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.

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.

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.

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.

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.

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.

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.

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.

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..

Respiratory syncytial virus bronchiolitis among refugees and asylum seekers from war-torn countries.

BACKGROUND: Bronchiolitis is a prominent illness in children with a high burden in the developing world. Our objective was to assess bronchiolitis severity among infants and toddlers of refugees and asylum seekers who fled from developing countries with high disease burden to a developed country. STUDY DESIGN: A retrospective cohort comparative-group study of children 0-24 months of age who were admitted with a diagnosis of respiratory syncytial virus (RSV) bronchiolitis to a tertiary university-affiliated medical center during 2009-2017. The refugees and asylum seekers (study group) were mostly from war-torn African countries, and the control group was comprised of children from Israel (controls), a developed country. The primary outcome was length of stay (LOS), and the secondary outcomes were nutritional support and disease characteristics. RESULTS: A total of 185 patients were included (92 refugees and 93 controls). The mean LOS was higher for the former compared to the latter (4.7 ± 3.2 vs. 3.5 ± 2 days, respectively, p = .004). More hospitalized refugees required nutritional support compared to controls (48% vs. 24%, respectively, p = .002). No differences were found in vital signs, physical findings and symptoms, laboratory results, or complications. CONCLUSIONS: Refugees and asylum seekers from developing countries presented with a longer RSV bronchiolitis disease course and required more nutritional support compared to their Israeli counterparts. This may suggest a more severe disease.

High fractional exhaled nitric oxide levels in asthma patients: Does size matter?

BACKGROUND: Fractional exhaled nitric oxide (FeNO) is a biomarker for eosinophilic inflammation used for diagnosis and monitoring of asthma. High FeNO indicates significant airway eosinophilia and steroid-responsive airway inflammation. Some children with asthma have extremely high FeNO levels, but whether these levels represent a different asthma phenotype compared with those with mildly elevated FeNO is unclear. The objective of this study is to investigate whether the extent of high FeNO levels correlates with clinical phenotype, asthma control, comorbidity, and pulmonary function test (PFT) findings in children with asthma. METHODS: Anthropometric data, daytime and nighttime symptoms, controller treatment, comorbidity, and PFT findings were retrieved from the Pediatric Pulmonology Unit database (2014-2020) and correlated with FeNO levels in pediatric asthma patients with high FeNO levels. RESULTS: Two-hundred children and adolescents with high FeNO levels (range 36-227 ppb) were included. Within this range, higher FeNO levels positively correlated with increased daytime and nighttime symptoms (p = .013 and p = .01, respectively) and poorly controlled asthma (p = .034). A FeNO level of ≥80 ppb was the cutoff for significantly more severe daytime and nighttime symptoms and very poorly controlled asthma compared with levels <80 ppb (p = .004, p = .005, and p = .036, respectively). No correlation was found between FeNO and controller treatment, comorbidity, and PFT performance. CONCLUSION: In pediatric asthma patients, high FeNO levels correlate with increased symptom severity and poor asthma control. A FeNO level of ≥80 ppb may serve as an objective indicator for severe asthma.

Real-time effects of COVID-19 pandemic lockdown on pediatric respiratory patients.

OBJECTIVE: A national lockdown was implemented in Israel to slow the viral spread of COVID-19. We assessed the real-time effects of the lockdown on disease expression and lifestyle modifications in pediatric patients with chronic respiratory disorders. METHODS: An anonymous electronic questionnaire was distributed during lockdown (March-May 2020) to caregivers of patients with chronic respiratory disorders. The primary outcome was change in disease expression and the secondary outcomes were changes in lifestyle and caregivers' emotional status. RESULTS: The clinical status of one-third of the 445 participating patients (age 0-18 years) reportedly improved, including decreased respiratory symptoms (n = 133, 33%), exacerbation frequency (n = 147, 35%), and use of reliever medications (n = 101, 27.4%). The condition of ~10% of the patients worsened. Clinical improvement was noted mostly in young patients <5 years (p = .001), asthmatic patients (p = .033), and patients with multiple underlying respiratory disorders (p = .015). Patients whose condition significantly worsened were more likely to be >5 years (p < .001), had increased screen time, decreased physical activity, and shorter sleep duration compared to their younger counterparts (p = .008, <.001, and .001, respectively). Caregivers' reports (n = 236 [58%]) of their own anxiety levels and perceptions of the patients' elevated health risk were increased, regardless of the children's actual clinical status. CONCLUSION: COVID-19 lockdown was associated with clinical improvement/stability for most of the surveyed children; however, their caregivers' anxiety level was heightened. An increased sedentary lifestyle was reported mostly in older children.

