Inspiratory muscle training in children with neuromuscular disorders.

Background: Progressive respiratory muscle weakness and ineffective cough contribute to morbidity and mortality in children with neuromuscular disorders (NMD). Inspiratory muscle training (IMT) aims to preserve or improve respiratory muscle strength and reduce respiratory morbidity. This study aimed to determine the safety and efficacy of IMT in children with NMD. Methods/design: A randomised cross-over study compared a 3-month intervention (IMT) with control periods (no IMT). Children diagnosed with NMD (5 years - 18 years) performed 30 breaths (at 30% of maximum inspiratory mouth pressure [Pimax]) with an electronic threshold device, twice daily. During the control period, participants did not perform any IMT. Discussion: Twenty three children (median [interquartile range {IQR}] age of 12.33 [10.03-14.17] years), mostly male (n = 20) and non-ambulant (n = 14) participated. No adverse events related to IMT were reported. No difference in median patient hospitalisation and respiratory tract infection (RTI) rates between non-training and intervention periods (p = 0.60; p = 0.21) was found. During IMT, Pimax and peak cough flow improved with a mean ± standard deviation (s.d.) of 14.57 ± 15.67 cmH2O and 32.27 ± 36.60 L/min, compared to 3.04 ± 11.93 cmH2O (p = 0.01) and -16.59 ± 48.29 L/min (p = 0.0005) during the non-training period. Similar to other studies, spirometry did not show a significant change. Conclusion: A 3-month IMT programme in children with NMD appears safe and well-tolerated, with significant improvement in respiratory muscle strength and cough efficacy. Clinical implications: Inspiratory muscle training could be considered a cost-effective adjunct to respiratory management in children with NMD. Trial Registration: Pan African Clinical Trial Registry, PACTR201506001171421, https://pactr.samrc.ac.za.

Performance of upper limb entry item to predict forced vital capacity in dysferlin-deficient limb girdle muscular dystrophy.

Dysferlin-deficient limb girdle muscular dystrophy (LGMD R2), also referred to as dysferlinopathy, can be associated with respiratory muscle weakness as the disease progresses. Clinical practice guidelines recommend biennial lung function assessments in patients with dysferlinopathy to screen for respiratory impairment. However, lack of universal access to spirometry equipment and trained specialists makes regular monitoring challenging. This study investigated the use of the Performance of Upper Limb (PUL) clinical scale entry item as a low-cost screening tool to identify patients with dysferlinopathy at risk of respiratory impairment. Using data from 193 patients from the Jain Foundation's International Clinical Outcomes Study, modelling identified a significant positive relationship between the PUL entry item and forced vital capacity (FVC). Eighty-eight percent of patients with the lowest PUL entry item score of 1 presented with FVC % predicted values of <60 %, suggestive of respiratory impairment. By contrast, only 10 % of the remainder of the cohort (PUL entry item of 2 or more) had an FVC of <60 %. This relationship also held true when accounting for ambulatory status, age, and sex as possible confounding factors. In summary, our results suggest that the PUL entry item could be implemented in clinical practice to screen for respiratory impairment where spirometry is not readily available.

Participation patterns of children with cerebral palsy: A caregiver's perspective.

Background: Participation in activities of daily living (ADL), education, leisure and play in children living with cerebral palsy (CP) may be affected by various factors, as outlined in the International Classification of Functioning, Disability and Health Framework (ICF). The aim of this study was to describe the participation patterns of a group of these children. Objectives: This study aimed to describe participation patterns in ADL, education, leisure and play activities of children living with CP in Modimolle. Method: An exploratory-descriptive qualitative (EDQ) study design was used. A researcher-constructed bio-demographic data sheet and a semi-structured interview schedule were used to collect data from the primary caregivers of children (5-17 years) living with CP in Modimolle. Interviews were transcribed verbatim, translated from Sepedi to English and analysed using the content analysis approach and NVivo software. Results: The findings of this study indicated that children living with CP in Modimolle require set-up and assistance to participate in various ADL such as self-care, family and community activities. They also participate in formal and informal educational programmes as well as active and passive leisure and play activities. However, at the moment, they have limited opportunities to participate because of resource constraints and inaccessible infrastructure. Conclusion: Although children with CP in Modimolle perform some ADL, and participate in educational, leisure and play activities, they are not fully integrated into their community. Legislative support and policy implementation are required to improve participation and integration of children living with CP. Further studies on community-specific integrative strategies to enhance participation among children living with disabilities are recommended. Contribution: This paper provides valuable information on the participation patterns of children with CP living in a rural area of South Africa. The findings can assist with development and implementation of community-specific, integrative health and social care strategies to enhance participation among children living with disabilities.

