229P Home mechanical ventilation in paediatric neuromuscular disorders in a resource limited setting

Neuromuscular disorders (NMD) have seen a revolution in the availability of novel disease-modifying therapies (DMT) in the last few years. Yet affected individuals often gain suboptimal improvement despite these, due to the paucity of robust allied supportive specialities such as sleep medicine. Sleep Disordered Breathing (SDB) is fairly common in NMD and is the leading cause of death in them. Hence, non-invasive ventilation (NIV) is a crucial part of SDB management. This is often challenging where resources are limited. Here we describe the approach of our referral neuromuscular centre in establishing home mechanical ventilation (HMV) and respiratory care including cough assist. Clinical details of children with neuromuscular diseases attending our centre between January 2018 and August 2022 with SDB were collected by retrospective data review. Level 1 polysomnography (PSG) results of those who underwent the test were retrieved and analysed. 296 children (male: 67.2%, female: 22.8%) with NMD were thus included. The majority of our cohort had spinal muscular atrophy (SMA,165/296), followed by Duchenne muscular dystrophy (DMD, 67/296) and the remaining had other NMD (64/296). 164/296 (55.4%) subjects underwent a PSG. The median Apnoea Hypopnea Index (AHI) was 8.0 (0.3 - 78) per hour of total sleep time. Interestingly, 59.14% of our cohort had moderate to severe OSA. PSG could not be carried out for all individuals with clinical symptoms of SDB due to economic constraints, lack of resources and availability in carrying out PSG in children at a tertiary care unit with its attendant waiting times. Hence we initiated HMV in all subjects with NMD either when they had clinically overt symptoms of SDB or when the PSG supported it or both. HMV was initiated in 243/296 (82.1%) of the subjects included in our cohort. 14 of these individuals underwent a tracheostomy, 32 used cough assist, 22 were on nasogastric tube feeds and 20 underwent gastrostomy with fundoplication. In the subset with SMA: 69/165 (41.87%) children had received one of the DMTs like gene therapy (32/69) or Risdiplam (17/69) or Spinraza (20/69). Initiation of HMV reduced hospital admissions and improved quality of life. 15 children succumbed due to respiratory failure. The use of HMV and cough assist is well established in resource-equipped settings, but is extremely challenging in resource-limited ones, where affected individuals often receive DMT, due to various charitable initiatives without the benefit of adequate respiratory support. But even with these challenges such as long waiting times for PSG, the financial and logistical challenges including those for BiPAP (Bilevel Positive Airway pressure) as well as the cough assistive devices, it is still possible to ensure that subjects have access to them to gain meaningful outcomes from DMTs. In the long run, early initiation reduces the economic burden, both in terms of morbidity and mortality. A low threshold for initiation of BiPAP in those with SDB without a PSG initially is a feasible practical approach in resource-limited settings.