Spurious Asthma Presentation during COVID-19.

The use of face masks as a means for preventing the spread of SARS-CoV-2 is now a common practice world-wide. Three children presented to our specialty clinic with respiratory complaints during protective face mask wearing. They were diagnosed as asthma and referred to our specialist clinic for further evaluation after asthma treatments were ineffective. Full details and a video clip demonstrating the effects of wearing the mask is presented for the first patient. The detailed evaluation confirmed the diagnosis of hyperventilation. Conclusions: In the current era of the daily use of masks, pediatricians should be aware of potential anxiety and hyperventilation while the mask is being worn, causing symptoms that mimic common respiratory disorders, such as asthma.

Nusinersen for spinal muscular atrophy type 1: Real-world respiratory experience.

BACKGROUND: The emergence of new treatments for spinal muscular atrophy (SMA) is revolutionary, especially for SMA type 1 (SMA1). Data on respiratory outcomes remain sparse and rely mostly on randomized clinical trials. We report our experience of Nusinersen-treated SMA1 patients in real-world settings. METHODS: Data from SMA1 patients treated with Nusinersen were prospectively collected between 1/2017 and 1/2020. Respiratory variables included the use of assisted ventilation, the use of mechanical insufflation-exsufflation (MIE), respiratory complications, and death or treatment cessation due to respiratory reasons. RESULTS: Twenty SMA1 patients were assessed before and after 2 years of Nusinersen treatment which was initiated at a median age of 13.5 months (range, 1-184). At baseline, 16 patients were using assisted ventilation, eight noninvasive and eight invasive. Twelve patients were using permanent ventilation and four partial ventilation. After 2 years of treatment, there was no change in respiratory support among ventilated patients. All four patients who were free from respiratory support at baseline required the initiation of assisted ventilation during the study period. All 20 patients used MIE after 2 years of treatment. Two patients died from acute respiratory failure and one sustained severe brain injury. Four patients had chronic and/or recurrent atelectasis. CONCLUSION: Most of our patients were stable in their need for assisted ventilation and did not worsen as expected in SMA1, nor did they improve as might be hoped. Future studies are needed to determine if earlier treatment with Nusinersen might result in respiratory outcomes superior to those reported in this real-life study.

Author Correction: Inhaled nitric oxide therapy in acute bronchiolitis: A multicenter randomized clinical trial.

An amendment to this paper has been published and can be accessed via a link at the top of the paper.

Inhaled nitric oxide therapy in acute bronchiolitis: A multicenter randomized clinical trial.

Currently, there are no approved treatments for infants with acute bronchiolitis, the leading cause for hospitalization of infants worldwide, and thus the recommended approach is supportive. Inhaled Nitric oxide (iNO), possesses anti-viral properties, improves oxygenation, and was shown to be safe in infants with respiratory conditions. Hospitalized infants with acute bronchiolitis were therefore recruited to a prospective double-blinded, multi-center, randomized controlled pilot study. They received intermittent high dose iNO (160 ppm) plus oxygen/air for 30 min or oxygen/air alone (control), five times/day, up to 5 days. Sixty-nine infants were enrolled. No difference was observed in frequencies of subjects with at least one Adverse Event (AE) in iNO (44.1%) vs. control (55.9%); neither was Methemoglobin >7% safety threshold. No drug-related serious AEs (SAEs) were reported. Analysis of Per-Protocol population revealed that length of stay (LOS), time to SpO2 ≥92%, and time to mTal clinical score ≤5 improved by 26.7 ± 12.7 (Welch's t-test p = 0.04), 20.8 ± 8.9 (p = 0.023), and 14.6 ± 9.1 (p = 0.118) hours, respectively, in the iNO group compared to the control. Overall, high dose iNO (160ppm) was safe, well-tolerated, reduced LOS and showed rapid improvement of oxygen saturation, compared to the standard therapy. Further investigation in larger cohorts is warranted to validate these encouraging efficacy outcomes. (Trial registration: NCT03053388).

Choosing Life with Spinal Muscular Atrophy Type 1.

This article is co-authored by the mother of a patient with spinal muscular atrophy (SMA), two pediatric pulmonologists and the pediatric neurologist in the team. It describes the patient and their family's experience of living with SMA. This commentary describes the mother's experience of the diagnosis and treatment process of her daughter's SMA in an era of emerging treatments for a disease which was until recently considered incurable. SMA diagnosis and management in the context of the patient mother's experiences is discussed.