Peak Cough Flow Research Articles

The Functional Capacity of the Upper Airway in Older Adults with Chronic Stroke

(1) Background: Older adults with chronic stroke may experience compromised upper airway functions due to stroke-related changes and aging. This study aimed to evaluate the functional capacity of the upper airway in older adults with chronic stroke. (2) Methods: A total of 44 patients (22 in each group) were included in the study. The respiratory assessment involved measuring forced vital capacity, forced expiratory volume in one second, maximum voluntary ventilation, and peak cough flow. The voice assessment recorded intensity, frequency, shimmer, and the harmonics-to-noise ratio during a monologue task. Additionally, the maximum phonation time of /a/ and /s/ was recorded. The swallowing assessment included the Eating Assessment Tool—10 and the Swallowing Quality of Life questionnaire. (3) Results: Significant differences were found in the experimental group compared to the control group in maximum voluntary ventilation (44.59 ± 15.61 vs. 58.50 ± 28.08, p = 0.049) and peak cough flow (173.64 ± 101.09 vs. 291.59 ± 176.58, p = 0.009). Additionally, the experimental group showed poorer results than the control group in monologue intensity (66.60 ± 3.72 vs. 114.72 ± 63.09, p = 0.001), the harmonics-to-noise ratio (9.08 ± 2.06 vs. 10.26 ± 1.59, p = 0.042), and the maximum phonation time of /s/ (4.36 ± 1.67 vs. 8.09 ± 4.07, p < 0.001). Patients with stroke also had significantly lower values for swallowing efficiency and safety compared to the control group (7.05 ± 8.44 vs. 2.23 ± 4.14, p = 0.021) and reported poorer quality of life related to swallowing difficulties (185.50 ± 23.66 vs. 200.32 ± 19.60, p = 0.029). (4) Conclusions: Older adults with chronic stroke exhibited significantly reduced cough strength, voice intensity, maximum phonation time, and swallowing function compared to controls.

Effect of five different body positions on lung function in stroke patients with tracheotomy

ABSTRACT Background In stroke patients with tracheotomy, reduced lung function heightens pulmonary infection risk. Body position can affect lung function; however, its impact in stroke patients with tracheostomy remains unclear. Objective To investigate the influence of five body positions on pulmonary function in stroke patients with tracheotomy. Methods Pulmonary function was assessed in five body positions (supine, supine 30°, supine 60°, sitting, and prone) in 47 stroke patients who underwent tracheotomy. Diaphragmatic excursion during quiet breathing (DEQ), diaphragmatic thickening fraction during quiet breathing (DTFQ), and diaphragmatic excursion during coughing (DEC) were measured using ultrasound. Peak cough flow (PCF) was measured using an electronic peak flow meter. Results Different positions had a significant impact on DEQ, DEC, and PCF in stroke patients with tracheotomy, although not on DTFQ. DEQ showed no significant differences between supine 60°, sitting, and prone positions. Both DEC and PCF reached their maximum values in the sitting position. In the sub-group analysis, DEQ in females did not show significant differences across different positions. Both males and females exhibited significantly higher PCF in the sitting compared to supine position. The lung function of obese patients was significantly better in the sitting than in the supine and supine 30° position. Regardless of the patient’s level of consciousness and whether the brainstem was injured, lung function in the sitting position was significantly higher than in the supine position. Conclusions Body posture influences lung function in stroke patients with tracheotomy. Patients should adopt a sitting position to enhance pulmonary function.

The Impact of Nusinersen Treatment on Respiratory Function in Patients with Spinal Muscular Atrophy: A Systematic Review.

