Treatment Delays May Be Less Important Than Family Support

Marisa Wexler MS avatar

by Marisa Wexler MS |

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family support | SMA News Today | illustration of someone pushing a wheelchair with a child

The COVID-19 pandemic led to delays in treatment with Spinraza (nusinersen) for many children with spinal muscular atrophy (SMA). However, these delays seem to have a lower impact on children’s functional skills when compared with family support, according to a small study in Italy.

The study, “Effect of the COVID-19 Pandemic on Children With SMA Receiving Nusinersen: What Is Missed and What Is Gained?,” was published in Frontiers in Neurology.

Spinraza, marketed by Biogen, is the first disease-modifying treatment widely approved to treat SMA. In Italy, the medication first became available in October 2017. Spinraza is administered as an intrathecal injection, or an injection directly into the spinal canal and given every four months as a maintenance therapy.

The COVID-19 pandemic has caused widespread disruptions in medical care. Now, a team of Italian researchers reported clinical data on 25 children with SMA (median age 8 years, range 2–15) who were being treated with Spinraza when the pandemic hit. The team conducted interviews with some of the children and their parents to understand its effects.

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About a third of the 25 children — eight, or 32% — experienced a delay in planned Spinraza infusions due to a pandemic-induced lockdown. Delays ranged from 26 to 91 days, with a median of 58 days (about two months).

The team noted that there was no clear association between these treatment delays and changes in functional scores.

“Although a detailed statistical analysis was not possible due to the small sample size, no correlation between a delayed treatment and changes of functional scores emerged over the short-period evaluations ([approximately] 2 months after the end of the first lockdown) and the immediately following months,” the researchers wrote.

Of the children assessed, three experienced marked declines in functional ability in the first several months of the pandemic. One had scoliosis that worsened shortly before the pandemic hit, and another had a generally severe disease course. The third patient was not compliant with the recommended care “due to a poor social context,” the researchers wrote.

The team also noted that two patients experienced substantial improvements in function compared with scores before the lockdown. These improvements were “due to great family support,” they wrote.

“Children who had both parents caring for them and helping each other during the lockdown showed the best improvement in motor function, while in the presence of social issues, above all in single-parent families, children suffered a lack of home institutional help,” the team wrote.

Concerning the interviews, five out of 25 parents did not perceive a change in their child’s muscle strength, 12 reported a worsening, and eight felt an improvement. Regarding their child’s breathing function, 80% of the parents (20) did not perceive a change, and 88% (22) did not perceive a change in swallowing abilities.

Most parents interviewed believed that the worsening seen was mainly “due to the suspension of outpatient physiotherapy (10 out of 25, 40%) or to a delay in nusinersen infusion (6 out of 25, 24%),” the researchers wrote.

Among the 13 children interviewed, 10 (77%) did not perceive a change in their muscle strength, two (15%) felt a worsening, and one (8%) reported a great improvement. Most children (92%, 12 children) did not perceive a change in swallowing or breathing function (69%, nine children).

According to the questionnaires, what children missed more during the lockdown was going to school and seeing their friends (61%) or taking lessons (23%).

Overall, the researchers suggested that “the great worsening [in functional scores] was seen in both the short- and long-term evaluations and cannot be attributed to the delay in infusions but to a difficult family situation of the patients,” they wrote.