Erratum: The role of physiotherapy in the respiratory management of children with neuromuscular diseases: A South African perspective.

[This corrects the article DOI: 10.4102/sajp.v77i1.1527.].

Inspiratory muscle training in children and adolescents living with neuromuscular diseases: A pre-experimental study.

BACKGROUND: Children with neuromuscular diseases (NMD) are at risk of morbidity and mortality because of progressive respiratory muscle weakness and ineffective cough. Inspiratory muscle training (IMT) aims to preserve or improve respiratory muscle strength, thereby reducing morbidity and improving health-related quality of life (HRQoL). OBJECTIVES: To describe the safety and feasibility of a 6-week IMT programme using an electronic threshold device (Powerbreathe®). Any adverse events and changes in functional ability, spirometry, peak expiratory cough flow (PECF), inspiratory muscle strength and HRQoL (Pediatric Quality of Life [PedsQL]) were recorded. METHODS: A convenience sample of eight participants (n = 4 boys; median [interquartile range {IQR}] age: 12.21 [9.63-16.05] years) with various NMD were included in a pre-experimental, observational pre-test post-test feasibility study. Training consisted of 30 breaths, twice daily, 5 days a week, for 6 weeks. RESULTS: There were significant pre- to post-intervention improvements in upper limb function and coordination (p = 0.03) and inspiratory muscle strength: maximum inspiratory mouth pressure (Pimax) (p = 0.01); strength-index (p = 0.02); peak inspiratory flow (PIF) (p = 0.02), with no evidence of change in spirometry, PECF or HRQoL. No adverse events occurred and participant satisfaction and adherence levels were high. CONCLUSION: Inspiratory muscle training (at an intensity of 30% Pimax) appears safe, feasible and acceptable, in a small sample of children and adolescents with NMD and was associated with improved inspiratory muscle strength, PIF and upper limb function and coordination. CLINICAL IMPLICATIONS: Larger, longer-term randomised controlled trials are warranted to confirm the safety and efficacy of IMT as an adjunct respiratory management strategy in children with NMD.

The role of physiotherapy in the respiratory management of children with neuromuscular diseases: A South African perspective.

BACKGROUND: Respiratory morbidity is common in children with neuromuscular diseases (NMD) owing to chronic hypoventilation and impaired cough. Optimal, cost-effective respiratory management requires implementation of clinical practice guidelines and a coordinated multidisciplinary team approach. OBJECTIVES: To explore South African physiotherapists' knowledge, perception and implementation of respiratory clinical practice guidelines for non-ventilated children with NMD. METHODS: An online survey was conducted amongst members of the South African Society of Physiotherapy's Cardiopulmonary Rehabilitation (CPRG) and Paediatric special interest groups and purposive sampling of non-member South African physiotherapists with respiratory paediatrics expertise (N= 481). RESULTS: Most respondents worked in private healthcare, with 1-10 years' experience treating patients with NMD. For acute and chronic management, most participants recommended nebulisation and 24-h postural management for general respiratory care. Percussions, vibrations, positioning, adapted postural drainage, breathing exercises and manually assisted cough were favoured as airway clearance techniques. In addition, participants supported non-invasive ventilation, oscillatory devices and respiratory muscle training for chronic management. CONCLUSION: Respondents seemed aware of internationally-endorsed NMD clinical practice guidelines and recommendations, but traditional manual airway clearance techniques were favoured. This survey provided novel insight into the knowledge, perspectives and implementation of NMD clinical practice guidelines amongst South African physiotherapists. CLINICAL IMPLICATIONS: There is an urgent need to increase the abilities of South African physiotherapists who manage children with NMD, as well as the establishment of specialised centres with the relevant equipment, ventilatory support and expertise in order to provide safe, cost-effective and individualised patient care.

Cough augmentation techniques for people with chronic neuromuscular disorders.

