Long-term Breathing Help in SMA Rose in Last 10 Years in UK: Survey

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by Steve Bryson PhD |

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The use of long-term breathing help, or ventilation, has markedly increased among children with spinal muscular atrophy (SMA) type 1 and other conditions since 2008, according to a survey of ventilation centers in the U.K.

This increase coincided with the expanded use of various types of non-invasive masks as a method of long-term ventilation — defined as daily breathing support for at least three months.

In addition, due to the effectiveness of new disease-modifying therapies (DMTs) for SMA, approved in the last decade, more doctors are now offering breathing help for children with the rare genetic disorder, the survey found.

“The potential to now survive with this condition into later childhood has altered clinicians’ approach to ventilatory support,” the researchers noted.

“The results of this survey support the argument for a national database of children and young people using mechanical ventilation at home to inform future recommendations and assist in resource allocation planning,” the team wrote.

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Survey: more children need breathing help

The survey was published in the Archives of Disease in Childhood, in the study “Changes in UK paediatric long-term ventilation practice over 10 years.”

Breathing difficulties are common in children with the most severe types of SMA. Some patients may need support at night to assist with breathing during sleep, while those with more severe cases may require long-term ventilation (LTV), either invasively with a breathing tube placed in the windpipe (tracheostomy) or non-invasively with masks.

National surveys conducted in the U.K. in 1998 and 2008 highlighted the increasing numbers of children dependent on long-term ventilation, as well as the need for at-home breathing help. Such  support has now been implemented nationwide.

With significant improvements in managing children with complex neurological conditions, such as SMA, the use of long-term ventilation for children has continued to evolve over the last 10 years.

Recent studies demonstrated that long-term ventilation improves quality of life and extends survival in children with various disorders with breathing challenges.

Still, the current status of children in the U.K. undergoing long-term ventilation needs to be updated to include the changes in treatment and breathing support that took place over the last decade, according to the researchers, based at the Sheffield Children’s Hospital NHS Foundation Trust.

“The aim of this survey therefore was to collect data on the use of LTV in the paediatric population at a single time point and to compare the results with data collected 10 and 20 years previously,” they wrote.

The team culled data on the 2,383 children and adolescents from 25 long-term ventilation centers in the U.K. who were receiving LTV on Sept. 30, 2019. The median age of this group was 9, with a range from 0–20 years.

In comparison, 141 youths were on long-term ventilation in 1998, and there were 933 in 2008, representing a 2.5-fold increase between 2008 and 2019.

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‘Marked increase’ for type 1 children

Among children with SMA type 1, in particular, there has been a marked increase in the overall number of patients being ventilated, and in the proportion of those using mask ventilation. In 2008, 12 of these patients received long-term ventilation, compared to 87 in 2019. Likewise, seven children (58%) with SMA used masks for long-term ventilation in 2008 versus 83 (95%) in 2019.

A similar number of children with SMA type 2 or type 3 required long-term ventilation between surveys, with 67 in 2008 compared to 72 in 2019.

The proportion of patients treated at home between 1998 and 2008 increased, but there was little change between 2008 and 2019. Increases in long-term ventilation were seen in youths with other conditions affecting breathing, including central hypoventilation syndrome, a chronic lung disease of prematurity, those with birth injury/cerebral palsy, and airway blockages.

Tracheostomy was used in 471 children. The most common reason for tracheostomy was the duration of ventilation over 24 hours, followed by pre-existing airway problems, and non-invasive interface failure. Non-invasive masks were used in 1,819 individuals, with a nasal mask that fits over the nose being the most commonly used, followed by full-face masks, and nasal pillows.

Because disease-modifying therapies to treat conditions like SMA have only been introduced recently, there was no comparison with the previous surveys regarding the number of treated children. Current data showed out of 317 children diagnosed with a neuromuscular disease, 115 received a DMT.

Among children treated with the approved SMA therapy Spinraza (nusinersen), clinicians said the use of this therapy influenced or may have influenced their decision to start long-term ventilation in 35% of cases.

In contrast, the use of other treatments such as Translarna (ataluren) for Duchenne muscular dystrophy and Myozyme (avalglucosidase alfa) in Pompe disease, both neuromuscular conditions, did not influence this decision.

“With the prevalence and survival of children with life-limiting conditions increasing, the number of patients receiving LTV will continue to grow, resulting in technology-dependent children who eventually require transition to adult services,” the team concluded.

“It is therefore important to accurately understand the evolution of paediatric LTV, in order to recognise what impact this intervention may have on both paediatric and adult teams,” the team added. This will help LTV centers “plan optimal inpatient and community services to support the healthcare needs of this growing population.”

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