Background/Objectives: This systematic review evaluated the impact of nusinersen therapy on respiratory health and function in individuals with spinal muscular atrophy (SMA) and determined whether nusinersen improves pulmonary function, focusing on differences based on patient age and the timing of treatment initiation. Methods: A systematic search of PubMed, Ovid Medline, ScienceDirect, and Web of Science databases was conducted up to January 2024 in accordance with the PRISMA guidelines. Thirteen studies were included, comprising clinical trials, observational studies, and case series that focused on respiratory outcomes in SMA patients treated with nusinersen. The data on study design, participant characteristics, nusinersen intervention, respiratory outcomes, and adverse events were extracted. The Joanna Briggs Institute Critical Appraisal Tool was used to assess study quality. A narrative synthesis was conducted to address the heterogeneity of the studies. Results: This review found a general trend of improvement in pulmonary function, specifically in forced vital capacity (FVC), although the extent and duration of improvement varied across the studies. Peak cough flow (PCF) and peak expiratory flow (PEF) showed positive trends in some studies, although the results were not consistently significant. Respiratory function improvements were frequently observed, particularly in younger patients and those treated earlier. Conclusions: Nusinersen appears to enhance respiratory function and improve motor outcomes in SMA patients, especially with early treatment. However, further research is needed to fully understand its mechanisms and long-term effects on respiratory health in SMA.

The home-based breathing and chest mobility exercise improves cardiorespiratory functional capacity in long COVID with cardiovascular comorbidities: a randomized study

BackgroundBeyond the acute phase, a considerable proportion of patients recovering from the coronavirus disease 2019 (COVID-19) experience long-term sequelae that profoundly impact their quality of life, particularly their physical fitness. This study aims to assess the effect of home-based breathing and chest mobility exercise on the cardiorespiratory functional capacity of long COVID with cardiovascular comorbidity.MethodsIn this randomized, controlled, single-blind clinical trial, 46 long COVID with cardiovascular comorbidities were randomly assigned to either intervention or control group. The intervention group (n = 23) received additional home-based breathing and chest mobility exercise 3x/week for 12 weeks supervised by attending physicians, whereas the control group only received a home-based cardiac rehabilitation program. Baseline and post-intervention assessments consisted of laboratory (D-Dimer and CRP levels) and functional capacity, assessed through 6-minute walking test (6-MWT), exercise stress test’s metabolic equivalents (METS), and predicted peak oxygen consumption (VO2 peak), peak expiratory flow rate (PEFR), peak cough flow (PCF), chest expansion, and EuroQoL’s quality of life. Intention to treat analysis was performed.ResultsAt the 12th week, intervention subjects had significantly greater functional capacity with higher mean PEFR (p = .031) and PCF (p = .016). Similarly, 6-MWT was higher in the group receiving home-based breathing and chest mobility training (p = .032). The bottom part of the chest circumference was statistically different between the two groups (p = .01). METS and predicted VO2 peak were also higher in the intervention group. However, laboratory parameters and quality of life did not differ markedly (p > .05).ConclusionsHome-based respiratory and chest mobility exercise could be an adjunct to cardiac rehabilitation in long COVID with cardiovascular comorbidities for improving cardiorespiratory functional capacity.Trial registrationThe study protocol was registered at http://ClinicalTrial.gov.id NCT05077943 (14/10/2021).

Dyspnea, respiratory muscle strength, cough capacity, and sleep quality in individuals with substance use disorder

There is evidence that substance use and smoking cause some adverse effects on the respiratory system. The aim of this study was to assess dyspnea severity, respiratory muscle strength, cough capacity, and sleep quality in people with substance use disorder (SUD). Forty eight individuals with SUD and 28 active cigarette smokers participated in the study. Participants’ dyspnea severity was assessed using the Modified Medical Research Council Scale, respiratory muscle strength was measured with a portable electronic mouth pressure device, peak cough flow was assessed with a Peak Flow Meter, and sleep quality was determined using the Pittsburgh Sleep Quality Index (PSQI). The amount of daily cigarette smoking and dyspnea severity were significantly higher in individuals with SUD (p < .001). Peak cough flow values, maximum inspiratory pressure (MIP), maximum expiratory pressure (MEP), MIP (%predicted), and MEP (%predicted) were not significantly different between the SUD patients and the active cigarette smokers (p > .05). However, PSQI sub-parameters such as subjective sleep quality, sleep latency, habitual sleep efficiency, use of sleeping medication, and total scores showed significant differences between the SUD patients and the active cigarette smokers (p < .05, p < .001, p = .03, p < .001, p < .001, respectively). Individuals with SUD were found to have higher dyspnea and poorer sleep quality than active smokers. However, respiratory muscle strength and cough capacities were similar.

