULTS: Fifty-six patients were enrolled. The only significant demographic difference between the severe outcomes patients (n = 14) and the home discharge patients (n = 42) was age (64.21 ± 13.97 vs. 53.98 ± 15.57 years, respectively, P = .04). The admission FeNO measurement was significantly lower in the former group compared with the latter group (15.86 ± 14.74 vs. 25.77 ± 13.79, parts per billion [PPB], respectively, P = .008). Time to severe outcome among patients with FeNO measurements ≤11.8 PPB was significantly shorter compared with patients whose FeNO measured >11.8 PPB (19.25 ± 2.96 vs. 24.41 ± 1.09 days, respectively, 95% confidence interval [CI] 1.06 to 4.25). An admission FeNO ≤11.8 PPB was a significant risk factor for severe outcomes (odds ratio = 12.8, 95% CI: 2.78 to 58.88, P = .001), with a receiver operating characteristics curve of 0.752. CONCLUSIONS: FeNO measurements by the Vivatmo-me handheld device can serve as a biomarker and COVID-19 support tool for medical teams. These easy-to-use, portable, and noninvasive devices may serve as valuable ED bedside tools during a pandemic.

Weaning Strategy of Diuretics in Outpatient Preterm Infants with Bronchopulmonary Dysplasia: A National Survey.

OBJECTIVE: Long-term diuretic treatment in patients with bronchopulmonary dysplasia (BPD) is common despite lack of data that support its use. We aimed to characterize the commonly used diuretics weaning strategies for outpatient clinically stable preterm infants with BPD. STUDY DESIGN: We conducted a cross-sectional web-based survey among all pediatric pulmonologists and neonatologists in Israel. Questionnaire included data regarding practitioners' different diuretics-weaning practice in this population. RESULTS: The response rate for pulmonologists and neonatologists were 35/50 (70%) and 36/120 (30%), respectively. When both oxygen and diuretics are used, 59% wean oxygen first and 32% wean diuretics first. If patients are solely on diuretics, 27% discontinue instantly, 34% decrease the dosage gradually, and 34% outgrow the discharge dosage. Significantly more pulmonologists decrease the dosage gradually, while more neonatologists discontinue at once (p < 0.001). Most participants (94%) reported being unsatisfied with the existing data and guidelines regarding these issues. CONCLUSION: Our results showed a wide range of practice patterns in the weaning strategy of diuretics in outpatient preterm infants with BPD. Pulmonologists and neonatologists differ significantly in their weaning strategy. A prospective larger controlled study to explore the outcome of gradual tapering versus discontinuation without weaning is warranted. KEY POINTS: · Diuretic treatment in patients with BPD is common despite lack of data that support its use.. · We demonstrated a wide range of practice patterns in the weaning strategy of diuretics in outpatients' BPDs.. · Pulmonologists and neonatologists differ significantly in their weaning strategy.. · Most participants are unsatisfied with the existing data and guidelines regarding these issues..

Systematic Analysis of CCNO Variants in a Defined Population: Implications for Clinical Phenotype and Differential Diagnosis.

Reduced generation of multiple motile cilia (RGMC) is a novel chronic destructive airway disease within the group of mucociliary clearance disorders with only few cases reported. Mutations in two genes, CCNO and MCIDAS, have been identified as a cause of this disease, both leading to a greatly reduced number of cilia and causing impaired mucociliary clearance. This study was designed to identify the prevalence of CCNO mutations in Israel and further delineate the clinical characteristics of RGMC. We analyzed 170 families with mucociliary clearance disorders originating from Israel for mutations in CCNO and identified two novel mutations (c.165delC, p.Gly56Alafs*38; c.638T>C, p.Leu213Pro) and two known mutations in 15 individuals from 10 families (6% prevalence). Pathogenicity of the missense mutation (c.638T>C, p.Leu213Pro) was demonstrated by functional analyses in Xenopus. Combining these 15 patients with the previously reported CCNO case reports revealed rapid deterioration in lung function, an increased prevalence of hydrocephalus (10%) as well as increased female infertility (22%). Consistent with these findings, we demonstrate that CCNO expression is present in murine ependyma and fallopian tubes. CCNO is mutated more frequently than expected from the rare previous clinical case reports, leads to severe clinical manifestations, and should therefore be considered an important differential diagnosis of mucociliary clearance disorders.

