Table 1.
Summary of time-to-event data for respiratory failure/support
| Author year | Data source, territory, population | N | Age | Outcomes |
| Aguerre 2020 [31] | A third-level pediatric hospital in Argentina | 59 | Children | Mean age at developing respiratory failure (SD), months |
| Type 1 SMA | •Type 1b SMA: 5.9 (2.32) | |||
| •Type 1c SMA: 13.8 (5.6) | ||||
| Kaneko 2017 [14] | Japanese individuals enrolled by questionnaire who completed the questionnaire | 112 | Adults and children | Median age at start of non-invasive positive pressure ventilation (range), months: |
| Types 1–3 SMA | •Type 1 : 47 | •Type 1a SMA (n = 3): 14 (5–51) | ||
| •Type 2 : 42 | •Type 1b SMA (n = 2): 23 (15–29) | |||
| •Type 3 : 23 | •Type 2a SMA (n = 6): 105.5 (27–379) | |||
| •Type 2b SMA (n = 16): 37 (24–160) | ||||
| •Type 3a SMA (n = 2): 336 and 468 (two patients) | ||||
| •Type 3b SMA (n = 1): 600 (single patient) | ||||
| Median age at start of TPPV (range), months: | ||||
| •Type 1a SMA (n = 35), 6 (2–51) | ||||
| •Type 1b SMA (n = 3), 12 (11–122) | ||||
| Wadman 2021 [13] | Data from a single-center retrospective study in the UK and a prospective multicenter study in Italy | 146 | Children and adolescents | Median age at start of NIV (range), years |
| Type 2 SMA | •Total cohort (n = 146): 4 (1–17) | |||
| •UK (n = 72): 6 (2–17) | ||||
| •Italy (n = 74): 3 (1–11) | ||||
| Wadman 2017 [8] | Dutch patient organization for neuromuscular diseases | 200 | Children and adolescents | Mean age at start of NIV (range), years: |
| The Netherlands Types 0–4 SMA | •Type 0/1a: 3 | •Type 0/1a SMA (n = 3): 0 | ||
| •Type 1b: 16 | •Type 1b SMA (n = 16): 0.8 (0.6–1.1) | |||
| •Type 1c: 23 | •Type 1c SMA (n = 23): 11 (1.3–36) | |||
| •Type 2a: 49 | Mean age at start of ventilatory support at night (range), years: | |||
| •Type 2b: 38 | •Type 2a SMA (n = 49): 14 (2–38) | |||
| •Type 3a: 33 | •Type 2b SMA (n = 38): 19 (2–66) | |||
| •Type 3b: 33 | •Type 3a SMA (n = 33): 46 (15–61.5) | |||
| •Type 4 : 5 | •Type 3b SMA (n = 33): 40 (39.6) | |||
| Mean age at start of ventilatory support > 16 hours/day (range), years: | ||||
| •Type 2a SMA (n = 49): 20 (16–24) | ||||
| •Type 2b SMA (n = 38): 31 (31) | ||||
| •Type 3a SMA (n = 33): 34 (20–47) | ||||
| •Type 3b SMA (n = 33) 0 |
Abbreviations: NIV = non-invasive ventilation; SD = standard deviation; SMA = spinal muscular atrophy; SMN = survival of motor neuron; TPPV = tracheostomy positive pressure ventilation.