Safety and efficacy of viltolarsen in ambulatory and nonambulatory males with Duchenne muscular dystrophy.

Duchenne muscular dystrophy (DMD) is an X-linked recessive disease characterized by mutations in the dystrophin gene, causing motor and pulmonary function decline. Viltolarsen is indicated for patients with dystrophin gene mutations amenable to exon 53 skipping. Here, we report safety, motor function, and the first pulmonary function results from the open-label, phase II Galactic53 trial of viltolarsen (NCT04956289). Male participants aged ≥ 8years with DMD received 80mg/kg intravenous viltolarsen once weekly for 48weeks. Results from participants receiving viltolarsen were compared with an external control cohort group-matched for multiple variables. All treatment-emergent adverse events were mild or moderate, 4 were considered treatment-related, and no participants discontinued. Participants receiving viltolarsen experienced clinically meaningful benefits in pulmonary function with higher percent predicted forced vital capacity and higher peak cough flow at Week 49 compared with the control cohort for both ambulatory and nonambulatory participants. Viltolarsen also stabilized upper limb motor function over the Treatment Period. These results support viltolarsen as an important part of the treatment armamentarium for both ambulatory as well as nonambulatory patients with DMD.

The impact of expiratory muscle strength training on voluntary cough effectiveness in Huntington's disease.

Dysfunction of the airway defence system in Huntington's disease (HD) is a significant but often overlooked problem. Although expiratory muscle strength training (EMST) is frequently utilized in cough effectiveness treatment, its specific impact in HD patients has not yet been explored. This study investigated the effects of EMST on voluntary peak cough flow (vPCF) in HD patients and evaluated the retention of potential gains post-intervention. In this prospective case-controlled trial, 29 HD patients completed an 8-week wait-to-start period, which served to identify the natural development of expiratory muscle strength and vPCF. This was followed by 8 weeks of EMST training and an additional 8 weeks of follow-up. The study's outcome parameters, vPCF and maximum expiratory pressure (MEP), were measured against those of age- and sex-matched healthy controls. Huntington's disease patients had significantly lower MEP (p < 0.001) and vPCF (p = 0.012) compared to healthy controls at baseline. Following the EMST, significant improvements in MEP (d = 1.39, p < 0.001) and vPCF (d = 0.77, p = 0.001) were observed, with HD patients reaching the cough performance levels of healthy subjects. However, these gains diminished during the follow-up, with a significant decline in vPCF (d = -0.451, p = 0.03) and in MEP (d = -0.71; p = 0.002). Expiratory muscle strength training improves expiratory muscle strength and voluntary cough effectiveness in HD patients, but an ongoing maintenance programme is necessary to sustain the improvements.

Effects of Home-Based Daily Respiratory Muscle Training on Swallowing Outcomes in Patients with Chronic Stroke: Protocol for a Randomized Controlled Trial.

(1) Background: Swallowing disorders are common following a stroke. This study aims to evaluate the effects of a home-based daily intervention focused on inspiratory and expiratory muscle training on swallowing outcomes in patients with chronic stroke. (2) Methods: This manuscript presents the protocol of a single-blind randomized clinical trial. Patients with chronic stroke will be randomly assigned to either an experimental or a control group. The experimental group will undergo daily home-based respiratory muscle training in addition to standard speech and language therapy, while the control group will receive only the standard intervention. The main outcome measures will include the aspiration risk, the strength of respiratory muscles, and peak cough flow. (3) Results: It is hypothesized that patients receiving home-based respiratory training in addition to standard therapy will achieve significant improvements in aspiration risk, respiratory muscle strength, and cough efficacy in comparison with those included in the control group. The results will be published as a manuscript. (4) Conclusions: This study aims to provide evidence on the effectiveness of home-based respiratory muscle training in enhancing swallowing function and respiratory parameters in patients with chronic stroke.