Cough ability measurements and recurrent respiratory symptoms in individuals with Ataxia Telangiectasia.

OBJECTIVES: Ataxia-Telangiectasia (A-T) individuals often present with respiratory muscle weakness, causing recurrent respiratory system infections, asthma-like symptoms, and chronic cough life-threatening events. The cough flow volume maneuver may reveal powerless airflow needed for efficient cough. The study aims to explore cough ability in relation to the flow/volume maneuver. METHODS: Data collected retrospectively from clinical charts of 35 A-T patients (age 12.7 ± 4.9 years) included forced expiratory and cough flow/volume maneuvers performed on the same day. Analysis compared among the maneuvers matching indices, numbers of cough-spikes, flow rate decay, and the reference data of similar ages. Adjusted to age, BMI, and number of hospitalizations prior to the tests, values were correlated with the cough indices. RESULTS: Cough peak-flow (C-PF) was propagated within 90 ± 20 ms compared with peak expiratory flow (PEF > 200 ms). C-PF measured values were higher than expiratory peak-flow measured values (3.27 ± 1.53 L/s versus 3.02 ± 1.52 L/s, respectively, but C-PF (%predicted) values were significantly lower than expiratory peak-flow (%predicted) (46 ± 15 versus 68 ± 20 %predicted, respectively, p < 0.002). The number of spikes/maneuver was low when compared with reference (2.0 ± 0.8 versus 6-12 spikes) and cough vital-capacity was lower than expiratory vital capacity (0.95 ± 0.43 versus 1.03 ± 0.47; p < 0.01). Inefficient C-PF was more prevalent in patients suffering from recurrent respiratory illness. The length of wheelchair confinement duration mostly influenced the C-VC level. CONCLUSIONS: The cough flow-volume curve can be applied as a method to follow cough ability in patients with A-T who showed a significantly reduced cough capacity. Further studies are needed to establish if the findings may aid decisions regarding cough assistance.

Nocardia Colonization: A Risk Factor for Lung Deterioration in Cystic Fibrosis Patients?

BACKGROUND: Cystic fibrosis (CF) patients are predisposed to infection and colonization with different microbes. Some cause deterioration of lung functions, while others are colonizers without clear pathogenic effects. Our aim was to understand the effects of Nocardia species in sputum cultures on the course of lung disease in CF patients. MATERIAL AND METHODS: A retrospective study analyzing the impact of positive Nocardia spp. in sputum of 19 CF patients over a period of 10 years, comparing them with similar status patients without Nocardia growth. Pulmonary function tests (PFTs) are used as indicators of lung disease severity and decline rate in functions per year is calculated. RESULTS: No significant difference in PFTs of CF patients with positive Nocardia in sputum was found in different sub-groups according to number of episodes of growth, background variables, or treatment plans. The yearly decline in PFTs was similar to that recognized in CF patients. The control group patients showed similar background data. However, a small difference was found in the rate of decline of their PFTs, which implies a possibly slower rate of progression of lung disease. CONCLUSIONS: The prognosis of lung disease in CF patients colonized with Nocardia does not seem to differ based on the persistence of growth on cultures, different treatment plans or risk factors. Apparently, Nocardia does not cause a deterioration of lung functions with time. However, it may show a trend to faster decline in PFTs compared to similar status CF patients without isolation of this microorganism in their sputum.

IV Penicillin G is as effective as IV cefuroxime in treating community-acquired pneumonia in children.