BACKGROUND: People with neuromuscular disorders may have a weak, ineffective cough predisposing them to respiratory complications. Cough augmentation techniques aim to improve cough effectiveness and mucous clearance, reduce the frequency and duration of respiratory infections requiring hospital admission, and improve quality of life. OBJECTIVES: To determine the efficacy and safety of cough augmentation techniques in adults and children with chronic neuromuscular disorders. SEARCH METHODS: On 13 April 2020, we searched the Cochrane Neuromuscular Specialised Register, CENTRAL, MEDLINE, Embase, CINAHL, and ClinicalTrials.gov for randomised controlled trials (RCTs), quasi-RCTs, and randomised cross-over trials. SELECTION CRITERIA: We included trials of cough augmentation techniques compared to no treatment, alternative techniques, or combinations thereof, in adults and children with chronic neuromuscular disorders. DATA COLLECTION AND ANALYSIS: Two review authors independently assessed trial eligibility, extracted data, and assessed risk of bias. The primary outcomes were the number and duration of unscheduled hospitalisations for acute respiratory exacerbations. We assessed the certainty of evidence using GRADE. MAIN RESULTS: The review included 11 studies involving 287 adults and children, aged three to 73 years. Inadequately reported cross-over studies and the limited additional information provided by authors severely restricted the number of analyses that could be performed. Studies compared manually assisted cough, mechanical insufflation, manual and mechanical breathstacking, mechanical insufflation-exsufflation, glossopharyngeal breathing, and combination techniques to unassisted cough and alternative or sham interventions. None of the included studies reported on the primary outcomes of this review (number and duration of unscheduled hospital admissions) or listed 'adverse events' as primary or secondary outcome measures. The evidence suggests that a range of cough augmentation techniques may increase peak cough flow compared to unassisted cough (199 participants, 8 RCTs), but the evidence is very uncertain. There may be little to no difference in peak cough flow outcomes between alternative cough augmentation techniques (216 participants, 9 RCTs). There was insufficient evidence to determine the effect of interventions on measures of gaseous exchange, pulmonary function, quality of life, general function, or participant preference and satisfaction. AUTHORS' CONCLUSIONS: We are very uncertain about the safety and efficacy of cough augmentation techniques in adults and children with chronic neuromuscular disorders and further studies are needed.

Assisted autogenic drainage in infants and young children hospitalized with uncomplicated pneumonia, a pilot study.

BACKGROUND AND PURPOSE: Pneumonia is the most important respiratory problem in low-to-middle income countries. Airway clearance therapy continues to be used in children with pneumonia and secretion retention; however, there is lack of evidence to support or reject this treatment. This study aimed to investigate the feasibility of a randomized controlled trial (RCT) on the efficacy and safety of assisted autogenic drainage (AAD) compared to standard nursing care in children hospitalized with uncomplicated pneumonia. METHODS: A single-blinded pilot RCT was conducted on 29 children (median age 3.5 months, IQR 1.5-9.4) hospitalized with uncomplicated pneumonia. The intervention group received standard nursing care with additional bi-daily AAD, for 10 to 30 min. The control group only received standard nursing care, unless otherwise deemed necessary by the physician or physiotherapist. The primary outcome measure was duration of hospitalization. The secondary outcome measures included days of fever and supplemental oxygen support; respiratory rate (RR) and heart rate adjusted for age; RR and oxygen saturation pre-, post-, and 1-hr post-treatment; oxygen saturation; adverse events; and mortality. RESULTS: No difference was found for duration of hospitalization (median 7.5 and 7.0 days for the control and intervention groups, respectively); however, Kaplan-Meier analysis revealed a strong tendency towards a shorter time to discharge in the intervention group (p = .06). No significant differences were found for the other outcome measures at time of discharge. No adverse events were reported. Within the intervention group, a significant reduction in RR adjusted for age was found. DISCUSSION: As no adverse events were reported, and AAD did not prolong hospitalization; AAD might be considered as safe and effective in young children with uncomplicated pneumonia. However, a larger multicentred RCT is warranted to determine the efficacy of AAD compared to standard nursing care.

Inspiratory muscle training for children and adolescents with neuromuscular diseases: A systematic review.

Patients with neuromuscular diseases are at risk of morbidity and mortality due to respiratory compromise caused by respiratory muscle weakness. A systematic review was performed using pre-specified search strategies to determine the safety of inspiratory muscle training (IMT) and whether it has an impact on inspiratory muscle strength and endurance, exercise capacity, pulmonary function, dyspnoea and health-related quality of life. Randomised, quasi-randomised, cross-over and clinical controlled trials were included if they assessed the use of an external IMT device compared to no, sham/placebo, or alternative IMT treatment in children aged 5-18 years with neuromuscular diseases. Seven full-text articles and two on-going trials (n = 168) were included. Most studies used threshold IMT devices over a medium to long-term period, and none reported any adverse events. Studies differed regarding intensity, repetitions, frequency, rest intervals and duration of IMT. Six studies reported no significant improvement in pulmonary function tests following IMT. Two comparable studies reported significant improvement in inspiratory muscle endurance and four studies reported significantly greater improvement in inspiratory muscle strength in experimental groups. The latter was confirmed in a meta-analysis of two comparable studies (overall effect p < 0.00001). Other outcome measures could not be pooled. There is currently insufficient evidence to guide clinical IMT practice, owing to the limited number of included studies; small sample sizes; data heterogeneity; and risk of bias amongst included studies. Large sample randomised controlled trials are needed to determine safety and efficacy of IMT in paediatric and adolescent patients with neuromuscular diseases.