Evaluation and management of dyspnea as the dominant presenting feature in neuromuscular disorders.

Dyspnea is a common symptom in neuromuscular disorders and, although multifactorial, it is usually due to respiratory muscle involvement, associated musculoskeletal changes such as scoliosis or, in certain neuromuscular conditions, cardiomyopathy. Clinical history can elicit symptoms such as orthopnea, trepopnea, sleep disruption, dysphagia, weak cough, and difficulty with secretion clearance. The examination is essential to assist with the diagnosis of an underlying neurologic disorder and determine whether dyspnea is from a cardiac or pulmonary origin. Specific attention should be given to possible muscle loss, use of accessory muscles of breathing, difficulty with neck flexion/extension, presence of thoraco-abdominal paradox, conversational dyspnea, cardiac examination, and should include a detailed neurological examination directed at the suspected differential diagnosis. Pulmonary function testing including sitting and supine spirometry, measures of inspiratory and expiratory muscle strength, cough peak flow, sniff nasal inspiratory pressure, pulse oximetry, transcutaneous CO2, and arterial blood gases will help determine the extent of the respiratory muscle involvement, assess for hypercapnic or hypoxemic respiratory failure, and qualify the patient for noninvasive ventilation when appropriate. Additional testing includes dynamic imaging with sniff fluoroscopy or diaphragm ultrasound, and diaphragm electromyography. Polysomnography is indicated for sleep related symptoms that are not otherwise explained. Noninvasive ventilation alleviates dyspnea and nocturnal symptoms, improves quality of life, and prolongs survival. Therapy targeted at neuromuscular disorders may help control the disease or favorably modify its course. For patients who have difficulty with secretion clearance, support of expiratory function with mechanical insufflation-exsufflation, oscillatory devices can reduce the aspiration risk.

Two-year survival after scheduled extubation in patients with pneumonia or ARDS: a prospective observational study

PurposeTo report two-year survival after scheduled extubation in patients with pneumonia or acute respiratory distress syndrome (ARDS).MethodsThis was a prospective observational study performed in a respiratory ICU of a teaching hospital. Pneumonia or ARDS patients who successfully completed a spontaneous breathing trial were enrolled. Data were collected before extubation. Patients were followed up to two years by phone every 3 months.ResultsA total of 230 patients were enrolled in final analysis. One-, 3-, 6-, 12-, and 24-month survival was 77.4%, 63.8%, 61.3%, 57.8%, and 47.8%, respectively. Cox regression shows that Charlson comorbidity index (hazard ratio: 1.20, 95% confidence interval: 1.10–1.32), APACHE II score before extubation (1.11, 1.05–1.17), cough peak flow before extubation (0.993, 0.986–0.999), and extubation failure (3.96, 2.51–6.24) were associated with two-year mortality. To predict death within two years, the area under the curve of receiver operating characteristic was 0.79 tested by Charlson comorbidity index, 0.75 tested by APACHE II score, and 0.75 tested by cough peak flow. Two-year survival was 31% and 77% in patients with Charlson comorbidity index ≥ 1 and < 1, 28% and 62% in patients with APACHE II score ≥ 12 and < 12, and 64% and 17% in patients with cough peak flow > 58 and ≤ 58 L/min, respectively.ConclusionsComorbidity, disease severity, weak cough and extubation failure were associated with increased two-year mortality in pneumonia or ARDS patients who experienced scheduled extubation. It provides objective information to caregivers to improve decision-making process during hospitalization and post discharge.