Overuse of broad-spectrum antimicrobials has resulted in bacterial resistance and increasing use of relatively expensive antibiotics for community-acquired pneumonia (CAP). We hypothesized that CAP requiring parenteral medication is still curable with narrow-spectrum and inexpensive penicillin G. A prospective, randomized study was performed on 58 children aged 3 months to 15 years with CAP. Children were randomly assigned to receive low-dose penicillin G, high penicillin G, or cefuroxime intravenously for 4-7 days. The course of illness was monitored clinically and with predetermined laboratory and radiological indices for 30 days. The children recovered at the same rate with no significant differences in time to defervescence or duration of hospitalization. Observed differences in leukocyte counts and C-reactive protein at discharge were of questionable clinical significance. Penicillin G is as effective and safe as cefuroxime for CAP in otherwise healthy children, even in moderate doses.

Non-ionizing measurement and quantification of bell-shaped chests in spinal muscular atrophy: a pilot study.

Background: Spinal Muscular Atrophy (SMA) is manifested by deformation of the chest wall, including a bell-shaped chest. We determined the ability of a novel non-ionizing, non-volitional method to measure and quantify bell-shaped chests in SMA. Methods: A 3D depth camera and a chest x-ray (CXR) were used to capture chest images in 14 SMA patients and 28 controls. Both methods measure the distance between two points, but measurements performed by 3D analysis allow for the consideration of the curve of a surface (geodesic measurements), whereas the CXR allows solely for the determination of the shortest path between two points, with no regard for the surface (Euclidean measurements). The ratio of the upper to lower chest distances was quantified to distinguish chest shape in imaging by both the 3D depth camera and the CXR, and the ratios were compared between healthy and SMA patients. Results: The mean 3D Euclidean ratio of distances measured by 3D imaging was 1.00 in the control group and 0.92 in the SMA group (p = 0.01), the latter indicative of a bell-shaped chest. This result repeated itself in the ratio of geodesic measurements (0.99 vs. 0.89, respectively, p = 0.03). Conclusion: The herein-described novel, noninvasive 3D method for measuring the upper and lower chest distances was shown to distinguish the bell-shaped chest configuration in patients with SMA from the chests of controls. This method bears several advantages over CXR and may be readily applicable in clinical settings that manage children with SMA.

Progressive flow-to-volume dysanapsis in cystic fibrosis: a predictor for lung transplantation?

RATIONALE: Airways obstruction and lung volume restriction, major features of lung disease in cystic fibrosis (CF), may regress independently, causing dysanapsis between these parameters. OBJECTIVES: To explore the significance of dysanapsis (FEF(25-75)/FVC) ratio in CF. METHODS: Yearly best spirometry data, collected during 8.6 ± 1 year per patient, was determined from 93 patients with CF. Three groups were formed according to initial FEV(1). Group-N (n = 35; control, FEV(1) above 80%predicted); Group-B (n = 38; FEV(1) below 80% predicted); and Group-LT (n = 20; data collected before lung transplantation). The yearly decline in spirometry indices was defined in relation to the preceding year. Decline exceeding -2 z scores from Group-N in each index was considered "rapid decline." MEASUREMENTS AND MAIN RESULTS: Group-N's yearly decline of FEV(1), FEF(25-75), and FEF(25-75)/FVC were similar and reached -1.88 ± 2.93%, -1.41 ± 3.37%, and -1.81 ± 4.48%, respectively. Rapid decline was equal to -6.5%, -10.8%, and -8.1%, respectively. Group-B's indices declined faster than that of Group-N, but did not exceed 1 z score. Group-LT showed a rapid decline solely in FEF(25-75)/FVC (mean z score = -6.4 ± 2.5; P < 0.0001), which sprouted abruptly from the regular course of regression 4 ± 1.3 years before transplantation. The rapid decline in FEF(25-75)/FVC was found in 19 of 20 patients from Group-LT and five patients from other groups (now waiting for transplantation). The phenomenon did not correlate with initial FEV(1) (%predicted) or age. Having airway hyperreactivity increased the risk of rapid decline in FEF(25-75)/FVC. CONCLUSIONS: Roughly 4 years before lung transplantation and independent of FEV(1) (%predicted), FEV(1) decline rate, or age, an abrupt rapid dysanapsis occurs. Findings imply insufficient ventilation/lung volume unit and insinuate a powerful marker for estimating lung translation time in CF.

