A pilot study of an integrated, personalized, respiratory and motor telerehabilitation program for pediatric patients with hereditary neuromuscular disorders.

INTRODUCTION: Telerehabilitation provides physical training to patients through telecommunication networks. We examined the feasibility, safety, and efficacy of an integrated, personalized, respiratory and motor telerehabilitation program for pediatric patients with hereditary neuromuscular disorders (NMDs). METHODS: Stable pediatric patients were recruited for a 16-week home training program with personalized pulmonary, upper and lower limb exercises. Patients reviewed instructional videos at home and attended bi-weekly follow-ups through video or audio calls, text messages, or emails. The primary outcomes were respiratory function, Medical Research Council (MRC) grading, hand/pinch strength, 6-minute walk test, and Pediatric Quality-of-Life Inventory 3.0 Neuromuscular Module survey. The secondary outcomes were study compliance and user feedback. RESULTS: Patients with spinal muscular atrophy (n = 4), congenital myasthenic syndrome (n = 2), and Duchenne muscular dystrophy (n = 2) completed the program. The median weekly exercise time was 101.3 min (range: 30.0-266.9). No extra face-to-face physiotherapy sessions were requested by the patients. No adverse events were reported. After the study, patients showed improvements in maximal expiratory pressure (35.0 vs. 47.5 cm H2O, p = .028) and maintained their MRC grade, hand/pinch strength, and walking distance. Patients also reported improvements in the Pediatric Quality-of-Life Inventory 3.0 Neuromuscular Module survey score (74.5 vs. 87.0, p = .036). Patients rated the overall program highly (mean: 4.00/5.00) and recommended it as a standard of care (mean: 4.38/5.00). DISCUSSION: Our telerehabilitation program was feasible, safe, and possibly effective for this pilot cohort of stable pediatric patients with hereditary NMDs. Larger-scale studies for longer periods are warranted to confirm the results.

Long-Term Management for Ventilator-Assisted Children in Hong Kong: 2 Decades' Experience.

BACKGROUND: The population of children receiving long-term mechanical ventilation is growing worldwide, but only limited data exist in Asian regions. The objective of the study was to review the management of these children in Hong Kong over the past 2 decades. METHODS: This was a retrospective cohort study. Hospital records from subjects receiving mechanical ventilation for >3 months were retrieved. Demographic characteristics and medical information of subjects (≤21 y old) under the care of the ventilator program at the Duchess of Kent Children's Hospital between 1997 and 2015 were reviewed. RESULTS: There were a total of 96 subjects, with male predominance (75%). Twenty-five subjects (26%) required invasive mechanical ventilation via tracheostomy, and the median age of starting ventilation was much lower than for subjects receiving noninvasive ventilation (1.6 vs 11.2 y, P .003). Underlying causes included neuromuscular diseases (50%); abnormal ventilatory control related to neurological, metabolic, or genetic conditions (25%); airway problems (18%); and chronic lung diseases (6%). Comorbidities were frequent, with significantly more subjects receiving invasive mechanical ventilation who required gastrostomy or nasogastric tube feeding and had intellectual impairment. Despite their medical complexities, most of our subjects (87.5%) could be discharged and resided in the community. Social reasons were the obstacles for discharge in 12 subjects receiving invasive mechanical ventilation. Seventeen subjects who died were receiving noninvasive ventilation, and most had progressive neuromuscular diseases. Only 8% subjects could be weaned off of ventilator support. At the time of the report, about one third of the subjects have reached adult age. CONCLUSIONS: Our study showed a steady growth in the number of children receiving long-term mechanical ventilation, similar to that in western countries, yet these children have distinct demographic characteristics, underlying diseases, comorbidities, and outcomes. Home care is feasible in the majority of these children despite a lack of in-home nursing care provision. Prolonged survival of these subjects necessitates the enhancement of transitional care to adult service.

Adenovirus respiratory infection in hospitalized children in Hong Kong: serotype-clinical syndrome association and risk factors for lower respiratory tract infection.

Lower respiratory tract infections (LRTI) caused by adenovirus can be severe with resultant chronic pulmonary sequelae. More than 50 serotypes have been recognized; however, the exact association of serotype with clinical phenotype is still unclear. There have been no reports on the adenovirus serotype pattern in Hong Kong, and their relationships with disease manifestations and complications are not known. Clinical and epidemiological data on 287 children (<6 years old) admitted with adenovirus respiratory infections from 2001 to 2004 were reviewed. Common presenting symptoms included fever (97.9 %) and cough and rhinitis (74 %). Extra-pulmonary manifestations were present in 37.3 %. The clinical picture mimicked bacterial infection for its prolonged high fever and neutrophilic blood picture. Forty-two patients (14.6 %) had LRTI, either pneumonia or acute bronchiolitis, but none had severe acute respiratory compromise. Children aged 1 to 2 years old were most at risk for adenovirus LRTI (adjusted p = 0.0165). Serotypes 1 to 7 could be identified in 93.7 % of the nasopharyngeal specimens, with serotypes 2 and 3 being the most prevalent. Different serotypes showed predilection for different age groups and with different respiratory illness association. The majority of acute bronchiolitis (71.4 %) were associated with serotype 2 infection, and this association was statistically significant (p < 0.0001). Serotype 3 infection accounted for over half of the pneumonia cases (57-75 %) in those aged 3-5 years old. Only one patient developed mild bronchiectasis after serotype 7 pneumonia. Children aged 1 to 2 years old were the at-risk group for adenovirus LRTI, but respiratory morbidity was relatively mild in our locality. There was an apparent serotype-respiratory illness association.

Successful use of BiPAP in infants with congenital myotonic dystrophy.

Reported herein are two cases of severe phenotype of congenital myotonic dystrophy (CDM) with presentation of respiratory insufficiency at birth. The infants were successfully managed with bi-level positive airway pressure (BiPAP) via nasal mask. The use of BiPAP in infants with CDM has not been reported before. The rationale for using BiPAP is discussed. BiPAP may be more effective than continuous positive airway pressure in managing respiratory insufficiency, especially in infants with the more severe phenotype of CDM.