Structured different exercise protocols improve lung function, respiratory muscle strength, and thickness in stroke patients. A randomized controlled trial

ABSTRACT Background The effect of core stabilization exercises (CSE) alone, or in combination with neuromuscular electrical stimulation (NMES) and Kinesio taping (KT) on lung function, respiratory muscle strength, and thickness in patients with stroke is not fully known. Objective To compare the efficacy of NMES and KT applied with CSE on lung functions, respiratory muscle strength, and thickness in patients with stroke. Methods A total of 45 stroke patients were randomly assigned to the core stabilization exercises (CSE) group, CSE+KT group or CSE+NMES group, respectively. All groups received the training protocol for 30–45 minutes, 3 days a week, for 6 weeks. Lung functions were measured using portable spirometry. Respiratory muscle strength was assessed using an analog manometer to measure maximal inspiratory pressure (MIP) and maximal expiratory pressure (MEP). Peak cough flow (PCF) was measured with a peak flow meter. Respiratory muscles thickness were determined using ultrasonography. Results Except for FVC (%pred) (F=4.432, p=0.018, np2=0.174), FEV1(%pred) (F=3.725, p=0.032, np2=0.151), and MEP (F=3.861, p=0.029, np2=0.155), the overall group by time interaction for rmANOVA showed that there was no statistically significant difference between groups (p>0.05). After post hoc analysis, it was determined that there was no statistically significant difference between the groups in terms of FVC (%pred), FEV1(%pred) and MEP (p>0.025). Conclusions The addition of NMES or KT to core stabilization exercises did not appear to provide additional benefit in improving lung function, respiratory muscle strength, and thickness in stroke patients.

Hybrid SPEAK OUT! protocol improves aerodynamic measurements and PROMs in Parkinson's disease.

SPEAK OUT! has been shown to enhance various aspects of voice such as intensity, prosody, voice quality and perception of voice. However, their impacts on expiration and communication effectiveness have not yet been evaluated. This study aimed to evaluate the effectiveness of the Hybrid SPEAK OUT! method on aerodynamic measurements and patient-reported outcome measures (PROMs) in individuals with Parkinson's disease (PD). This study included 157 participants with PD who underwent an intensive 8-week multidisciplinary treatment program. The Hybrid SPEAK OUT! method consisted of three sessions per week, including two group sessions and one individual treatment session. Voice-related aerodynamic measures included maximum phonation time (MPT), vocal cord function using the S/Z ratio, and expiratory flow measures including peakexpiratoryflow (PEF) and peak cough flow (PCF). Two PROMs were included: the Hebrew version of the voice handicap index-10 and communication effectiveness questionnaire. The results of 111 participants were analysed. MPT duration increased, PEF and PCF increased, and better scores were found in PROMs. These findings would appear to support the effectiveness of the Hybrid SPEAK OUT! methods improving function, activity and participation in individuals with PD. However, further research is needed. What is already known on the subject Prior research has demonstrated the effectiveness of behavioral therapies, including the SPEAK OUT! program, in managing speech symptoms in individuals with Parkinson's disease (PD). These therapies have shown improvements in voice intensity, fundamental frequency, voice quality, and voice perception. However, the impact on aerodynamic measures, expiratory flow, and patient-reported outcome measures has not been extensively studied. What this paper adds to existing knowledge This study adds to the existing knowledge by demonstrating that a Hybrid SPEAK OUT! approach, can lead to improvements in aerodynamic measures, including maximum phonation time (MPT), expiratory and cough peak flow. In terms of motor learning, we found that two non-speech exhalation-related tasks that were not directly trained improved following the intensive speech training protocol. Furthermore, it shows positive changes in patient-reported outcome measures, with reduced voice-related disability and improved communication efficiency. What are the potential or actual clinical implications of this work? The findings of this study have important clinical implications for the management of speech symptoms in individuals with PD. The Hybrid SPEAK OUT! program, which combines group and individual sessions, can effectively improve voice, aerodynamic measurements and patient-reported outcome measures (PROMs), ultimately enhancing the overall quality of life for patients. These findings support the effectiveness of the Hybrid SPEAK OUT! methods for improving function, activity, and participation in individuals with PD.