Inhaled Nitric Oxide for the Treatment of Acute Bronchiolitis: A Multicenter Randomized Controlled Clinical Trial to Evaluate Dose Response.

Rationale: Inhaled nitric oxide (iNO) has potential antiinflammatory, antimicrobial, and antiviral properties for patients with lower respiratory tract infections. Objectives: We compared the safety and efficacy of iNO administered in two concentrations in addition to standard supportive treatment (SST) compared with SST alone with the aim of improving clinical outcomes of infants with bronchiolitis. Methods: In this prospective, multicenter, double-blind, randomized controlled study, 89 infants hospitalized with moderate to severe bronchiolitis were randomly assigned to three treatment groups: 150 ppm NO plus SST (group 1), 85 ppm NO plus SST (group 2), and the control treatment (O2/air plus SST) (group 3). Treatment was given for 40 minutes, four times each day, for up to 5 days. The primary endpoint was time to reach "fit for discharge." This was a composite endpoint composed of both reaching a sustained oxygen saturation ≥92% on room air and reaching a clinical score ⩽5. Secondary endpoints included time to reach sustained oxygen saturation ≥92% on room air, time to clinical score ⩽5, and time to hospital discharge. Safety was assessed by the number of treatment-related adverse events (AEs) or serious AEs. Time-to-event efficacy outcomes were analyzed using a Cox proportional hazards regression model. Hazard ratios (HR) describe how many times more likely an individual is to experience an event, if such an individual receives NO rather than the control treatment during the observational period. Results: Group 1 demonstrated significant efficacy for time to reach fit to discharge compared with groups 2 (HR, 2.11; P = 0.041) and 3 (HR, 2.32; P = 0.049). Group 1 also demonstrated significant efficacy for time to hospital discharge compared with groups 2 (HR, 2.01; P = 0.046) and 3 (HR, 2.28; P = 0.043). No significant differences were observed between groups 2 and 3 for either endpoint. There were no differences between treatment groups in time to reach a clinical score ⩽5. The iNO therapy was well tolerated, with no treatment-related serious AEs. Conclusions: Treatment with high-dose intermittent iNO at 150 ppm showed reduced time to clinical improvement compared with 85 ppm or control treatment of hospitalized infants with acute bronchiolitis. The 150-ppm iNO dose is well tolerated, with significant benefit compared with both standard therapy and 85 ppm iNO, improving respiratory outcomes and reducing length of stay. Clinical trial registered with www.clinicaltrials.gov (NCT04060979).

High flow nasal cannula therapy in the pediatric home setting.

BACKGROUND: High-flow nasal cannula (HFNC) therapy may be better tolerated than traditional noninvasive ventilation (NIV) and is rapidly gaining acceptance in pediatric acute care. In Israel, HFNC is approved for domestic use. We aim to describe its indications, efficacy, parental satisfaction, and safety. METHODS: Retrospective study of children treated with home HFNC therapy in three pediatric centers. Data included demographic parameters, indication of use, weight and days of hospitalization before and after initiation. Safety, tolerability, and parental satisfaction were assessed via standardized telephone questionnaire. RESULTS: Median (interquartile range [IQR]) age of initiating home HFNC in 75 children was 8.3 (2.2, 29.6) months. Indications were obstructive sleep apnea (33; 44%), airway malacia (19; 25%), chronic lung disease (15; 20%), neuromuscular disease (4; 5%), and postextubation support (4; 5%). Weight standard deviation score rose from -2.3 pre-HFNC to -1.7 at 6.7 months post-HFNC initiation, p < 0.001. Hospital admission days during the 2 months pre- versus post-HFNC initiation were 22 (5.5, 60) and 5 (0, 14.7) respectively, p < 0.008. Median (IQR) parental satisfaction score was 5/5 (4, 5). Fifty of 60 (83%) respondents would recommend home HFNC to other families in a similar situation. There were no serious adverse events. CONCLUSION: In our population, domestic HFNC appeared safe and well tolerated for a variety of indications. Its introduction was associated with improved weight gain, fewer hospitalization days and high parental satisfaction. Further work is required to characterize groups of children most likely to benefit from HFNC, as opposed to traditional modes of NIV.

