Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2023 Jan 30;68(2):247-254.
doi: 10.4187/respcare.10353.

Management of Critically Ill Patients With Spinal Muscular Atrophy Admitted With Acute Respiratory Failure

Awni M Al-Subu  1 , Carolyn K Adams  2 , Scarlett A Dykstra  2 , Miranda R Langkamp  2 , Rhonda A Yngsdal-Krenz  3 , Rand M Al Subu  3 , Zhumin Zhang  4 , Kimberly N Baker  5 , Thomas B Brazelton  3
Affiliations

Management of Critically Ill Patients With Spinal Muscular Atrophy Admitted With Acute Respiratory Failure

Awni M Al-Subu et al. Respir Care. .

Abstract

Background: Spinal muscular atrophy (SMA) is a neurodegenerative disease that results in progressive muscular atrophy and weakness. The primary cause of morbidity and mortality in these children is pulmonary disease due to poor airway clearance that leads to acute respiratory failure. There is a paucity of literature on the treatment of children with SMA and acute respiratory failure.

Methods: We performed a retrospective chart review of pediatric patients with SMA type I or II admitted to the PICU in a tertiary-care children's hospital with acute respiratory failure who required mechanical ventilation and/or aggressive airway clearance.

Results: The study included 300 unique encounters among 137 unique subjects. All the subjects received airway clearance at home before admission, and 257 encounters (85.7%) were supported with noninvasive ventilation (NIV) before admission. Sixty-eight subjects (49.6%) required endotracheal intubation on admission or at some point during their PICU stay. The median (interquartile range [IQR]) time to intubation was 0 (0-20) h, and the median (IQR) duration of mechanical ventilation was 2 (1-5) d on invasive mechanical ventilation with no statistical difference between type I and type II (P = .89). Of those, 65 (91.1%) were successfully extubated to NIV on the first attempt, whereas 4 subjects (5.8%) required multiple extubation attempts and 3 subjects (4.4%) required subsequent tracheostomy. For the subjects who were intubated, both PICU and hospital length of stay were longer (P < .001) when compared with the subjects managed by NIV alone. The subjects with SMA type I had a longer PICU length of stay, with a median (IQR) of 5 (3-11) d versus 4 (2-7) d (P = .002). The hospital length of stay and duration of invasive mechanical ventilation were not statistically different (P = .055 and P = .068, respectively).

Conclusions: The subjects with SMA types I or II can be treated successfully with NIV and aggressive airway clearance during acute respiratory failure. Similarly, when intubation is required, successful extubation can be achieved with NIV transitional support combined with aggressive airway clearance maneuvers.

Keywords: SMA; mechanical ventilation; mucociliary clearance; neuromuscular diseases; pediatrics; spinal muscular atrophy.

PubMed Disclaimer

Conflict of interest statement

The authors have disclosed no conflicts of interest.

Figures

Fig. 1.
Fig. 1.
Hospital encounters for subjects admitted with spinal muscular atrophy (SMA) type I or II over the study period.
See this image and copyright information in PMC

References

    1. Nicolau S, Waldrop MA, Connolly AM, Mendell JR. Spinal muscular atrophy. Semin Pediatr Neurol 2021;37:100878. - PubMed
    1. Chen T-H, Hsu J-H. Noninvasive ventilation and mechanical insufflator-exsufflator for acute respiratory failure in children with neuromuscular disorders. Front Pediatr 2020;8:593282. - PMC - PubMed
    1. Mercuri E, Finkel RS, Muntoni F, Wirth B, Montes J, Main M, et al. ; SMA Care Group. Diagnosis and management of spinal muscular atrophy: part 1: recommendations for diagnosis, rehabilitation, orthopedic and nutritional care. Neuromuscul Disord 2018;28(2):103-115. - PubMed
    1. Wang CH, Finkel RS, Bertini ES, Schroth M, Simonds A, Wong B, et al. ; Participants of the International Conference on SMA Standard of Care. Consensus statement for standard of care in spinal muscular atrophy. J Child Neurol 2007;22(8):1027-1049. - PubMed
    1. Finkel RS, McDermott MP, Kaufmann P, Darras BT, Chung WK, Sproule DM, et al. . Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurology 2014;83(9):810-817. - PMC - PubMed

LinkOut - more resources