Prediction of cough effectiveness in amyotrophic lateral sclerosis patients assessed by ultrasuond of the diaphragm during the cough expiration phase

Assessing cough effectiveness, using Cough Peak Flow, is crucial for patients with Neuromuscular Diseases, such as Amyotrophic Lateral Sclerosis. Impaired cough function can contribute to respiratory decline and failure. The goal of the study is to determine the correlation between diaphragmatic excursion and cough expiratory phase, potentially utilizing ultrasonographic indices to estimate Cough Peak Flow in these patients. Twenty-two patients were enrolled in this study. The upward displacement of the diaphragm was measured with ultrasonography during voluntary cough expiration and Cough Peak Flow was simultaneously measured. A multivariable linear regression model was built to quantify the association between Cough Peak Flow and diaphragm expiratory excursion. There is significative relationship between Cough Peak Flow and diaphragm excursion with a Pearson’s r coefficient of 0.86 observed in the patients group. Multiple linear regression analysis for Cough Peak Flow (Adjusted R2 = 0.86) revealed significant associations between Cough Peak Flow and expiratory excursion (adjusted β-coefficient: 64.78, 95 %, CI: 51.50–78.07, p<0.001) and sex (adjusted β-coefficient: −69.06; 95 % CI: −109.98 to −28.15, p=0.001). Our results predict the cough effectiveness by using M-mode diaphragmatic sonography with a potentially significant impact on therapeutic choices.

REVEALS—a longitudinal cohort study of multifaceted respiratory assessment in ALS

Objective To systematically assess decline in respiratory measures in amyotrophic lateral sclerosis (ALS) and to examine the impact of sex, disease onset type and baseline morbidity on progression. Methods The REVEALS study (Registry of Endpoints and Validated Experiences in ALS) was conducted between April 2018 and February 2021 in six European ALS centers. Slow and forced vital capacity (S/FVC), sniff nasal inspiratory pressure (SNIP), peak cough flow, amyotrophic lateral sclerosis functional rating scale-revised (ALSFRS-R), and respiratory morbidity were collected. Data were analyzed using a Bayesian multiple outcomes random effects model. Results Two hundred and eighty participants had a median of three assessments (IQR 2.0, 5.0) over a median of 8 months (IQR 2.3, 14.1). There were 974 data collection timepoints. Differences in respiratory measures and rates of decline between disease-onset and sex subgroups were identified. Females had lower scores in all respiratory measures and females with bulbar onset ALS had faster decline compared with other sub-groups. These differences were not detected by the ALSFRS-r respiratory subscale. Dyspnea, orthopnea, and a higher King’s stage at baseline were associated with lower respiratory scores throughout follow-up, while having a regular productive cough at baseline was associated with lower peak cough flow scores. Conclusion Respiratory function declines more quickly in females with ALS compared with males when measured by FVC, SVC, SNIP, or PCF, but not the ALSFRS-R respiratory sub-score. Higher baseline King’s staging and the presence of clinical respiratory symptoms at baseline were associated with worse respiratory function. The ALSFRS-R respiratory sub-score is poorly correlated with objective respiratory measurements.

Reliability and validity of cough peak flow measurements in myasthenia gravis

Decreased cough strength in myasthenia gravis (MG) leads to aspiration and increases the risk of MG crisis. The aim of this study was to clarify the reliability and validity of cough peak flow (CPF) measurements in MG. A total of 26 patients with MG who underwent CPF measurements using the peak flow meter by themselves were included. MG symptoms were evaluated by pulmonary function tests and clinical MG assessment scales before and after immune-treatments. The relationship between CPF and pulmonary function tests and MG comprehensive were assessed. The cut-off value of CPF for aspiration risk was determined and the area under the curve (AUC) was calculated. The intraclass correlation coefficient was more than 0.95 for pre-and post-treatment. Positive correlations were found between CPF and almost all spirometric values as well as between the differences of pre-and post-treatment in CPF and quantitative myasthenia gravis score. The CPF for identifying the aspiration risk was used to calculate the CPF cut-off value of 205 L/min with a sensitivity of 0.77, specificity of 0.90, and AUC of 0.85. The CPF, a convenient measure by patients themselves, has a high reliability in patients with MG, and is a useful biomarker reflecting MG symptoms.