Translation of the quality of life questionnaire for primary ciliary dyskinesia (QOL-PCD) into Hebrew: The Israeli experience.

BACKGROUND: Patients with primary ciliary dyskinesia (PCD) have chronic morbidities affecting their quality of life (QOL). Health-related QOL (HRQOL) has been recognized as an important tool for assessing the burden of PCD on patients and their families. A PCD-specific HRQOL questionnaire (QOL-PCD) was developed and validated for the English-speaking populations. Still, it has not yet been translated into Hebrew or adapted for Israeli PCD patients. METHODS: This describes our translation of the original English version of QOL-PCD into a Hebrew version. The process involved forward translation by independent translators, construction of a consensus version, back-translation into the original English version, analysis by the expert committee, and a pretest. The pretest was administered to 20 participants with PCD (the patients and the parents of minor patients) to assess the feasibility and practicality of the tool. Scale and item indices (scale-level content validity index [S-CVI] and item-level content validity index [I-CVI]) were calculated for content validity. RESULTS: All study participants understood the final Hebrew version that the expert committee had approved with no difficulty. The instrument had satisfactory (>0.80) content validity, with S-CVI indexes of 0.99 for the adult version, 0.86 for the parental version, 0.95 for the adolescent version, and 1.00 for the child version. CONCLUSION: The English version of a QOL-PCD was translated into Hebrew and adapted in Israel. This translation is a valid instrument to assess HRQOL in Hebrew-speaking patients with PCD and their family members. It may be helpful for PCD patient management and research in Israel.

A case of an unfortunate tooth fairy visit to a ventilator-dependent child.

An 8-year-old boy recently sustained a cerebellar arteriovenous malformation rupture, and subsequently suffered from severe neurological injury and became ventilator-dependent through a tracheostomy. During a routine clinic visit, the parents reported that a loose baby tooth had fallen out and disappeared 7 days earlier. The physical examination was unremarkable, but a chest X-ray demonstrated a foreign body in the left lung and secondary atelectasis. A rigid bronchoscopy extracted what turned out to be a tooth from the left lower lobe bronchus, with no associated sequelae. Aspiration of a tooth is rare, and it is mostly seen in children and elderly patients following trauma, endotracheal intubation, and dental procedures. Only a few previous studies emphasized the increased risk of foreign body aspiration among neurological impaired children. This unique report describes a child in his physiological exfoliation period, which is characterized by the spontaneous shedding of 20 teeth over the course of several years. In severely neurologically impaired children, this period carries a risk of aspiration of teeth and secondary pulmonary damage. Therefore, treating physicians and caregivers must be aware of this risk, and routine dental check-ups are advised in a neurological impaired child during this period.

Lockdown Fatigue in Pediatric Respiratory Patients: Lessons from the First COVID-19 Year.

