Impact of COVID-19 pandemic on access to online therapeutic education programs for children with sickle cell disease.

BACKGROUND: Sickle cell disease (SCD) is a lifelong disease for which outcomes may be influenced by patients' self-care knowledge. Therapeutic education (TPE) is a patient-centered teaching instrument based on patient's adaptative processes and needs. TPE was developed in the Paris area by a pediatric health network using interactive face-to-face meetings. The COVID-19 pandemic has impacted the TPE modalities by promoting online training. Our aims were to evaluate the accessibility of patients with SCD to online TPE. METHODS: We compared sessions of TPE before and after the onset of the pandemic: the number of sessions, performed face-to-face or online, individual or in a group. We also recorded the number of participants in each session and their age, school level, and department in France. FINDINGS: We observed an increase in the total number of trained children, but participation varied greatly according to the geographical area of residence, with a decrease from 22.4% to 4.9% in the proportion of attendees living in the most socio-economically deprived French departments. DISCUSSION: Online TPE is feasible for patients with SCD but with unequal access according to socio-economic status. APPLICATION TO PRACTICE: Access to TPE needs to be improved for patients living in socially disadvantaged areas.

Non-invasive Ventilation in Children With Neuromuscular Disease.

The respiratory muscles are rarely spared in children with neuromuscular diseases (NMD) which puts them at risk of alveolar hypoventilation. The role of non-invasive ventilation (NIV) is then to assist or "replace" the weakened respiratory muscles in order to correct alveolar hypoventilation by maintaining a sufficient tidal volume and minute ventilation. As breathing is physiologically less efficient during sleep, NIV will be initially used at night but, with the progression of respiratory muscle weakness, NIV can be extended during daytime, preferentially by means of a mouthpiece in order to allow speech and eating. Although children with NMD represent the largest group of children requiring long term NIV, there is a lack of validated criteria to start NIV. There is an agreement to start long term NIV in case of isolated nocturnal hypoventilation, before the appearance of daytime hypercapnia, and/or in case of acute respiratory failure requiring any type of ventilatory support. NIV is associated with a correction in night- and daytime gas exchange, an increase in sleep efficiency and an increase in survival. NIV and/or intermittent positive pressure breathing (IPPB) have been shown to prevent thoracic deformities and consequent thoracic and lung hypoplasia in young children with NMD. NIV should be performed with a life support ventilator appropriate for the child's weight, with adequate alarms, and an integrated (±additional) battery. Humidification is recommended to improve respiratory comfort and prevent drying of bronchial secretions. A nasal interface (or nasal canula) is the preferred interface, a nasobuccal interface can be used with caution in case of mouth breathing. The efficacy of NIV should be assessed on the correction of alveolar ventilation. Patient ventilator synchrony and the absence of leaks can be assessed on a sleep study with NIV or on the analysis of the ventilator's in-built software. The ventilator settings and the interface should be adapted to the child's growth and progression of respiratory muscle weakness. NIV should be associated with an efficient clearance of bronchial secretions by a specific program on the ventilator, IPPB, or mechanical insufflation-exsufflation. Finally, these children should be managed by an expert pediatric multi-disciplinary team.

Non-invasive Ventilation and CPAP Failure in Children and Indications for Invasive Ventilation.

Non-invasive ventilation (NIV) and continuous positive airway pressure (CPAP) are effective treatments for children with severe sleep disordered breathing (SBD). However, some patients may present too severe SDB that do not respond to NIV/CPAP or insufficient compliance to treatment. A careful revaluation of the interface and of ventilator settings should be performed before considering alternative treatments. In patients with obstructive sleep apnea (OSA), alternatives to CPAP/NIV rely on the underlying disease. Ear-nose-throat (ENT) surgery such as adeno-tonsillectomy (AT), turbinectomy or supraglottoplasty represent an effective treatment in selected patients before starting CPAP/NIV and should be reconsidered in case of CPAP failure. Rapid maxillary expansion (RME) is restricted to children with OSA and a narrow palate who have little adenotonsillar tissue, or for those with residual OSA after AT. Weight loss is the first line therapy for obese children with OSA before starting CPAP and should remain a priority in the long-term. Selected patients may benefit from maxillo-facial surgery such as mandibular distraction osteogenesis (MDO) or from neurosurgery procedures like fronto-facial monobloc advancement. Nasopharyngeal airway (NPA) or high flow nasal cannula (HFNC) may constitute efficient alternatives to CPAP in selected patients. Hypoglossal nerve stimulation has been proposed in children with Down syndrome not tolerant to CPAP. Ultimately, tracheostomy represents the unique alternative in case of failure of all the above-mentioned treatments. All these treatments require a multidisciplinary approach with a personalized treatment tailored on the different diseases and sites of obstruction. In patients with neuromuscular, neurological or lung disorders, non-invasive management in case of NIV failure is more challenging. Diaphragmatic pacing has been proposed for some patients with central congenital hypoventilation syndrome (CCHS) or neurological disorders, however its experience in children is limited. Finally, invasive ventilation via tracheotomy represents again the ultimate alternative for children with severe disease and little or no ventilatory autonomy. However, ethical considerations weighting the efficacy against the burden of this treatment should be discussed before choosing this last option.

Follow-Up and Monitoring of Children Needing Long Term Home Ventilation.

Once continuous positive airway pressure (CPAP) or noninvasive ventilation (NIV) is started in a child, and the child is discharged home, follow-up needs to be organized with regular visits in order to check the tolerance and efficacy of the treatment. But there is a lack of validated clinical guidelines, mainly because of the heterogeneity of the ventilator servicing, the costs and health care systems among countries. Therefore, visits timing and strategies to monitor CPAP/NIV are not clearly defined. Moreover, depending on various factors such as the underlying disorder, the medical stability, the age of the child, and socio-economic factors, follow-up usually ranges between 1 month and 3-6 months, or even 1 year following treatment initiation, with an overnight hospital stay, an out-patient visit, a home visit, via telemonitoring or telemedicine, alone or in combination. Apart from clinical evaluation, nocturnal oximetry and capnography monitoring and/or poly(somno)graphy (P(S)G) are usually carried out during the follow-up visits to monitor the delivered pressure, leaks, residual respiratory events and synchrony between the patient and the ventilator. Built-in software data of CPAP/NIV devices can be used to assess the adherence of treatment, to monitor pressure efficiency, leaks, asynchronies, and to estimate the presence of residual respiratory events under CPAP/NIV if P(S)G is not available or in alternance with P(S)G. The possibility of CPAP/NIV weaning should be assessed on a regular basis, but no criteria for the timing and procedures have been validated. Weaning timing depends on the clinical condition that justified CPAP/NIV initiation, spontaneous improvement with growth, and the possibility and efficacy of various upper airway, maxillofacial and/or neurosurgical procedures. Weaning may be allowed in case of the disappearance of nocturnal and daytime symptoms of sleep-disordered breathing (SDB) after several nights without CPAP/NIV and the objective correction of SDB on a P(S)G. But no parameters are defined. In any case, a long term follow-up is necessary to ascertain the weaning success. Large prospective studies, together with international and national guidelines, are required in order to build evidence for standardizing practice for the follow-up and weaning of CPAP/NIV in children.