Effect of Inspiratory Muscle Training on Cough Strength in Older People With Frailty: A Single-Blind Randomized Controlled Trial

ObjectiveTo investigate the effect of inspiratory muscle training (IMT) on cough strength in older people with frailty. DesignSingle-blind randomized controlled trial. SettingDay health care centers at 2 sites. ParticipantsOlder people with frailty (N=60). InterventionsEligible people were randomly assigned to receive IMT program in addition to general exercise training (IMT group), or general exercise training alone (control group). The IMT group performed training using a threshold IMT device with the load set at 30% of maximum inspiratory mouth pressure in addition to the general exercise training program throughout the 8 weeks. The IMT took place twice a day and each session consisted of 30 breaths. Main Outcome MeasuresPrimary outcome was cough strength, measured as the cough peak flow (CPF), at the beginning and the end of the program. ResultsData from 52 participants (26 in each group) were available for the analysis. The mean age was 82.6 years; 33% were men. The change in CPF at the end of the program was 28.7±44.4 L/min in the IMT group and −7.4±26.6 L/min in the control group. A linear regression model showed that the presence or absence of IMT was associated with changes in CPF (mean difference between groups, 36.3; 95% confidence interval, 16.7-55.9; effect size, 0.99). ConclusionsIMT may be a useful intervention to improve cough strength in frail older people.

Therapeutic Role of Nusinersen on Respiratory Progression in Pediatric Patients With Spinal Muscular Atrophy Type 2 and Nonambulant Type 3.

Nusinersen has shown significant functional motor benefit in the milder types of spinal muscular atrophy (SMA). Less is known on the respiratory outcomes in patients with nusinersen-treated SMA. The aim of this study was to describe changes in respiratory function in pediatric patients with SMA type 2 and 3 on regular treatment with nusinersen within the iSMAc international cohort and to compare their trajectory with the natural history (NH) data published by the consortium in 2020. This is a 5-year retrospective observational study of pediatric SMA type 2 and nonambulant type 3 (age ≤18 years) treated with nusinersen. The primary objective was to compare the slopes of decline in forced vital capacity % predicted (FVC% pred.), FVC, and age when FVC dropped below 60% between the treated patients and a control group from the natural history cohort. Data on peak cough flow and the use of noninvasive ventilation (NIV) and cough assist were collected. Data were available for 69 treated patients, 53 were SMA type 2 and 16 type 3. The mean (SD) age at first injection was 8.5 (3.2) and 9.7 (3.7) years, respectively. The median (interquartile range) treatment duration was 1 (0.7; 1.9) and 1.2 (0.9; 1.9) years, respectively. At the time of the first nusinersen injection, 24 of 52 (46%) patients with SMA type 2 and 2 of 16 (13%) patients with SMA type 3 were on NIV. Forty-three of 53 (81%) and 4 of 16 (25%) patients used cough device. FVC% pred. in treated patients with SMA type 2 declined annually by 2.3% vs 3.9% in NH (p = 0.08) and in treated patients with type 3 by 2.6% vs 3.4% NH (p = 0.59). Patients treated reached FVC <60% later than untreated (12.1 vs 10 years, p = 0.05). A higher percentage of treated vs untreated patients maintained FVC% pred. equal/above their baseline after 12 (65% vs 36%) and 24 (50% vs 24%) months, respectively. NIV use among treated did not significantly change throughout 1-year follow-up. This study included the largest real-world cohort of pediatric patients with milder SMA types. The results suggest a positive role of nusinersen in delaying the respiratory decline in patients treated longer than 1 year when compared with natural history. Larger cohorts and longer observation are planned. This study provided Class III evidence that nusinersen slows progression for patients with SMA types 2 and 3 compared with a natural history cohort.

A Preliminary Study of Voluntary Cough Motor Performance and Learning With Skill Training and Biofeedback.