Lockdown policies have been implemented worldwide to limit the spread of COVID-19. "Lockdown fatigue" is a state of exhaustion related to the accumulating effects of repeated lockdowns. The aim of the current study was to examine the effects of repeated lockdowns on children with respiratory disorders. Data on children aged 0−18 years old with respiratory disorders were collected by an electronic survey during the third lockdown in Israel. The retrieved information included demographics and baseline medical status, respiratory clinical status during the third lockdown compared to pre-lockdown periods, lockdown adherence, lifestyle modifications and caregiver emotional status. The results were compared to those of a similar questionnaire distributed during the first lockdown. A total of 234 patients (62% males, 37% females, mean age 6.8 years (confidence interval 6.1−7.5)) were enrolled. Respiratory symptoms and exacerbation frequency were reduced in 76 (35.5%) and 58 (27.4%) patients, respectively, compared to the pre-lockdown period and similar to the first lockdown (p = 0.840 and p = 0.063, respectively). However, compared to the first lockdown, the third lockdown was associated with greater use of reliever medications (p = 0.006), less use of inhaled corticosteroids as routine treatment (p = 0.027), and more pediatric emergency room visits and hospitalizations (p = 0.001 and p < 0.001, respectively). The results also showed an increase in sedentary lifestyle (e.g., reduced physical activity (p = 0.025), less healthy eating habits (p = 0.001)) and reduced adherence to lockdown policies (p < 0.001). These data show that the continuing stability of clinical improvement during lockdown was accompanied by deleterious sequelae that potentially indicate "lockdown fatigue" among children with respiratory disorders.

Asthma risk after a pediatric intensive care unit admission for respiratory syncytial virus bronchiolitis.

BACKGROUND: Admission to a pediatric intensive care unit (PICU) has been associated with respiratory consequences in children with asthma and carries major implications for management control. Whereas respiratory syncytial virus (RSV) bronchiolitis has been associated with increasing intensity of wheezing, the relationship between RSV-bronchiolitis PICU admission and future asthma is unclear. This retrospective case-control study evaluated whether hospitalization in the PICU due to RSV bronchiolitis is more likely to be associated with future asthma in early life compared with hospitalization in a general pediatric ward. METHODS: Children hospitalized due to RSV bronchiolitis between 2007 and 2019 in the PICU (study group) were compared to those hospitalized in a general pediatric ward (controls). Asthma prevalence was assessed by a follow-up questionnaire based on The International Study of Asthma and Allergies in Childhood questionnaire. RESULTS: Sixty-three PICU patients and 66 controls were included. The PICU patients presented with more severe disease during RSV hospitalization. At follow-up, significantly more PICU patients aged 3-6 years had physician-diagnosed asthma, respiratory symptoms during the previous 12 months, and underwent respiratory treatment since hospital discharge compared to controls (14 [60.9%] vs. 4 [18.2%] patients; 15 [65.2%] vs. 6 [27.3%]; and 16 [69.6%] vs. 8 [36.4%]; respectively). These differences were no longer observed after 6 years of age. CONCLUSIONS: Children admitted to the PICU for RSV bronchiolitis are at higher risk for asthma in subsequent pre-school years and will require close respiratory follow-up than those admitted to general pediatric wards. Admission venue should be queried when asthma is suspected.

Unforeseen changes in seasonality of pediatric respiratory illnesses during the first COVID-19 pandemic year.

OBJECTIVES: To investigate whether the three nationwide coronavirus disease 2019 (COVID-19) lockdowns imposed in Israel during the full first pandemic year altered the traditional seasonality of pediatric respiratory healthcare utilization. METHODS: Month by month pediatric emergency department (ED) visits and hospitalizations for respiratory diagnoses during the first full COVID-19 year were compared to those recorded for the six consecutive years preceding the pandemic. Data were collected from the patients' electronic files by utilizing a data extraction platform (MDClone© ). RESULTS: A significant decline of 40% in respiratory ED visits and 54%-73% in respiratory hospitalizations during the first COVID-19 year compared with the pre-COVID-19 years were observed (p < 0.001 and p < 0.001, respectively). The rate of respiratory ED visits out of the total monthly visits, mostly for asthma, peaked during June 2020, compared with proceeding years (109 [5.9%] versus 88 [3.9%] visits; p < 0.001). This peak occurred 2 weeks after the lifting of the first lockdown, resembling the "back-to-school asthma" phenomenon of September. CONCLUSIONS: This study demonstrates important changes in the seasonality of pediatric respiratory illnesses during the first COVID-19 year, including a new "back-from-lockdown" asthma peak. These dramatic changes along with the recent resurgence of respiratory diseases may indicate the beginnings of altered seasonality in pediatric pulmonary pathologies as collateral damage of the pandemic.

