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Spinal muscular atrophy type 1: avoidance of hospitalization by respiratory muscle support
Giancarlo Ottonello1, Chiara Mastella, Alessia Franceschi
1U.O. Anestesia e Rianimazione IRCCS G Gaslini, Genoa, Italy.
Insights
A protocol using noninvasive ventilation (NIV) and mechanically assisted coughing (MAC) effectively prevented acute respiratory failure and hospitalizations in young children with spinal muscular atrophy type 1. Caregivers can successfully implement this respiratory support strategy at home.
Area of Science:
- Pediatric Pulmonology
- Neuromuscular Disorders
- Respiratory Care
Background:
- Spinal muscular atrophy type 1 (SMA1) presents significant respiratory challenges in young children.
- Respiratory decompensation often leads to continuous noninvasive ventilation (NIV) dependence and oxyhemoglobin desaturation.
- Effective management strategies are crucial to prevent acute respiratory failure and hospitalizations.
Purpose of the Study:
- To evaluate an oximetry protocol utilizing continuous NIV and mechanically assisted coughing (MAC).
- To assess the protocol's efficacy in preventing acute respiratory failure and hospitalizations in children with SMA1 under 3 years.
- To determine the feasibility of implementing this protocol with trained caregivers in a home setting.
Main Methods:
- Retrospective chart review of 16 pediatric patients with SMA1 under 3 years.
- Patients were consecutively referred due to respiratory decompensations and NIV dependence.
- Protocol involved training parents in NIV, MAC, and basic life support for home use.
Main Results:
- 49 acute respiratory episodes occurred (1.20/patient/year), with 43 (87.8%) meeting criteria for avoided hospitalization.
- Only 6 episodes (0.15/patient/year) necessitated hospitalization.
- Four patients required endotracheal intubation (0.1/patient/year); three were successfully extubated to NIV and MAC.
Conclusions:
- A protocol combining high-span bilevel positive airway pressure (NIV) and MAC effectively manages airway secretions and normalizes oxygen saturation.
- Trained caregivers can successfully implement this protocol to avoid acute respiratory failure and hospitalizations in young children with SMA1.
- This approach offers a viable strategy for home-based respiratory support in SMA1.
Objective:
The aim of this study was to report the outcomes of an oximetry protocol using up to continuous full ventilator-setting noninvasive ventilation (NIV) and mechanically assisted coughing (MAC) to avoid episodes of acute respiratory failure and hospitalizations for children with spinal muscular atrophy type 1 under 3 yrs of age.
Design:
This study was a retrospective chart review of 16 patients with spinal muscular atrophy type 1 under 3 yrs of age consecutively referred for respiratory decompensations resulting in continuous NIV dependence and oxyhemoglobin desaturation. An avoided hospitalization was defined by the need for continuous NIV using high span bilevel positive airway pressure and reversal of desaturations by MAC in the home setting. The protocol included training and equipping parents to use NIV, MAC, and basic life support.
Results:
There were 49 acute episodes (1.20/patient per year), of which 43 met the criteria for an avoided hospitalization. Therefore, only six episodes (0.15/patient per year) required hospitalization, and four required endotracheal intubation (0.1/patient per year). Three of the four were extubated after 4, 9, and 15 days, respectively, to full NIV support and aggressive MAC. The fourth patient, for whom NIV could not be provided, underwent an emergency intubation at home and died at the age of 40 mos.
Conclusions:
A protocol including high span bilevel positive airway pressure along with MAC to expel airway secretions and normalize oxyhemoglobin saturation can be used by trained caregivers to avoid episodes of acute respiratory failure and hospitalization for children with spinal muscular atrophy type 1 under 3 yrs of age.
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