Sensorimotor cough skill training (CST) has been shown to improve cough strength, as well as facilitate changes during training (i.e., motor performance) and generalization to untrained tasks (i.e., motor learning). However, there is a gap in our understanding of the effects of voluntary CST (without sensory stimuli) on motor performance and learning. Furthermore, the contribution of physiologic factors, such as lung volume, a driver of cough strength in healthy adults, and treatment-specific factors, such as biofeedback, remains unexamined. Twenty individuals with Parkinson's disease (PD) completed pre- and post-CST single voluntary, sequential voluntary, and reflex cough testing. Participants were randomized to biofeedback or no biofeedback groups. They completed one CST session involving 25 trials of voluntary coughs, with the treatment target set 25% above baseline peak flow. Participants were instructed to "cough hard" to exceed the target. In the biofeedback group, participants received direct visualization of the target line in real time. Cough peak flow showed positive improvements in motor performance (β = .02; 95% credible interval [CI]: 0.01, 0.03) and learning (β = .26; 95% CI: 0.03, 0.47). Changes in lung volume from pre- to post-CST did not predict treatment response. No differences in treatment response were detected between the biofeedback groups. A single session of voluntary CST improved voluntary cough motor performance and learning. Although lung volume increased during CST, changes to lung volume did not predict treatment response. These findings demonstrate the potential of voluntary CST to improve motor performance and motor learning among individuals with PD and cough dysfunction. https://doi.org/10.23641/asha.25447444.

Waves of Precision: A Practical Guide for Reviewing New Tools to Evaluate Mechanical In-Exsufflation Efficacy in Neuromuscular Disorders.

Mechanical insufflation-exsufflation (MI-E) is essential for secretion clearance, especially in neuromuscular disorders. For the best outcomes, initiation of MI-E should be started at the correct time with regular evaluation to the response to treatment. Typically, cough peak flow has been used to evaluate cough effectiveness with and without MI-E. This review highlights the limitations of this and discussed other tools to evaluate MI-E efficacy in this rapidly developing field. Such tools include the interpretation of parameters (like pressure, flow and volumes) that derive from the MI-E device and external methods to evaluate upper airway closure. In this review we pinpoint the differences between different devices in the market and discuss new tools to better titrate MI-E and detect pathological responses of the upper airway. We discuss the importance of point of care ultrasound (POCUS), transnasal fiberoptic laryngoscopy and wave form analysis in this setting. To improve clinical practice newer generation MI-E devices should allow real-time evaluation of waveforms and standardize some of the derived parameters.

Functional abilities, respiratory and cardiac function in a large cohort of adults with Duchenne muscular dystrophy treated with glucocorticoids.

The transition to adult services, and subsequent glucocorticoid management, is critical in adults with Duchenne muscular dystrophy. This study aims (1) to describe treatment, functional abilities, respiratory and cardiac status during transition to adulthood and adult stages; and (2) to explore the association between glucocorticoid treatment after loss of ambulation (LOA) and late-stage clinical outcomes. This was a retrospective single-centre study on individuals with Duchenne muscular dystrophy (≥16 years old) between 1986 and 2022. Logistic regression, Cox proportional hazards models and survival analyses were conducted utilizing data from clinical records. In all, 112 individuals were included. Mean age was 23.4 ± 5.2 years and mean follow-up was 18.5 ± 5.5 years. At last assessment, 47.2% were on glucocorticoids; the mean dose of prednisone was 0.38 ± 0.13 mg/kg/day and of deflazacort 0.43 ± 0.16 mg/kg/day. At age 16 years, motor function limitations included using a manual wheelchair (89.7%), standing (87.9%), transferring from a wheelchair (86.2%) and turning in bed (53.4%); 77.5% had a peak cough flow <270 L/min, 53.3% a forced vital capacity percentage of predicted <50% and 40.3% a left ventricular ejection fraction <50%. Glucocorticoids after LOA reduced the risk and delayed the time to difficulties balancing in the wheelchair, loss of hand to mouth function, forced vital capacity percentage of predicted <30% and forced vital capacity <1 L and were associated with lower frequency of left ventricular ejection fraction <50%, without differences between prednisone and deflazacort. Glucocorticoid dose did not differ by functional, respiratory or cardiac status. Glucocorticoids after LOA preserve late-stage functional abilities, respiratory and cardiac function. It is suggested using functional abilities, respiratory and cardiac status at transition stages for adult services planning.