Nonrespiratory complications of nusinersen-treated spinal muscular atrophy type 1 patients.

BACKGROUND: Emergence of new treatments for spinal muscular atrophy type 1 (SMA1) has led to dramatic improvements in respiratory failure and survival. However, these "treated" patients sustain major problems in other organ systems, which may directly or indirectly affect their respiratory function. We observed three main nonrespiratory manifestations in these patients comprised of facial deformities, feeding problems, and spinal deformities. OBJECTIVE: To investigate these three main sequelae in nusinersen-treated SMA1 patients. METHODS: Data on nusinersen-treated SMA1 patients were prospectively collected throughout a 3-year period, with special focus upon nonrespiratory features of the disease. RESULTS: Twenty nusinersen-treated SMA1 patients were included (eight males, median age 13.5 months, interquartile range: 4-56.2 months), among whom 17 survived after 3 years of follow-up. At follow-up, 15 (88%) patients were diagnosed with facial weakness, hypoplasia, or deformity. All but one patient (94%) were fed invasively by percutaneous endoscopic gastrostomy or nasogastric tube feeding. Four patients (25%) had maintained oral feeding in parallel to gastrostomy feeding and had clinical and radiologic evidence of aspirations. Fifteen (88%) patients were diagnosed with scoliosis, of whom seven had undergone or were scheduled to undergo corrective surgery. CONCLUSIONS: Nusinersen-treated SMA1 patients may sustain facial deformities, feeding problems, and severe scoliosis, all of which affect their respiratory system. Strict surveillance of these complications is essential to avoid further respiratory morbidity.

Hospital versus home treatment of respiratory exacerbations in cystic fibrosis.

BACKGROUND: Treatment of respiratory exacerbations in Cystic Fibrosis (CF) is important in slowing disease progression. The treatment may be given either at home or at the hospital. The aim of our study was to compare both treatment settings. MATERIAL/METHODS: We retrospectively analyzed data of 139 treatments in 54 CF patients (age range 12-47 y) treated for respiratory exacerbations at the hospital (n = 84) and/or at home (n = 55). Primary outcomes were improvement in pulmonary function tests (PFTs), weight gain and duration of treatment in relation to treatment setting. Secondary outcomes were these same parameters, but in relation to different clinical preconditions and CF-related complications. RESULTS: Mean improvement in FEV1 (% predicted) was similar between the hospital and home treatments (14.3 ± 34.4% vs. 14.3 ± 20.2%, respectively; NS), yet treatment duration was significantly shorter at the hospital (9.7 ± 6.7 vs. 16.3 ± 9.1 days, respectively; P < 0.02), especially for patients colonized with Pseudomonas aeruginosa (11.1 ± 5.5 vs. 18.0 ± 11.0 days, respectively; p<0.01). At the hospital, a subgroup of patients with CF-related complications improved their FEV1 significantly more than those at home (13.1 ± 19.4% vs. 1.9 ± 14.9%, respectively; P < 0.02), particularly patients with CF-related diabetes mellitus (CFRDM) (11.4 ± 18.7% vs. 1.7 ± 14.6%, respectively; P < 0.05). Patients tended to gain more weight at the hospital compared to home treatment (1.36 ± 4.6 kg and 0.49 ± 3.6 kg respectively; P = 0.06). CONCLUSIONS: Hospital treatment for acute respiratory exacerbations in CF may be superior to home treatment, as indicated by a shorter duration of hospitalization, better improvement in FEV1 in patients with CF-related complications, CFRDM in particular and a trend toward better weight gain.