Palliative care for babies with spinal muscular atrophy (SMA) type 1 has improved in recent decades, with greater involvement of dedicated pediatric medical teams, a new French study suggests.
As more treatments become available, ethical questions about the appropriate level of care will need to be addressed, researchers say.
The study, “Palliative Care in SMA Type 1: A Prospective Multicenter French Study Based on Parents’ Reports,” was published in the journal Frontiers in Pediatrics.
Treatments for SMA type 1 have only become available recently. Providing palliative care has largely been considered the most ethical option for healthcare providers in France, while in the U.S., non-invasive ventilation and feeding support have been the preferred treatment at earlier stages of the disease.
Even with available therapies, patients may still require medical support that raises ethical considerations for parents and healthcare professionals about their use.
Researchers in France reviewed the state of palliative care for SMA type 1, with a particular emphasis on the views of parents of affected children.
Most data came from a prospective clinical trial (NCT01862042), which enrolled 37 children (20 girls, 17 boys, median age at diagnosis was nearly 3 months) with SMA type 1 who received palliative care at one of 17 centers in France between 2012 and 2016. Importantly, this timeframe is before the approval of the first SMA therapy, Spinraza (nusinersen, by Biogen), which was approved in Europe in 2017.
The children’s parents were given a health book that contained both guidance on care for SMA type 1, as well as questionnaires about the palliative care their children were receiving. They were encouraged to fill out the questionnaires at least once per month (though they could do so as often as they desired), and to solicit input from healthcare professionals.
Palliative care typically involved therapies to aid in breathing (e.g. oxygen therapy), to improve nutritional intake (use of a nasogastric feeding tube), to help with motor function (physical therapy), and to minimize pain (with analgesics, for example).
Care given to these children was compared to that given to 43 other children with SMA who were also treated at centers in France during the same time period (data collected through the French Pediatric Neuromuscular Network). Seven of these children had been treated with Spinraza.
Results showed no significant differences in patients not receiving Spinraza in the trial to those in the other group.
While the number of infants on Spinraza was small, they were more likely to receive non-invasive ventilation and feeding support but used less analgesics than children not receiving the medication.
These findings were also compared to data from 222 children with SMA type 1 who received palliative care between 1989 and 2009. While the course of the disease did not change, the researchers noted an increase in use of at-home hospital settings to provide care, and in the involvement of teams of healthcare workers dedicated to providing pediatric palliative care. These changes may be attributed to changes in French law regarding end-of-life care in the mid-2000s, the scientists said.
“Whereas, natural history has not evolved since 1989 in France for SMA-1 patients, improvements concerning integrated palliative supportive care have been made, enabling more coordinated medical support, as well as more well-defined implication of parents as everyday-life caregivers to their child,” they wrote.
The extent to which patients who receive recent treatments will need further care is still an open question, the researchers noted. As this continues to be evaluated, it will raise further ethical questions about care and therapy access.
“In that context,” the researchers wrote, “parents need to be clearly informed on the different existing options with the remaining unknowns, before they consent to any treatment option, including their mandatory implication in their child’s care and evaluation.”
Of note, three of the study’s authors were consultants or participated in advisory boards for Biogen, or received conference participation fees from the company.
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