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Long-term Noninvasive Ventilation, CPAP Use Up Among Children in France with Neuromuscular Diseases


This new report also shows the value of national databases tracking the use of ventilation support.

A new study of French children with neuromuscular disorders (NMD) shows that long-term use of noninvasive ventilation (NIV) increased significantly in the past 20 years and that spinal muscular atrophy (SMA) was the most common diagnosis for children receiving the treatment

The report, which includes data from 27 pediatric university medical centers in France, helps to show how use of ventilation support has changed over time. The study was published in Neuromuscular Disorders.

The authors wrote that NIV, which can include continuous positive airway pressure (CPAP), is a way to offset the risk of alveolar hypoventilation caused by NMD.

“The role of NIV is to assist or ‘replace’ the weakened respiratory muscles to correct alveolar hypoventilation by maintaining an adequate tidal volume and minute ventilation,” the authors said.

NIV is initially used at night, when breathing tends to be less physiologically efficient, the authors said. Over time, however, patients may require breathing assistance during the daytime.

France has an NIV network that tracks data regarding pediatric use of NIV and/or CPAP. A 2021 study by the network showed that 1447 children younger than age 20 were using NIV/CPAP for at least 3 months on June 1, 2019. Of those, 28% had NMDs.

In the new report, the investigators wanted to offer a detailed analysis of that NMD cohort to better understand which patients were using NIV.

The authors found that 63% of the children with NMD using long-term NIV in 2019 were boys and that the entire cohort had an average age of 11.2 years. Of those, 33% had a diagnosis of SMA, 30% had congenital myopathy/dystrophy, 20% had Duchenne muscular dystrophy, and 7% had Steinert myotonic dystrophy. The remaining 9% had other NMDs, the authors said.

Of all of the patients receiving NIV, just 6% were receiving CPAP treatment. On average, children using NIV/CPAP used it for 8.0 hours per day, they noted.

The study authors added that the data show a large increase in the number of children with NMD receiving long-term NIV/CPAP in France over the past 2 decades, rising from just 35 in 2000 to 387 in 2019.

“However, although the number of NMD patients has risen sharply, the percentage of patients with NMDs treated with long-term NIV/CPAP has remained relatively stable, from 34% in 2000 to 27% in 2019, due to the large increase in children treated with CPAP for severe upper airway obstruction,” they wrote.

They said similar increases have been seen globally.

In 85% of cases, treatment was initiated electively. The authors said that data point suggests that screening for nocturnal hypoventilation is working, since NIV/CPAP is only being started because of a respiratory exacerbation in 15% of cases.

The authors said the study shows the value of data sets like the NIV network in France. They said such networks will become even more important as disease-modifying therapies become more widely available for NMDs like SMA.

“Surveys and registers also contribute to provide optimal medical care and to help to define recommendations and guidelines for the NIV/CPAP initiation, follow-up, and weaning of this increasingly challenging population,” they concluded.


Allaer L, Khirani S, Griffon L, et al. Long term noninvasive ventilation and continuous positive airway pressure in children with neuromuscular diseases in France. Neuromuscul Disord. Published online September 24, 2022. doi:10.1016/j.nmd.2022.09.008

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