Table 2.
Noninvasive airway approaches for patients with NMD with acute on chronic respiratory failure.
| References | Study Design | Number of NMD patients (age) | NMD diagnosis (n) | ARF types* (n, %) | NIV/interface/ secretion clearance | Success rate and main findings | Predictor of NIV failure | NIV Complications (n) | Limit |
|---|---|---|---|---|---|---|---|---|---|
| Padman et al. (39) | Monocenter retrospective study | 11 patients; (range: 4-21 y) | DMD (7), SMA (2), SCI (1), nonspecific myopathy (l) | Type 2 (11, 100%) | BLPAP via nasal mask | • NIV success rate (no intubation): 91 % • Improved RR, PaCO2, serum bicarbonate, and length of hospitalization after NIV use |
None identified | No major complications | Hypoxic ARF and significant difficulty handling secretions |
| Birnkrant et al. (55) | Monocenter retrospective study | 8 patients (range 1-18 y) | DMD(5), SMA(3) | Undefined ARF, including 3 post-extubation ARF | BLPAP via nasal interface |
• Allowed weaning from an invasive airway: 100% effective in avoiding ETI or facilitating extubation |
None identified | NA | Non described |
| Niranjan and Bach (40) | Monocenter retrospective study | 10 patients (median: 17 y; range: 13-21 y) vs. 7 historical controls | DMD (8), SMA (1), SCI (1) | Type 2 (10, 100%), including 6 post-extubation ARF | BLPAP via mouthpiece or nasal interface + MI-E |
• NIV success rate (no intubation): 100% • Shorter hospital stay in NIV group than historical control |
None identified | NA | Non described |
| Bach et al. (56) | Monocenter retrospective study | 11 children with 28 ARF episodes (median: 6 m; range: 2-11 m) | SMA type 1 (11) | Post-extubation ARF (28, 100%) | BLPAP via nasal interface+ MI-E for post-extubation support |
• NIV success rate (no intubation): 82 % • NIV can facilitate extubation for type 1 SMA children even with severe bulbar muscle weakness |
None identified | NA | Non described |
| Vianello et al. (36) | Monocenter prospective case-control study | 14 patients (median: 24 y; range: 10-69 y) vs. 14 historical controls | DMD (7), ALS (4), CMD(1), HMSN (1), CM(1) | Type 2 (14, 100%) | E = BLPAP via nasal interface + cricothyroid-mini-tracheostomy; C = IMV via ETI |
• NIV success rate (no intubation): 71% (14% mortality rate) vs. 21% of controls (57% mortality rate) • Lower mortality and complications, and shorter ICU stay of NIV group than controls • NIV combined with cricothyroid-mini-tracheostomy for secretion clearance was well tolerated without significant complications |
None identified | No major complications | Severe bulbar involvement |
| Vianello et al. (37) | Monocenter prospective case-control study | 11 patients (median: 31 y; range: 16-64 y) vs. 16 historical controls | DMD (4), SMA (3), ALS (2), LGMD(1), FSHD (1) | Type 2 (11, 100%) | E = BLPAP via nasal interface+ MI-E+CPT; C = BLPAP+CPT |
• NIV success rate (no intubation): 82 vs. 37% of controls • No serious side effects and well-tolerated in all subjects with MI-E use |
None identified | Gastric distension (1), epistaxis (1) | |
| Servera et al. (41) | Monocenter prospective cohort study | 17 patients (48.7 ± 20.9 y) | ALS (11), DMD (4), transverse myelitis (1), nonspecific myopathy (1) | Type 2 ARF (17, 100%) | BLPAP via nasal/oronasal interfaces + MI-E |
• NIV success rate (no intubation): 79.2% • Severe bulbar involvement limited NIV effectiveness |
Bulbar dysfunction | NA | Severe bulbar involvement NIV/MI-E performed in non-ICU settings |
| Piastra et al. (42) | Monocenter prospective observational cohort study | 10 children (4.1 ± 4.5 y; range 3 m-12 y) | SMA type 1(2), CMD –Ullrich (1), CM-nemaline CM (1), MG (2), mitochondrial myopathy (1), spinal cord hamartomatosis (1), nonspecific myopathies (2) | Type 2 (5, 50%); Type 1 (2, 20%); mixed/undefined (3, 30%) | BLPAP via facial mask or helmet+ CPT |
• NIV success rate (no intubation): 80% • Hypercarbic ARF resolved within 6 h of NIV use • Oxygenation markers improved rapidly after NIV introduction |
Airway obstruction | No major complications | Copious tracheal secretion needing frequent suction |
| Dohna-Schwake et al. (53) | Monocenter retrospective study | 15 children (median: 6 y) | SMA (6), DMD (3), Pompe disease (2); CMD (2), myopathy (1), myotonic dystrophy (1) | Undefined ARF, including 2 post-extubation ARF | CPAP via mask |
• NIV success rate (no intubation): 87% • Improved HR, RR, blood pH, PaCO2, and SaO2 after 1-2 h of NIV use in the success group |
Low pH at 1–2 h after NIV | midface skin ulcers and gastric distension | 3 patients requested “do-not-intubate-status” |
| Chen et al. (57) | Monocenter prospective observational cohort study | 15 children with 16 ARF episodes (mean: 8.1 y; range 3 m- 18 y) | SMA (6), DMD (2), CM (2), MM (2), HMSN (2), LGMD 2I (1) | Type 2 (15, 94%) including 1 post-extubation ARF; Type 1 (1, 6%) | BLPAP via nasal/oronasal or facial mask + MI-E |
• NIV success rate (no intubation): 75% • Improved blood pH, and PaCO2 after 12 h of NIV use in the success group |
Fewer decrement of RR after 3 h of NIV use | No major complications | |
| Chen et al. (52) | Monocenter prospective observational cohort study | 56 NMD patients (44 children) with 62 ARF episodes; median: 13 y; range: 2 m-39 y) | SMA (32), DMD (14), CM (6), CMD (4), MM (4), HMSN (1), SMARD (1) | Type 2 ARF (53, 85%) including 23 post-extubation failure; Type 1 ARF (9, 15%) | BLPAP via nasal/oronasal or facial mask + MI-E |
• NIV success rate (no intubation): 86% • Improved HR, RR, blood pH, and PaCO2 after 4 h of NIV use in the success group • Shorter PICU and hospital stay of success group |
RR decreased at 4 h; pH increased, and PaCO2 decreased at 4-8 h after NIV | No major complications | Initial checking blood gases at a later point of 4–8 h after NIV |
*
Type 1 ARF, Hypoxemic ARF; Type 2 ARF, hypercapnic ARF.
NMD, neuromuscular disorders; NIV, non-invasive ventilation; ARF, acute respiratory failure; BLPAP, bi-level positive airway pressure; MI-E, Mechanical insufflator-exsufflator; E, experiment; C, control; CPT, chest physical treatments; DMD, Duchenne muscular dystrophy; SMA, spinal muscular atrophy; SCI, spinal cord injury, HMSN, hereditary motor and sensory neuropathy; CMD, congenital Muscular Dystrophy; CM, congenital Myopathy; MG, myasthenia gravis; MM, mitochondrial myopathy; SMARD, spinal muscular atrophy with respiratory distress; LGMD 2I, limb-girdle muscular dystrophy type 2I; NA, Not available.
CPAP, Continuous positive airway pressure.